A proposed staging system for amyotrophic lateral sclerosis
Roche, José C. (Hospital Universitario Miguel Servet (Saragossa))
Rojas-Garcia, Ricardo (Institut d'Investigació Biomèdica Sant Pau)
Scott, Kirsten M. (MRC King's College London)
Scotton, William (King's College London)
Ellis, Catherine E. (King's College Hospital NHS Foundation Trust)
Burman, Rachel (King's College Hospital NHS Foundation Trust)
Wijesekera, Lokesh (King's College London)
Turner, Martin R. (Oxford University)
Leigh, P.Nigel (University of Sussex)
Shaw, Christopher E. (King's College London)
Al-Chalabi, Ammar (King's College London)
Universitat Autònoma de Barcelona. Departament de Medicina

Date:	2012
Abstract:	Amyotrophic lateral sclerosis is a neurodegenerative disorder characterized by progressive loss of upper and lower motor neurons, with a median survival of 2-3 years. Although various phenotypic and research diagnostic classification systems exist and several prognostic models have been generated, there is no staging system. Staging criteria for amyotrophic lateral sclerosis would help to provide a universal and objective measure of disease progression with benefits for patient care, resource allocation, research classifications and clinical trial design. We therefore sought to define easily identified clinical milestones that could be shown to occur at specific points in the disease course, reflect disease progression and impact prognosis and treatment. A tertiary referral centre clinical database was analysed, consisting of 1471 patients with amyotrophic lateral sclerosis seen between 1993 and 2007. Milestones were defined as symptom onset (functional involvement by weakness, wasting, spasticity, dysarthria or dysphagia of one central nervous system region defined as bulbar, upper limb, lower limb or diaphragmatic), diagnosis, functional involvement of a second region, functional involvement of a third region, needing gastrostomy and non-invasive ventilation. Milestone timings were standardized as proportions of time elapsed through the disease course using information from patients who had died by dividing time to a milestone by disease duration. Milestones occurred at predictable proportions of the disease course. Diagnosis occurred at 35% through the disease course, involvement of a second region at 38%, a third region at 61%, need for gastrostomy at 77% and need for non-invasive ventilation at 80%. We therefore propose a simple staging system for amyotrophic lateral sclerosis. Stage 1: symptom onset (involvement of first region); Stage 2A: diagnosis; Stage 2B: involvement of second region; Stage 3: involvement of third region; Stage 4A: need for gastrostomy; and Stage 4B: need for non-invasive ventilation. Validation of this staging system will require further studies in other populations, in population registers and in other clinic databases. The standardized times to milestones may well vary between different studies and populations, although the stages themselves and their meanings are likely to remain unchanged. © The Author (2012).
Grants:	Ministerio de Ciencia e Innovación BA10/00047
Rights:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original.
Language:	Anglès
Document:	Article ; recerca ; Versió publicada
Subject:	Amyotrophic lateral sclerosis ; El Escorial criteria ; Motor neuron disease ; Natural history ; Staging
Published in:	Brain, Vol. 135 Núm. 3 (march 2012) , p. 847-852, ISSN 1460-2156

DOI: 10.1093/brain/awr351
PMID: 22271664

6 p, 176.9 KB

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Research literature > UAB research groups literature > Research Centres and Groups (research output) > Health sciences and biosciences > Institut de Recerca Sant Pau
Articles > Research articles
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