Sleep disturbance in mucopolysaccharidosis type III (Sanfilippo syndrome): a survey of managing clinicians.
Sanfilippo syndrome (mucopolysaccharidosis type III) is the commonest mucoploysaccharidosis. It causes neurodegeneration with often profound sleep and behavioral disturbance. Management of the sleep disturbance is difficult and inconsistent. In this study, we surveyed clinicians with particular expertise in the management of individuals with mucopolysaccharidoses. We found that sleep problems are almost universal in this patient population and that no one treatment is consistently viewed as beneficial. Among the clinicians surveyed, melatonin is reported as the medication most likely to be of benefit. Benzodiazepines, chloral hydrate, antihistamines and antipsychotic agents are overall reported as less efficacious. The major side-effect of the medications as a group was reported to be daytime somnolence. Based on this study, recommendations are given regarding the approach to sleep disturbance in Sanfilippo syndrome.
Fraser
J
J
Genetic Health Services Victoria, Royal Children's Hospital, Victoria, Australia.
Wraith
J E
JE
Delatycki
M B
MB
D001521:Behavior Therapy; D003625:Data Collection; D006801:Humans; D009084:Mucopolysaccharidosis III / Q000150:complications*; D010820:Physicians; D012893:Sleep Wake Disorders / Q000150:complications* / Q000188:drug therapy / Q000628:therapy