EFFICACY OF HUMAN RECOMBINANT GROWTH HORMONE THERAPY IN CHILDREN WITH PRADER–WILLI SYNDROME AT THE VIETNAM NATIONAL CHILDREN’S HOSPITAL
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Abstract
Objectives: To describe human recombinant growth hormone (rhGH) therapy and evaluate treatment outcomes in children with Prader–Willi syndrome (PWS) at the Vietnam National Children’s Hospital.
Methods: Case series study was conducted in 48 children with genetically confirmed PWS who received GH therapy for at least 12 months at the Vietnam National Children’s Hospital between January 2022 and June 2026.
Results: Among the 48 patients, 56.2% were female, and 95.8% had the deletion subtype. The median age at diagnosis and GH initiation was 11,4 and 14,1 months, respectively. The mean treatment duration was 2.6 ± 1.0 years and the average GH dose is 0.43 mg/m²/day. Six patients have discontinued treatment. Height SDS improved significantly from −1.16 ± 1.14 to −0.38 ± 0.99 (Δ = 0.78 ± 1.11, p = 0.001). BMI SDS increased from −0.56 ± 3.00 to 2.44 ± 2.33 (Δ = 2.96 ± 2.36, p < 0.001). Earlier initiation of GH therapy was independently associated with greater improvement in Height SDS after adjusting for baseline height Z-score, treatment duration, and GH dose (β = −0.661, p = 0.001).
Conclusions: GH therapy significantly improved linear growth in children with PWS. Earlier initiation of GH therapy was independently associated with greater height improvement. Changes in BMI SDS during treatment reflect the complex nutritional characteristics of children with PWS and highlight the need for long-term nutritional monitoring and multidisciplinary management.
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Keywords
Prader-Willi syndrome, growth hormone, Height SDS, BMI SDS, children
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