• Acta Medica Port · Feb 2023

    A Case Series Study on Growth Hormone Therapy in Children with Prader-Willi Syndrome in Portugal.

    • Madalena Meira Nisa, Miguel Vieira Martins, Bárbara Barroso de Matos, Joana Simões Monteiro, Catarina Marques Duarte, Brígida Robalo, Carla Pereira, and Lurdes Sampaio.
    • Serviço de Pediatria. Centro Hospitalar Tondela-Viseu. Viseu; Unidade de Endocrinologia Pediátrica. Departamento de Pediatria. Hospital de Santa Maria. Centro Hospitalar Universitário Lisboa Norte. Lisboa. Portugal.
    • Acta Medica Port. 2023 Feb 1; 36 (2): 889588-95.

    IntroductionPrader-Willi syndrome is a multisystemic genetic disorder associated with shorter adult height. Nowadays, all paediatric Prader-Willi syndrome patients are considered for growth hormone treatment. We present the experience of this treatment at a Portuguese paediatric endocrinology unit and intend to emphasise the importance of creating a follow-up national network of these patients.Material And MethodsLongitudinal, retrospective, analytical study of Prader-Willis syndrome patients using data between 1989 and 2021. Growth hormone therapy was offered to eligible patients. The analysis included all Prader-Willis syndrome patients, with a comparison between treated and untreated patients; a longitudinal analysis of patients receiving growth hormone therapy (baseline, 12 and 36 months of follow-up) was also carried out. The statistical analysis was carried out using STATA® v13.0.ResultsOut of 38 patients with Prader-William syndrome, 61% were male. The median age at diagnosis was four months and 61% received growth hormone therapy. The patients who reached adulthood, or 18 years old, had a median near-adult height, Z-score of -2.71, and their median body mass index indicated class 2 obesity, regardless of growth hormone therapy. Patients had a lower body mass index in the growth hormone group (35 vs 51 kg/m2, p < 0.042) near-adult height.ConclusionThis case series represents the first national study that included patients on growth hormone therapy after the National Health Service started supporting the treatment for Prader-Willi syndrome patients and supports its use, reinforcing the positive effects on growth and body mass index. Longer follow-up studies are needed to analyse the effect of growth hormone on patient metabolic profiling, body composition and cognitive level.

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