What are the treatments for Prader-Willi syndrome (PWS)?

Parents can enroll infants with PWS in early intervention programs. However, even if a PWS diagnosis is delayed, treatments are valuable at any age.

The types of treatment depend on the individual’s symptoms. The healthcare provider may recommend the following:

  • Use of special nipples or tubes for feeding difficulties. Difficulty in sucking is one of the most common symptoms of newborns with Prader-Willi syndrome. Special nipples or tubes are used for several months to feed newborns and infants who are unable to suck properly, to make sure that the infant is fed adequately and grows. To ensure that the child is growing properly, the health care provider will monitor height, weight, and body mass index (BMI) monthly during infancy.
  • Strict supervision of daily food intake. Once overeating starts between ages 2 and 4 years, supervision will help to minimize food hoarding and stealing and prevent rapid weight gain and severe obesity. Parents should lock refrigerators and all cabinets containing food. No medications have proven beneficial in reducing food-seeking behavior.

    A well-balanced, low-calorie diet and regular exercise are essential and must be maintained for the rest of the individual's life. People with PWS rarely need more than 1,000 to 1,200 calories per day. Height, weight, and BMI should be monitored every 6 months during the first 10 years of life after infancy and once a year after age 10 for the rest of the person's life to make sure he or she is maintaining a healthy weight. Ongoing consultation with a dietitian to guarantee adequate vitamin and mineral intake, including calcium and vitamin D, might be needed.
  • Growth Hormone (GH) therapy. GH therapy has been demonstrated to increase height, lean body mass, and mobility; decrease fat mass; and improve movement and flexibility in individuals with PWS from infancy through adulthood.1,2,3 When given early in life, it also may prevent or reduce behavioral difficulties. Additionally, GH therapy can help improve speech, improve abstract reasoning, and often allow information to be processed more quickly. It also has been shown to improve sleep quality and resting energy expenditure.2,3,4 GH therapy usually is started during infancy or at diagnosis with PWS. This therapy often continues during adulthood at 20% to 25% of the recommended dose for children.
  • Treatment of eye problems by a pediatric ophthalmologist. Many infants have trouble getting their eyes to focus together. These infants should be referred to a pediatric ophthalmologist who has expertise in working with infants with disabilities.
  • Treatment of curvature of the spine by an orthopedist. An orthopedist should evaluate and treat, if necessary, curvature of the spine (scoliosis). Treatment will be the same as that for people with scoliosis who do not have PWS.
  • Sleep studies and treatment. Sleep disorders are common with PWS. Treating a sleep disorder can help improve the quality of sleep. The same treatments that healthcare providers use with the general population can apply to individuals with PWS.
  • Physical therapy. Muscle weakness is a serious problem among individuals with PWS. For children younger than age 3, physical therapy may increase muscular strength and help such children achieve developmental milestones. For older children, daily exercise will help build lean body mass.
  • Behavioral therapy. People with PWS have difficulty controlling their emotions. Using behavioral therapy can help. Stubbornness, anger, and obsessive-compulsive behavior, including obsession with food, should be handled with behavioral management programs using firm limit-setting strategies. Structure and routines also are advised.1,5
  • Medications. Medications, especially serotonin reuptake inhibitors (SRIs), may reduce obsessive-compulsive symptoms. SRIs also may help manage psychosis.
  • Early interventions/Special needs programs. Individuals with PWS have varying degrees of intellectual difficulty and learning disabilities. Early intervention programs, including speech therapy for delays in acquiring language and for difficulties with pronunciation, should begin as early as possible and continue throughout childhood.6

    Special education is almost always necessary for school-age children. Groups that offer training in social skills may also prove beneficial. An individual aide is often useful in helping PWS children focus on schoolwork.
  • Sex hormone treatments and/or corrective surgery. These treatments are used to treat small genitals (penis, scrotum, clitoris).
  • Replacement of sex hormones. Replacement of sex hormones during puberty may result in development of adequate secondary sex characteristics (e.g., breasts, pubic hair, a deeper voice).
  • Placement in group homes during adulthood. Group homes offer necessary structure and supervision for adults with PWS, helping them avoid compulsive eating, severe obesity, and other health problems.2

Citations

  1. Cassidy, S. B., & Schwartz, S. (2009). Prader-Willi syndrome. In Pagon, R. A., Bird, T. D., Dolan, C. R., Stephens, K., Adam, M. P. (Eds.). Gene reviews. Seattle, WA: University of Washington. Retrieved June 13, 2012, from http://www.ncbi.nlm.nih.gov/books/NBK1330.
  2. Burman, P., Ritzén, E. M., & Lindgren, A. C. (2001). Endocrine dysfunction in Prader-Willi syndrome: A review with special reference to GH. Endocrine Reviews, 22, 787-799. Retrieved June 13, 2012, from https://pubmed.ncbi.nlm.nih.gov/11739333/
  3. Carrel, A. L., Myers, S. E., Whitman, B. Y., & Allen, D. B. (2002). Benefits of long-term GH therapy in Prader-Willi syndrome: A 4-year study. Journal of Clinical Endocrinology & Metabolism, 87, 1581-1585.
  4. Buiting, K., Dittrich, B., Gross, S., Lich, C., Färber, C., Buchholz, T., et al. (1998). Sporadic imprinting defects in Prader-Willi syndrome and Angelman syndrome: Implications for imprint-switch models, genetic counseling, and prenatal diagnosis. American Journal of Human Genetics, 63, 170-180.
  5. Holm, V. A., Cassidy, S. B., Butler, M. G., Hanchett, J. M., Greenswag, L. R., Whitman, B.Y., et al. (1993). Prader-Willi syndrome: Consensus diagnostic criteria. Pediatrics, 91, 398-402.
  6. Gunay-Aygun, M., Schwartz, S., Heeger, S., O'Riordan, M. A., & Cassidy, S. B. (2001). The changing purpose of Prader-Willi syndrome clinical diagnostic criteria and proposed revised criteria. Pediatrics, 108, e92. Retrieved June 13, 2012, from https://publications.aap.org/pediatrics/article/108/5/e92/63709/The-Changing-Purpose-of-Prader-Willi-Syndrome external link
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