Endocrine Abstracts

JOINT2663

Introduction: Despite the long-established use of recombinant human growth hormone (rhGH) in China for treating growth hormone deficiency (GHD), there still exists a significant gap in understanding its current status and the potential challenges with its effective use. This study aimed to explore the real-world status of rhGH treatment among Chinese children with short stature (SS) and to identify the associated factors.

Methods: In this multicenter, retrospective, observational study, we analyzed data from children receiving rhGH treatment from January 1, 2017, to April 30, 2024. The data was sourced from two registry databases in China (Chinese Society of Pediatric Endocrinology and Metabolism (CSPEM) Growth Study Database (CGSD) and Long-term Efficacy and Safety Evaluation of Growth Hormone in Children in China (CGLS) and included eligible naïve children diagnosed with GHD, idiopathic SS (ISS) or small for gestational age (SGA), and who were treated with at least one rhGH and with height standard deviation score (Ht-SDS). All the data including baseline characteristics, type of rhGH treatment received (long-acting [LAGH] or short-acting [SAGH]), and treatment compliance were collected and analyzed.

Results: A total of 41, 942 children (15, 975 with GHD, 24, 445 with ISS, and 1, 522 with SGA) with a median age of 8. 29 (5. 88–10. 64) years were included. Mean Ht-SDS and BMI-SDS were -2. 51 ± 0. 42 and -0. 48 ± 1. 11, respectively. Short-acting growth hormone (SAGH) was the most preferred option in children than long-acting growth hormone (LAGH) (79. 53% vs. 20. 47%). The overall bone age (BA) – chronological age (CA) was reported as -1. 43 (1. 06). Treatment initiation with an appropriate dose was observed in children with SGA and GHD compared with ISS (33. 26%) with an insufficient dose. Irregular administration often precedes discontinuation, and it was identified as the main reason for long-term withdrawal in both the LAGH and SAGH groups. Treatment discontinuation was observed in 42. 1% of the children mainly due to irregular administration (18. 3%), limited medication access (16. 9%), and refusal by patients or families (11. 2). Overall, the LAGH group exhibited a significantly longer duration of therapy compared to the SAGH group (1. 75 [1. 71, 1. 79] vs. 1. 58 [1, 56, 1. 61]; P < 0. 001).

Conclusion: In China, children with SS tended to start rhGH treatment at a relatively older age and exhibited a high rate of treatment discontinuation. To achieve better treatment outcomes for children with SS, good adherence and guideline-recommended medication patterns need to be adhered to.