OBJECTIVES:

To conduct a responder analysis from data generated from a patient reported outcome, the Patient Life Interference (LI) questionnaire reported by patients with paediatric growth hormone deficiency (pGHD) in a phase 3 trial, to support and understand its interpretation.

METHODS:

A phase 3 (NCT03831880) open-label, crossover study, where non-naïve patients with pGHD (3 to <18 years) were randomized 1:1 to receive: Sequence 1: 12 weeks of Genotropin then 12 weeks of somatrogon; or Sequence 2: 12 weeks of somatrogon then 12 weeks of Genotropin. The primary endpoint was treatment burden assessed as the difference in mean overall LI total scores between weekly and daily injection schedules, completed after each 12-week treatment period. A post-hoc responder analysis of LI scores using an anchor-based approach was conducted on patients who completed the patient LI questionnaire at Baseline, Week 12, and Week 24. For each injection schedule, responder was defined as decrease of > 18 points from Baseline on Overall LI total score. A non-responder was defined as increase >0 or decrease < 18 points from Baseline on Overall LI total score.

RESULTS:

Of the 87 randomized patients, 78 completed the LI questionnaire at Baseline, W12 and W24 and were included in the responder analysis. During treatment with Genotropin, 13 patients (16.7%) were responders and 65 (83.3%) were non-responders. During somatrogon treatment, 37 (47.4%) were responders and 41 (52.6%) were non-responders. The proportion of responders is significantly different (p<0.0001) between each treatment, in favour of somatrogon. This supports the phase 3 primary endpoint results.

CONCLUSIONS:

A significantly higher proportion of patients treated with once-weekly somatrogon reported meaningful reductions in treatment burden. This could lead to improved adherence and better outcomes compared to Genotropin once a day.