OBJECTIVES

To evaluate patient and caregiver perceptions of the treatment burden associated with weekly somatrogon compared to daily somatropin injection in subgroups identified as ‘Not Burdened’ or ‘Burdened’ at baseline (BL).

METHODS : A phase 3 (NCT03831880) open-label, crossover study, randomized non-naïve pediatric pts (3 to <18 years) with GHD 1:1 to receive either Sequence 1: 12 weeks of somatropin then 12 weeks of somatrogon; or Sequence 2: 12 weeks of somatrogon then 12 weeks of somatropin. Study assessments were administered via validated dyad questionnaires. The primary endpoint was treatment burden assessed as the difference between mean overall Life Interference (LI) total scores after each 12-week treatment period of somatrogon and somatropin experienced in each treatment sequence. Pts were categorised at BL based on how often they were bothered by injections in the past 4 weeks: Not Burdened (Sometimes, Often, Always) or Burdened (Never, Rarely).

RESULTS

Eighty-seven pts were randomized; Sequence 1 (N=43), Sequence 2 (N=44); 85 completed. At BL, 51 pts were ‘Not Burdened’ and 31 were ‘Burdened’. In both the ‘Not Burdened’ and the ‘Burdened’ group, the difference between the mean overall LI total score was significantly lower for the somatrogon group (Not Burdened’ group: somatrogon (6.77) vs somatropin (17.31) mean difference: -10.54; two-sided 95% CI: [-14.59, -6.49]; P<0.0001 ; ‘Burdened’ group : somatrogon (9.80) vs somatropin (34.06) mean difference: -24.27; two-sided 95% CI: [-33.02, -15.51]; P<0.0001).

CONCLUSIONS

In paediatric pts with GHD, compared with daily somatropin, weekly somatrogon had a lower treatment burden as shown by less LI, regardless if patients were categorized as ‘Not Burdened’ or ‘Burdened’ at BL. The introduction of a weekly treatment could benefit all patients who currently use daily GH.