J Clin Endocrinol Metab. 2025 Nov 15:dgaf626. doi: 10.1210/clinem/dgaf626. Online ahead of print.
ABSTRACT
CONTEXT: In children with idiopathic isolated growth hormone deficiency (IIGHD), GH secretion often normalizes by near adult height (NAH). Whether rhGH treatment can be safely discontinued earlier remains unclear.
OBJECTIVE: To investigate if withdrawing rhGH treatment from mid-puberty onwards had no negative effect on attained NAH in adolescents who, after retesting, were no longer GH deficient.
DESIGN: Prospective multicenter patient preference study (2017-2024) with follow-up until NAH (SEENEZ GH Study).
SETTING: Pediatric endocrinology departments in multiple centers.
PATIENTS OR OTHER PARTICIPANTS: 127 adolescents (95 male, 75%) with childhood IIGHD (GH peak 1.7-10 µg/L) who tested GH sufficient (GH peak >6.7 µg/L) at mid-puberty. 44 continued rhGH (GHcont), 83 discontinued (GHstop). 99% of patients completed the study.
INTERVENTIONS: RhGH treatment continuation vs discontinuation from mid-puberty until NAH.
MAIN OUTCOME MEASURES: Primary: NAH-SDS minus target height (TH)-SDS. Secondary: NAH-SDS, total pubertal growth (TPG), and predicted vs attained height gain.
RESULTS: Mean (SD) NAH-SDS minus TH-SDS was -0.17 (0.60) in the GHcont and -0.18 (0.62) in the GHstop group (P=.96). Mean NAH-SDS was -0.91 (0.76) (GHcont) vs -0.78 (0.76) (GHstop) (P=.35). Mean (SD) TPG (from start of puberty) in males was 27.5 cm (7.0; GHcont) vs 25.9 cm (6.2; GHstop) (P =.25) and in females 20.5 cm (5.7; GHcont) vs 20.9 cm (7.6; GHstop) (P = .90). Predicted vs attained height gain based on the prediction model did not differ between groups.
CONCLUSIONS: In adolescents with transient IIGHD, rhGH treatment can be stopped at mid-puberty. These findings support reducing rhGH treatment duration, lowering patient burden and healthcare costs.
PMID:41239863 | DOI:10.1210/clinem/dgaf626
