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Gepubliceerd in: Quality of Life Research 12/2018

24-07-2018 | Review

Measuring quality of life in children with spinal muscular atrophy: a systematic literature review

Auteurs: Shalvaree Vaidya, Stefan Boes

Gepubliceerd in: Quality of Life Research | Uitgave 12/2018

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Abstract

Objectives

Spinal muscular atrophy (SMA) is a rare, hereditary, autosomal recessive neuromuscular disorder that, in its most severe forms, impacts infants and children. Once symptomatic, it is characterized clinically by a distinct inability to achieve motor milestones, such as the ability to lift the head, sit, stand, or walk. Quality of life (QOL) measurement in very young infants presents a particular challenge. Therefore, this review aims to highlight commonly used measurement tools and identifies future research opportunities for QOL measurement in SMA.

Methods

A systematic literature review was carried out focusing on the various tools used to measure QOL in children < 18 years of age with formally diagnosed SMA type I, II, or III. Although the disease area of interest was SMA, data on Duchenne’s muscular dystrophy were also included because of the rare nature of SMA.

Results

The Pediatric Quality of Life Inventory was the most commonly utilized tool to measure QOL in children; this included the generic and neuromuscular modules. No disease-specific tool to capture QOL in children with SMA was identified. Additionally, no measurement tools exist for very young infants (i.e., under 12 months) with SMA Type 1.

Conclusions

Evolving standards of care will lead to increased interest by stakeholders, on the methods used to measure QOL in infants and children across all types of SMA. Generic tools may not adequately capture QOL changes in SMA, especially given the age group affected by the disease. Further research is required to explore the scope for a disease-focused approach.

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Metagegevens
Titel
Measuring quality of life in children with spinal muscular atrophy: a systematic literature review
Auteurs
Shalvaree Vaidya
Stefan Boes
Publicatiedatum
24-07-2018
Uitgeverij
Springer International Publishing
Gepubliceerd in
Quality of Life Research / Uitgave 12/2018
Print ISSN: 0962-9343
Elektronisch ISSN: 1573-2649
DOI
https://doi.org/10.1007/s11136-018-1945-x