ESPE Abstracts (2023) 97 RFC14.4

ESPE2023 Rapid Free Communications Late Breaking (6 abstracts)

Evaluating the Effect of Recombinant Human Growth Hormone Treatment on Sleep-related Breathing Disorders in Toddlers with Prader–Willi Syndrome: a one-year retrospective cohort study.

Ruoqian Cheng 1 , Chengjun Sun 1 , Haiyan Guo 2,3 , Jinrong Fu 4 , Yufeng Zhou 5,3 , Jing Wu 1 , Zhenran Xu 1 , Jinwen Ni 1 & Feihong Luo 1


1Department of Pediatric Endocrinology and Inherited Metabolic Diseases, Children’s Hospital of Fudan University, Shanghai, China. 2nstitute of Pediatrics, Children's Hospital of Fudan University, National Children's Medical Center, and the Shanghai Key Laboratory of Medical Epigenetics, International Co-laboratory of Medical Epigenetics and Metabolism, Ministry of Science and Technology, Institutes of Biomedical Sciences, Fudan University, Shanghai, China. 3National Health Commission (NHC) Key Laboratory of Neonatal Diseases, Fudan University, Shanghai, China. 4Department of General Medicine, Children's Hospital of Fudan University, Shanghai, China. 5Institute of Pediatrics, Children's Hospital of Fudan University, National Children's Medical Center, and the Shanghai Key Laboratory of Medical Epigenetics, International Co-laboratory of Medical Epigenetics and Metabolism, Ministry of Science and Technology, Institutes of Biomedical Sciences, Fudan University, Shanghai, China


Keywords: Sleep-Related Breathing Disorders, recombinant human Growth Hormone treatment, Prader–Willi syndrome, Toddlers.

Background: Recombinant human growth hormone (rhGH) therapy is beneficial for children with Prader–Willi syndrome (PWS) in improving short stature and metabolism, but the effect of early rhGH treatment on respiratory and sleep parameters for the PWS children under three years old remains elusive. Thus, this study aimed at investigating the impact of rhGH treatment on sleep-related breathing disorders (SRBDs) for toddlers with PWS.

Methods: We recruited 17 age-matched rhGH-treated PWS patients (rhGH group) and 17 none-rhGH-treated control (non-rhGH group) between October 2018 and January 2023. We collected data related to polysomnography-polygraphy (PSG) and serum level of insulin-like growth factor (IGF-1) and insulin-like growth factor binding protein 3 (IGFBP-3).

Results: The mean age in the rhGH group was 20.76 ± 9.22 months which was similar with that of the non-rhGH group (25.23 ± 13.81 months). The demographic and anthropometric parameters were similar across the two groups after 52 weeks of treatment. Treatment with rhGH on toddlers did not exert adverse effects on the obstructive apnea-hypopnea index (OAHI), central apnea index (CAI), oxygen desaturation index (ODI), mean percutaneous oxygen saturation (SpO2), lowest SpO2, duration when SpO2 is lower than 90%, and proportion of the patients with SpO2 lower than 90%. The increased IGF-1 and IGFBP-3 did not worsen SRBDs.

Conclusion: Treatment with rhGH for 52 weeks on young toddlers with PWS showed no deleterious effects on SRBDs. This shed more light on the importance of initiating rhGH therapy early in PWS patients.

Volume 97

61st Annual ESPE (ESPE 2023)

The Hague, Netherlands
21 Sep 2023 - 23 Sep 2023

European Society for Paediatric Endocrinology 

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