How is Prader-Willi syndrome (PWS) treated?

Updated: Oct 14, 2020
  • Author: Ann Scheimann, MD, MBA; Chief Editor: Luis O Rohena, MD, MS, FAAP, FACMG  more...
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Patients with Prader-Willi syndrome (PWS) frequently require medical care for the following [6, 9] :

  • Initial management of hypotonia or poor feeding
  • Evaluation for hypogonadism or hypopituitarism
  • Management of obesity
  • Monitoring for scoliosis
  • Therapy for behavioral issues

On June 20, 2000, the US Food and Drug Administration (FDA) approved the use of growth hormone in children with genetically confirmed Prader-Willi syndrome and evidence of growth failure. [10, 11, 12]  A study by Bakker et al indicated that growth hormone therapy improves the health-related quality of life (HRQOL) in children with Prader-Willi syndrome. The study included annual HRQOL assessments over an 11-year period. [56]

A prospective cohort study by Donze et al reported that in infants and toddlers with Prader-Willi syndrome who underwent 3 years of growth hormone therapy, mental and motor development tended to see a greater increase in patients who began treatment at a younger age and who had lower psychomotor development at baseline. Using the Bayley Scales of Infant Development II, the investigators also found that over the 3-year treatment course, mental development increased from a mean (standard error of the mean [SEM]) baseline value of 58.1% to 79.6%, while mean (SEM) motor development rose from 41.9% at baseline to 78.2%. [57]

A study by Scheermeyer et al indicated that in infants (aged 2-12 mo) with Prader-Willi syndrome, the effects of low-dose growth hormone treatment (4.5 mg/m2/wk) on growth and development are comparable to those of higher-dose treatment (7 mg/m2/wk). Moreover, although IGF levels rose in response to the lower-dose therapy, the increase was not excessive, signaling that the lower-dose approach may decrease long-term treatment risks. [58]

A double-blind, placebo-controlled, crossover study by Miller et al suggested that low-dose intranasal oxytocin therapy may reduce appetite drive in patients with Prader-Willi syndrome, as well as improve socialization, anxiety, and repetitive behaviors in these individuals. [59]

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