Background: According to international guidelines Prader-Willi children during GH treatment must be closely monitored by polysomnography, ENT evaluation and IGF1 levels.
Objective and hypotheses: The study aims to determine whether the modulation of GH therapy in children and adolescents with Prader-Willi Syndrome with a specific decisional score (POI score; Salvatoni A., Horm Res Paediatr. 2012) changes and to what extent the results of the therapy.
Method: We compared retrospectively 40 prepubertal children (21 boys), aged 4.1 (3.8) year, with genetically confirmed Prader-Willi syndrome, in treatment with GH for at least 3 years. Twenty patients (group A) were treated with a GH standard dose of 0.09 U/kg per day, and treatment was discontinued according to the Italian pharmaceutical agency (AIFA) recommendations (BMI over 95th percentile and/or OSAS); in the other 20 patients (group B) the dosage of GH treatment was modulated according to the POI score. We compared in the two groups of patients the trend of the following aspects in the last 2 years of GH treatment: times of discontinuation of the treatment, dosage of GH, IGF1 levels, height-SDS and BMI-SDS. The results are reported as (median (IQR). Mann-Whitney Rank test was used for statistical analysis.
Results: The group B resulted to be treated with significant lower dose of GH (0.08 (0.06) mg/kg per week vs 0.31 (0.03) mg/kg per week; P<0.0001)). The two groups showed at the end the 2 years of treatment similar changes in BMI-SDS (group A+1.2(1.6) vs group B+0.1 (2.6); P=ns), height-SDS (group A+0.66 (1.36) vs group B+0.10 (1.01); P=ns) and times of discontinuation of the treatment (one case in each group). The change in IGF1-SDS resulted significantly higher in group A (group A 2.39 (1.78) vs group B 1.36 (1.82); P<0.02).
Conclusion: The use of the POI score even reducing GH doses and IGF1 levels does not significantly alter the therapeutic results.
Conflict of interest: The study was supported by a grant Provided by Pfizer.
Funding: This work was supported by Pfizer - Ref Pfizer WI180070.
01 - 03 Oct 2015
European Society for Paediatric Endocrinology