Autologous stem cell transplantation for progressive systemic sclerosis: a prospective non-interventional study from the European Society for Blood and Marrow Transplantation Autoimmune Disease Working Party
Haematologica, vol. 106, pp. 375–383
Abstract
Three randomized controlled trials in early severe systemic sclerosis demonstrated that autologous hematopoietic stem cell transplantation was superior to standard cyclophosphamide therapy. This European Society for Blood and Marrow Transplantation multi-center prospective non-interventional study was designed to further decipher efficacy and safety of this procedure for severe systemic sclerosis patients in real-life practice and to search for prognostic factors. All consecutive adult systemic sclerosis patients undergoing a first autologous hematopoietic stem cell transplantation between December 2012 and February 2016 were prospectively included in the study. Primary endpoint was progression free survival. Secondary endpoints were overall survival, non-relapse mortality, response and incidence of progression. Eighty systemic sclerosis patients were included. Median follow-up duration was 24 (6-57) months after stem cell transplantation using cyclophosphamide plus antithymocyte globulins conditioning for all, with CD34+ selection in 35 patients. At 2 years, progression free survival was 81.8%, overall survival was 90%, response was 88.7% and incidence of progression was 11.9%. The 100 days non-relapse mortality was 6.25% (n=5) with four deaths from cardiac event, including three due to cyclophosphamide toxicity. Modified Rodnan skin score and forced vital capacity improved with time (p< 0.001). By multivariate analysis, baseline skin score >24 and older age at transplant were associated with lower progression free survival (Hazard ration 3.32) and 1.77, respectively). CD34+-selection was associated with better response (Hazard ration: 0.46). This study confirms the efficacy of autologous stem cell transplantation in real-life practice for severe systemic sclerosis using non myeloablative conditioning. Careful cardio-pulmonary assessment to identify organ involvement at patient referral, reduced cyclophosphamide doses and CD34+ selection may improve outcomes. The study was registered at ClinicalTrials.gov: NCT02516124.
Authors 23
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Universitätsklinikum Tübingen · University of Tübingen
Affiliation as printed
University Hospital Tuebingen; Intenal Medicine II, Tuebingen, Germany
Intenal Medicine II, Tuebingen, Germany
University Hospital Tuebingen
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Affiliation as printed
University of São Paulo, Ribeirão Preto, Brazil
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Hôpital Saint-Antoine · Université Pierre-et-Marie-Curie
Affiliation as printed
Saint Antoine Hospital, Université Pierre et Marie Curie, Paris, France
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Hôpital Saint-Antoine · European Society for Blood and Marrow Transplantation
Affiliation as printed
EBMT Paris Study Office, Hôpital St Antoine, Paris, France
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Leiden University Medical Center
Affiliation as printed
Leiden University Medical Center, Department of Rheumatology; Leiden, Netherlands
Leiden University Medical Center, Department of Rheumatology
Leiden, Netherlands
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Affiliation as printed
Scleroderma Clinic, Osp. G. Pini, Department of Rheumatology, Milan, Italy
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Affiliation as printed
University and University Hospital of Basel, Department of Rheumatology, Basel, Switzerland
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Affiliation as printed
University Hospital of Wuerzburg, Department of Rheumatology/Immunology, Wuerzburg, Germany
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University Hospitals of the Ruhr-University of Bochum
Affiliation as printed
University Hospital of Bochum, Med. Klinik, Bochum, Germany
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Hôpitaux Universitaires de Strasbourg
Affiliation as printed
Service de Medecine Interne et Immunologie Clinique, Hopitaux Universitaires de Strasbourg, France
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Centre Hospitalier Universitaire de Toulouse · Hôpital Purpan
Affiliation as printed
CHU de Toulouse, Hopital Purpan, Service de Medecine Interne, Toulouse, France
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Affiliation as printed
Dept. of Hematology, Ribeirão Preto Medical School, University of São Paulo, Brazil
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Hôpital Saint-Louis · Université Paris Diderot
Affiliation as printed
Dept. of Hematology, Hopital Saint Louis and Université Paris 7, Denis Diderot, Paris, France
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Leiden University Medical Center
Affiliation as printed
Leiden University Medical Center, Department of Hematology, Leiden, Netherlands
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Hôpitaux Universitaires de Strasbourg
Affiliation as printed
Strasbourg University Hospital, Department of Hematology, Strasbourg, France
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Centre Hospitalier Universitaire de Clermont-Ferrand
Affiliation as printed
CHU de Clermont Ferrand, Department of Hematology, Clermont Ferrand, France
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Sheffield Teaching Hospitals NHS Foundation Trust
Affiliation as printed
Dept. of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK
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Affiliation as printed
Department of Hematology, Hospital Clínic of Barcelona, Barcelona, Spain
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Institut universitaire du cancer de Toulouse Oncopole
Affiliation as printed
UCT Oncopole, Department of Haematology, Toulouse, France
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University of Milan · Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Affiliation as printed
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, University of Milan, Milano Italy
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Affiliation as printed
University Hospital Tuebingen, Department of Internal Medicine II, Tuebingen, Germany
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Affiliation as printed
Saint Antoine Hospital, Department of Haematology, Paris, France
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Assistance Publique – Hôpitaux de Paris · Hôpital Saint-Louis
Affiliation as printed
Assistance Publique-Hopitaux de Paris, Saint-Louis Hospital, Paris, France
Cited by 119 stored of 124
Cited by patents worldwide 2 (Lens.org)
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