Title :

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

Author(s) :

Abdallah, N. A. [Auteur]
Service de Médecine Interne [Saint-Louis]
Institut de Recherche Saint-Louis - Hématologie Immunologie Oncologie (Département de recherche de l’UFR de médecine ; ex- Institut Universitaire Hématologie-IUH) [IRSL]
Centre de référence des maladies auto-immunes systémiques rares d'Île-de-France / National Reference Center for Rare Systemic Autoimmune Diseases
Wang, M. A. B. [Auteur]
Lady Davis Institute for Medical Research [Montréal]
Lansiaux, P. [Auteur]
Institut de Recherche Saint-Louis - Hématologie Immunologie Oncologie (Département de recherche de l’UFR de médecine ; ex- Institut Universitaire Hématologie-IUH) [IRSL]
Puyade, M. [Auteur]
CIC Poitiers – Centre d'investigation clinique de Poitiers [CIC 1402]
Centre hospitalier universitaire de Poitiers = Poitiers University Hospital [CHU de Poitiers [La Milétrie]]
Berthier, S. [Auteur]
Centre Hospitalier Universitaire de Dijon - Hôpital François Mitterrand [CHU Dijon]
Terriou, Louis [Auteur]
Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286
Charles, C. [Auteur]
Institut de Recherche Saint-Louis - Hématologie Immunologie Oncologie (Département de recherche de l’UFR de médecine ; ex- Institut Universitaire Hématologie-IUH) [IRSL]
Burt, R. K. [Auteur]
Hudson, M. [Auteur]
Farge, D. [Auteur]
McGill University Health Center [Montreal] [MUHC]
Hopital Saint-Louis [AP-HP] [AP-HP]
Institut de Recherche Saint-Louis - Hématologie Immunologie Oncologie (Département de recherche de l’UFR de médecine ; ex- Institut Universitaire Hématologie-IUH) [IRSL]

Journal title :

Bone Marrow Transplantation

Abbreviated title :

Bone Marrow Transplant.

Volume number :

-

Pages :

-

Publication date :

2021-06-20

ISSN :

0268-3369

HAL domain(s) :

Sciences du Vivant [q-bio]

English abstract : [en]

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 ...
Show more >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, p = 0.018), mRSS (Δ mRSS: −16 versus −9, p = 0.02), and HAQ-DI (ΔHAQ-DI: −0.89 versus −0.2, p = 0.05) with no significant difference in OS, EFS, lung, heart and kidney function between the groups. In conclusion, this study demonstrates long term benefits of non-myeloablative AHSCT when assessed by the five longitudinal measures within GRCS affording direct primary endpoint comparison between ASTIS and SCOT.Show less >

Language :

Anglais

Peer reviewed article :

Oui

Audience :

Internationale

Popular science :

Non

Administrative institution(s) :

Université de Lille
Inserm
CHU Lille

Collections :

• Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286

Submission date :

2024-01-12T06:46:00Z
2024-02-27T09:33:21Z