Treatment Burden of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Randomized Study

Abstract

Context Somatrogon is a long-acting recombinant human growth hormone treatment developed as a once-weekly treatment for pediatric patients with growth hormone deficiency (GHD). Objective Evaluate patient and caregiver perceptions of the treatment burden associated with the once-weekly somatrogon injection regimen vs a once-daily Somatropin injection regimen. Methods Pediatric patients (≥3 to <18 years) with GHD receiving once-daily somatropin at enrollment were randomized 1:1 to Sequence 1 (12 weeks of once-daily Somatropin, then 12 weeks of once-weekly somatrogon) or Sequence 2 (12 weeks of once-weekly somatrogon, then 12 weeks of once-daily Somatropin). Treatment burden was assessed using validated questionnaires completed by patients and caregivers. The primary endpoint was the difference in mean overall life interference (LI) total scores after each 12-week treatment period (somatrogon vs Somatropin), as assessed by questionnaires. Results Of 87 patients randomized to Sequence 1 (n = 43) or 2 (n = 44), 85 completed the study. Once-weekly somatrogon had a significantly lower treatment burden than once-daily Somatropin, based on mean overall LI total scores after somatrogon (8.63) vs Somatropin (24.13) treatment (mean difference –15.49; 2-sided 95% CI –19.71, –11.27; P < .0001). Once-weekly somatrogon was associated with greater convenience, higher satisfaction with treatment experience, and less LI. The incidence of treatment-emergent adverse events (TEAEs) for Somatropin and somatrogon was 44.2% and 54.0%, respectively. No severe or serious AEs were reported. Conclusion In pediatric patients with GHD, once-weekly somatrogon had a lower treatment burden and was associated with a more favorable treatment experience than once-daily Somatropin.

Treatment burden, adherence, and quality of life in children with daily GH treatment in France

“…This result is consistent with data from a previous discrete choice experiment study having showed that patients prefer a less frequent injection regimen for treating GHD ( 31 ). Our mean overall life interference total score related to daily injections of somatropin was consistent with the one found in the NCT03831880 trial (27.7 and 24.13), and three times higher than the mean score related to weekly injections of somatrogon (8.63, P < 0.0001 between groups) ( 17 ). As reported in other conditions, a reduced treatment burden may result in improvement treatment adherence ( 12 ).…”

“…After first results from a crossover phase III trial which showed that treatment burden of weekly injections of somatrogon was lower than that of somatropin administered once daily in children with GHD (NCT03831880 trial) ( 17 ), the real-world cross-sectional French QOLITHOR study, conducted in 275 pediatric patients including 166 children with GHD, provides complementary information on patient experience as recommended by French, European, and US Health Authorities ( 18 , 19 , 20 , 21 ).…”

“…Since treatment burden of the rhGH injection is often a shared experience between patient and caregiver, it was assessed using a Dyad Clinical Outcomes Assessment (DCOA) questionnaire specifically developed and validated prior to this trial to measure rhGH injection treatment burden, with a simultaneous assessment using a parent–child dyad (Life Interference Questionnaire for Growth Hormone Deficiency (LIQ-GHD)) ( 16 ). First results showed that treatment with somatrogon administered once-weekly improved the mean overall life interference total score (primary outcome) after 12 weeks of treatment compared to somatropin administered once-daily (mean respective overall life interference total scores: 8.63 and 24.13; difference between mean scores: –15.49; 95% CI: (–19.71, –11.27); P < 0.0001) ( 17 ).…”

Persistence with daily growth hormone among children and adolescents with growth hormone deficiency in the UK

“…Long-acting GH preparations (LAGH), injected once weekly as opposed to once daily, have recently been approved for use in the US, EU, Japan, Canada, and Australia and are believed to be a potential solution for improvements in patient adherence ( 18 ). Although no published data have emerged on the impact of LAGH on adherence from real-world use, a recently published clinical trial demonstrated that treatment burden, as evaluated by life interference, was significantly reduced when patients were treated with somatrogon once weekly compared to once daily Genotropin, and the overall treatment experience for somatrogon was preferred by patients and caregivers ( 19 ). Another recent study found a preference for LAGH and reduced treatment burden in patients that switched from daily somatropin to weekly lonapegsomatropin ( 20 ).…”

Long acting growth hormone (LAGH), an update

“…Factors contributing to poor adherence include the burden of daily injections, pain caused by injections, storage issues, the need to reconstitute solutions, and managing injections while traveling ( 6 , 12 , 13 ). There is, therefore, a need for GH therapies that require a reduced injection frequency, while still being effective and having a tolerable safety profile compared to daily hGH ( 14 ).…”