Linear growth and metabolic outcomes of growth hormone treatment in childhood-onset craniopharyngioma: the OUTSPREAD study.

Abstract

Growth hormone (GH) deficiency is common following craniopharyngioma resection. This study aimed to compare growth status and metabolic parameters before and after GH treatment for patients with craniopharyngioma. This retrospective study included 134 patients with multicenter study for Ideal Outcomes of Pediatric Rare Endocrine and Metabolic Diseases in Korea (OUTSPREAD study), who received GH treatment for ≥1 year. Data on growth and metabolic parameters before and after GH treatment (at 1, 2, and 3 years, and at the end of treatment) were collected. The mean age at diagnosis was 7.6 years, with GH treatment initiated at 9.4 years and concluded at 15.7 years. Before GH treatment, height and weight were below average, while body mass index (BMI) was above average. Following GH treatment, height and weight significantly increased while BMI decreased. Analysis using a sex-, age- and steroid dose- adjusted linear mixed model showed significantly decreased aspartate aminotransferase, alanine aminotransferase and total cholesterol levels after treatment. Random glucose levels increased slightly but remained within the normal range, and glycosylated hemoglobin levels remained stable. This is the first longitudinal report showing the positive effects of GH treatment for craniopharyngioma in Korean patients. After surgery for craniopharyngioma, GH treatement is essential for patients with GH deficiency. In this first multicenter Korean cohort evaluating long-term growth and metabolic outcomes, GH treatment significantly improved height, weight, and BMI SDS. Hepatic indices (AST/ALT) and lipid parameters (total cholesterol) decreased during treatment. Random glucose increased slightly but remained within the normal range, and HbA1c showed no significant change. These findings support GH treatment as a key component of long-term management with benefits beyond linear growth.