Effects of Recombinant Human Growth Hormone Treatment, Depending on the Therapy Start in Different Nutritional Phases in Paediatric Patients with Prader-Willi Syndrome: A Polish Multicentre Study.

Agnieszka Lecka-Ambroziak,Katarzyna Doleżal-Ołtarzewska,Maciej Hilczer,Anna Noczyńska,Agata Zygmunt-Górska,Barbara Rabska-Pietrzak,Renata Stawerska,Teresa Żak,Anna Wędrychowicz,Mieczysław Szalecki,Elżbieta Gołębiowska,Dorota Birkholz-Walerzak,Monika Obara-Moszyńska,Marta Wysocka-Mincewicz,Adam Dudek,Elżbieta Petriczko

doi:10.3390/jcm10143176

Abstract

Recombinant human growth hormone (rhGH) treatment is an established management in patients with Prader–Willi syndrome (PWS), with growth promotion and improvement in body composition and possibly the metabolic state. We compared anthropometric characteristics, insulin-like growth factor 1 (IGF1) levels, metabolic parameters and the bone age/chronological age index (BA/CA) in 147 children with PWS, divided according to age of rhGH start into four groups, corresponding to nutritional phases in PWS. We analysed four time points: baseline, rhGH1 (1.21 ± 0.81 years), rhGH2 (3.77 ± 2.17 years) and rhGH3 (6.50 ± 2.92 years). There were no major differences regarding height SDS between the groups, with a higher growth velocity (GV) (p = 0.00) and lower body mass index (BMI) SDS (p < 0.05) between the first and older groups during almost the whole follow-up. IGF1 SDS values were lower in group 1 vs. other groups at rhGH1 and vs. groups 2 and 3 at rhGH2 (p < 0.05). Glucose metabolism parameters were favourable in groups 1 and 2, and the lipid profile was comparable in all groups. BA/CA was similar between the older groups. rhGH therapy was most effective in the youngest patients, before the nutritional phase of increased appetite. We did not observe worsening of metabolic parameters or BA/CA advancement in older patients during a comparable time of rhGH therapy.

Highlights

Recombinant human growth hormone treatment is a well-established proceeding in many indications in paediatric endocrinology
The main results of our study show that Recombinant human growth hormone (rhGH) therapy is most effective in the youngest patients, before the nutritional phase of increased appetite, we can still observe stable body mass index (BMI) standard deviation scores (SDS) maintenance and improvement in height SDS in the first period of the treatment, commenced in the early phase of increased appetite
We tried to look at the clinical settings of rhGH action in children with Prader–Willi syndrome (PWS), the disease with a background of multiple hypothalamic disorders, with a special insight into different nutritional phases reported in PWS

Summary

Introduction

Recombinant human growth hormone (rhGH) treatment is a well-established proceeding in many indications in paediatric endocrinology. It is important in the management of patients with growth hormone deficiency (GHD) and patients with with genetic disorders, such as Turner syndrome, Noonan syndrome or Prader–Willi syndrome (PWS) [1,2]. The treatment for patients with PWS is beneficial from different aspects of rhGH mechanisms of action. PWS is a rare genetic imprinting disease with hypothesised primary hypothalamic disorder caused by a lack of paternally inherited genes on chromosome 15q11-q13. The imprinted genes on chromosome 15q11-q13 include small nuclear ribonucleoprotein polypeptide

Methods

Discussion

Conclusion