Somapacitan

Somapacitan is a once-weekly GH treatment that has shown efficacy and safety profiles equivalent to daily GH in children with GH deficiency (GHD).

To investigate long-term safety, efficacy, and treatment burden associated with somapacitan after 7 years (364 weeks) of treatment.

REAL 3 (NCT02616562) was a phase 2, randomized, open-label trial investigating the efficacy and safety of somapacitan vs daily GH in children with GHD. After 156 weeks, participants entered a 208-week safety extension. Children in cohort I (age 2.5-10.0 years) previously completed 3 years in the trial; cohort II (age <2.5 years) and cohort III (age 9.0-17.0 years) entered the trial at week 156 for safety-only assessment. All participants received somapacitan 0.16 mg/kg/week. Height velocity (HV), HV SD score (SDS), height SDS, IGF-I SDS, incidence of adverse events (AEs), and treatment burden were assessed.

Overall, 43 participants (73%) in cohort I, 1 participant (100%) in cohort II, and 11 participants (69%) in cohort III completed the 208-week safety extension. Consistent increments in HV and HV SDS were seen in cohort I, and height SDSs at week 364 were close to 0. The incidence of AEs was aligned with previous investigations across all cohorts; 3 serious AEs in cohort I were considered probably/possibly related to treatment. Treatment burden was reduced in children who switched from daily GH to somapacitan.

After 7 years of treatment, children with GHD receiving somapacitan experienced consistent increases in height SDS and reduced treatment burden. No new safety concerns were identified.

Craniopharyngiomas are frequently diagnosed during childhood and adolescence, crucial periods for physical and psychosocial development. Despite improvements in survival rates, patients with childhood-onset craniopharyngioma face a wide array of lifelong complications, which emerge or worsen during adolescence, complicating the transition to adult care. Nevertheless, the transition age (15-25 years) remains an understudied phase in clinical practice. This narrative review synthesises current literature on the endocrine, neurocognitive, and metabolic consequences of transition-age childhood-onset craniopharyngiomas, providing a practical clinical approach to their diagnosis and management, along with an overview of emerging treatment strategies. Childhood-onset craniopharyngiomas are frequently associated with pituitary hormone deficiencies, which typically worsen post-treatment. While replacement protocols largely mirror those for adult patients, particular emphasis should be placed on patient education and optimal timing of treatment, especially regarding puberty induction and growth hormone replacement. Emerging therapies, such as long-acting growth hormone and modified-release hydrocortisone, should be considered to improve compliance. Hypothalamic dysfunction, both pre- and post-treatment, can lead to obesity, sleep disorders, and cognitive impairment. GLP-1 receptor agonists and melanocortin receptor agonists have recently shown promise in managing hypothalamic obesity. Sleep disturbances and cognitive impairment, often overlooked in clinical practice, should be systematically assessed in patients with hypothalamic involvement. Cardiovascular and bone health complications should be proactively addressed to improve long-term outcomes. Childhood-onset craniopharyngioma survivors require multidisciplinary care, particularly during the transition to adulthood. Timely endocrine management, individualised treatment strategies, and emerging targeted therapies are crucial for optimising quality of life and metabolic and neurocognitive outcomes.