ESPE Abstracts

Background: Due to its short half-life, conventional biosynthetic human growth hormone needs to be given daily which may represent a burden for patients and their families. Novel long-acting growth hormone (GH) formulations are under development and enable reduced injection frequency through once weekly administration. Two surveys were designed to collect first insights and expectations towards new long-acting formulations.

Methods: Between June and October 2021, two surveys were performed among pediatric endocrinologists and parents/caregivers of children receiving daily growth hormone treatment, addressing treatment experiences and attitudes/expectations towards long-acting GH formulations. In total, 20 pediatric endocrinologists and 41 parents/caregivers participated.

Results: Pediatric endocrinologists: Around half (55%) of the endocrinologists expected equivalent efficacy from long-acting GH formulations whereas only 10% expected improved efficacy as compared to daily injections. More than half of the respondents thought that only 70-90% of prescribed doses were actually administered and anticipated improvement of adherence from long-acting formulations (83%). Furthermore, 33% of physicians regarded these new formulations as an advantage for patients with needle phobia and the majority expected significant improvement in the quality of life. Nevertheless, around 50% had concerns regarding pharmacokinetics that might cause unphysiological IGF1 dynamics with implications for safety such as hitherto unknown long-term effects.

Patients/caregivers: Over 40% of patients/caregivers stated that their child was hurt most when excluded from activities because of her/his height although most reported no social or emotional problems. Besides that, a great confidence in growth hormone treatment and very high adherence rates (administration frequency 6.8 days per week) were reported. Overall, most respondents regarded a change in injection frequency from once daily to once weekly very positively, not least due to increase in flexibleness whereas some were concerned quitting the child´s routine with daily injections.

Conclusions: Both surveys showed that most pediatric endocrinologists and patients/caregivers were positive about long-acting GH formulations and expect significant improvement in quality of life. However, there are still some concerns regarding safety and efficacy which need to be evaluated in long-term studies.