Growth Hormone Deficiency Learning Zone
Transcript: Real-world evidence
Professor Mehul Dattani, Dr. Charlotte Höybye, Professor Steven Simoens, Dr. Shankar Kanumakala and Dr. Robert Murray
Interview recorded Jul 2023. All transcripts are created from interview footage and directly reflect the content of the interview at the time. The content is that of the speaker and is not adjusted by Medthority.
We'll move on to the next section which is "Real-world evidence of growth hormone treatment and patient/caregiver shared decision-making." So thank you very much Charlotte for your talk. Thank you for the introduction and here are my disclosures. And as we have already heard, there are several studies showing that growth hormone injections are effective in increasing height and height velocity as well as beneficial effects on body composition. But the individual outcomes are highly variable and the factors that affect treatment outcome are aetiology of growth hormone deficiency and the age when growth hormone treatment is started. So the effect is better in prepubescent children, and also the parental height and height SDS, and weight when growth hormone treatment is initiated are important. And daily growth hormone treatment has a very good safety profile. We have the well-known mild side effects with muscle and joint pain, and headache. And less frequent intracranial hypertension, slipped capital femoral epiphysis, and progressive scoliosis. Over time, there has been some safety concerns about impaired glucose metabolism and diabetes or neoplasm but the evidence is limited except in patients with pre-existing risk factors. Adherence as you have already said and heard this afternoon, adherence is very important to get the best outcome of growth hormone treatment. And treatment adherence varies very much across studies from 15 to 98%, and it is also known that adherence decline with time.
And in this study, it was shown that adherence decrease from 78.2% during the first year of treatment to 68.1% during the third year. And the factors that may reduce adherence include discomfort or pain with the injections, not being satisfied with the outcome, fear of injection or phobia, needle phobia, and interference with daily activities, not feeling involved in the treatment or being unwilling or not interested to continue treatment. Other challenges are limited access to certain growth hormone or devices due to funding or regulatory issues. It can also be the goals and expectations of patients and parents, and the burden of needing daily growth hormone treatment or injections. How can we improve the adherence? One way is to use a SHARE method which is a 5-step process with the aim to improve the patient and parents' knowledge of growth hormone deficiency, and to involve them in growth hormone treatment from start by sharing decisions and responsibility. And the S stands for Seek patient's participation. H to Help the patient decide on which devices are best. A to Assess patient's values and preferences.
R to Reach a decision with the patient. And E to Evaluate the patient's decision. Reducing the number of injections is another way and it has been shown that the weekly long-acting growth hormone preparations that Shankar told us about reduce treatment burden. It also improves the overall experience of growth hormone treatment and result in less interference with daily activities. It has also been shown that the effect and safety profile of the weekly injections are non-inferior or similar to daily growth hormone treatment. However, we need cost-benefit analysis and long-term studies of outcomes and safety so surveillance registers are suggested. So I'll continue with the discussion. Do you have any comments on this? Is it the adherence problem for you, you think? Yes, and I think adherence is, I was about to say, is something that until you've really gone into. We've all mentioned it a little bit but I think it is a very, very significant problem with real world, you know, growth hormone use. And I think we all underestimate it because we like to think we're doing a little bit better than we really are. And you know, there are these children where you go up and up higher and higher on the dose, and yet the responsiveness is not there. Just this morning, I was in with a patient consultation and they told me that they'd been given growth hormone on alternate days for three years without my realising. So, and the child was just about trickling along the bottom centile. You know, it is very difficult. You think you know it and you don't. I dunno whether others have similar experiences.
Shankar. Yeah. Can I ask a question that is sort of dual-edged? So children who are non-adherent, are not doing the injections for whatever reason. Number one, do you think long acting growth hormone would be an option or alternate option? And number two, considering that they are actually not using it appropriately, is there a rule to discontinue treatment for them because they are not using it? Is it a question to me? I think it would be worth just to check why they're not taking it as they're supposed to do and try to see if there's something that you can help them with to improve adherence. So can I bring choice in at this stage? Because for a number of years, we've sort of grown up on the dogma of giving patients choice to make sure that their adherence is improved. I personally think it's not as simple as that. And then we're gonna hear from Steven about economics. But you know, actually one has to think about it, you know, where there's almost, you know, a 50, 60% difference in price between products occasionally. And if you go by patient choice, is it worth that extra cost if they want to choose a more expensive medication? Are they really going to use it better? I don't know. So it'd be good to hear people's thoughts on that. Shankar. I mean, patient choice is important, but like all of us, our choices will also evolve over time. You might like pink colour now and so you want a pink pen now. But you might not want a pink pen after a while. So I think thinking about patient choice is very important, but I think recognising it is not one patient choice is the end of it is not the thing. I think within the system we need to have some degree of flexibility. There are... It is clunky in order to change from one growth hormone preparation to another one.
But I think we need to be aware that patient choices evolve over time and whatever they are very important to them may no longer be important as we go along in time. Yeah, I mean I think you are right. Things do change, their preferences will change. And I know that some people then, go back onto the web and say, "Well, we'd prefer this device now after a few years." And then as adolescents, the children will have more autonomy- Yeah. In what they want as well. So then again, that's a different time. Yeah. Rob, can I just ask you. How do you tackle the problems of adherence in adults who are severely GHD? Wow. I suppose it's less of a problem in adults and the adults are also a bit more honest, I suspect, about it. So because it's one of those things in the UK, we treat just for wellbeing. If the patients don't take it, they often just tell me and therefore we stop it. So because it's not one of those that's critical, I suspect we have more trouble with some of the other pituitary hormones where they are more critical and we need the patient to take them. But growth hormone, because it's always let's just try it for your wellbeing. If the patient doesn't see that, they stop it. And I'm quite happy with that. It's rare if it makes a big difference to the patient they would want to stop it, that adherence would be there. And I suspect that's in part, what I wonder with the children is. Are these the children who have already have a reasonable height and therefore don't see too much benefit from the growth hormone who are more likely to be non-adherent rather than a really short child? Not always. I mean, sometimes they are pretty short and they just don't wanna be bothered with injections. And that, then, makes it more challenging.
I mean if someone's done very well and they've got a reasonable height and then they wanna stop a little bit earlier, that doesn't worry us that much. But it's the ones that are quite short and then just decide they just don't want it. I mean all the disease about growth hormone deficiency round table, I had a set of patients who were one of the twin was SGA and the other twin was not an SGA. And the SGA twin became eligible for growth hormone based on the set criteria. And the parents actually... There was a height difference but the parents were really struggling and they made a final decision not to treat one child with growth hormone rather than go ahead with treatment. And I did follow that child for probably about four/five years in order to give them an opportunity if they want to change their mind as the child became more shorter and shorter. But the child continued to be a bit short but not not enough in terms of between the two twins. And they were very happy between the two twins that one child is not treated. I mean that was their choice. It was a difficult choice but that's what they stuck with. And in this scenario that the child did not become progressively shorter, but in GHD there will be a situation where the child is going to be progressively shorter. Yeah, I think that's right. That's right. SGA is a little bit... Yeah, if they don't want it, then you'd say well that's the choice. Yeah. Okay, I think we better wrap up and thank you again Charlotte for a very nice overview.
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