Long term outcomes of the French ASTIS systemic sclerosis cohort using the global rank composite score
- 9 June 2021
- journal article
- research article
- Published by Springer Science and Business Media LLC in Bone Marrow Transplantation
- Vol. 56 (9), 2259-2267
- https://doi.org/10.1038/s41409-021-01355-1
Abstract
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.Keywords
This publication has 26 references indexed in Scilit:
- Cardiac involvement and treatment-related mortality after non-myeloablative haemopoietic stem-cell transplantation with unselected autologous peripheral blood for patients with systemic sclerosis: a retrospective analysisThe Lancet, 2013
- Haematopoietic SCT in severe autoimmune diseases: updated guidelines of the European Group for Blood and Marrow TransplantationBone Marrow Transplantation, 2011
- Autologous non-myeloablative haemopoietic stem-cell transplantation compared with pulse cyclophosphamide once per month for systemic sclerosis (ASSIST): an open-label, randomised phase 2 trialThe Lancet, 2011
- Clinical prediction of 5-year survival in systemic sclerosis: validation of a simple prognostic model in EUSTAR centresAnnals Of The Rheumatic Diseases, 2011
- Causes and risk factors for death in systemic sclerosis: a study from the EULAR Scleroderma Trials and Research (EUSTAR) databaseAnnals Of The Rheumatic Diseases, 2010
- Autologous hematopoietic stem cell transplantation for autoimmune diseases: an observational study on 12 years' experience from the European Group for Blood and Marrow Transplantation Working Party on Autoimmune DiseasesHaematologica, 2009
- SclerodermaThe New England Journal of Medicine, 2009
- Validation of French version of the Scleroderma Health Assessment Questionnaire (SSc HAQ)Clinical Rheumatology, 2004
- The use of the health assessment questionnaire (HAQ) to determine physical disability in systemic sclerosisArthritis & Rheumatism, 1991
- D-Penicillamine Therapy in Progressive Systemic Sclerosis (Scleroderma)Annals of Internal Medicine, 1982