Long-term effects of GH therapy in adult patients with Prader-Willi syndrome: a longitudinal study

Frontiers in Endocrinology

Endocrinology

Davide Soranna¹  Giuseppe Zampino²  Antonino Crinò²  Antonella Zambon³  Graziano Grugni⁴  Alessandro Sartorio⁵  Lucia Grugni⁶ 

[1] Biostatistics Unit, Istituto Auxologico Italiano, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Milan, Italy;Center for Rare Diseases and Congenital Defects, Fondazione Policlinico Universitario A. Gemelli - Research Institute, Rome, Italy;Department of Statistics and Quantitative Methods, University of Milano-Bicocca, Milan, Italy;Division of Auxology, Istituto Auxologico Italiano, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Piancavallo-Verbania, Italy;Experimental Laboratory for Auxo-Endocrinological Research, Istituto Auxologico Italiano, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Milan, Italy;School of Medicine and Surgery, University of Tor Vergata, Rome, Italy;

关键词: Prader-Willi syndrome;    adults;    GH deficiency;    GH therapy;    obesity;   

DOI  :  10.3389/fendo.2023.1198616

 received in 2023-04-01, accepted in 2023-05-10,  发布年份 2023

来源: Frontiers

【 摘 要 】

IntroductionPrader-Willi syndrome (PWS) is a complex disorder resulting from the failure of expression of paternal alleles in the PWS region of chromosome 15. The PWS phenotype resembles that observed in the classic non-PWS GH deficiency (GHD), including short stature, excessive fat mass, and reduced muscle mass. To date, a small number of studies on the long-term effects of GH treatment are available in adult subjects with PWS.MethodsIn this longitudinal study, 12 obese subjects with PWS (GHD/non-GHD 6/6) were treated for a median of 17 years, with a median GH dose of 0.35 mg/day. The median age was 27.1 years. Anthropometric, body composition, hormonal, biochemical, and blood pressure variables were analyzed in all subjects.ResultsWaist circumference was significantly lower at the end of the treatment period (p-value=0.0449), while body mass index (BMI) did not differ significantly. Compared to the baseline, a highly significant reduction of Fat Mass % (FM%) was observed (p-value=0.0005). IGF-I SDS values significantly increased during GH therapy (p-value=0.0005). A slight impairment of glucose homeostasis was observed after GH therapy, with an increase in the median fasting glucose levels, while insulin, HOMA-IR, and HbA1c values remained unchanged. Considering GH secretory status, both subjects with and without GHD showed a significant increase in IGF-I SDS and a reduction of FM% after GH therapy (p-value= 0.0313 for all).DiscussionOur results indicate that long-term GH treatment has beneficial effects on body composition and body fat distribution in adults with PWS associated with obesity. However, the increase in glucose values during GH therapy should be considered, and continuous surveillance of glucose metabolism is mandatory during long-term GH therapy, especially in subjects with obesity.

【 授权许可】

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Copyright © 2023 Grugni, Sartorio, Soranna, Zambon, Grugni, Zampino and Crinò