ESPE2024 Poster Category 1 GH and IGFs 2 (11 abstracts)
1Department of Paediatric Endocrinology, Diabetology and Gynaecology, Necker Children’s University Hospital, Assistance Publique Hôpitaux de Paris (APHP), Paris, France. 2Université Paris Cité, Paris, France. 3Department of Paediatric Endocrinology and Diabetology, Angers University Hospital, Angers, France. 4The Netherlands Organization for Applied Scientific Research TNO, Leiden, Netherlands. 5Medical Affairs Department Endocrinology, Merck Santé S.A.S., (an affiliate of Merck Healthcare KGaA, Darmstadt, Germany), Lyon, France. 6Medical Affairs Department Endocrinology, Merck Santé S.A.S., (an affiliate of Merck Healthcare KGaA, Darmstadt, Germany), Paris, France. 7Medical Affairs Department Endocrinology, Merck Serono S.A.S., (an affiliate of Merck Healthcare KGaA, Darmstadt, Germany), Lyon, France. 8Global Medical Affairs, Cardiometabolic & Endocrinology, Merck Healthcare KGaA, Darmstadt, Germany. 9Department of Paediatric Endocrinology, Obesity, Bone diseases, Genetic and Gynaecology, Toulouse University Hospital, Toulouse, France. 10Department of Paediatric Endocrinology and Diabetology and Department of Adolescent Medicine, Bicêtre Paris-Saclay Hospital, Paris Saclay University, Assistance Publique Hôpitaux de Paris (APHP), Le Kremlin-Bicêtre, France
Background: The Study and Collection of Observational data for Patients with easypod® Connect (SCOPE) French retrospective study follows children with growth disorders treated with recombinant human growth hormone (r-hGH) therapy via the easypod® connected injection device for up to 5 years.
Aim: To show the results from the SCOPE study analysing the adherence to r-hGH therapy in a real-world setting and its effect on catch-up growth in patients with growth disorders.
Methods: French patients aged between 4–17 years at GH treatment initiation and <18 years during treatment were selected for SCOPE study. Adherence and height data up to 36 and 30 months of treatment, respectively were extracted from easypod® connect ecosystem between June 2018 – December 2023. Adherence was classified as optimal (≥85% of injections) and suboptimal (<85% of injections). To evaluate catch-up growth (Δ height standard deviation score [HSDS]), broken-stick method was used to calculate mean ΔHSDS at 6, 12, 18, and 24 months.
Results: Adherence data for 481 patients with 44.9% females and 55.1% males (53.8% Growth hormone deficiency, 32.6% Small for gestational age, 2.7% Turner Syndrome, and 10.8% other or unknown) were available. Overall, 2,163 height measurements from 472 patients were available. At treatment initiation, mean (SD) age and mean (SD) HSDS were 9.9 (2.9) years and −2.4 (0.8), respectively. Proportion of patients with optimal adherence was 90.6% in first year (n = 436/481), 86.1% in second year (n = 341/396), and 81.1% in third year (n = 181/223). Adherence was significantly lower during summer holidays (July–August) (82.0% [n = 378/461]) than during academic year (September–June) (88.5% [n = 408/461]) (P <0.001, in patients with data available for both periods; n = 461). Mean ΔHSDS was 0.31, 0.54, 0.72, and 0.86 after 6, 12, 18, and 24 months of treatment, respectively. When initiating treatment with r-hGH at age <7 years, mean ΔHSDS was 0.33, 0.60, 0.82, and 1.01 after 6, 12, 18, and 24 months, respectively. Patients with optimal adherence exhibited higher catch-up growth, with a mean ΔHSDS at 0–12 months of 0.55 and 0.46 in optimal (n = 429) and suboptimal (n = 43) adherence groups, respectively, and at 13–24 months of 0.33 and 0.28 in optimal (n = 341) and suboptimal (n = 55) adherence groups, respectively.
Conclusion: Adherence rates to r-hGH delivered via easypod® were high and remained optimal over the first 3 years of treatment in France. Optimal treatment adherence and early initiation of treatment positively affected growth response in the first 2 years.