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Transplantation
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
Ferrata Storti Foundation
Haematologica 2021 Volume 106(2):375-383
Joerg Henes,1 Maria Carolina Oliveira,2 Myriam Labopin,3 Manuela Badoglio,4 Hans Ulrich Scherer,5 Nicoletta Del Papa,6 Thomas Daikeler,7 Marc Schmalzing,8 Roland Schroers,9 Thierry Martin,10 Gregory Pugnet,11 Belinda Simoes,12 David Michonneau,13 Erik W.A. Marijt,14 Bruno Lioure,15 Jacques Olivier Bay,16 John A Snowden,17 Montserrat Rovira,18 Anne Huynh,19 Francesco Onida,20 Lothar Kanz,21 Zora Marjanovic22 and Dominique Farge23 for the NISSC1 members
1Center for Interdisciplinary Clinical Immunology, Rheumatology and Auto-inflammatory Diseases and Department of Internal Medicine II (Oncology, Hematology, Immunology and Rheumatology), University Hospital, Tübingen, Germany; 2Ribeirão Preto Medical School, University of São Paulo, Ribeirão Preto, Brazil; 3EBMT Paris Study Office, Saint Antoine Hospital, Université Pierre et Marie Curie, INSERM UMR 938, Department of Haematology, Paris, France; 4EBMT Paris Study Office, Hôpital St Antoine, INSERM UMR 938, Paris, France; 5Leiden University Medical Center, Department of Rheumatology, Leiden, the Netherlands; 6Scleroderma Clinic, Ospedale Pini, Department of Rheumatology, Milan, Italy; 7University and University Hospital of Basel, Department of Rheumatology, Basel, Switzerland; 8University Hospital of Würzburg, Department of Rheumatology/Immunology, Würzburg, Germany 9Universitäts Klinik of Bochum, Med. Klinik, Bochum, Germany; 10Hôpitaux Universitaires de Strasbourg, Service de Médecine Interne et Immunologie Clinique, Centre de Référence des Maladies Auto-Immunes Systémiques Rares Est-Sud-Ouest (RESO), Strasbourg, France; 11CHU de Toulouse, Hôpital Purpan, Service de Médecine Interne, Toulouse, France; 12Ribeirão Preto Medical School, University of São Paulo, Division of Hematology, Ribeirão Preto, Brazil; 13Hôpital Saint Louis & Université Paris 7, Denis Diderot, Department of Hematology, Paris, France; 14Leiden University Medical Center, Department of Hematology, Leiden, the Netherlands; 15Strasbourg University Hospital, Department of Hematology, Strasbourg, France; 16CHU de Clermont Ferrand, Department of Hematology, Clermont Ferrand, France; 17Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK; 18Hospital Clínic of Barcelona, Department of Hematology, Barcelona, Spain; 19UCT Oncopole, Department of Hematology, Toulouse, France; 20Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico, University of Milan, Milan, Italy; 21University Hospital Tübingen, Department of Internal Medicine II (Oncology, Haematology, Immunology and Rheumatology), Tübingen, Germany 22Saint Antoine Hospital, Department of Hematology, Paris, France; 23Hôpital Saint-Louis, Assistance Publique des Hôpitaux de Paris, Centre de Référence des Maladies Auto- Immunes Systémiques Rares d’Ile-de-France, Filière ‘FAI2R’, Unité de Médecine Interne: Maladies Auto-immunes et Pathologie Vasculaire (UF 04), Université de Paris, EA 3518, Paris, France and 24Department of Medicine, McGill University, Montreal, Quebec, Canada
ABSTRACT
Three randomized controlled trials in early severe systemic sclerosis demonstrated that autologous hematopoietic stem cell transplan- tation 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 con- secutive adult patients with systemic sclerosis undergoing a first autolo- gous 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 over- all survival, non-relapse mortality, response and incidence of progression. Eighty patients with systemic sclerosis were included. The median dura-
Correspondence:
DOMINIQUE FARGE
dominique.farge-bancel@aphp.fr; dominique.farge@mcgill.ca
Received: June 22, 2019. Accepted: January 9, 2020. Pre-published: January 16, 2020.
https://doi.org/10.3324/haematol.2019.230128
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