Real-world adherence to growth hormone treatment and catch-up growth in children with growth disorders in France: An interim analysis from the SCOPE study (abstract)

Background: The SCOPE French retrospective study follows children affected with growth hormone (GH) disorders treated with recombinant human GH (r-hGH) therapy via the easypod® connected injection device for up to 5 years. Aim: To show the results from an interim analysis of the SCOPE study analysing adherence to r-hGH therapy in a real-world setting and its effect on catch-up growth. Methods: Adherence data up to 36 months and height data up to 18 months of treatment were extracted from the easypod® connect ecosystem between July 2018 and December 2022. Adherence was classified as optimal (≥85% of injections recorded) versus suboptimal (<85%). To study catch-up growth (Δ height standard deviation score [HSDS]), patients aged ≥2 years at treatment start, with a HSDS <-1 at treatment start and ≥1 HSDS measurement between 0-18 months of treatment were selected. A linear mixed-effect model was applied to calculate the predicted mean ΔHSDS at 6, 12 and 18 months. Results: In total, 363 children with adherence data and 199 patients with HSDS data were available from 466 patients. At treatment start, mean age (SD) was 9.7 (3.1) years and mean HSDS (SD) was -2.5 (0.7). Among patients with adherence data available, the proportion of patients with optimal adherence was 94.8% in the first year (n=344/363), 91.1% in the second year (n=195/214) and 85.7% in the third year (n=90/105). Adherence was significantly lower during the summer holidays (July and August) (86.2%) compared with the academic year (September to June) (94.5%) (P<0.001, in patients with data available for both time periods; n=311). Predicted ΔHSDS was 0.31, 0.54 and 0.70, respectively, after 6, 12 and 18 months of treatment. ΔHSDS was higher when the patient started treatment at a younger age; e.g., when initiating treatment with r-hGH at age 6, predicted ΔHSDS was 0.38, 0.68 and 0.86, respectively, after 6, 12 and 18 months. Due to the high proportion of patients with optimal adherence, adherence did not have a significant effect on ΔHSDS. Conclusion: Adherence rates to r-hGH delivered via easypod® are high and remain optimal over 36 months in France. Lower adherence during the summer holidays may reflect difficulties to remain adherent to treatment when not in school or may be due to treatment holidays during the summer. These initial results confirmed the growth response to r-hGH in children and showed that starting GH therapy at a younger age is associated with improved height outcomes.