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Licensed Unlicensed Requires Authentication Published by De Gruyter July 4, 2019

Safety and effectiveness of growth hormone therapy in infants with Prader-Willi syndrome younger than 2 years: a prospective study

Raquel Corripio ORCID logo, Carla Tubau, Laura Calvo, Carme Brun, Núria Capdevila, Helena Larramona and Elisabeth Gabau

Abstract

Background

There is little evidence of the effects of early treatment with growth hormone (GH) in infants with Prader-Willi syndrome (PWS). A prospective study was conducted to assess the safety of GH therapy in infants younger than 2 years of age with PWS.

Methods

A total of 14 patients with PWS started treatment with GH under the age of 2 years and were followed over a 2-year period. A deletion of chromosome 15 was present in nine infants (64.3%) and maternal uniparental disomy 15 in five infants (35.7%). The median age at start of GH treatment was 9.6 months (interquartile range [IQR] 9.0–18.3 months). Changes in height standard deviation score (SDS), body mass index (BMI) SDS and subcapsular and tricipital skinfolds in the follow-up period were evaluated with a mixed-model regression analysis using the Package R.

Results

There were no fatal adverse events. A significant decrease (p < 0.001) in tricipital and subcapsular skinfold thickness, with an upward trend of height SDS and a downward trend of BMI SDS, was observed. Infants who started GH before 15 months of age started walking at a median of 18.0 [17.0–19.5] months vs. 36.6 [36.3–37.8] months for those who began treatment with GH after 15 months of age (p = 0.024).

Conclusions

GH treatment in infants with PWS less than 2 years of age is safe and improved body composition. Infants who received GH before the age of 15 months started to walk earlier.


Corresponding author: Dra. Raquel Corripio, MD, PhD, Service of Pediatric Endocrinology, Parc Taulí Hospital Universitari, Institut d’Investigació i Innovació Parc Taulí I3PT, Universitat Autónoma de Barcelona, C/ Parc Taulí 1, E-08208 Sabadell, Barcelona, Spain, Phone: +34 93 74558269, Fax: +34 93 7160646, E-mail: .

Acknowledgments

The authors thank Marta Pulido, MD, for editing the manuscript and editorial assistance.

  1. Author contributions: All the authors have accepted responsibility for the entire content of this submitted manuscript and approved submission.

  2. Research funding: None declared.

  3. Employment or leadership: None declared.

  4. Honorarium: None declared.

  5. Competing interests: The funding organization(s) played no role in the study design; in the collection, analysis, and interpretation of data; in the writing of the report; or in the decision to submit the report for publication.

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Received: 2018-12-08
Accepted: 2019-05-08
Published Online: 2019-07-04
Published in Print: 2019-08-27

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