Preprint Article Version 1 Preserved in Portico This version is not peer-reviewed

Transient Isolated Idiopathic Growth Hormone Deficiency – a Self-Limiting Pediatric Disease with Male Predominance or a Diagnosis Based on Uncertain Criteria? Lesson from 20-Years’ Real-World Experience with Retesting in One Center

Version 1 : Received: 21 April 2024 / Approved: 7 May 2024 / Online: 7 May 2024 (13:39:50 CEST)

A peer-reviewed article of this Preprint also exists.

Smyczyńska, J.; Hilczer, M.; Smyczyńska, U.; Lewiński, A.; Stawerska, R. Transient Isolated, Idiopathic Growth Hormone Deficiency—A Self-Limiting Pediatric Disease with Male Predominance or a Diagnosis Based on Uncertain Criteria? Lesson from 20 Years’ Real-World Experience with Retesting at One Center. Int. J. Mol. Sci. 2024, 25, 5739. Smyczyńska, J.; Hilczer, M.; Smyczyńska, U.; Lewiński, A.; Stawerska, R. Transient Isolated, Idiopathic Growth Hormone Deficiency—A Self-Limiting Pediatric Disease with Male Predominance or a Diagnosis Based on Uncertain Criteria? Lesson from 20 Years’ Real-World Experience with Retesting at One Center. Int. J. Mol. Sci. 2024, 25, 5739.

Abstract

In the majority of children with growth hormone (GH) deficiency (GHD), normal GH secretion may occur before the attainment of final height. The aim of the study was to assess the incidence of persistent and transient GHD and the effectiveness of recombined human GH (rhGH) therapy in children with isolated idiopathic GHD, with respect to the moment of therapy withdrawal and according to different diagnostic criteria of GHD. Analysis included 260 patients (173 boys, 87 girls) with isolated idiopathic GHD who completed rhGH therapy and have reassessed GH and IGF-1 secretion. The incidence of transient GHD with respect to different pre- and post-treatment criteria was compared together with assessment of GH therapy effectiveness. Incidence of transient GHD, even with respect to pediatric criteria was very high. Normal GH secretion occurred before the attainment of near-final height. Application of more restricted criteria decreased the number of children diagnosed with GHD but not the incidence of transient GHD among them. Poor response to GH therapy was observed mainly in patients with normal IGF-1 before treatment, suggesting that diagnosis of GHD may be false positive. Further efforts should be made to avoid overdiagnosis GHD and overtreatment of the patients.

Keywords

growth hormone deficiency; growth hormone therapy; near-final height; retesting; insulin-like growth factor-1

Subject

Medicine and Pharmacology, Endocrinology and Metabolism

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