A critical review of patient and parent caregiver oriented tools to assess health-related quality of life, activity of daily living and caregiver burden in spinal muscular atrophy

Marika Pane, Eugenio Maria Mercuri, Anna Lia Frongia, Laura Antonaci, Maria Carmela Pera, Giorgia Coratti, Sonia Messina, Amy Pasternak, Matthew Civitello, Jacqueline Montes, Anna Mayhew, Richard Finkel, Francesco Muntoni

Research output: Contribution to journalArticle

Abstract

The positive outcome of different therapeutic approaches for spinal muscular atrophy (SMA) in clinical trials and in clinical practice have highlighted the need to establish if functional changes are associated with possible changes of patient health-related quality of life or have an effect on activities of daily living and caregiver burden. The aim of this paper is to provide a critical review of the tools previously or currently used to measure quality of life, activity of daily living, and caregiver burden in SMA. We identified 36 measures. Only 6 tools were specifically developed for SMA while the others had been used and at least partially validated in wider groups of neuromuscular disorders including SMA. Twelve of the 36 focused on health-related quality of life, 5 on activities of daily living and 9 on caregiver burden. Ten included a combination of items. The review provides a roadmap of the different tools indicating their suitability for different SMA types or age groups. Scales assessing activities of daily living and care burden can provide patients and carers perspective on functional changes over time that should be added to the observer rated scales used in clinic.
Original languageEnglish
Pages (from-to)940-950
Number of pages11
JournalNeuromuscular Disorders
Volume29
DOIs
Publication statusPublished - 2019

Keywords

  • Activity of daily living
  • Care burden, Personalized medicine
  • Spinal muscular atrophy
  • Quality of life
  • Health related quality of life

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