Real-Life Growth Hormone Treatment Patterns in Children from China: A Report from Two Databases

Although recombinant human growth hormone (rhGH) treatment has been established in China for many years for treating short stature (SS), there exists a lacuna on the current status and potential problems associated with effective usage of rhGH. The objective of this study is to investigate the real-world status of rhGH treatment in Chinese children with SS and its associated factors.

This multicenter, retrospective, observational study included children treated with rhGH from January 1, 2017, to April 30, 2024, from two registry databases in China, which included children who were treated with at least one rhGH owing to GH deficiency (GHD), small for gestational age (SGA), or idiopathic short stature (ISS). Data collected included baseline characteristics, the type of rhGH treatment [long-acting (LAGH) or short-acting (SAGH)], and treatment compliance.

A total of 41,942 children (15,975 with GHD, 24,445 with ISS, and 1522 with SGA) with a median age of 8.29 (5.88-10.64) years were included. The mean Ht SDS and BMI-SDS were - 2.51 ± 0.42 and -0.48 ± 1.11, respectively. More children started using SAGH than LAGH (79.53% vs. 20.47%). Although most children with SGA and GHD initiated with an appropriate dose, 33.26% of children with ISS started with an insufficient dose. In the total population, 42.1% of children discontinued medication. The most common reasons were irregular administration, limited medication access, refusal by patients or families, and observational discontinuation. In addition, the LAGH group exhibited a significantly longer duration of therapy compared with the SAGH group.

In China, children with SS tended to start rhGH treatment at a relatively older age and to exhibit 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 followed.