Literature DB >> 28762856

Exploring the diagnosis delay and ALS functional impairment at diagnosis as relevant criteria for clinical trial enrolment.

Bello Hamidou1,2, Benoit Marin1,2,3, Geraldine Lautrette4, Marie Nicol1,4, William Camu5, Philippe Corcia6, Marie-Christine Arnes-Bes7, Christine Tranchant8, Pierre Clavelou9, Didier Hannequin10, Giroud Maurice11, Katell Beauvais12, Jean-Christophe Antoine13, Véronique Danel-Brunaud14, Fausto Viader15, Pierre-Marie Preux1,2,3, Philippe Couratier1,2,4.   

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.

Entities:  

Keywords:  ALSFRS-R slope; Amyotrophic lateral sclerosis; clinical trial; diagnosis delay; phenotype

Mesh:

Year:  2017        PMID: 28762856     DOI: 10.1080/21678421.2017.1353098

Source DB:  PubMed          Journal:  Amyotroph Lateral Scler Frontotemporal Degener        ISSN: 2167-8421            Impact factor:   4.092


  3 in total

1.  Epidemiological, Clinical and Genetic Features of ALS in the Last Decade: A Prospective Population-Based Study in the Emilia Romagna Region of Italy.

Authors:  Giulia Gianferrari; Ilaria Martinelli; Elisabetta Zucchi; Cecilia Simonini; Nicola Fini; Marco Vinceti; Salvatore Ferro; Annalisa Gessani; Elena Canali; Franco Valzania; Elisabetta Sette; Maura Pugliatti; Valeria Tugnoli; Lucia Zinno; Salvatore Stano; Mario Santangelo; Silvia De Pasqua; Emilio Terlizzi; Donata Guidetti; Doriana Medici; Fabrizio Salvi; Rocco Liguori; Veria Vacchiano; Mario Casmiro; Pietro Querzani; Marco Currò Dossi; Alberto Patuelli; Simonetta Morresi; Marco Longoni; Patrizia De Massis; Rita Rinaldi; Annamaria Borghi; Amedeo Amedei; Jessica Mandrioli
Journal:  Biomedicines       Date:  2022-03-31

2.  Intraspinal Transplantation of the Adipose Tissue-Derived Regenerative Cells in Amyotrophic Lateral Sclerosis in Accordance with the Current Experts' Recommendations: Choosing Optimal Monitoring Tools.

Authors:  Magdalena Kuzma-Kozakiewicz; Andrzej Marchel; Anna Kaminska; Malgorzata Gawel; Jan Sznajder; Anna Figiel-Dabrowska; Arkadiusz Nowak; Edyta Maj; Natalia Ewa Krzesniak; Bartlomiej H Noszczyk; Krystyna Domanska-Janik; Anna Sarnowska
Journal:  Stem Cells Int       Date:  2018-08-12       Impact factor: 5.443

3.  High-dose pharmaceutical grade biotin (MD1003) in amyotrophic lateral sclerosis: A pilot study.

Authors:  Raul Juntas-Morales; Nicolas Pageot; Abdelkarim Bendarraz; Sébastien Alphandéry; Frédéric Sedel; Stéphanie Seigle; William Camu
Journal:  EClinicalMedicine       Date:  2020-01-27
  3 in total

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