Strategies for sustained engagement and adherence in long-acting GH therapy
- [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.
