ESPE Abstracts (2021) 94 P2-309

1Department of Pediatrics, 2nd Faculty of Medicine, Charles University and University Hospital in Motol, Prague, Czech Republic; 2Department of Children and Adolescents, 3rd Faculty of Medicine, Charles University and University Hospital in Královské Vinohrady, Prague, Czech Republic; 3Institute of Endocrinology, Prague, Czech Republic; 4Department of Pediatrics, Faculty of Medicine, University of Ostrava and University Hospital, Ostrava, Czech Republic; 5Department of Pediatrics, Hospital České Budějovice, České Budějovice, Czech Republic; 6Department of Pediatrics, Faculty of Medicine, Masaryk University and University Hospital Brno, Brno, Czech Republic; 7Department of Pediatrics, Faculty of Medicine, Charles University and University Hospital Hradec Králové, Hradec Králové, Czech Republic; 8Department of Pediatrics, Faculty of Medicine, Charles University and University Hospital Pilsen, Pilsen, Czech Republic; 9Department of Pediatrics, Masaryk Hospital, Ústí nad Labem, Czech Republic; 10Department of Pediatrics, Faculty of Medicine, Palacky University and Olomouc University Hospital, Olomouc, Czech Republic; 11Institute of Biostatistics and Analyzes, s.r.o., Brno, Czech Republic

Background: Following a phase of feeding difficulties and underweight in early life, children with Prader-Willi syndrome (PWS) develop hyperphagia and a tendency towards severe obesity. Growth hormone therapy (GH) has been approved in PWS to compensate their growth failure but may exert also additional effects on muscle component and body composition as well.

Objectives: We aimed to test if an early initiation of GH therapy within the first two years of life may positively influence the trajectory of body mass index (BMI) in PWS, if compared to PWS children with later therapy onset.

Methodology: We analyzed individual trajectories of BMI in 98 patients with PWS from the REPAR – Czech national registry of patients treated with GH (54 boys and 44 girls; mean age at start of treatment 5.5 ± 4.3 years; mean ± SD). Patients were divided into two groups according to age at the start of GH treatment: early-onset GH therapy – treatment started till 2 years of age (n = 55; age 0.8 ± 0.4 years; mean ± SD), late-onset GH therapy – treatment started thereafter (n = 43; age 7.0 ± 4.6 years; mean ± SD).

Results: Children within the late-onset GH therapy group were obese already at treatment initiation (SDS BMI 2.9 ± 2.1; mean ± SD). Within the first year of GH treatment, their BMI significantly declined by 0.9 SDS (p = 0.009). After five years, mean BMI decreased below the obesity limit to 1.7 (± 1.8) SDS. On the opposite, children with early-onset GH therapy were underweight before GH treatment (mean SDS BMI -1.0 ± 1.2). Between 3rd and 7th year of treatment, their BMI significantly increased to borderline obesity range (2.1 ± 2.6 SDS; P < 0.001). After 5 years of therapy, BMI-SDS did not differ between both groups (p = 0.45). However, most children with an early-onset GH therapy apparently avoided the phase of severe obesity.

Conclusion: Early initiation of GH administration in children with PWS has the potential to prevent transition to severe obesity within their entire childhood, at least in 60 % of children. Following five years of GH therapy, no differences persist in BMI between those who were treated early or late. Nevertheless, the long-term effective prevention of obesity in PWS unavoidably also requires adjustment of eating habits.

Volume 94

59th Annual ESPE (ESPE 2021 Online)

22 Sep 2021 - 26 Sep 2021

European Society for Paediatric Endocrinology 

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