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World Journal of Pediatrics
Published in: World Journal of Pediatrics 5/2023

Open Access 24-12-2022 | Original Article

Early recombinant human growth hormone treatment improves mental development and alleviates deterioration of motor function in infants and young children with Prader–Willi syndrome

Authors: Ruo-Qian Cheng, Yan-Qin Ying, Zheng-Qing Qiu, Jun-Fen Fu, Chun-Xiu Gong, Yan-Ling Yang, Wei Shi, Hui Li, Ming-Sheng Ma, Chang-Yan Wang, Min Liu, Jia-Jia Chen, Chang Su, Xiao-Ping Luo, Fei-Hong Luo, Wei Lu

Published in: World Journal of Pediatrics | Issue 5/2023

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Abstract

Background

Recombinant human growth hormone (rhGH) therapy has shown to improve height and body composition in children with Prader–Willi syndrome (PWS), the evidence of early rhGH treatment on motor and mental development is still accumulating. This study explored the time effect on psychomotor development, anthropometric indexes, and safety for infants and young children with PWS.

Methods

A phase 3, single-arm, multicenter, self-controlled study was conducted in six sites. Patients received rhGH at 0.5 mg/m2/day for first four weeks, and 1 mg/m2/day thereafter for up to 52 weeks. Motor development was measured using Peabody Developmental Motor Scales-second edition, mental development using Griffiths Development Scales-Chinese (GDS-C). Height standard deviation score (SDS), body weight SDS, and body mass index (BMI) SDS were also assessed.

Results

Thirty-five patients were enrolled totally. Significant improvements were observed in height, body weight, and BMI SDS at week 52; GDS-C score showed significant improvement in general quotient (GQ) and sub-quotients. In a linear regression analysis, total motor quotient (TMQ), gross motor quotient (GMQ), and fine motor quotient were negatively correlated with age; however, treatment may attenuate deterioration of TMQ and GMQ. Changes in GQ and locomotor sub-quotient in < 9-month group were significantly higher than ≥ 9-month group. Mild to moderate severity adverse drug reactions were reported in six patients.

Conclusion

Fifty-two-week treatment with rhGH improved growth, BMI, mental development, and lessened the deterioration of motor function in infants and young children with PWS. Improved mental development was more pronounced when instituted in patients < 9 months old.
Appendix
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Metadata
Title
Early recombinant human growth hormone treatment improves mental development and alleviates deterioration of motor function in infants and young children with Prader–Willi syndrome
Authors
Ruo-Qian Cheng
Yan-Qin Ying
Zheng-Qing Qiu
Jun-Fen Fu
Chun-Xiu Gong
Yan-Ling Yang
Wei Shi
Hui Li
Ming-Sheng Ma
Chang-Yan Wang
Min Liu
Jia-Jia Chen
Chang Su
Xiao-Ping Luo
Fei-Hong Luo
Wei Lu
Publication date
24-12-2022
Publisher
Springer Nature Singapore
Published in
World Journal of Pediatrics / Issue 5/2023
Print ISSN: 1708-8569
Electronic ISSN: 1867-0687
DOI
https://doi.org/10.1007/s12519-022-00653-y

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