Prader-Willi syndrome: who can have growth hormone?

Area of Science:

• Pediatric Endocrinology
• Sleep Medicine
• Genetics

Background:

• Growth hormone (GH) is approved for Prader-Willi syndrome (PWS) to improve various health aspects.
• Concerns exist regarding sudden death in PWS patients initiating GH, potentially linked to severe obesity and sleep-disordered breathing (SDB).
• PWS patients are susceptible to SDB due to autonomic dysfunction and upper airway obstruction, which GH might exacerbate.

Purpose of the Study:

• To evaluate the evidence for GH use in PWS, focusing on respiratory risks.
• To propose a management strategy for identifying and monitoring PWS patients at risk during GH therapy.
• To determine if GH therapy can be safely administered to obese PWS patients with SDB.

Main Methods:

• Review of current literature on GH therapy in PWS and its association with SDB.
• Analysis of potential mechanisms by which GH may worsen respiratory function in PWS.
• Proposal of a clinical pathway for managing respiratory risks in PWS patients undergoing GH treatment.

Main Results:

• GH therapy may exacerbate SDB through adenotonsillar hypertrophy, increased metabolic rate, and fluid shifts.
• Continuous positive airway pressure (CPAP) or bilevel positive airway pressure (BiPAP) can mitigate respiratory risks.
• Evidence suggests GH can be safely used in severely obese PWS children if SDB is managed.

Conclusions:

• With appropriate respiratory support, GH therapy is considered safe for obese PWS patients.
• A proactive approach to managing SDB is crucial for the safe initiation and continuation of GH treatment in PWS.
• Withholding beneficial GH therapy due to unmanaged respiratory risks is not supported by current evidence.