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  • Review Article
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The emerging agenda of stratified medicine in neurology

Nature Reviews Neurology volume 10, pages 15–26 (2014)Cite this article

Subjects

Key Points

  • Stratified medicine focuses on the scientifically based classification of disease and treatment response, and is well suited to the practice of neurology

  • Stratification of patients on the basis of genetic and clinical characteristics and imaging has already had an impact on the cost-effectiveness of preventative and therapeutic interventions in patients with primary and secondary stroke

  • Pharmacogenetic stratification has been introduced to manage warfarin treatment in patients at risk of stroke and to reduce risks of severe cutaneous reactions in patients treated with antiepileptic drugs

  • Stratification of patients with multiple sclerosis on the basis of baseline characteristics and short-term pharmacodynamic responses could increase treatment benefits, limit adverse effects and reduce the costs of treatment

  • Brain imaging and other biomarkers enable specific diagnoses of dementia syndromes, and could be used to stratify healthy individuals at risk of developing dementia for early preventative interventions

  • Major challenges to clinical implementation of stratified neurology exist, but its high value, together with technological and scientific advances, justify a concerted effort to accelerate development in this area

Abstract

Stratified medicine can reduce the costs of neurological care, bringing benefits to both patients and physicians. The availability of routine genetic testing, new biomarkers and advanced imaging, as well as new technologies for patient-centred data collection, has expanded the potential for patient stratification. Several neurology subspecialities, including stroke, epilepsy and behavioural neurology, have already applied stratification for disease prognosis, optimization of disease management and reduction of treatment-related adverse events. Stratification approaches could improve the cost-effectiveness of neurological care that involves treatments with high costs or risks of adverse reactions, as well as guide the use of emerging, highly individualized therapies. There are still major challenges in the development of clinically actionable stratification concepts, and practical barriers can limit adoption of these concepts into clinical practice. However, improved technologies and disease understanding are making more precise stratification practical. We believe that neurologists should become leaders in the development and validation of these practices, and that use of these approaches should be part of a broader strategy for addressing both the growing needs of an ageing population and the rising pressures for rapid improvements in the cost-effectiveness of therapeutics.

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Figure 1: The range of factors that should be addressed by stratified medicine approaches.
Figure 2: Stratification of patients for efficacy can substantially reduce the number needed to treat for benefit.
Figure 3: PET imaging in the diagnosis of Alzheimer disease.

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Acknowledgements

P. M. Matthews is grateful for support from the Medical Research Council to develop the OPTIMISE consortium for stratified medicine in MS. He received considerable input concerning stratified medicine approaches from many involved in the consortium, particularly R. Horwitz and J. Abell (GSK), R. Hyde (Biogen), G. Giovanonni (Queen Mary College London), L. Fugger and M. Craner (University of Oxford), C. Green (Exeter University), M. P. Sormani (Genoa University), D. Miller (University College London) and S. Baranzini (UCSF). This work also benefited from support to Imperial College London from the UK NIHR Biomedical Research Centres Scheme.

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Authors and Affiliations

  1. Division of Brain Sciences, Department of Medicine, Imperial College London, Burlington Danes, Hammersmith Hospital, DuCane Road, London, W12 0NN, UK

    Paul M. Matthews

  2. Neurology Imaging Unit, Hammersmith Hospital, DuCane Road, London, W12 0NN, UK

    Paul Edison

  3. Stroke Unit, Charing Cross Hospital, St Dunstan's Road, London, W6 8RP, UK

    Olivia C. Geraghty

  4. Department of Neurology, Charing Cross Hospital, St Dunstan's Road, London, W6 8RP, UK

    Michael R. Johnson

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All authors researched data for the article and made substantial contributions to discussion of the content, writing of the article and to review and/or editing of the manuscript before submission.

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Correspondence to Paul M. Matthews.

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Competing interests

P. M. Matthews is a part-time employee of GlaxoSmithKline Research and Development, Ltd, and holds stocks and options in the company. He has received speakers honoraria from Novartis and Biogen. P. Edison has received consultancy honoraria from Piramal Life Sciences. M. R. Johnson has received Advisory Board and speakers honoraria from GlaxoSmithKline, UCB Pharma and Esai.

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Matthews, P., Edison, P., Geraghty, O. et al. The emerging agenda of stratified medicine in neurology. Nat Rev Neurol 10, 15–26 (2014). https://doi.org/10.1038/nrneurol.2013.245

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