Health utility decreases with increasing clinical stage in amyotrophic lateral sclerosis

Abstract

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease typically causing death within three years. Understanding the impact of disease on patients using health utility at different stages of ALS would allow meaningful cost-benefit analysis of new potential therapies. A common health-related quality of life measurement, developed and validated for the UK, is the EQ-5D. Using clinical trial data from the LiCALS study, we calculated health utility using the EQ-5D for each King's ALS clinical stage from 214 patients. We analysed whether health utility, and other health-related measures, significantly changed between each of the clinical stages. Results showed that mean health utility decreased by 0.487 (the scale runs from 1 to – 0.594) between clinical stages 2A and 4. Emotional states, measured using the Hospital Anxiety and Depression Scale (HADS), showed worsening depression and anxiety scores as ALS progressed. Age of onset, disease onset, gender and treatment group were not predictors of EQ-5D, depression or anxiety.

In conclusion, increasing severity of King’s ALS Clinical Stage is associated with a progressive decrease in EQ-5D health utility. This is useful for cost-benefit analysis of new therapies and validates this ALS clinical staging system.

Key words::

• Clinical staging
• amyotrophic lateral sclerosis
• health utility
• quality of life
• EQ-5D

Acknowledgements

We thank the patients with ALS/MND who participated in the LiCALS trial with the support of their families. We thank the Motor Neurone Disease Association of England, Wales and Northern Ireland, the UKMND-LiCALS study group, the ALS Association, National Institute for Health Research (NIHR) Dementias and Neurodegenerative Disease Research Network (DeNDRoN), the Angel Fund and the ALS Therapy Alliance for support. This work received funding from the European Community's Health Seventh Framework Programme. AAC receives salary support from the NIHR Dementia Biomedical Research Unit at South London and Maudsley NHS Foundation Trust and King's College London. MRT receives funding from the Medical Research Council and Motor Neurone Disease Association UK Lady Edith Wolfson Fellowship. PJS is supported as an NIHR Senior Investigator. The views expressed are those of the authors and not necessarily those of the National Health Service, the NIHR, or the Department of Health.

Declaration of interest: Carolyn Young receives personal payment for consultancy to Biogen Idec including ALS Staging Workshop and institutional payment as grant income from MNDA and trials re-imbursement from commercial organizations.

Pamela Shaw receives consultancy payments from Biogen Idec, Cytokinetics Inc., Sanofi-Aventis, Vertex Pharmaceuticals, ONO Pharma.

P. Nigel Leigh receives consultancy fees and expenses from GSK, Biogen-Idec and NeuroNov, for five years.

Martin R. Turner has received paid consultancy from Biogen Idec for ALS Staging Workshop.

Ammar Al-Chalabi has received personal payment for consultancy to Biogen Idec and Cytokinetics Inc. He has received royalties from books – The Brain (Oneworld Publications) and Complex Disease Genetics, a Laboratory Manual (Cold Spring Harbor Laboratory Press). Institutional payments include grant income from various charities and governmental organizations.

The authors alone are responsible for the content and writing of the paper.