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GH therapy improves linear growth, adult height in Prader-Willi syndrome
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Prepubertal and adolescent children with Prader-Willi syndrome treated with recombinant human growth hormone therapy before the onset of puberty saw increased linear growth and adult height over at least 3 years of treatment, according to a worldwide, retrospective cohort study.
“Long-term data of a large group of GH-treated prepubertal children with [Prader-Willi syndrome] have not been reported,” N.E. Bakker, MD, of the Dutch Growth Research Foundation in Rotterdam, the Netherlands, and colleagues wrote. “Results of nationwide studies from the USA, the Netherlands and Switzerland are reassuring, but have the limitation of small patient numbers because [Prader-Willi syndrome] is a rare syndrome. Therefore, the present study was undertaken to evaluate growth during 3 years of GH treatment and adult height data in a large group of children with [Prader-Willi syndrome], by using the Pfizer International Growth Database (KIGS), containing data from 1987 to 2012.”
Using the KIGS database, Bakker and colleagues analyzed data from 522 prepubertal children with Prader-Willi syndrome treated with GH (Genotropin, Pfizer) for 3 years (54.8% boys), as well as 173 adolescents with Prader-Willi syndrome treated with GH therapy at least 2 years before puberty onset, with treatment continuing until patients reached near-adult height (46.8% boys). A safety analysis included all 2,332 children in the database. Primary outcome was prepubertal growth over 3 years of GH therapy, as measured by height and BMI standard deviation of samples (SDS), as well as near-adult height data after GH therapy in adolescents; secondary outcomes included adverse events in children treated with GH therapy.
In prepubertal children, height increased from –2.05 SDS at baseline to –0.31 SDS at 3 years (P < .05), whereas BMI SDS increased according to age-matched reference data from 1.11 SDS to 1.53 SDS at 3 years.
In the adolescent group, height increased from –2.14 SDS at baseline to –0.22 SDS at the start of puberty (P < .05 vs. baseline). However, delta height SDS declined from the start of puberty for a mean adult height SDS of –1.19. BMI SDS did not change in this group; mean BMI SDS was 1.78 at adult height.
In multiple regression analysis, height SDS and BMI SDS at the start of GH therapy were negatively associated with adult height SDS (beta = –0.3 and –0.4, respectively; P < .01 for both).
Scoliosis was the most frequently reported adverse event during GH treatment in patients (n = 154). Twelve children died, 10 were receiving GH treatment at time of death. Causes of death included gastric perforation, pneumonia, accident, immunodeficiency and cardiorespiratory arrest.
“During GH treatment, BMI SDS remained on average below the + 2 SDS,” the researchers wrote. “The number of reported deaths in GH-treated children with [Prader-Willi syndrome] in KIGS seems low compared to the high annual mortality rate of 3% in untreated [Prader-Willi syndrome] patients. However, safety issues, such as diabetes, sleep-related breathing disorders, gastric problems and infections, should be closely monitored in children with [Prader-Willi syndrome], with and without GH treatment.” – by Regina Schaffer
Disclosure: Pfizer supported this study. Four of the authors were employees of Pfizer at the study; one author is a member of the KIGS Steering Committee.
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