SAT643 Patient Preference Questionnaire Response After Switching To Somapacitan After Taking Daily Gh: 2-year Results From REAL4

Abstract

Abstract Disclosure: J. Blair: Advisory Board Member; Self; Novo Nordisk. Speaker; Self; Novo Nordisk, Ipsen. Other; Self; Novo Nordisk. B.S. Miller: Consulting Fee; Self; Abbvie, Bristol-Myers Squibb, Novo Nordisk, Pfizer, Inc., EMD Serono, Endo Pharmaceuticals. Grant Recipient; Self; Alexion Pharmaceuticals, Inc., Abbvie, Amgen Inc, Novo Nordisk, Pfizer, Inc. M. Hojby: Employee; Self; Novo Nordisk. Stock Owner; Self; Novo Nordisk. A.K. Maniatis: Research Investigator; Self; Novo Nordisk, Pfizer, Inc. J. Mori: Advisory Board Member; Self; Novo Nordisk. Speaker; Self; Novo Nordisk, Pfizer, Inc. V. Boettcher: Advisory Board Member; Self; Merck. Speaker; Self; Novo Nordisk, Merck. Other; Self; Ferring Pharmaceuticals, Lilly USA, LLC, Merck, Novo Nordisk. H. Kim: None. R. Beck Bang: Employee; Self; Novo Nordisk. Stock Owner; Self; Novo Nordisk. L. De Fries Jensen: Employee; Self; Novo Nordisk. Stock Owner; Self; Novo Nordisk. M. Polak: Advisory Board Member; Self; Ipsen, Novo Nordisk, Pfizer, Inc. Grant Recipient; Self; Ipsen, Novo Nordisk, Pfizer, Inc., Sandoz, Sanofi, Merck. Speaker; Self; Novo Nordisk, Ipsen. R. Horikawa: Advisory Board Member; Self; Novo Nordisk, Pfizer, Inc. Grant Recipient; Self; Sandoz. Speaker; Self; Novo Nordisk, Pfizer, Inc. Growth hormone (GH) replacement therapy in children requires daily subcutaneous (s.c.) injections. Although beneficial to growth and other health related outcomes, daily injections can be burdensome for patients and their caregivers. This is acutely apparent in pediatric patients, such as children with GH deficiency (GHD), where both the child and parent/caregiver’s lifestyle are affected. Indeed, concerns have been raised that this burden is linked to lower adherence rates for daily GH treatment and a subsequent reduction in the efficacy of treatment. Somapacitan is a long-acting reversible albumin-binding human GH derivative in development for once-weekly s.c. administration in children with GHD. REAL4 is an ongoing multi-national, randomized, open labelled phase 3 trial consisting of a 52-week main phase and three-year extension period (NCT03811535). During the main phase, results showed that once-weekly s.c. somapacitan (0.16 mg/kg/week) has a similar efficacy and safety profile compared to daily GH (0.034 mg/kg/day Norditropin®) treatment (non-inferiority confirmed). Following the main phase, patients who had been taking daily GH (n=68) switched to once-weekly s.c. somapacitan. After 4 weeks (week 56), the parents/caregivers of these patients were asked to complete a patient preference questionnaire. Results from this questionnaire are presented here. The first question asked was: Which treatment do you prefer? Responses showed 45/50 (90%) preferred once-weekly somapacitan, 5/50 (10%) had no preference, and no respondents preferred daily GH treatment. Of the 45 respondents who preferred the somapacitan treatment regime, 38 (84.4%) ‘very strongly’ or ‘strongly’ preferred it, while 7 (15.6%) had a not very strong preference. Among the reasons respondents preferred somapacitan were: number of times needing to do injections (27/45; 60%); less worried about remembering to do injections (21/45; 46.7%); and child less worried about getting injections and child less annoyed about getting injections (both 15/45; 33.3%), as well as others. Finally, when asked which treatment they would be most adherent to, most (35/45; 77.8%) answered that they would be more adherent to once-weekly somapacitan compared to the daily GH treatment regime. In conclusion, these results indicate that once-weekly somapacitan treatment is preferred by parents/caregivers with a child that has switched from daily GH. The main reason for this preference being a reduction in the number of injections needed. Notably, these respondents believe they will be more adherent to GH replacement therapy with once-weekly somapacitan. Presentation: Saturday, June 17, 2023