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

Prader-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 mas...

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Published in:	Frontiers in endocrinology (Lausanne) Vol. 14; p. 1198616
Main Authors:	Grugni, Graziano, Sartorio, Alessandro, Soranna, Davide, Zambon, Antonella, Grugni, Lucia, Zampino, Giuseppe, Crinò, Antonino
Format:	Journal Article
Language:	English
Published:	Switzerland Frontiers Media S.A 26-05-2023
Subjects:	adults Endocrinology GH deficiency GH therapy Glucose Humans Insulin-Like Growth Factor I Longitudinal Studies obesity Obesity - complications Obesity - drug therapy Prader-Willi syndrome Prader-Willi Syndrome - complications Prader-Willi Syndrome - drug therapy GH deficiency GH therapy Prader-Willi syndrome obesity adults
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Summary:	Prader-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. In 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. Waist 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). Our 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.
Bibliography:	ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23 Reviewed by: Marko Stojanovic, University of Belgrade, Serbia; Nevena Folic, University of Kragujevac, Serbia; Sandra Stankovic, Clinical Center Niš, Serbia; Sanja Panic Zaric, The Institute for Health Protection of Mother and Child Serbia, Serbia Edited by: Rade Vukovic, The Institute for Health Protection of Mother and Child Serbia, Serbia
ISSN:	1664-2392 1664-2392
DOI:	10.3389/fendo.2023.1198616