Psychotropic Treatments in Prader-Willi Syndrome: A Critical Review of Published Literature

Eur J Pediatr. 2016 Jan;175(1):9-18. doi: 10.1007/s00431-015-2670-x. Epub 2015 Nov 19.


Prader-Willi syndrome (PWS) is a rare genetic syndrome. The phenotype includes moderate to intellectual disability, dysmorphia, obesity, and behavioral disturbances (e.g., hetero and self-injurious behaviors, hyperphagia, psychosis). Psychotropic medications are widely prescribed in PWS for symptomatic control. We conducted a systematic review of published literature to examine psychotropic medications used in PWS. MEDLINE was searched to identify articles published between January 1967 and December 2014 using key words related to pharmacological treatments and PWS. Articles with original data were included based on a standardized four-step selection process. The identification of studies led to 241 records. All selected articles were evaluated for case descriptions (PWS and behavioral signs) and treatment (type, titration, efficiency, and side effects). Overall, 102 patients were included in these studies. Treatment involved risperidone (three reports, n = 11 patients), fluoxetine (five/n = 6), naltrexone (two/n = 2), topiramate (two/n = 16), fluvoxamine (one/n = 1), mazindol (one/n = 2), N-acetyl cysteine (one/n = 35), rimonabant (one/n = 15), and fenfluramine (one/n = 15).

Conclusion: We identified promising treatment effects with topiramate for self-injury and impulsive/aggressive behaviors, risperidone for psychotic symptoms associated with uniparental disomy (UPD), and N-acetyl cysteine for skin picking. The pharmacological approach of behavioral impairment in PWS has been poorly investigated to date. Further randomized controlled studies are warranted.

What is known: Behavioral disturbances in Prader-Willi syndrome including aggressive reactions, skin picking, and hyperphagia might be very difficult to manage. Antipsychotic drugs are widely prescribed, but weight gain and increased appetite are their major side effects.

What is new: Topiramate might be efficient for self-injury and impulsive/aggressive behaviors, N-acetyl cysteine is apromising treatment for skin picking and Antidepressants are indicated for OCD symptoms. Risperidone is indicated in case of psychotic symptoms mainly associated with uniparental disomy.

Keywords: Antidepressor; Antipsychotic; Methylphenidate; Prader-Willi syndrome; Risperidone; Topiramate.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review
  • Systematic Review

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Cystine / analogs & derivatives
  • Cystine / therapeutic use
  • Fructose / analogs & derivatives
  • Fructose / therapeutic use
  • Humans
  • Prader-Willi Syndrome / drug therapy*
  • Psychotropic Drugs / therapeutic use*
  • Risperidone / therapeutic use
  • Topiramate


  • Psychotropic Drugs
  • Topiramate
  • Fructose
  • Cystine
  • Risperidone
  • N-monoacetylcystine