ESPE Abstracts (2022) 95 P2-147

ESPE2022 Poster Category 2 GH and IGFs (14 abstracts)

Perception of long-acting growth hormone- data from two surveys in Austria

Jelena Belic 1 , Stefan Riedl 2,3 & Manfred Windisch 1

1Pfizer Corporation Austria Gesellschaft M.B.H., Vienna, Austria; 2St Anna Children's Hospital, Medical University of Vienna, Vienna, Austria; 3Department of Pediatric Pulmonology, Allergology and Endocrinology, Medical University of Vienna, Vienna, Austria

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 childĀ“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.

Volume 95

60th Annual ESPE (ESPE 2022)

Rome, Italy
15 Sep 2022 - 17 Sep 2022

European Society for Paediatric Endocrinology 

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