Sleep Disturbance in Sanfilippo Syndrome: a Parental Questionnaire Study

Overview

Journal Arch Dis Child

Specialty Pediatrics

Date 2005 Nov 23

PMID 16301549

Citations 21

Authors

J Fraser

A A Gason

J E Wraith

M B Delatycki

Affiliations

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Abstract

Aims: To determine the incidence, manifestations, and best management of sleep disturbance in Sanfilippo syndrome (mucopolysaccharidosis (MPS) type III).

Methods: Families were ascertained through the MPS societies of Australasia, the UK, and the USA. Questionnaires were sent by mail and were answered anonymously. Identical questions regarding sleep disturbance were asked about unaffected siblings to provide control data. Sleep disturbance was quantified by a total sleep disturbance score.

Results: A total of 141 responses were received; 91.5% of children with Sanfilippo syndrome had sleep disturbance and this was significantly higher than for their unaffected sibs; 77.5% of parents had used medication for this problem, with melatonin and antihistamines being most commonly used. Melatonin and benzodiazepines were reported as the most efficacious. Many different environmental modifications had been employed for this problem and some parents reported success with behavioural therapies.

Conclusions: Sleep disturbance is common, severe, and difficult to manage in Sanfilippo syndrome. Based on the parental responses and its safety profile, melatonin is the first line drug that should be tried. Behavioural therapy should be tried in all with Sanfilippo syndrome and sleep disturbance.

Citing Articles

Sanfilippo syndrome: consensus guidelines for clinical care.

Muschol N, Giugliani R, Jones S, Muenzer J, Smith N, Whitley C Orphanet J Rare Dis. 2022; 17(1):391.

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Abramova A, Attarian H, Dolgova S, Belyakova-Bodina A, Iakovenko E, Broutian A Sleep Sci. 2021; 14(Spec 1):97-100.

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Oxidative Stress in Mucopolysaccharidoses: Pharmacological Implications.

Pierzynowska K, Gaffke L, Cyske Z, Wegrzyn G, Buttari B, Profumo E Molecules. 2021; 26(18).

PMID: 34577086 PMC: 8468662. DOI: 10.3390/molecules26185616.

Parent Experiences of Sanfilippo Syndrome Impact and Unmet Treatment Needs: A Qualitative Assessment.

Porter K, ONeill C, Drake E, Parker S, Escolar M, Montgomery S Neurol Ther. 2020; 10(1):197-212.

PMID: 33263924 PMC: 8139994. DOI: 10.1007/s40120-020-00226-z.

Challenging behavior in mucopolysaccharidoses types I-III and day-to-day coping strategies: a cross sectional explorative study.

Hoffmann F, Hoffmann S, Kunzmann K, Ries M Orphanet J Rare Dis. 2020; 15(1):275.

PMID: 33008435 PMC: 7532084. DOI: 10.1186/s13023-020-01548-9.

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