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Effective GH Replacement With Somapacitan in Children With GHD: REAL4 2-year Results and After Switch From Daily GH

Authors
 Bradley S Miller  ;  Joanne C Blair  ;  Michael Højby Rasmussen  ;  Aristides Maniatis  ;  Jun Mori  ;  Volker Böttcher  ;  Ho-Seong Kim  ;  Rikke Beck Bang  ;  Michel Polak  ;  Reiko Horikawa 
Citation
 JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM, Vol.108(12) : 3090-3099, 2023-11 
Journal Title
JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM
ISSN
 0021-972X 
Issue Date
2023-11
MeSH
Body Height ; Child ; Dwarfism, Pituitary* / drug therapy ; Growth Disorders / drug therapy ; Growth Hormone / therapeutic use ; Human Growth Hormone* / adverse effects ; Humans ; Insulin-Like Growth Factor I
Keywords
growth hormone ; growth hormone deficiency ; growth hormone replacement therapy ; long-acting growth hormone ; somapacitan
Abstract
Context Somapacitan is a long-acting GH derivative for treatment of GH deficiency (GHD). Objective Evaluate the efficacy and tolerability of somapacitan in children with GHD after 2 years of treatment and after the switch from daily GH. Design A randomized, multinational, open-labelled, controlled parallel group phase 3 trial, comprising a 52-week main phase and 3-year safety extension (NCT03811535). Setting Eighty-five sites across 20 countries. Patients A total of 200 treatment-naive prepubertal patients were randomized and exposed; 194 completed the 2-year period. Interventions Patients were randomized 2:1 to somapacitan (0.16 mg/kg/wk) or daily GH (0.034 mg/kg/d) during the first year, after which all patients received somapacitan 0.16 mg/kg/wk. Main outcome measures Height velocity (HV; cm/year) at week 104. Additional assessments included HV SD score (SDS), height SDS, IGF-I SDS, and observer-reported outcomes. Results HV was sustained in both groups between 52 and 104 weeks. At week 104, mean (SD) for HV between weeks 52 and 104 was 8.4 (1.5) cm/year after continuous somapacitan treatment and 8.7 (1.8) cm/year after 1 year of somapacitan treatment following switch from daily GH. Secondary height-related endpoints also supported sustained growth. Mean IGF-I SDS during year 2 was similar between groups and within normal range (-2 to +2). Somapacitan was well tolerated, with no safety or tolerability issues identified. GH patient preference questionnaire results show that most patients and their caregivers (90%) who switched treatment at year 2 preferred once-weekly somapacitan over daily GH treatment. Conclusions Somapacitan in children with GHD showed sustained efficacy and tolerability for 2 years, and after switching from daily GH. Patients/caregivers switching from daily GH expressed a preference for somapacitan.
Files in This Item:
T992023040.pdf Download
DOI
10.1210/clinem/dgad394
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Pediatrics (소아과학교실) > 1. Journal Papers
Yonsei Authors
Kim, Ho Seong(김호성) ORCID logo https://orcid.org/0000-0003-1135-099X
URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/199305
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