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Orphanet Journal of Rare Diseases

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Open Access 01-12-2019 | Research

An observational study of pediatric healthcare burden in Angelman syndrome: results from a real-world study

Authors: Nasreen Khan, Raquel Cabo, Wen-Hann Tan, Regina Tayag, Lynne M. Bird

Published in: Orphanet Journal of Rare Diseases | Issue 1/2019

Abstract

Background

The objective of this study is to describe variations in the healthcare resource utilization (HRU) among individuals with Angelman syndrome (AS) over the first 12 years of life. Data for this study were drawn from the AS Natural History study (ASNHS), which is an observational study on the developmental progress, behavior, and medical morbidity of individuals with AS conducted over eight years. Caregiver-reported information on hospitalization, surgery, and medication utilization was used to assess HRU. Repeated measures mixed effect models were used to assess the relationship between age and probability of hospitalization, surgery, and prescription medication utilization.

Results

Mean age at study enrollment was 6 years of age and both sexes were equally represented. The mean number of visits per participant was three. Results from this study suggest that individuals with AS have a high HRU burden. Hospitalization and surgery burden were highest in the first year of life. Use of medications for seizures and sleep disturbance increased over time.

Conclusions

The study highlights the significant healthcare burden among individuals with AS. Future studies that estimate cost and caregiver burden associated with AS are needed to assess the lifelong economic impact of AS on families and healthcare system.

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Title: An observational study of pediatric healthcare burden in Angelman syndrome: results from a real-world study
Authors: Nasreen Khan
Raquel Cabo
Wen-Hann Tan
Regina Tayag
Lynne M. Bird
Publication date: 01-12-2019
Publisher: BioMed Central
Published in: Orphanet Journal of Rare Diseases / Issue 1/2019
Electronic ISSN: 1750-1172
DOI: https://doi.org/10.1186/s13023-019-1210-6

Keynote webinar | Spotlight on sleep in brain health

Springer Medicine

An observational study of pediatric healthcare burden in Angelman syndrome: results from a real-world study

Abstract

Background

Results

Conclusions

Keynote webinar | Spotlight on sleep in brain health

Springer Medicine

Abstract

Background

Results

Conclusions

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