Weekly somapacitan in GH deficiency : 4-year efficacy, safety, and treatment/disease burden results from REAL 3

Sävendahl, Lars (avtor), Battelino, Tadej (avtor), Højby Rasmussen, Michael (avtor), Brod, Meryl (avtor), Röhrich, Sebastian (avtor), Saenger, Paul (avtor), Horikawa, Reiko (avtor)

Context: Growth hormone deficiency (GHD) in children is currently treated with daily injections of GH, which can be burdensome for patients and their parents/guardians. Somapacitan is a GH derivative in development for once-weekly treatment of GHD. Objective: This work aimed to assess the efficacy and safety of somapacitan, and associated disease/treatment burden, after 4 years of treatment and 1 year after switching to somapacitan from daily GH. Methods: This long-term safety extension of a multicenter, controlled phase 2 trial (NCT02616562) took place at 29 sites in 11 countries. Patients were prepubertal, GH-naive children with GHD. Fifty patients completed 4 years of treatment. Patients in the pooled group received somapacitan (0.04, 0.08, 0.16 mg/kg/week) for 1 year, followed by the highest dose (0.16 mg/kg/week) for 3 years. Patients in the switched group received daily GH 0.034 mg/kg/day for 3 years, then somapacitan 0.16 mg/kg/week for 1 year. Main outcome measures were height velocity (HV), change from baseline in HV SD score (SDS), change from baseline in height SDS, disease burden, and treatment burden for patients and parents/guardians. Results: Changes from baseline in HV and HV SDS were similar and as expected in both groups. Observer-reported outcomes showed that patients and parents/guardians seem to have experienced a reduced treatment burden when switching from daily GH to somapacitan. Most parents/guardians (81.8%) strongly/very strongly preferred somapacitan over daily GH. Conclusions: Somapacitan showed similar efficacy and safety in patients who continued somapacitan treatment and those who switched from daily GH to somapacitan. Once-weekly injections may lead to a reduced treatment burden relative to once-daily injections. A plain-language summary of this work is available.

• Jezik: Angleški jezik
• Ključne besede: childhood growth hormone deficiency, growth hormone treatment, daily growth hormone, somapacitan, long-acting growth hormone
• Vrsta gradiva: Članek v reviji
• Tipologija: 1.01 - Izvirni znanstveni članek
• Organizacija: MF - Medicinska fakulteta
• Status publikacije: Objavljeno
• Različica publikacije: Objavljena publikacija
• Leto izida: 2023
• Št. strani: Str. 2569–2578
• Številčenje: Vol. 108, iss. 10
• PID: 20.500.12556/RUL-152415
• UDK: 616-053.2
• ISSN pri članku: 0021-972X
• DOI: 10.1210/clinem/dgad183
• COBISS.SI-ID: 154830083
• Datum objave v RUL: 24.11.2023

Gradivo je del revije

• Naslov: The journal of clinical endocrinology & metabolism
• Skrajšan naslov: J. clin. endocrinol. metab.
• Založnik: Oxford University Press, Endocrine Society
• ISSN: 0021-972X
• COBISS.SI-ID: 60951

Licence

• Licenca: CC BY-NC-ND 4.0, Creative Commons Priznanje avtorstva-Nekomercialno-Brez predelav 4.0 Mednarodna
• Povezava: http://creativecommons.org/licenses/by-nc-nd/4.0/deed.sl
• Opis: Najbolj omejujoča licenca Creative Commons. Uporabniki lahko prenesejo in delijo delo v nekomercialne namene in ga ne smejo uporabiti za nobene druge namene.

Projekti

• Financer: Drugi - Drug financer ali več financerjev
• Program financ.: Novo Nordisk