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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.
Henes, Joerg; Oliveira, Maria Carolina; Labopin, Myriam; Badoglio, Manuela; Scherer, Hans Ulrich; Del Papa, Nicoletta; Daikeler, Thomas; Schmalzing, Marc; Schroers, Roland; Martin, Thierry; Pugnet, Gregory; Simoes, Belinda; Michonneau, David; Marijt, Erik W A; Lioure, Bruno; Olivier Bay, Jacques; Snowden, John A; Rovira, Montserrat; Huynh, Anne; Onida, Francesco; Kanz, Lothar; Marjanovic, Zora; Farge, Dominique.
Afiliación
  • Henes J; University Hospital Tuebingen; Intenal Medicine II, Tuebingen, Germany.
  • Oliveira MC; University of São Paulo, Ribeirão Preto, Brazil.
  • Labopin M; Saint Antoine Hospital, Université Pierre et Marie Curie, Paris, France.
  • Badoglio M; EBMT Paris Study Office, Hôpital St Antoine, Paris, France.
  • Scherer HU; Leiden University Medical Center, Department of Rheumatology; Leiden, Netherlands.
  • Del Papa N; Scleroderma Clinic, Osp. G. Pini, Department of Rheumatology, Milan, Italy.
  • Daikeler T; University and University Hospital of Basel, Department of Rheumatology, Basel, Switzerland.
  • Schmalzing M; University Hospital of Wuerzburg, Department of Rheumatology/Immunology, Wuerzburg, Germany.
  • Schroers R; University Hospital of Bochum, Med. Klinik, Bochum, Germany.
  • Martin T; Service de Medecine Interne et Immunologie Clinique, Hopitaux Universitaires de Strasbourg, France.
  • Pugnet G; CHU de Toulouse, Hopital Purpan, Service de Medecine Interne, Toulouse, France.
  • Simoes B; Dept. of Hematology, Ribeirão Preto Medical School, University of São Paulo, Brazil.
  • Michonneau D; Dept. of Hematology, Hopital Saint Louis and Université Paris 7, Denis Diderot, Paris, France.
  • Marijt EWA; Leiden University Medical Center, Department of Hematology, Leiden, Netherlands.
  • Lioure B; Strasbourg University Hospital, Department of Hematology, Strasbourg, France.
  • Olivier Bay J; CHU de Clermont Ferrand, Department of Hematology, Clermont Ferrand, France.
  • Snowden JA; Dept. of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK.
  • Rovira M; Department of Hematology, Hospital Clínic of Barcelona, Barcelona, Spain.
  • Huynh A; UCT Oncopole, Department of Haematology, Toulouse, France.
  • Onida F; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, University of Milan, Milano Italy.
  • Kanz L; University Hospital Tuebingen, Department of Internal Medicine II, Tuebingen, Germany.
  • Marjanovic Z; Saint Antoine Hospital, Department of Haematology, Paris, France.
  • Farge D; Assistance Publique-Hopitaux de Paris, Saint-Louis Hospital, Paris, France.
Haematologica ; 106(2): 375-383, 2021 02 01.
Article en En | MEDLINE | ID: mdl-31949011
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.
Asunto(s)

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Esclerodermia Sistémica / Enfermedades Autoinmunes / Trasplante de Células Madre Hematopoyéticas / Esclerodermia Difusa Tipo de estudio: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Adult / Aged / Humans Idioma: En Revista: Haematologica Año: 2021 Tipo del documento: Article País de afiliación: Alemania

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Esclerodermia Sistémica / Enfermedades Autoinmunes / Trasplante de Células Madre Hematopoyéticas / Esclerodermia Difusa Tipo de estudio: Clinical_trials / Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Adult / Aged / Humans Idioma: En Revista: Haematologica Año: 2021 Tipo del documento: Article País de afiliación: Alemania