Long term outcomes of the French ASTIS systemic sclerosis cohort using the global rank composite score

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TitreLong term outcomes of the French ASTIS systemic sclerosis cohort using the global rank composite score
Type de publicationJournal Article
Year of Publication2021
AuteursAbdallah NAit, Wang M, Lansiaux P, Puyade M, Berthier S, Terriou L, Charles C, Burt RK, Hudson M, Farge D
JournalBONE MARROW TRANSPLANTATION
Volume56
Pagination2259-2267
Date PublishedSEP
Type of ArticleArticle
ISSN0268-3369
Résumé

{Two randomised trials (ASTIS, SCOT) of Autologous Hematopoietic Stem Cell Transplantation (AHSCT) versus monthly Cyclophosphamide for severe Systemic Sclerosis (SSc) patients used similar inclusion criteria, but different primary endpoints: event-free-survival (EFS) at 24 months in ASTIS versus the global rank composite score (GRCS) at 54 months in SCOT. Here we analysed the French ASTIS cohort (n = 49) outcome using the same GRCS endpoint as reported in SCOT. All patients, randomised to AHSCT (n = 26) or Cyclophosphamide (n = 23), were evaluated for the non-parametric GRCS endpoint based on: death, EFS, forced vital capacity (FVC), Health Assessment Questionnaire Disability Index (HAQ-DI) and modified Rodnan skin score (mRSS) at 60 months. Secondary endpoints were: EFS, overall survival (OS), HAQ DI and organ status. In intention-to-treat analysis, the GRCS demonstrated superiority for AHSCT (median: 9 versus -19
DOI10.1038/s41409-021-01355-1