Long-term side effects of growth hormone treatment in children with Prader–Willi syndrome

Abstract

The main motivations of growth hormone (GH) treatment of Prader–Willi syndrome (PWS) are the stimulation of growth and lean muscle mass. Furthermore GH therapy in Prader–Willi children seems to favorably affect their behavior and mental performances. It is still a matter of discussion whether GH therapy in PWS should be considered responsible for specific adverse events. The most significant of them are scoliosis and breathing disorders, the latter considered being responsible for some deaths, reported in children with PWS, mainly at the beginning of GH therapy. Obstructive sleep apnea was occasionally reported also in patients treated with GH for several years. The review reports and discusses the latest data related to side effects of long-term GH treatment in children with PWS.

Keywords::

• body composition
• growth hormone
• Prader–Willi
• short stature
• side effects
• therapy

Financial & competing interests disclosure

A Salvatoni has received three grants (2005–2008–2014) from Pfizer to study the side effects of GH treatment in patients with Prader–Willi syndrome. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

Key issues

• It is still under investigation whether or not growth hormone (GH) therapy in Prader–Willi syndrome could be responsible for major long-term adverse side effects.

• Polysomnographic and otolaryngology control at least once a year is suggested during long-term GH treatment.

• Annual or closer orthopedic evaluation for possible developing or worsening of scoliosis during GH therapy particularly in fast-growing subjects.

• Periodical assessment of glucose homeostasis in particular in overweight PWS patients receiving GH.

• Periodical assessment of adrenal and thyroid function in all PWS in treatment with GH.

• No adverse psychic side effects were reported; on the contrary beneficial effect of GH therapy was reported on mental performances and behavior in PWS children, but more studies are needed.

• Possible long-term neoplastic risk in PWS subjects treated with GH in childhood requires further investigations.