Growth Hormone Deficiency Learning Zone
Transcript: Sustained engagement and adherence in long-acting GH therapy
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Recognizing syndromic and secondary causes of GHD
Lars Sävendahl, MD, PhD, Bradley S. Miller, MD, PhD
- [Brad] Hi, my name is Brad Miller and I'm a pediatric endocrinologist at the University of Minnesota Medical School and M Health Fairview Masonic Children's Hospital. Today I'd like to introduce the podcast, "Bridging growth hormone deficiency gaps: From complexity to continuity". Today's topic is recognizing syndromic and secondary causes of growth hormone deficiency, and I'm joined by Lars Sävendahl.
- [Lars] Hi, I am Lars from Karolinska Institute in Stockholm, Sweden. I'm pediatric endocrinologist, and I'm also senior physician at Astrid Lindgren Children's Hospital at Karolinska University Hospital in Stockholm. Pleasure to meet you, Dr. Miller. - So great to talk to you tonight. What I'd like to start with is which clinical features should prompt clinicians to look beyond isolated growth hormone deficiency and consider syndromic or secondary causes? - To me, looking at the growth curve and losing growth percentiles between one and two years of age is to me a quite strong indication that there might be a syndromatic short stature. We all know that growth hormone deficiency rarely presents with growth failure before one year of age. And of course a familial short stature can also present second year of life. But a clear loss of growth precentiles during second year of life is suggestive of a syndrome. And if you look for example, at Turner Syndrome patients, most of them have lost growth potential during their second year.
- [Brad] Excellent. The other things that we think about as early warning signs, aside from the growth pattern, what are some of the other symptoms or physical changes that we would look for to identify those other conditions?
- [Lars] Of course, developmental problems, neurodevelopmental problems. We always look for any syndromic features, malformations, micro or macrocephaly for example. And of course all the syndromatic, we have full pediatric endocrinologist and geneticist. We have a quite systematic way of going through the patient. I usually go from top to down and starting with the hairline and then the forehead and the eyes and going down and ticking off. And I think if you do that systematically, you are quite likely to pick up any syndrome, even if you can't really point out what syndrome, if you have done this systematically in many patients, you are quite good at this.
- [Brad] When we think about the very early patients who have growth hormone deficiency, are there some key signs that we should be looking for for those congenital syndromes or genetic syndromes that have a growth hormone problem?
- [Lars] I mean, congenital growth hormone deficiency, which is often malformations, boys micropenis is what we look for. Symptom could be hypoglycemia, especially if there is a multiple pituitary hormone deficiency where you have both cortisol and growth hormone deficiency. So there are ways to diagnose based just on clinical signs and symptoms, pituitary deficiency including GHD, very early on in life and during first year of life, as I said, we cannot really rely on growth.
- [Brad] What are the family history diagnostic clues? Are there any connections we can make from the family related to growth hormone deficiency? And how often is that growth hormone deficiency itself a genetic condition that you see?
- [Lars] Yeah, of course if the patient grow much below the genetic height, we always calculate the mid parental target height. And if the patient has a growth trajectory, which is clearly below more than minus two standard deviation below the target, that suggests a problem, right, which could be syndromatic. Then of course if there is any history of a genetic disorder in the family is very important. It could be a recessive, it could be dominant, it could be X-linked, right? So you always should ask for this in your family history. And when taking family history, do it in three generations. I always plot a growth chart, three generation growth chart. And if you do that graphically, you can quite quickly pinpoint whether this is a genetic short stature or whether this might be an autosomal dominant disorder, for example. And other questions I usually ask because I also run a tall stature clinic is or a sudden death in any family member at a young age right? Which can indicate the syndrome when it's on the tall side is Marfan syndrome, but there're also other syndromes where you may die early on, right, from a syndrome.
- [Brad] No, I think, no, very good point. And I've definitely had families where I've seen the adult and then the child, who both have the same genetic cause for growth hormone deficiency. And so they weren't necessarily syndromic, if you will, but they had, as you mentioned, a genetic cause of a growth hormone problem. So when do you think genetics is necessary in our evaluation for children for growth in general and then more specifically growth hormone deficiency?
- [Lars] I think it is, you need to be very careful when performing genetics and I think it is good to follow algorithm when it comes to performing genetic workup. I strongly recommend the newly published international guidelines published in the European Journal of Endocrinology just a few months ago here in 2026 by Andrew Dauber and many international co-authors. And they give guidance on when to perform genetic testing and what genetic testing and an algorithm. So depending on what symptoms you have and likelihood to have a genetic course, you perform different testing and they classify patients into isolated short stature, those who might have skeletal dysplasia, those patients who might have defects in growth hormone IGF axis, and those who might have syndromic short stature. And as we discussed earlier, if you have for example, neuro developmental disorders or major malformations, then you should consider referring to clinical geneticist. And depending on them, whether it's recognized or unrecognized syndrome, we can do either targeted testing or consider referral to a medical geneticist to perform more advanced genetic testing.
- [Brad] I think it's an area where we as pediatric endocrinologists are becoming more involved and one of the challenges is access to that medical geneticist or genetic testing, whether it's because of insurance barriers or referral challenges. And so I think we as pediatric endocrinologists need to learn more and arm ourselves for interpreting the test, but continue to partner, as you said, with our genetics colleagues for phenotyping as well as interpretation and recommending of the proper testing. So definitely.
- [Lars] Testing becomes less and less expensive and we need to really be aware of the potential benefits and risks of performing a test. And we need to always involve the family and the patient in that decision-making, whether to perform the testing or not, because the false positive is quite high in these genetic testing when you can make a lot of secondary findings, which is irrelevant.
- [Brad] Yeah, let's transition now to secondary causes of growth hormone deficiency. And I know that you have been involved in caring for children who have developed a growth hormone problem because of their medical conditions that they've experienced. So what are the things that we need to focus on there?
- [Lars] Of course, secondary then we need that there's another cause, right? Like a damage or disorder of the pituitary or hypothalamus rather than a genetic, right? And this could be, for example, craniopharyngioma, it could be post-radiation for neuro brain tumor or post-surgery. In some cases also it could be post-traumatic, these secondary growth hormone deficiencies. And here of course we'll have a lot of help from just a medical history from neuroimaging of course and the physical exam and labs. But here often we need to include neuroimaging in evaluation.
- [Brad] Yeah, I think traumatic brain injury is an unrecognized potential cause I've taken care of a few children who had long-term growth failure after a significant traumatic event that wasn't recognized as related to growth hormone deficiency. The other piece, intrathecal chemotherapy has been recognized over time as being an a risk factor in some of our cancer survivors. And so in addition to radiation, I think those are key questions.
- [Lars] And one other important underlying cause is glucocorticoids. That's my research taught an area actually, negative consequences of glucocorticoid treatment on growth. And many of these patients have been treated on quite high doses of GCs, glucocorticoids. So always look at the medicines, whether there is any treatment that might affect growth negatively. Even inhaled steroids, even the asthma patients on high doses of glucocorticoids often present with the growth failure and sometimes end up in peds endo clinic. So that's of course one important differential.
- [Brad] Good question. When you're thinking about the diagnostic workup for syndromic or secondary growth hormone deficiency, what are the things that you should be focusing on?
- [Lars] Neuroimaging, as I said, is of course important there and then we need to do the labs as well, right? But of course if we have a history of brain surgery or radiation and we know based on the radiation dose, that is very likely that the patient will develop growth hormone deficiency and the patient present with short stature and low IGF1, we do not necessarily need to do full pituitary testing. So then diagnosis is quite easy.
- [Brad] What are some of the limitations of the tests that we use, particularly in individuals with secondary growth hormone deficiency? I find that sometimes our testing isn't as helpful as we'd like it to be.
- [Lars] I entirely agree. Entirely agree.
- [Brad] So the growth hormone deficiency stimulation tests sometimes is normal in children who have a clear damage to the hypothalamus and pituitary.
- [Lars] Correct.
- [Brad] Sometimes if they're overweight, their IGF1s aren't as low as we would expect them to be. So those I think are some of the things that I've noticed in my clinical experience.
- [Lars] I totally agree.
- [Brad] I mentioned earlier how we capture these patients and part of the challenge is how do we talk to our colleagues to make sure that these children are being referred, whether it's the geneticist, the neurologist, or the oncologist, and how can we make improve that process?
- [Lars] I think multidisciplinary team conference, we always have that actually every morning in our peds endo clinic, where we discuss new referrals and who to see the patient and what team to see the patient. So individualize and sometimes you need all these specialists, sometimes you do not need it. So individualize your team members.
- [Brad] For us, the oncology team is one of the key partners. And personally I am in the clinic with them every week as part of watching children as they're going through their therapy early in the diagnosis and then continuing to follow them afterwards. But it's interesting, that seems to work for our brain tumor patients, but less so for our leukemia patients. And so sometimes I'm not there on the day that the leukemia patients are there. And so I think it's really important to develop those workflows with the teams to make sure that we're catching everybody.
- [Lars] Yeah we have a similar setup here, regular conferences with our oncologists and I think that is really advisable to have.
- [Brad] The bigger challenge is the primary care provider who's seeing patients in the community, what has worked for your system in terms of educating and communicating with them to make sure the patients come to see us when they should.
- [Lars] Either by regular meetings, where we invite primary care physicians and importantly local regional guidelines. So how to work up the patient in primary care and when to refer the patient to the specialist. Such written guidelines, I feel is very beneficial. And if you have those guidelines, you can ensure that the patient always arrives in your clinic with quite complete growth chart, for example. And the medical history information that you need. And also some basic workup that we stress the primary care physicians to do prior to referring to exclude other causes of short stature. I mean many chronic disorders we know including family psychological problems may present as with short stature. And they, I personally believe should not end up in peds endo specialist clinic. They should be handled by primary care.
- [Brad] When you think of the children with syndromic causes of growth hormone, hormone deficiency, they often have other associated genetic or birth defects. How does your team partner with trying to make sure that they're seeing all of their specialists in a coordinated way?
- [Lars] We have clinical guidelines for each of these syndromes detailing how often and when to meet these different specialists. So it's kind of an algorithm and an Excel sheet. So not to miss anything. And this is also shared with the families and patients. So they are also aware and I think to involve the families is important because then we reduce further the risk that we miss something.
- [Brad] Yeah how do you, in your system, is that, is the nurse in charge of that? Is there a complex scheduler that keeps track of that or is that more on the families? What's worked in how your system tries to coordinate that care?
- [Lars] So for each, I mean for this guidelines, each specialist clinic have their own system to ensure that you have the ophthalmology checkup every year. They have the cardiology every year, et cetera. But we have always one kind of primary specialist who take care of the patient. Often it's the peds endo to ensure that these, all these other specialist visits take place at the right time point. But often it works very well because everyone is aware of these guidelines and automatically call the patient back. I think it works pretty well.
- [Brad] I think that is a challenge in different systems to figure out who is in charge of those pieces and making sure, you know, as a pediatric endocrinologist, I think of myself as a complex pediatrician for, you know, the quarterback for many of the children with rare diseases. And so I find myself referring them back to the cardiologist because they haven't been to see them for some time, et cetera. So I think it's an important thing to decide who is that person for each individual condition.
- [Lars] Agree.
- [Brad] Are there any other key points that you would wanna bring up for either helping that long-term monitoring or the initial diagnosis or the acquired growth hormone deficiency? Any other thoughts that you'd like to share?
- [Lars] I think we have covered this quite well, Dr. Miller today and to you who listened to this podcast, I really encourage you to look into this clinical guidelines by Andrew Dauber in European Journal of Endocrinology, published earlier this year, 2026, the international guideline on testing of children with short stature genetic testing and follow the algorithm there, which is very much in line of what we have discussed today in our podcast, Dr. Miller.
- [Brad] Absolutely. No, I think that the genetics of all of our conditions is gonna be really important. We, as pediatricians, sub-specialists, et cetera, are going to be learning how we incorporate the genetic tests into the care, individualized care of our patients over time. So I think it's a really key topic and an excellent consensus group that came together specifically related to growth.
- [Lars] What I should also mention, Dr. Miller, is that artificial intelligence, there are ways now to diagnose syndromes. There are legal aspects there to be cleared out and which is often a problem. But if you can solve this and use, there are commercial tools and also free tools to diagnose syndromes just based on pictures. Geneticists use this quite frequently. I've tried that in my own clinic with quite good success.
- [Brad] Well, thank you very much Dr. Sävendahl for this conversation today and look forward to talking again.
- [Lars] Very nice talking to you, Dr. Miller, and have a nice day today. Bye.
The importance of early referral and multidisciplinary care
Lars Sävendahl, MD, PhD, Bradley S. Miller, MD, PhD
- [Brad] Hi, my name is Brad Miller, and I'm a Pediatric Endocrinologist from the University of Minnesota Medical School and the M Health Fairview Masonic Children's Hospital. We'll be talking today about "Bridging GHD gaps: From complexity to continuity". This is a Medthority sponsored podcast. The title today is "The importance of early referral in multidisciplinary care". Joining me today is Lars Savendahl.
- [Lars] Hi, thank you very much, Dr. Miller, for introducing. I'm Lars Savendahl. I'm a Professor of Pediatric Endocrinology at Karolinska Institute in Stockholm, Sweden. I'm also Senior Physician at the Astrid Lindgren Children's Hospital and Karolinska University Hospital in Stockholm. It's a pleasure to record this today with you, Dr. Miller.
- [Brad] Excellent, so what I'd like to start with is why is early identification and timely referral important in the management of children with growth hormone deficiency?
- [Lars] Well, first of all, early diagnosis is much easier than having a patient approaching puberty. The diagnosis tests have much better specificity at a young age. It is a great advantage to be able to start treatment early as we know that the long-term growth outcome is significantly better the earlier you start. So from a life perspective, you do need more growth hormone milligram than if you start later, but the growth outcome is better.
- [Brad] Excellent. What are some of the challenges in getting to see the kids earlier for us as pediatric endocrinologists, and how can we fix that?
- [Lars] It's difficult. I mean, it might be parental delay, it might be delay in the primary care physician or pediatrician as sometimes there are financial issues, for example, in United States, less in many European countries. And I think it's very important to have podcasts like this and other kind of information directed to HCPs and also to the general public discussing the importance of early referral when child has growth problems,
- [Brad] What are some of the early signs that somebody might have growth hormone deficiency, particularly in infancy or even as young children, toddlers?
- [Lars] Well, in the newborn, of course, we always look for hypoglycemia, developmental problems, otherwise during childhood and infancy, early growth failure. But it's important to note that during first year life, it's very difficult to diagnose a growth hormone deficiency based on growth failure. That usually will not present until the second year of life.
- [Brad] So what are the things that the pediatrician should be watching for as they're trying to pick up children who require a referral to the endocrinologist?
- [Lars] Always analyze and look at the growth chart carefully and look at the weight and high development together. And if you lose significantly more in height than weight, this, of course, suggests an endocrine problem, and it could be a growth hormone deficiency.
- [Brad] I live in Minnesota, which I call the land of tall people, and I know Sweden is similar. How much does the role of the family's height play in your evaluation and where the child is compared to that?
- [Lars] Of course, it plays very important role and we always calculate mid parental target height. And as you know, most children they grow within +/- 1 SD from their mid parental target height. And if the current height is significantly deviant from the family height and that could be a sign of, for example, a growth hormone deficiency.
- [Brad] Is there an age at which that becomes really helpful? I know and as you said in the infants, it's a little harder to gauge where they should be, is that more second or third year of life where you feel like- - Yeah, yeah. - Mid parental- - Second, third life, that's usually when you see these children present. However, if there is, for example, an underlying syndrome, I worked a lot with Turner syndrome during my fellowship in North Carolina and we looked at the growth pattern of children with Turner syndrome and they usually lose between first and second year of life, so syndromatic short statue often present earlier, second year of life.
- [Brad] And I think it's really important as we talk about trying to identify children with growth hormone deficiency that we may identify other conditions that are not growth hormone deficiency. - Correct. - And have to separate them. So yeah. - Yeah.
- [Brad] So what roles do the primary care physicians play in that early stages of growth hormone deficiency evaluation? What are the things that you should think they should be doing to evaluate children early?
- [Lars] First of all, exclude other causes of short stature. Everything from psychological problems, family problems to other chronic disorders, which is, of course, much more common than growth hormone deficiency. So here in Sweden, we have in the clinical guidelines how to evaluate children with short stature in primary care. So I think, and there are also several both national and international guidelines and it's very good to adhere to these.
- [Brad] One of the challenges we have in evaluating whether a child has a chronic problem is knowing which tests to get. And I know there have been literature saying cost-effective approaches are really important as well. So what are things that you recommend that people do besides a good history and a good physical examination and looking at the growth chart?
- [Lars] Of course, a basic laboratory workup, hopefully the primary physician has already made it, right? And that includes, of course, looking at other hormones like thyroid hormone. Thyroid hormone deficiency is very important to exclude. We always look at IGF-1, IGFBP-3, and also a quite wide battery of other basic labs. I think it's good to have an algorithm or guideline to follow when you work up a child with short stature. Several have been published recently. There is a very recent paper published in European Journal of Endocrinology by Andrew Dauber presenting an algorithm for how to work out a child with short stature and more specifically how to use genetic testing in a wise way in a child presenting with short stature. It's important to note not all children should undergo genetic testing. You need to be quite selective there because there are both pro and cons with genetic testing. - Absolutely. - But I highly recommend this paper by Dr. Andrew Dauber, an international consortium, international guidelines in European Journal of Endocrinology published earlier this year.
- [Brad] I think it is really important, and one of the things that they highlight in that paper was that there are genetic forms of growth hormone deficiency, but there are also many different genes that regulate our growth that have nothing to do with the growth hormone and IGF-1 pathway, and some that we would expect affect the growth plate and others that we wouldn't understand before we knew that they were involved in growth, that they could impact it, so a very important paper. - Indeed.
- [Brad] One of the keys I thought in that paper was they also talk about the developmental issues that you mentioned earlier as being an important part of deciding whether or not a child would warrant genetic testing.
- [Lars] And then, of course, dysmorphic features, microcephaly, macrocephaly, for example, of other signs that could warrant genetic workup.
- [Brad] Are there particular physical differences aside you mentioned the hypoglycemia is a symptomatic one that picks up growth hormone deficiency. Are there physical changes that you would say are more common in a child with growth hormone deficiency?
- [Lars] Yeah, in the newborn, a micropenis is we always look for in the males that could suggest growth hormone deficiency. And of course there could be signs of other pituitary hormone deficiencies also that can present together with growth hormone deficiency in the newborn.
- [Brad] Yeah, I think of midline defects as something that we some watch for as well in those children. The children with optic nerve abnormalities are ones that we keep a track of as well. - Yeah. - With concerns either early on or developing over time.
- [Lars] Yeah, yeah. But not all of the children with septic optic dysplasia have growth hormone deficiency, a subgroup there.
- [Brad] Definitely agree. No, I think that's a key important, like these are risk factors but don't make the diagnosis for us. So looking forward, once you have the patients identified with growth hormone deficiency, what's your team look like in terms of caring for children with growth hormone deficiency and what are the different roles of the people on the team?
- [Lars] We have a team, our peds endo clinic, we have pediatric endocrine nurses and doctors. We also have geneticists, we have dieticians, we have psychologists. And depending on the family situation, we engage more or less of these team members. Every child, every family regularly meets the nurse in separate nurses clinics. And generally, every other clinic visit here in Stockholm is a nurses visit and every other a doctor's visit, and thereby we can see the patients more frequently. I know that in the United States, where I also practiced before, patients more frequently see the doctor. In many places in Europe, this is alternating between nurses and doctors. And I think personally that's a very efficient way. Nurses often have more time and can bring up issues that maybe sometimes the doctor for time reason cannot discuss.
- [Brad] No, I think it's you highlight differences in the system> Here in the states, I partner with a nurse practitioner that we alternate visits with the patients, but she also cares for a number of patients independently as well. So it's a good team, but we also include our endocrine nurses in the mix as well. Do you have a pharmacist that is part of team as a regular component?
- [Lars] We have a pharmacist who is linked or associated with our team and that we on demand can consult, but the pharmacist is not involved in every patient.
- [Brad] And that's similar for us as well. - Yeah, yeah.
- [Brad] When you think about the differences that people bring to the team, you mentioned the endocrine nurses, what are the special areas that you ask them to be involved with?
- [Lars] I mean, the more looking into the social parts, half of the families do not live together, the parents, and they alternate between often one week at father's, one week at mother's home, and there are challenges there and which is linked to the adherence of treatment. And of course, the new possibility of using long-acting growth hormone with an injection once a week could be an advantage to such families where it works a little bit better with one parent than the other.
- [Brad] For sure, I think that's something that we're all exploring now and learning more about is how much will that impact adherence or compliance and what are the unique circumstances where it may be beneficial? And you talked about multidisciplinary involvement and mentioned the psychologist. How much does that individual, either a social worker or counselor, psychologist play in your system?
- [Lars] The psychologist should play a stronger role here. As I mentioned, I did my fellowship in North Carolina in the '90s and there, we had a psychologist, Brian Stabler, who saw every patient with short stature first visit and before a decision-making on starting growth hormone treatment. And I think that was very wise because many, many times it's not an issue about growth hormone, it's psychological issues behind the patient and the family can be as much help by such psychological support rather than growth hormone. Unfortunately, we don't really have the same resources here.
- [Brad] Yeah, it's interesting. We don't have those resources as available as we would like them either. And I don't think they're as much a part of a team as we would like them to be. So how does coordinated multidisciplinary care support effective long-term management during the pediatric years? And so how do we communicate amongst ourselves to make sure that we're keeping patients on treatment and helping them stay on it longer?
- [Lars] I think it differs between teams, but we have a systematic way of communication between team members to facilitate adherence, and we monitor adherence at every clinical visit and we recorded also in the hospital records, so I think this needs to be worked on. We all know that adherence will decrease over time if we are not very, very persistent emphasizing the importance of adherence.
- [Brad] What are the ways you track adherence? In our clinic, we ask the patients and they always tell us they're taking their medicine, so what ways do you- - I agree. - Find to be effective?
- [Lars] I believe they are quite honest. And I always ask that in every clinic visit. There, of course, we are have other ways, looking at growth IGF to see if they're not telling the truth and we can also see whether they do not ask for refill of prescriptions, so you can count backwards, so I think that there are ways to look through this.
- [Brad] Yeah, I think the pharmacy has been a helpful component for us as well. - Yeah, yeah. - And I'm hoping that as we move forward with technology, that that'll be more seamless in terms of being able to identify people who are struggling with compliance, and it's usually, as you said, it's not intentional, it's the multiple other barriers that get in the way, but figuring those out are really key.
- [Lars] Yeah, and of course, electronic devices could potentially facilitate adherence.
- [Brad] I think there are some digital technologies moving forward that may help, so I think that's an exciting area. The last topic we wanted to talk about today is the transitioning and how do we prepare our patients for that transition to adulthood. Some of our patients will need growth hormone long-term, and what does that look like in your system?
- [Lars] First of all, we prepare the families that growth hormone treatment is always reevaluated every year and will be reevaluated before when growth is finished and before any transition. We also ask the patients too as early as possible to take responsibility for injections themselves to prepare for transition and adult life later on. I personally try to always be with the adult endocrinologist in the first transition clinic. I think it's very good to have a formalized transition clinic, but this is problematic transition, and we know that adherence may decrease after being trans transitioned to adult medicine.
- [Brad] Are there things that you found to be effective aside from visiting with them together, other things that have helped that transition?
- [Lars] Not as I can remember right now. Maybe you, Dr. Miller, have any hints there?
- [Brad] No, yeah, I was going to share on my end, we do meet with the endocrinologist that they're going to be partnering with and, as you said, try and get the family to meet with them early in the process so that they knew who they're going to be transitioning to. We also start having the patient take responsibility not only for their injections, but for reporting their own healthcare, for making their appointments, et cetera, to get that to be more of their responsibility and ownership, if you will. So, but it's still a challenge, we have many of our patients go off to adulthood and then suddenly come back to us saying we didn't find the right fit, and so that can be a challenge
- [Lars] For here at Karolinska, an advantage we are just 50 meters from each other, adult and pediatric endocrinologists, so many of our patients steps by here even after turning 80. And so, but of course we always meet with the adult endocrinologist also prior to seeing the patient together, so to try to have that doctor-to-doctor communication, so. - Excellent. Well, I think this has been a great conversation about children and adolescents with growth hormone deficiency, and I appreciate your time today.
- [Lars] It's been a pleasure, Dr. Miller. Have a nice evening.
Why structured transition from pediatric to adult care matters
Bradley S. Miller, MD, PhD, Mehul Dattani, MD
- [Brad] My name is Brad Miller, and I'm a pediatric endocrinologist from the University of Minnesota and the M Health Masonic Children's Hospital. Today, the title of the series is "Bridging GHD gaps: From complexity to continuity" by Medthority," and the title of today's topic is "Why structured transition from pediatric to adult care matters," and I'm joined by Mehul Dattani.
- [Mehul] Hi, Brad, nice to meet you.
- [Brad] So Professor Dattani, please let me know your institution, where you're from and what your practice is. - So I'm a professor of pediatric endocrinology at Great Ormond Street Hospital in the Institute of Child Health in London, and my clinical practice covers clinics both at Great Ormond Street as well as University College London Hospital, so the practice at Great Ormond Street Hospital is in children below the age of 16 years, and the practice at UCLH is a transition clinic with patients anywhere between 13 years and 20 years.
- [Brad] Excellent, and I think the main focus of our conversation today is to dive into transition and really talk about the process of transition. To start with, why does this structured transition matter for young people with growth hormone deficiency entering adult care? And that includes children with hypopituitarism.
- [Mehul] I mean, I think the transition process is extremely important because it's well-known that young people tend to be lost to follow-up at this crucial time, there's a lot going on in their lives, and unfortunately, it's health that is often not prioritized as much as it should be. We know that adherence at the time of puberty can be a challenging issue, and we know that there is a very significant loss of follow-up in a number of other disorders, although the data for growth hormone deficiency aren't clear as yet, but up to 50% of patients are lost with some conditions such as diabetes or Turner syndrome, et cetera, so I think it's important to try and make sure that, at this stage, there is strict vigilance really of the young person's health, the family need to be on board and we need to understand the various things going on in their lives, which I'm sure we'll touch on as we talk, to ensure that they are moved safely from pediatrics to adult care.
- [Brad] How do the coexisting conditions, I mentioned hypopituitarism, but when we think of growth hormone deficiency isolated versus panhypopit, where does that grade of adherence or challenge increase?
- [Mehul] So I think with growth... I think growth hormone deficiency in isolation, the children usually want to grow, and once they're of a reasonable height, they then think, "Hang on, why am I doing this?" And then, they can often sometimes stop the medication. On the other hand, the hypopituitarism is extremely important, that the treatment is monitored very carefully because if, obviously, they are lost to follow-up and they don't take their medications, then they are at risk of becoming extremely ill, and so, I think it's extremely important to engage, you know, patients with both disorders, either isolated growth hormone deficiency or hypopituitarism, and ensure that the right assessments are made at transition and that the patients then move on at the right time to the right physicians in adult care.
- [Brad] One of the big challenges in our practice, the added layer, if you will, are children with septo-optic dysplasia with visual impairments and brain tumor survivors who may lack some challenges... Or lack some ability for independence, I just wanted to explore that with you in terms of what are the extra things in those populations that your system does to try and help improve success for them?
- [Mehul] So I think that is a very important area to cover, and we look after a large number of patients with septo-optic dysplasia and also hypopituitarism in isolation but with other learning difficulties and autism, and there is a very strong association between these conditions, so at transition, we're faced with the challenges of how do you try and achieve as much independence for these young people as you can, and yet keeping them safe and... With their treatments, essentially, so what we tend to do is try and time this over a number of years, the process begins at the age of around 13 years with us, and at Great Ormond Street Hospital, we look after children until the age of 16, maybe occasionally a little bit longer, but really by that stage we do need to transition to the adolescent service at UCLH, now, with that, they are often on multiple medications, they are visually impaired, a large proportion of them do have significant learning difficulties or autism, and so, it's really trying to individualize and personalize the approach to these children and young people, and one size is not gonna fit all, and we begin the process around about the age of 13 years, try and see if they can understand the condition, the need for the medication, try and see if you can try and get a reasonable amount of independence at that stage, some patients will actually do their own growth hormone injections, for instance, whereas others would be very reliant on their families, and I think one's gotta be sensitive to the dynamics within an individual family, 'cause these families are hard-pushed with a lot of things going on, the young people are often in quite a difficult place psychologically, and I think one needs to really take it very slowly, so we also try and move them to the adolescent clinic at UCLH where the clinical nurses there have the same experience as at Great Ormond Street, but obviously with older young people, and their role is extremely important in the whole process, in fact, my clinical nurse specialists know the young people and the families very well, both at GOSH, and then, when they move to UCLH. We often need to have a best interest meeting in the UK, I don't know whether you have a similar approach in the US, but it's... And that is really challenging because even a blood test in a young person over a certain age is gonna need to have a best interest meeting to make sure that you are doing it in the child's best... Or young person's best interest, so that is really a huge challenge for us and for the families, and it's difficult for the families because they now have to, you know, go through a number of legal processes for the... With these children, if they... The children can't care for themselves.
- [Brad] That is different in the States, it comes about closer to when they reach 18, so that's a interesting dynamic difference, and you've described a number of the principles of an amazing transition program that that you have there, and what I'd like to ask you is how does that structured transition protocol differ from informal transfer of care? You know, do you have some patients that are more on the informal side, or does everybody follow your program?
- [Mehul] So with isolated growth hormone deficiency the process can be a little bit simpler, and what we tend to do is retest most of these young people once they have finished their growth, and this will be once they have been moved to the adolescent center, because there, we do an insulin tolerance test, which we don't do at Great Ormond Street, for instance, so there are practical reasons why we need to transition them for the tests, and once they are there, then obviously, if they're still growth hormone deficient, we will recommence growth hormone after discussion with the young person, occasionally, they do want a period off the growth hormone treatment to see how they feel, and I think that that is absolutely acceptable provided that you keep a close eye on them with regular monitoring of the blood tests and the bone mineral density, and that they're fully aware of the pros and cons of treatment. If they then decide to go on growth hormone treatment, then you continue, and then, after the age of 18 years, I will transfer them directly to my adult endocrinology colleagues, and there's a range of them, so I just decide who would be the best for this young person. On the other hand, if a young person has hypopituitarism, then I follow the more structured transition pathway, and around about the age of 17, I will start seeing them with my adult endocrinology colleague, and there's one particular person who has an interest in hypopituitarism, and we do the clinics together, so we have around six clinics a year where we see these complex patients, and we'll see them either once if it's a relatively straightforward transition to adult services, but occasionally, the patients want a bit more time to get to know the adult services, so there are some that are transitioned, say, at the age of 20 years, 21 years and we'll see them together for a bit until everybody's happy and comfortable.
- [Brad] Yeah, I contrast this to our program, which we're accused, as pediatricians, of holding onto them forever and not wanting to let them go, and 18 is our typical time when we're discussing the transition with our patients, and the adult colleagues would actually prefer not to start seeing them until 21 in many circumstances, but we do have a regular transition meeting where we have conversations with the adult endocrinologist, but we're physically not located close to each other and insurance doesn't pay for us to both see them on the same day, so it kinda limits our transition possibilities, so we've worked a lot on trying to do it virtually and things of that nature, but I think these cores that you guys are doing really tell us how we should be trying to improve that process for our patients, whether it's GHD, panhypopit, or as you mentioned, type 1 diabetes and other chronic illnesses. So how does local implementation vary? You obviously have an amazing program at GOSH and UCL, what about other places in your country as well as across Europe?
- [Mehul] I think it's variable and there are some centers that will transition directly, I mean, simply because of the logistics as you've just said, but increasingly, there are a number of centers that actually link up with the adult services and either discuss the patients or... Virtually or whatever modality they have, or they will have joined clinics, as I've said, with us, but, you know, resources are tied everywhere, the adult endocrinologists are quite pushed, so it does vary hugely, and certainly within the UK, there's some centers that find it challenging to do this, and not all of my colleagues also have the facility to do the joint clinics with adult endocrinologists, so I'm fortunate because I have a contract at UCLH and it all works much better, but some of the others don't do that and they will transition directly, so even within one center, it can vary a little bit.
- [Brad] Okay, excellent. Back to the patients, how do you figure out for them when they're ready to make these transitions? And I know you mentioned your nursing team that works with them, you know, what are the processes that you use to get them ready for those different stages or assess their readiness for those transitions?
- [Mehul] So I think we know the families very well, we start the discussions very early on, and the assessment is a joint one. So clinical nurse specialists often sit in clinics with me with these complicated patients, and we start discussing with the families as to when they feel that they would be ready, and of course, the answer is often, "Never," so we have to get over that stumbling block and explain the whole process to them, so the whole process starts at around the age of 13, but very few are transitioned before 15, 16, and then, you know, the nurses will assess their readiness, if you like, in terms of understanding, in terms of likely independence, and a lot of young people will say to you that, "Actually, yes, we would like to move to an adolescent center where we don't have to sit in the waiting room with, you know, young people..." "Very young children" so yeah, and... You know, and I think at UCLH, they do see that it's a different environment. When we first started the process, I think a lot of the families were extremely anxious about the whole process, and we have a support group for septo-optic dysplasia and transition would often be the, you know, topic that was possibly the most controversial, and a lot of families would get very upset and angry about the process, over the years, they've realized that there is a structured process, that we do have an adult endocrinologist who's interested in the condition and is developing the appropriate expertise, and so, now, it's much smoother.
- [Brad] That's awesome. How often does it not go well? And what do you do in those circumstances where somebody makes the transition and bounces back? Or is that not an event anymore?
- [Mehul] So it's not an event anymore because once they do transition, it is difficult for them to then step back into pediatric care simply because of the logistics, but I sometimes... I do stay a little bit involved, and that's why, as I said, my colleague in adult endocrinology and I see them maybe for a few more years until they're absolutely comfortable, now, we have had a few that have just been lost to follow-up thereafter or they're moved to their local centers, which is also understandable, we ensure that they're in the right place locally in those circumstances, but the majority will stay with us and settle down, I mean, they have to get used to the fact that they are seen less frequently in the adult services than they will be seen in the pediatric services, so in the UK, that's very much a phenomenon. - [Brad] Yeah, the other thing that I hear from our patients is that there's less handholding in the adult services, if you forget to get your prescription or make your appointment, there aren't people chasing you down as much in the adult world as there are in pediatric world, and some of the patients really need that support, so those are really challenges that we have to navigate. So from a patient perspective, I just wanna focus in the last minute or so on why is that smooth and well-coordinated handover important for maintaining the continuity at the point of transfer? What do you see in terms of, and you highlighted it at the beginning of our conversation, of the outcomes if it's not done well?
- [Mehul] I mean, I think the transition process is critical because it's a time when we reassess the endocrinology, now, the adult endocrinologist will put me through my paces and say, "Well, how did you make that diagnosis of cortisol deficiency?" So it's a time when we go back and look over the records, and say, "Well, actually, these were the data," and sometimes, they will say, "Well, maybe we should retest them," and often, they retest them, and often, the hormone secretion is fine at that point, say, cortisol, growth hormone, usually, they are significantly deficient because of the sort of cohort we have, but the other hormones, they may be able to come off them, so I think it's a very, very important part of the process, that the two of you sit together, analyze this in detail, and then, work out the best way forward, I often switch a lot of my patients once they've finished going, for instance, to prednisolone instead of hydrocortisone, which gives them more autonomy, essentially, so again, transition is a time when you critically look at all that and make sure that they are on the right medication and the right preparation, and then, make sure that they're established under the care of the adult endocrinologists and not lost to follow-up, and I think that is the critical part of it.
- [Brad] Perfect. Well, thank you very much for your insights and look forward to more conversations.
- [Mehul] Thank you, Brad.
Practical transition protocols and models of care
Mehul Dattani, MD, Bradley S. Miller, MD, PhD
- [Brad] My name is Brad Miller, and I'm a pediatric endocrinologist from the University of Minnesota and M Health Masonic Children's Hospital in Minneapolis, Minnesota. The podcast series title is "Bridging GHD gaps: From complexity to continuity". The episode is "Practical transition protocols and models of care." And I'm joined by Mehul Dattani from Great Ormond Street. So Mehul, please introduce yourself.
- [Mehul] Hi, I'm Mehul Dattani. I'm a professor of pediatric and adolescent endocrinology based between Great Ormond Street Children's Hospital and the Institute of Child Health in London. And my pediatric practice is at Great Ormond Street Children's Hospital, whereas my adult adolescent practice is at University College London Hospitals. So at University College London Hospitals, we look after young people who are in the process of transition to adult care. And so there's a two-stage transition process from Great Ormond Street to UCLH and then to adult care over there.
- [Brad] Excellent. You know, so today we're gonna be talking about transition and how transition protocols can really impact that process. So how do you define your transition protocol and how does that practically affect the transition process? And we'll talk a little bit about how it differs in different places, but how did you guys develop yours and what are the key components of it?
- [Mehul] So the way we've developed our transition protocol is, at Great Ormond Street Hospital, we're supposed to start talking about transition and document that from the age of 12 to 13 years. And there's some key questions at that stage about capacity and readiness, et cetera. And then we are reviewing that every six months thereafter. So my personal practice is a two-stage transition, as I've said. So first stage is from pediatrics to adolescence, and then the second stage to adult care when I often see the patients jointly in specific transition clinics with my adult endocrinology colleague. Now, through the whole process, this is a multidisciplinary process essentially. So our clinical nurse specialists work very closely with us. They know the families even better than I do, and they will listen to the concerns of the families, and they will then gauge the right time for transition to University College London Hospital, for instance. And when I'm there, we have an adolescent clinical nurse specialist team who also get to know the patients very well. And in fact, before the patients are transitioned to UCLH, both sets of nurses have joint meetings about each patient that is being transitioned. - Excellent. - So there's a good handover.
- [Brad] No, that's excellent. How is that tracked in the electronic medical record? Do you have a transition pathway in your system to say we've met this milestone and beyond?
- [Mehul] Yeah, so we are working on that at the moment. So that's already started, but we're just refining it a little bit. But yes, so we do have that, which clinical nurse specialists usually do. They're amazing.
- [Brad] So as you're seeing the patient, you can see that those components have been already addressed as part of your evaluation and you can bring that up with the families at the different points to say, here's where we are, here are the things that we're concerned about. How does that change your care of the patients as they get older? We talked a little bit about patients giving their own injections, patients becoming more independent. Are those things that you're stressing as the child is getting closer to transition?
- [Mehul] Oh, yes. I mean, I think we try and encourage as much autonomy as possible from the ages about 14, 15 years. So, you know, I would expect them to know a little bit about their medications, about their conditions. So they often have sessions with our clinical nurse specialist where they can go over the condition itself and ask relevant questions. The parents obviously know about the condition pretty well and they will also then talk to the young people. We give them all the literature that we have created for the patient so that they can go and read about it. They have open access, so we have Epic, and they can question us through Epic on any matters that they need to. And our nurses are brilliant. They will answer what they can, and then they'll come back to me if there any questions that they can't. So there's a dialogue that's very open. And really, once we have a degree of independence, and once we feel that they will be comfortable in the adolescent setting, then that is a time we feel that they should transition out to University College London Hospitals. And we will, you know, be sensitive about that and explain the pros and cons of the process. And most young people are quite ready by then and will appreciate really the difference in setting in the adolescent unit as compared to the pediatric unit.
- [Brad] One of the things that our patients also we talk about them with is scheduling their own appointments and having some of the meeting without their parents present. So is that a component of your system as well? - [Mehul] So we start asking them if they want time on their own with us, but the parents are usually included in the consultations at the start. That's at Great Ormond Street. When we then move them to University College London Hospital, we start talking to the patient directly, and again, we'll offer them an opportunity to talk to us on their own. And my adult colleague will then explain to them that when they move to the adult endocrinology care, their parents will be excluded at the start of the consultation and brought in if the young person so desires. So they know that there will be that change as they move through the pathway really.
- [Brad] Yeah. So we've talked about some of the core elements that your team has in place, the education that you and the nurse specialists are doing, the readiness assessment, the documentation, the timelines. Are there any pieces that we're missing in that conversation of things that you think are key to the components of your transition?
- [Mehul] So I guess the confounder really is when a young person is under several different specialties at Great Ormond Street, for instance, and we do have a lot of complex young people with rare genetic conditions, et cetera. And then you have to be sensitive to the families because obviously if one component of the service is at University College London Hospital and the rest is all at GOSH, then that is difficult for the families. So I think that is one issue that sometimes holds back transition. And again, we will discuss it with the families and say, you know, "What do you feel?" And they will often say, "Can we just stay here for a little bit longer until we get more of the other parts sorted and transitioned as well?" So there is a little bit of flexibility in the system, and you usually have to then account to the managers and say, this is the reason why we're keeping this young person here, because we have the CQC, Care Quality Commission. And essentially they will come back to us and say, "Why are you holding onto young people too late?" So, you know, we are monitored very carefully, so we have to justify those actions. The other advantage, I guess, is that we have Epic at both sites, UCLH and at Great Ormond Street. So actually we can look at the notes across, although it's not as good as I'd like at the moment, but I think people are working on better communication really.
- [Brad] Excellent. So, we've talked about your model, and I know there are different models in different locations and the United States has a number of different models that depend upon the location and the resources as well. So when you talk about pediatric and adult joint clinics, shared care pathways, dedicated transition coordinators, what are things that other centers, if they can't find your system, should focus on to try and mimic or duplicate what you're doing?
- [Mehul] I mean, I think it obviously comes down to resource in the first instance and obviously time for particularly with the adult endocrinologist but also the pediatricians. Both have to make time to make sure that the process is smooth. And yes, it may be difficult if there is no sort of co-location of pediatric and adult services, and that is, you know, the case in many centers in the country, yet we have that issue as well. I mean, Great Ormond Street is a children's hospital, UCLH is about a mile away, which is not the end of the world, but you know, it is a little bit of away. So you have to make it work somehow. And I think these days with virtual sort of meetings, in fact that's what our CNS team do, the clinical nurse specialists. They will have virtual meetings with their counterparts at UCLH and ensure that all the patients are discussed appropriately. We've also now started to get together with the adult endocrinologists for a number of different conditions, such as CAH for example, and hypopituitarism is next on the list, where we have joint meetings and we bring their difficult patients, for instance, patients who are gonna be a real challenge to transition, to say, "Okay, how best can we help this family move across?" So there's also that avenue now that's opening up for us, but it needs effort and I think, you know, in the patient's interest, I think you just have to go that extra mile.
- [Brad] Yeah. Dedicated transition coordinators, it sounds like your system has a process in place where that may not be necessary because you have specific individuals with those duties. Our center has explored that as dedicated transition coordinators to work, just as you mentioned, particularly when the young individual has multiple different sub-specialists, a cardiologist, a endocrinologist, a nephrologist. We've also have explored that just for any child that is transitioning with a chronic condition, whether it's endocrine or cystic fibrosis or others. So how does that get incorporated at your center, if at all?
- [Mehul] So that is a bit of a challenge because we have to directly communicate with the different teams to find out when the patients are being transitioned. But again, I guess we don't have a formal transition coordinator, but our clinical nurse specialists act as transition coordinators. So as I've said, they will drive the process. They will discuss with the other clinical nurse specialists in the other team, say cardiology or respiratory, et cetera, and then work out what's happening, and then we can sort of come to some sort of decision. But the problem at the moment, it is that different teams will transition at different times, and it's really, that is a real challenge that we need to get on top of. The hospital now is very aware of that, and there is a whole move to sort of coordinate transition across specialty, so I think it will come.
- [Brad] Yeah. One of the things you mentioned earlier was the retesting of individuals as they get close to transition or after transition to make sure what endocrine needs they have. And that gets to the question of what are the key points that we as pediatric endocrinologists should be documenting and providing for our adolescent or adult endocrinology colleagues? So what are the pieces that we need to go back into our records and find and highlight for our partners in care?
- [Mehul] I think that's a very important question, and I think the answer to that must be that it's a lot of work, but you actually have to go back to the beginning and you have to go back to why that child was started on growth hormone, why that child was started on hydrocortisone and levothyroxine as well. And obviously puberty will be better documented because it is, you know, the time of transition anyway. But all of these other hormones you do need to go back and see because sometimes the evidence may not be as clear as to the need for the relevant medication. And you do need to look at the notes and try and understand why the clinician who started the medication at that point, that may not be you, did so in the first instance, okay? And so we go back with the data. And obviously Epic came in in 2019 just before COVID with us, so all the records before that are somewhere else. We can access them, but they're not perfect, and sometimes you just need to go through the lab records, which are there. So it is a lot of work, a high-level intensity, but it is so helpful because as I say, you know, I will be questioned by the adult endocrinologist who sits with me and said, "Okay, why did you make that decision?" "Are you sure that this person needs it at this particular time or should we retest?" And so if we have that information, we say, "Well, this is why we started it, okay?" And yes, there is a need for retest for some of the hormones, there's no doubt about it. And we have indeed found that the results have been normal in, you know, late adolescence, early adulthood.
- [Brad] No, I think that's key. The transition process that I've followed here in our system and my partners have as well is the term we like to use is wrap them up in a bow and have them ready when they're ready to transition. So essentially documenting what we've done, retesting if it's needed, and having them, particularly because not all of our adult centers have the capability of doing the dynamic testing that may be necessary. And so particularly if they're not at an academic center, that becomes a challenge. So the information that we include in that high-quality transfer, is that just part of your standard note or do you actually prepare a specific transition document that goes with the patient or is added to as the transition process grows?
- [Mehul] So what I do is then try and make sure that that last report when I see the patient for the last time at Great Ormond Street is very comprehensive and includes, you know, the original sort of diagnosis, the MRI for instance, the data on the biochemistry if I can find it from the very early on. Genetic data if I've got that as well. So everything will hopefully be in there and move to UCLH with the patient essentially.
- [Brad] When they make that transition, we've talked earlier about the challenges of transition in specific patient populations. What are some of the warning signs that you see in patients or characteristics of patients that are gonna be more difficult to transition?
- [Mehul] So first thing is I think once they've finished their growth, sometimes they'll say they will carry on with growth hormone treatment if they're severely deficient. But then you know that the prescriptions are not being asked for in a timely manner. So you realize that actually there's a problem there, okay? And I mean, my biggest worry I have to say is with things like hydrocortisone and thyroxine. Thyroxine is easy enough. You pick it up on the blood tests and you know that they're not taking it. And you can confront them and go through the reasons why they're not taking it and help them understand the need for it. Hydrocortisone is a little bit less obvious. And, you know, I have had people then going into crisis, et cetera, adrenal crisis and then they realize that actually they do need it. And that's why, I mean, I proactively, once they've finished their growth, discuss sort of prednisolone with them. There's also Efmody, which is becoming available. So we discuss these options with them and see if they would be preferred. But we engage with them the whole time, try and engage with them as much as we can.
- [Brad] In the last minute or so, I wanna just examine the ongoing feedback that you and your adult team have to improve your process. So what does that look like at your center in terms of meetings amongst the teams to say, how are we doing this? How can we do this better?
- [Mehul] So the adult endocrinologists and I obviously meet regularly, so we do discuss the transition. And are there any particular problems with any patients that have been raised? We haven't as yet got a formal feedback process for the patients, and that's what we are working on at the moment, and that's what we need to do really to try and improve the process. We also listen obviously to our clinical nurse specialists because they will have a very good idea of what's working and what isn't for specific patients. And you know, I do sometimes hear from some of my patients, say, "Well, this doesn't work well." And then I go back and say to the adult endocrinologist, well, they've expressed this concern, you know, that they haven't been seen in a timely manner or whatever. And I know you're busy, but you need to prioritize that. So I think sometimes you do do that, but the patients often have that relationship with you over several years and they feel comfortable to come back to you and say, actually can you help with this? So that does happen.
- [Brad] If you had advice for somebody trying to develop a program in their area, what would be the key steps that you would say to improve this process or set up a new process in the location that they're at?
- [Mehul] So if possible, dialogue with the adult endocrinologist at a very early stage really, and then you work out the process that fits both of you the best. And, you know, I really value the joint consultations because I learn a lot from the adult endocrinologist. They learn a lot from us if they don't know much about growth, et cetera. And they admit it. And obviously, I don't know about a lot of the things that they do. For instance, they will give prednisolone only once a day, and, you know, I balk at that and they say, no, it's fine. You don't need to worry about it. They're all fine. So you know, it's different processes and you need to understand each other. So that's the first step. And then the second thing is obviously bringing your team on board. And my team actually are much more advanced than me in thinking about transition. My nurses will come up and say, "What do you think? Ready now? We think they're ready." And then that is a hint saying, get on with it, you know. So I think all of those processes would be helpful. If they haven't got access to an adult endocrinologist locally, then obviously that is a little bit more challenging. But then they need to find someone, you know, within reasonable distance that they can communicate with electronically or, you know, virtually, however.
- [Brad] Sounds good.
- [Mehul] But those are the steps really that you would need to start off with.
- [Brad] Excellent. Well, I appreciate your time. This was a great conversation about how we can improve the transition process for our patients. So thank you very much.
- [Mehul] Thank you, Brad. It's a pleasure.
Barriers to GH therapy adherence across age groups – the role of digital support tools
Bradley S. Miller, MD, PhD, Paul Dimitri, BSc, MBChB, FRCPCH, PhD
- Welcome to the podcast entitled "Bridging GHD gaps: From complexity to continuity" from Medthority for Healthcare Providers. My name is Brad Miller and I'm a pediatric endocrinologist at the University of Minnesota Medical School and M Health Fairview Masonic Children's Hospital. Joining me today is Paul Dimitri and would love to get the discussions started.
- Thanks very much, Brad. So my name is Paul Dmitri. I'm professor of Child Health Technology at the University of Sheffield in the UK and I work as a pediatric endocrinologist at Sheffield Children's Hospital.
- Well, I'd like to start the conversation today to talk about the primary barriers to growth hormone therapy adherence across different age groups, and get your perspectives on the challenges at different ages.
- Yeah, thanks very much for the question. It's an interesting one, and it's fair to say that children and adolescents miss growth hormone doses for reasons that are far more developmental and behavioral than being deliberate. What you tend to find in the older group is routine shift with school transitions, social activities, increasing autonomy, adherence naturally becomes harder. Younger children depend entirely on adults to structure their health behaviors, whilst adolescents often test boundaries. They prioritize peer identity, spontaneity and privacy over medical routines. They've got emotional factors, things like needle anxiety, treatment fatigue, or a desire to feel normal also play a fundamental role, and these developmental dynamics mean that missed doses are really a sign of refusal. They're a predictable outcome of growing up and they highlight the need for age-responsive support and technology that adapts to developmental change. So, for the younger children, adherence is shaped almost entirely by the ecosystem around them. So their caregivers, they juggle work, childcare for other siblings, household pressures and unpredictable daily routines, all of which have the potential to influence whether injections happen consistently or happen randomly, and, you know, for those of us that are parents, that evening chaos, shared caregiving responsibilities or a parent who might work late, can easily disrupt dosing. And then there are other factors that play a role as well. So there are families that experience internal or external stress, they have low health literacy or potentially if they're using digital tools, low digital literacy, or they have limited support. They face even greater challenges, and so in this context, adherence becomes a family behavior, not an individual one. So tools that provide predictable routines, something that gives the caregiver a prompt, a digital tracking tool, that can transform continuity of care by essentially reducing the cognitive load and helping families maintain stability even when life is busy or unpredictable.
- Just as a follow up to that, do you think there are specific barriers because it's growth hormone versus other things that might be changing the dynamic?
- Yeah, it's an interesting question. I think it's possibly not necessarily unique to growth hormone, but I would suspect growth hormone falls into the challenge of taking a medication long term, so you have a medication that for some children, it's prescribed at a very early age and this is something that is potentially through the course of their, right their way through to their adolescence and even beyond that into adulthood. So both those internal factors and those external or extraneous factors then will challenge them at different points in their life as they grow up, as they develop autonomy. Then there's different factors challenge both those individuals and their families in different ways. - So how can digital tools be used to address these age-specific adherence challenges?
- Yeah, I think going back to those age-specific adherence challenges, they can smooth out, the digital tools can smooth out some of those adherence challenges by meeting children, adolescents, and young adults exactly where they are developmentally. So for younger children, and they're of course dependent on their families, the most effective features of those that will reduce that cognitive load and meet routines for those families and their children more predictable. So it's things within the digital tools, so well-timed reminders, visual countdowns, and then for the children themselves, character-based prompts that help them anticipate injections and reduce anxiety, but also eases the burden on busy caregivers. Then you've got gamification within digital tools, and I think this is fundamental, particularly for the young children. So, you know, things that have been used in the mainstream gaming world, like streaks, badges, progressive journeys, that can turn adherence into a shared family activity and more fun for the child rather than a nightly negotiation, which can occur with growth hormone therapy given that it's injectable. And then there's interactive content, so short animations, story-based explanations that will help children and young people understand and normalize treatment, especially when you pair that with reward-based routines that reinforce that consistency. And then when children move into adolescence and early adult tools, adulthood, sorry, digital tools shift from supporting routines really then to supporting autonomy. So adolescents will benefit from the platforms that will offer personalized dashboard, self-management modules and private spaces to track symptoms, mood, or progress, so these are features or tools that reinforce ownership rather than dependence. And so, then as they move into adulthood, these digital platforms will then be used to scaffold the transition to adult care, by providing structured transition pathways and may give them tools such as appointment management, secure messaging that encourages them to take the lead but still while feeling supported. The young adults navigating independence, tools that integrate medication reminders with their academic work, if they're at university, their work or their social challenges alongside a decision-support prompt will help them to maintain continuity as their life becomes more complex, and it almost certainly will become more complex as they move into adulthood. So I think collectively, at those different age groups, children, adolescents moving into becoming young people and young adults, these digital systems will involve with the child. It supports families early on, it empowers the adolescents and then enables that confident independence as they move into adulthood.
- Great, so what types of digital technologies are currently being used to support adherence in growth hormone therapy?
- So, I think it's fair to say that digital adherence support in growth hormone therapy has really evolved over time and it's involving into what's become a powerful ecosystem of connected injection devices that are aligned with mobile apps and clinical dashboards, and each of them is essentially designed to reduce missed doses and make treatment more predictable. What the connected devices do is automatically record injections, then transmit data securely and will flag gaps in dosing patterns, giving families and clinicians a real-time view of adherence. And the mobile apps extend this offering, extend it by offering reminders and routine-building tools, dose logging, animations for younger children and progress visualizations that will help those young people understand the impact of consistency. And then clinician dashboards will bring all these data streams together. It will enable early identification of risk, it will support targeted conversations, which I think these platforms are set out to do. It really facilitates the conversations that I and others have with young people and families, and it gives that approach to proactive support rather than waiting for growth filtering to appear months later, and more challenging questions at that later date. So what these technologies are offering is clear strengths that allow us to acquire objective data, it reduces the caregiver burden and opportunities for earlier intervention and improve communication between families and clinical teams, but it is fair to say they do have some limitations as well. It's important to remember that not all families have equal digital access, so some will find apps overwhelming, they may disengage with digital systems over time, and of course, it's important to remember that connected devices will only work if data transmission is reliable and the ecosystem is easy for those families and young people to use. And then when you look at the clinical side, clinicians may face dashboard fatigue if systems are poorly integrated and that there's disengagement from adolescents and families where there's little information for those clinicians to use. So I think, when we think about the next generation of solutions, that's gonna have to address these gaps. It's gonna have to move towards personalized age-responsive digital support, but also to make sure there's seamless interoperability and the tools will adapt to the realities of family life and developmental changes so there's sustained engagement on both sides, from children, young people and their families, but also from healthcare professionals as well.
- You talked a little bit about this already, but how you integrate that into your own clinical practice and the routine clinical care is a key component, and so it's both directions, how the families use it and how we use it, and so how do you do that in your practice or how do you foresee doing that as things evolve?
- Yeah, it's a really important question that, because when you integrate digital adherence tools, the effective way to do it is to introduce them as part of a collaborative family-centered conversation, not as an add-on or an obligation to use them. And it's important to frame these tools as something that reduces workload for the families, rather than something that monitors them. And what that does is it helps to build trust from the outset. So it's important to demonstrate that the Apple device during the consultation, it's important to demonstrate it so that you're setting up together and agree how reminders and notifications will be used to support smoother adoption. So essentially, for younger children where caregivers need confidence in how a tool fits into daily routines and for adults who will respond better when digital tools are positioned to supporting independence, it's really important to engender that ownership rather than a mode of surveillance. So, in practical terms, you need clear, simple onboarding, ideally supported by healthcare professionals, not just one individual, but a team. So for example, nursing colleagues as well to ensure that families leave the clinic knowing exactly what the tool does, how it benefits them, and who they can contact if they have some problems as they start to utilize that tool, and then once that tool is in use, the digital data can enrich follow-up discussions, and what it does is shift the focus from guesswork of adherence to objective in real-time insight. And what clinicians can do is use that, those adherence trends to guide counseling, to explore barriers to adherence and tailor intervention. And what that might mean is adjusting routines, addressing emotional factors or planning additional support. And again, that's done by both the negotiation and also by collaboration with the family and the young people. Not a process by which we push them because we say we're watching them. And then there's using it in practical terms from a pharmacist perspective. So pharmacists could link that adherence data to prescription management and also if the teams have psychologists, they can interpret behavioral trends that signal stress or treatment fatigue, and intervene when appropriate. So when that whole multidisciplinary team engages with the same digital ecosystem, what it creates is a consistent message that the tool is part of routine care. It's not an optional extra, but it will strengthen continuity across appointments and all the life stages that I spoke about earlier.
- A key question about integration for clinicians is how do different systems work with your healthcare system and information platform, and what barriers do you see there?
- Yeah, so integration is really important, and this is one of the most challenging, but in terms of those digital tools, that has to be system-wide interoperability. These digital tools can't be set up to operate with one system, because there are multiple different digital platforms that exist within trust, through, you know, different electronic patient records as an example, and even in the UK we have multiple different electronic patient records, so that integration of digital tools with systems where you require the transfer of information is fundamental. What exists at the moment is actually a digital tool that is independent of that, and so it doesn't require that intraoperability with clinical systems, so that it can be used system-wide without creating that fragmentation. But what's important is also the operability with uploading information through either a mobile network or through the internet, and so there needs to be both a credible and sustainable means of uploading information so that we don't essentially push people away from using the digital tools 'cause they can't transfer the information and transmit it in a way that's usable. - Are there any emerging digital innovations that you think are going to change how people adhere or improve the adherence better?
- Yeah, I think it's fair to say, this is a rapidly-evolving field, so I think these digital adherence tools for growth hormone therapy are moving beyond, or will move beyond simple dose line towards predictive personalized and fully-integrated ecosystems. There's gonna be AI-driven analytics and they'll begin to identify early behavioral patterns that precede non-adherence, enabling proactive intervention rather than retrospective correction. Then there's digital phenotyping using passive data that we can gather environmentally, sleep, activity or mood that may help clinicians understand when life-stage pressures or emotional factors are affecting their routines. And then there's the opportunity for virtual coaching, adaptive reminders, conversational agents which are being explored to provide age-specific support while augmented-reality explainers and interactive-learning tools may help younger children understand treatment in more engaging ways. And as these innovations mature, evidence and validation is going to be fundamental. This includes accuracy, safety, usability and equity. That has to be demonstrated before integrating these tools into clinical pathways, and there are also regulatory expectations for artificial intelligence in relation to transparency, data governance and how these tools can be adopted. So, in terms of the next frontier, there's going to be, I think, a seamless integration in tools across an entire care ecosystem, and what that gives us is the opportunity to link patient-facing digital tools with electronic health records, pharmacy systems, and multidisciplinary teams, whatever digital platforms they're using, so that adherence insights flow automatically to the right professionals at the right time.
- Excellent, well, I think this has been a great conversation, and I'm looking forward to hearing more as the field evolves, and excited to see some of these digital tools in action to help us partner with our patients and our care teams to help our kids, so thank you very much.
- Yeah, thanks very much.
Adherence in long-acting GH therapy
Bradley S. Miller, MD, PhD, Paul Dimitri, BSc, MBChB, FRCPCH, PhD
- [Brad] Welcome to the podcast series titled, "Bridging GHD gaps: From complexity to continuity" from Medthority for Healthcare Professionals. My name is Brad Miller, and I'm a pediatric endocrinologist from the University of Minnesota and M Health Masonic Children's Hospital in Minneapolis, Minnesota. Our guest today is Paul Dmitri.
- [Paul] Thanks very much, Brad. It's a pleasure to be here. My name is Paul Dimitri. I am a professor of child health technology at the University of Sheffield, and I'm a consultant in pediatric endocrinology at Sheffield Children's Hospital in the UK.
- [Brad] Well, let's get started. I'd like to ask you, what are the essential components of effective long-term monitoring in patients receiving long-acting growth hormone therapy?
- [Paul] So in terms of effective long-term monitoring in those receiving long-acting growth hormone therapy, that seems to work best when clinicians combine the review of clinical and anthropometric parameters and they recognize a suboptimal response relatively quickly. So that will in include routine monitoring typically of growth velocity of height, SDS, so standard deviation score, and pubertal staging, but also assessments of wellbeing and treatment burden so that you understand not just about the anthropometric parameters, but also what the causative reasons for possibly disengagement is. And then we need to look at this in respect to biochemical markers such as IGF-1, to evaluate treatment effect. So combine this information will help clinicians to understand whether the therapy itself is achieving its intended outcomes. In terms of the monitoring intervals for that, that will, I think, vary by age and also complexity. So, younger children or those early treatment may need more frequent review while stable older children, adolescents or adults may be seen between longer intervals. And then thinking about transition, so the movement of young people from pediatric to adult care, that's going to be a period where structure monitoring plans and shared care coordination become especially important to maintain that continuity. So we need to think about that as an essential component of their effective long-term monitoring to ensure that we don't lose into follow-up. In terms of early signs of suboptimal response, that really should prompt a closer evaluation even when using long-acting formulations. So that may include slowing growth velocity, unexpected changes in IGF-1 trends or reports of increasing treatment fatigue. What clinical teams can then do is use those signals to explore barriers, adjust routines, or consider whether additional assessments are needed. And nurses, pharmacists, and multidisciplinary colleagues will play key roles in reinforcing monitoring, supporting those families with practical changes and ensuring that concerns that are raised between visits not just at the clinical visit, are acted upon as well. So I think ultimately when that monitoring is consistent and collaborative, long-acting growth hormone therapy can deliver its full benefit across childhood adolescents and into adult life.
- [Brad] When there are challenges with the adherence, how do you or your team members approach that with the families? What are the best ways to have that conversation?
- [Paul] Yeah, that's a really important question, and I get asked that quite frequently in multiple different settings, particularly where there are cultural differences as well, because I think it's often seen that when we're monitoring adherence that we're going to have a conversation that is essentially going to feel like it's punitive, that we're telling people off because they are not taking their medication. But I think it's fair to say that any of us during our lifetime that have taken medication, at some point, have been non-adherent to medication. And I flip this conversation the other way and say, actually, this is not about being punitive, but this is about supporting the journey on a particular medication to make sure that we understand the factors that are challenging with them. And it comes from a concept of non-adherence being usually part of what happens as a consequence of lifestyle factors as opposed to being a deliberate action. There's very rarely a deliberate action here to take medication. So it's really important to see this as the complexity of life and to support them in those different aspects of life at different ages to make sure that we help them through those challenging parts of that growth hormone journey.
- [Brad] How can you tell the difference between somebody not taking the medicine and the medicine just not working, or the wrong dose, or the wrong type of medicine? Yeah. - Yeah, I think that's an interesting question, and I think it becomes more challenging without objective measures of adherence, but usually in terms of the wrong dose. It may be that when they're taking the wrong dose over a shorter period of time by escalating the dose that you'll see a response to therapy. Whereas for those taking a non-adherent to therapy, as you start to escalate the dose, what you should essentially see in the majority of cases is a response, but actually you start to escalate the dose and you don't see a response, that should raise thoughts about the possibility of non-adherence rather than problems with not responding to a dose.
- [Brad] What are the safety parameters that you should be monitoring during long-acting growth hormone therapy, and how can early identification of issues support adherence and treatment continuity? We talked a little bit about that already.
- [Paul] So I think it's important that clinicians focus on a small set of core evidence-based parameters. And what they do is they use them consistently over time. So I mentioned this already in the previous question, IGF-1 trends, growth velocity, pubertal progression, and also general wellbeing that will help clinicians understand whether therapy is being tolerated as expected. And part of that safety monitoring is about monitoring its symptom tracking as well, because growth hormone has complications and those might manifest in terms of headaches to signify possible intracranial hypertension, gait changes where it might signify slipped femoral epiphysis or signs of edema. And what that should do is prompt closer clinical review or additional evaluation, especially in the early months of therapy or during rapid growth phase. But what's important to qualify in that basis, that those complications are quite rare and I don't see them very often in my clinical practice, but what's important is to know about them because early recognition ensures that the concerns are addressed before they disrupt treatment or actually would still disrupt health.
- [Brad] Do you think that proactive safety monitoring impacts adherence? Do you think that's part of the equation that tells families what we're monitoring for and how to continue being adherent?
- [Paul] Yeah, it's hugely important. I think proactive safety monitoring really plays a crucial role in supporting adherence and long-term continuity. So when families and young people feel that side effects have been taken seriously and monitored well and managed promptly, I think their confidence in treatment increases and anxiety decreases. And I go back to the principle of the multidisciplinary team. So nurses, pharmacists, and other colleagues can really help support this collaborative anticipatory approach that not only protects patient's safety, but engenders trust and reduces treatment interruptions. And what that will ultimately do is support sustained engagement right away throughout that treatment journey through childhood adolescence and transition into adult care.
- [Brad] Do you see the growth response being a positive impact on adherence by itself? - [Paul] Oh, without a doubt, and I've seen that before in my clinical practice. And I think if patients and their families can see the response visually through showing them the growth chart and their increased height, they're much more likely to stay engaged. And just anecdotally, one story of a young man that I looked after who was needle phobic and actually went through a lot of psychological support to take his growth hormone when he saw his height over time, that's what maintained his engagement long-term, that he was growing, and that's exactly what he wanted to do.
- [Brad] Yeah, that's excellent. We also noticed that once the growth starts to slow, that treatment fatigue seems to be kicking in where kids feel like I've already caught up. And if I keep taking it, it's not gonna make as much a difference. So I think that's a difficult time to kind of come in and say, "We're still seeing growth. We're not seeing any safety concerns. We need to keep going."
- [Paul] Yeah, Although one of the things I try and emphasize through the journey on regular points is that growth hormone is not just about growth. It's got other- - Completely.
- [Paul] And one of the things, a statement that I've made in the past is the problem with growth hormone is it was called growth hormone because it's assumed that's all it's about, but it's got so many other important systemic factors as well.
- [Brad] Completely agree. So from your experience, what strategies support sustained patient and caregiver engagement throughout long-acting growth hormone therapy?
- [Paul] So I think sustained engagement in long-acting growth hormone therapy is strongest when clinicians combine clear education and this anticipatory support alongside shared decision-making from the very beginning. If you set up realistic expectations, this helps families understand that monitoring those adjustments or long-term follow-up are just normal parts of therapy rather than signs of problems. And so it's important that we explain how growth velocity, IGF-1 trends, and wellbeing assessments are guiding our decisions. And what that essentially translates to is building trust and reducing anxiety. And gotta remember this is a family unit. It's parents or caregivers and their children, and they'll benefit from developmentally tailored education that has to evolve as the child grows. So to begin with, when children are younger, simple visual explanations. And then as they move through childhood into adolescence, we start to build on those discussions around autonomy. And then as they move to adult care, that transition focus guidance as they move into employment or higher education in terms of psychosocial and practical barriers, we've got to sort of take into consideration what you mentioned earlier, Brad, which was treatment fatigue, busy routines, caregiver stress, needle anxiety, or just things that are going on in life, things that are potentially common and predictable. So if we can address those proactively by normalizing difficulties, exploring routines in detail, and using insights to identify early signs of disengagement, we can start to work with the families collaboratively to support them in those challenging times. And what that shared decision-making does is it invites families and young people to help shape how they're followed up, how their care is delivered. And we can, as clinicians and healthcare professionals, develop personalized support strategies that helps them to increase ownership of their care and reduces that resistance that could be met if we took a much more didactic approach. And I come back to that multidisciplinary approach again, that it's not just clinicians, it's also about the wider team to reinforce consistent messages, but also to maintain continuity. And so collaboration, I've mentioned collaboration a number of times. It's important that that collaboration is there, that it's personalized and it's responsive to real-world pressures to accept that long-term adherence can be challenging, but would become more sustainable if they have that wraparound collaborative support that they need at different parts of their journey and their life.
- [Brad] You mentioned psychosocial and practical barriers a minute ago, and cultural as well. How can we address some of those challenges? We have kids that are taking multiple different medicines. We have parents with separate households, we have language barriers and cultural barriers. What are some of the things that you found with your team that have been successful in those challenges?
- [Paul] Well, I think the first thing I'd say, Brad, is that you mentioned the word team, and I think that's the fundamental way of addressing these challenges. It's not one person that solves all those challenges, but that are team members with different skillset that will help to address those challenges. So I think taking a step back from looking at the team who should be involved in the team is to understand the nuances of those challenges that the young people or their caregivers or parents are being faced with. So it's about ultimately exploring the challenge first and then understanding the pathway to supporting those challenges. And some of them may be, as you say, psychosocial, some of them may be cultural, and it's about us as clinicians, but also our wider team working with the families to direct them to the right person. Or if we can't find the right person within the team, then we have to go outside the team to provide that additional support. And the nice thing about working in a hospital system is that whilst we work as a core team around the child with regards to pediatric endocrine and growth hormone care, that actually there are other members that we have in the hospital play therapists, those that work support in psychology or child adolescent mental health that they could access as well. But I think I go back to that word personalized, which is every issue has a personal slant to it. So we must make sure that we provide personalized and focused support within that multidisciplinary environment.
- [Brad] Do you find that long-acting growth hormone has any different challenges than daily growth hormone? Some people have said, "If I have to take it once a week, I'm more likely to forget it." Or, "Because of the flexibility of long-acting growth hormone, it's not so bad because I don't have to take it every day, every week on Sunday." Are there any nuances there?
- [Paul] It's a good question. I'm not certain that I'm seeing many nuances specific to growth hormone, long-acting growth hormone in relation to daily growth hormone. I think there is that balance between understanding whether adherence is better because you don't have to take something every day or whether you'll forget because it's not taken every day. And I think that will be very much a personalized issue. But one of the things we do in our own hospital is we give patients choice of their growth hormone therapy. And I think by empowering them with that choice, that will allow them to make the decision about what they want that fits in with their type of lifestyle. So we're already starting to address some of the potential challenges that the families or young people perceive by allowing them choice. So they may find that a weekly growth, I mean, for example, fits much better with their daily life through their routines because they have a much more challenging daily lifestyle so actually once a week is much easier for them. But I think it's important to remember with weekly growth hormone that it's not one injection. It's once a week. So, over time, it depends whether children, young people, and their families feel comfortable with that, with giving potentially two injections when they're there at the same time. So I think there are multiple factors to think about with both long-acting growth hormone therapy and also with daily growth hormone therapy. The important thing is to monitor these families and to support them to make sure that we start to pull out what challenges they have before they translate into a clinically negative effect.
- [Brad] And I think you really emphasized nicely the importance of the team and how all different parts of the team can help us with better outcomes for our patients. So thank you very much. That ends today's session and thank you very much Dr. Dimitri. This was a excellent conversation.
- [Paul] Thanks very much for your time, Brad. It's been a pleasure speaking to you.
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This content has been independently developed by Medthority with educational funding from Pfizer, to support healthcare professionals with access to the highest quality medical and scientific information.