Exploring the diagnosis delay and ALS functional impairment at diagnosis as relevant criteria for clinical trial enrolment.
Amyotroph Lateral Scler Frontotemporal Degener
; 18(7-8): 519-527, 2017 11.
Article
en En
| MEDLINE
| ID: mdl-28762856
ABSTRACT
Objectives were i) to describe the phenotypic heterogeneity of incident amyotrophic lateral sclerosis (ALS) patients diagnosed in 2012 in French ALS centres; ii) to look at the associations between ALSFRS-R score and ALSFRS-R slope (ΔFS) at time of diagnosis with diagnosis delay, ALS phenotypes and Airlie House diagnosis criteria (AHDC); iii) to describe the rate of progression on ΔFS, according to diagnosis delay. METHODS:
Incident ALS cases diagnosed in French ALS centres were included. The rate of progression was evaluated as follows ΔFS = (48 - ALSFRS-R at time of diagnosis)/duration from onset to diagnosis (months). Fast and slow progressors were defined by ΔFS >1 and <0.5, respectively.RESULTS:
At time of diagnosis, 476 patients were classified into eight phenotypes bulbar (33.0%), spinal lumbar (28.2%), spinal cervical (23.1%), flail leg (4.4%), ALS/FTD (4.2%), possible flail arm (4.0%), respiratory (2.1%), dropped-head (1.0%). Median ΔFS (n = 358/476) was 1.0 [0.5-2.0]. ΔFS was associated with AHDC (p = 0.009), but not with clinical phenotype (p = 0.902). Stratification on diagnosis delay (<12 months or ≥18 months) allowed to differentiate fast progressors from slow progressors.CONCLUSION:
At time of inclusion in therapeutic trial closed to diagnosis, ΔFS or diagnosis delay may discriminate the rate of progression.Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Ensayos Clínicos como Asunto
/
Selección de Paciente
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Progresión de la Enfermedad
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Técnicas de Diagnóstico Neurológico
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Diagnóstico Tardío
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Esclerosis Amiotrófica Lateral
Tipo de estudio:
Diagnostic_studies
/
Observational_studies
/
Prevalence_studies
/
Risk_factors_studies
Límite:
Aged
/
Female
/
Humans
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Male
/
Middle aged
País/Región como asunto:
Europa
Idioma:
En
Revista:
Amyotroph Lateral Scler Frontotemporal Degener
Año:
2017
Tipo del documento:
Article
País de afiliación:
Francia