Table 2.
Evidence to support medication/supplement management in PWS
| Medical intervention | Category | Indication | Special Considerations | References |
|---|---|---|---|---|
| Growth hormone | A | All patients throughout life for short stature, maintenance of lean body mass, cognitive benefits |
Potential risk of worsening OSA and link to sudden death (Tauber, Diene, Molinas, & Hebert, 2008) Recommend polysomnography pre–post initiation |
Bakker et al. (2017), Bakker, Kuppens, et al. (2015), Bohm, Ritzen, and Lindgren (2015), Coupaye et al. (2016), Dykens, Roof, Hunt‐Hawkins (2017), Hoybye (2015), Kuppens et al. (2017), Lo et al. (2015), Longhi et al. (2015) |
| HCG | C | Infant males with undescended testes/possibly in puberty | Transient increase in testosterone | Bakker, Wolffenbuttel, et al. (2015), Eiholzer et al. (2007) |
| Testosterone | C | Male hypogonadism | Potential increased aggression; consider initiating low disease and increasing slowly over time | Kido et al. (2013) |
| Estrogen/progesterone | C | Female hypogonadism | Potential worsening behavior and increased risk of blood clots; consider initiating low doses and increasing slowly over time | Eldar‐Geva et al. (2013) |
| Modafinil | B | Excessive daytime sleepiness/narcolepsy and impulsive behavior | Increased risk of severe skin rash can worsen anxiety in some, but some parents report improvement | De Cock et al. (2011), Weselake et al. (2014) |
| Topiramate | C | Severe skin picking | In neurodevelopmentally disabled children, associated with cognitive slowing | Shapira et al. (2004), Shapira et al. (2002), Smathers et al. (2003) |
| SSRIs | C | stubbornness, cognitive rigidity, anxiety, and OCD | Threshold of affect, start low and increase slowly | Dech and Budow (1991), Kohn, Weizman, and Apter (2001), Selikowitz, Sunman, Pendergast, and Wright (1990) |
| Antipsychotics | B | Aggression, impulsivity, magical thinking, psychosis | Increased risk of weight gain with some | Akca and Yilmaz (2016), Araki et al. (2010), Bonnot et al. (2016), Durst, Rubin‐Jabotinsky, Raskin, Katz, & Zislin (2000a, 2000b ), Dykens and Shah (2003), Elliott et al. (2015) |
| Metformin | B | Insulin resistance | GI side effects, lactic acidosis | Chan, Feher, and Bridges (1998), Miller, Linville, and Dykens (2014) |
| GLP‐1R agonists | B | Insulin resistance, obesity | Generally not recommended for age <14, GI side effects and possible pancreatitis, slows gastric emptying | Fintini et al. (2014), Salehi et al. (2016) |
| N‐acetylcysteine | C | Skin picking | Individuals seem to develop a tolerance | Miller and Angulo (2014) |
| Carnitine | C | Carnitine deficiency; Vegetarian/vegan diet | GI distress can be a side effect | Ma et al. (2012), Miller, Lynn, Shuster, and Driscoll (2011) |
| CoQ10 | C | Poor suck, low stamina | GI side effects | Butler et al. (2003), Eiholzer et al. (2008), Miller et al. (2011) |
| MCT oil | C | Infants with failure to thrive despite adequate calories, weight control in combination with exercise | Additional calories, clogs NG tubes | Ma et al. (2012) |
| DHA | C | Low‐fat diet, hyperlipidemia | GI side effects | No evidence |
| B12 | C | Low serum vitamin B12 levels, elevated mean corpuscular volume (MCV) and low energy levels, vegetarian/vegan diet | Can increase anxiety | No evidence |
| Bariatric surgery | D | Life‐threatening obesity, resistant to all other interventions | Increased risk of severe surgical morbidity and mortality | Fong, Wong, Lam, and Ng (2012), Marceau and Biron (2012), Scheimann, Miller, and Glaze (2017) |
Medications are listed with the indication and as applicable evidence‐based references. Categories of evidence—A: Good evidence to support a recommendation for use; B: Moderate evidence to support a recommendation for use; C: Poor evidence to support a recommendation for or against use; D: Moderate or good evidence to support a recommendation against use; and E: Good evidence to support a recommendation against use.
CoQ10: coenzyme Q10; DHA: docosahexaenoic acid; GH: growth hormone; GLP‐1R: glucagon‐like peptide‐1 receptor; HCG: human chorionic gonadotropin; MCT: medium‐chain triglycerides; OCD: obsessive–compulsive disorder; PWS: Prader–Willi syndrome; SSRI: selective serotonin reuptake inhibitor.