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
| Autor: | Henes, J., Oliveira, M.C., Labopin, M., Badoglio, M., Scherer, H.U., Papa, N. del, Daikeler, T., Schmalzing, M., Schroers, R., Martin, T., Pugnet, G., Simoes, B., Michonneau, D., Marijt, E.W.A., Lioure, B., Bay, J.O., Snowden, J.A., Rovira, M., Huynh, A., Onida, F., Kanz, L., Marjanovic, Z., Farge, D., NISSC1 Members |
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| Přispěvatelé: | University of São Paulo (USP), CHU Saint-Antoine [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Centre International des greffes [CHU Saint-Antoine] (EBMT), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-CHU Saint-Antoine [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Sorbonne Université (SU), Leiden University Medical Center (LUMC), University of Basel (Unibas), CHU Strasbourg, Hôpital Purpan [Toulouse], CHU Toulouse [Toulouse], Hopital Saint-Louis [AP-HP] (AP-HP), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), CHU Clermont-Ferrand, Institut Universitaire du Cancer de Toulouse - Oncopole (IUCT Oncopole - UMR 1037), Université Toulouse III - Paul Sabatier (UT3), Université Fédérale Toulouse Midi-Pyrénées-Université Fédérale Toulouse Midi-Pyrénées-CHU Toulouse [Toulouse]-Institut National de la Santé et de la Recherche Médicale (INSERM), Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico |
| Rok vydání: | 2021 |
| Předmět: |
Adult
medicine.medical_specialty Transplantation Conditioning Cyclophosphamide [SDV]Life Sciences [q-bio] medicine.medical_treatment Aucun Hematopoietic stem cell transplantation Transplantation Autologous MESH: Scleroderma Systemic Article Autoimmune Diseases 03 medical and health sciences 0302 clinical medicine Autologous stem-cell transplantation Bone Marrow MESH: Autoimmune Diseases Internal medicine Clinical endpoint Humans Medicine Prospective Studies Progression-free survival MESH: Hematopoietic Stem Cell Transplantation MESH: Transplantation Conditioning Aged MESH: Aged 030203 arthritis & rheumatology MESH: Humans Scleroderma Systemic business.industry Hazard ratio Hematopoietic Stem Cell Transplantation MESH: Cyclophosphamide MESH: Adult Hematology TRANSPLANTE AUTÓLOGO MESH: Transplantation Autologous MESH: Prospective Studies 3. Good health Transplantation MESH: Scleroderma Diffuse 030220 oncology & carcinogenesis Scleroderma Diffuse MESH: Bone Marrow Stem cell business medicine.drug |
| Zdroj: | Repositório Institucional da USP (Biblioteca Digital da Produção Intelectual) Universidade de São Paulo (USP) instacron:USP Haematologica, 106(2), 375-383. FERRATA STORTI FOUNDATION Haematologica Haematologica, Ferrata Storti Foundation, 2021, 106 (2), pp.375-383. ⟨10.3324/haematol.2019.230128⟩ |
| ISSN: | 0390-6078 1592-8721 |
| DOI: | 10.3324/haematol.2019.230128⟩ |
| Popis: | 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 multicenter, 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 patients with systemic sclerosis undergoing a first autologous hematopoietic stem cell transplant between December 2012 and February 2016 were prospectively included in the study. The primary endpoint was progression-free survival. Secondary endpoints were overall survival, non-relapse mortality, response and incidence of progression. Eighty patients with systemic sclerosis were included. The median duration of the follow-up was 24 (range, 6-57) months after stem cell transplantation using cyclophosphamide plus antithymocyte globulin conditioning for all, with CD34+ selection in 35 patients. At 2 years, the progression- free survival rate was 81.8%, the overall survival rate was 90%, the response rate was 88.7% and the incidence of progression was 11.9%. The 100-day non-relapse mortality rate was 6.25% (n=5) with four deaths from cardiac events, including three due to cyclophosphamide toxicity. Modified Rodnan skin score and forced vital capacity improved with time (P24 and older age at transplantation were associated with lower progression-free survival (hazard ratios 3.32 and 1.77, respectively). CD34+-cell selection was associated with better response (hazard ratio 0.46). This study confirms the efficacy of autologous stem cell transplantation, using nonmyeloablative conditioning, in real-life practice for severe systemic sclerosis. Careful cardio-pulmonary assessment to identify organ involvement at the time of the patient’s referral, reduced cyclophosphamide doses and CD34+-cell selection may improve outcomes. The study was registered at ClinicalTrials.gov: NCT02516124. |
| Databáze: | OpenAIRE |
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