Long term outcomes of the French ASTIS systemic sclerosis cohort using the global rank composite score.
Journal
Bone marrow transplantation
ISSN: 1476-5365
Titre abrégé: Bone Marrow Transplant
Pays: England
ID NLM: 8702459
Informations de publication
Date de publication:
09 2021
09 2021
Historique:
received:
19
02
2021
accepted:
11
05
2021
revised:
29
04
2021
pubmed:
11
6
2021
medline:
14
10
2021
entrez:
10
6
2021
Statut:
ppublish
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, 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.
Identifiants
pubmed: 34108673
doi: 10.1038/s41409-021-01355-1
pii: 10.1038/s41409-021-01355-1
doi:
Substances chimiques
Cyclophosphamide
8N3DW7272P
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
2259-2267Informations de copyright
© 2021. The Author(s), under exclusive licence to Springer Nature Limited.
Références
Hao Y, Hudson M, Baron M, Carreira P, Stevens W, Rabusa C, et al. Early mortality in a multinational systemic sclerosis inception cohort. Arthritis Rheumatol. 2017;69:1067–77.
doi: 10.1002/art.40027
Gabrielli A, Avvedimento EV, Krieg T. Scleroderma. N. Engl J Med 2009;360:1989–2003.
doi: 10.1056/NEJMra0806188
Elhai M, Meune C, Boubaya M, Avouac J, Hachulla E, Balbir-Gurman A, et al. Mapping and predicting mortality from systemic sclerosis. Ann Rheum Dis. 2017;76:1897–905.
doi: 10.1136/annrheumdis-2017-211448
Fransen J, Popa-Diaconu D, Hesselstrand R, Carreira P, Valentini G, Beretta L, et al. Clinical prediction of 5-year survival in systemic sclerosis: validation of a simple prognostic model in EUSTAR centres. Ann Rheum Dis. 2011;70:1788–92.
doi: 10.1136/ard.2010.144360
Steen VD, Medsger TA, Rodnan GP. D-Penicillamine therapy in progressive systemic sclerosis (scleroderma): a retrospective analysis. Ann Intern Med. 1982;97:652–9.
doi: 10.7326/0003-4819-97-5-652
Poole JL, Steen VD. The use of the health assessment questionnaire (HAQ) to determine physical disability in systemic sclerosis. Arthritis Care Res 1991;4:27–31.
doi: 10.1002/art.1790040106
Georges C, Chassany O, Mouthon L, Tiev K, Toledano C, Meyer O, et al. Validation of French version of the Scleroderma Health Assessment Questionnaire (SSc HAQ). Clin Rheumatol. 2005;24:3–10.
doi: 10.1007/s10067-004-0942-3
Mousseaux E, Agoston-Coldea L, Marjanovic Z, Stanciu R, Deligny C, Perdrix L, et al. Left ventricle replacement fibrosis detected by CMR associated with cardiovascular events in systemic sclerosis patients. J Am Coll Cardiol. 2018;71:703–5.
doi: 10.1016/j.jacc.2017.11.061
Tyndall AJ, Bannert B, Vonk M, Airo P, Cozzi F, Carreira PE, et al. Causes and risk factors for death in systemic sclerosis: a study from the EULAR Scleroderma Trials and Research (EUSTAR) database. Ann Rheum Dis. 2010;69:1809–15.
doi: 10.1136/ard.2009.114264
Burt RK, Shah SJ, Dill K, Grant T, Gheorghiade M, Schroeder J, et al. Autologous non-myeloablative haemopoietic stem-cell transplantation compared with pulse cyclophosphamide once per month for systemic sclerosis (ASSIST): an open-label, randomised phase 2 trial. Lancet. 2011;378:498–506.
doi: 10.1016/S0140-6736(11)60982-3
van Laar JM, Farge D, Sont JK, Naraghi K, Marjanovic Z, Larghero J, et al. Autologous hematopoietic stem cell transplantation vs intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis: a randomized clinical trial. JAMA. 2014;311:2490–8.
doi: 10.1001/jama.2014.6368
Sullivan KM, Goldmuntz EA, Keyes-Elstein L, McSweeney PA, Pinckney A, Welch B, et al. Myeloablative autologous stem-cell transplantation for severe scleroderma. N. Engl J Med. 2018;378:35–47.
doi: 10.1056/NEJMoa1703327
Duarte RF, Labopin M, Bader P, Basak GW, Bonini C, Chabannon C, et al. Indications for haematopoietic stem cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2019. Bone Marrow Transplant. 2019;54:1525–52.
doi: 10.1038/s41409-019-0516-2
Kowal-Bielecka O, Fransen J, Avouac J, Becker M, Kulak A, Allanore Y, et al. Update of EULAR recommendations for the treatment of systemic sclerosis. Ann Rheum Dis. 2017;76:1327–39.
doi: 10.1136/annrheumdis-2016-209909
Majhail NS, Farnia SH, Carpenter PA, Champlin RE, Crawford S, Marks DI, et al. Indications for Autologous and Allogeneic Hematopoietic Cell Transplantation: Guidelines from the American Society for Blood and Marrow Transplantation. Biol Blood Marrow Transplant. 2015;21:1863–9.
doi: 10.1016/j.bbmt.2015.07.032
Denton CP. Challenges in systemic sclerosis trial design. Semin Arthritis Rheumatism. 2019;49:S3–7.
doi: 10.1016/j.semarthrit.2019.09.019
Snowden JA, Saccardi R, Allez M, Ardizzone S, Arnold R, Cervera R, et al. Haematopoietic SCT in severe autoimmune diseases: updated guidelines of the European Group for Blood and Marrow Transplantation. Bone Marrow Transplant. 2012;47:770–90.
doi: 10.1038/bmt.2011.185
Steen VD, Medsger TA. Improvement in skin thickening in systemic sclerosis associated with improved survival. Arthritis Rheumatism. 2001;44:2828–35.
doi: 10.1002/1529-0131(200112)44:12<2828::AID-ART470>3.0.CO;2-U
Khanna D, Seibold J, Goldin J, Tashkin DP, Furst DE, Wells A. Interstitial lung disease points to consider for clinical trials in systemic sclerosis. Rheumatology. 2017;56:v27–32.
doi: 10.1093/rheumatology/kex203
Khanna D, Berrocal VJ, Giannini EH, Seibold JR, Merkel PA, Mayes MD, et al. The American College of Rheumatology Provisional Composite Response Index for Clinical Trials in Early Diffuse Cutaneous Systemic Sclerosis: ACR PROVISIONAL COMPOSITE INDEX FOR SCLERODERMA CLINICAL TRIALS. Arthritis Rheumatology 2016;68:299–311.
doi: 10.1002/art.39501
Khanna D, Huang S, Lin CJF, Spino C. New composite endpoint in early diffuse cutaneous systemic sclerosis: revisiting the provisional American College of Rheumatology Composite Response Index in Systemic Sclerosis. Ann Rheum Dis. 2021;80:641–50.
doi: 10.1136/annrheumdis-2020-219100
Finkelstein DM, Schoenfeld DA. Combining mortality and longitudinal measures in clinical trials. Stat Med. 1999;18:1341–54.
doi: 10.1002/(SICI)1097-0258(19990615)18:11<1341::AID-SIM129>3.0.CO;2-7
Burt RK, Oliveira MC, Shah SJ, Moraes DA, Simoes B, Gheorghiade M, et al. 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 analysis. Lancet. 2013;381:1116–24.
doi: 10.1016/S0140-6736(12)62114-X
Farge D, Burt RK, Oliveira M-C, Mousseaux E, Rovira M, Marjanovic Z, et al. Cardiopulmonary assessment of patients with systemic sclerosis for hematopoietic stem cell transplantation: recommendations from the European Society for Blood and Marrow Transplantation Autoimmune Diseases Working Party and collaborating partners. Bone Marrow Transplant. 2017;52:1495–503.
doi: 10.1038/bmt.2017.56
Farge D, Labopin M, Tyndall A, Fassas A, Mancardi GL, Van Laar J, et al. 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 Diseases. Haematologica. 2010;95:284–92.
doi: 10.3324/haematol.2009.013458
Burt RK, Farge D. Autologous HSCT is efficacious, but can we make it safer? Nat Rev Rheumatol. 2018;14:189–91.
doi: 10.1038/nrrheum.2018.34