Hematopoietic stem cell transplantation (HSCT) has evolved for >20 years as a specific treatment of patients with autoimmune disease (AD). Using European Society for Blood and Marrow Transplantation registry data, we summarized trends and identified factors influencing activity and outcomes in patients with AD undergoing first autologous HSCT (n = 1951; median age, 37 years [3-76]) and allogeneic HSCT (n = 105; median age, 12 years [<1-62]) in 247 centers in 40 countries from 1994 to 2015. Predominant countries of activity were Italy, Germany, Sweden, the United Kingdom, The Netherlands, Spain, France, and Australia. National activity correlated with the Human Development Index (P = .006). For autologous HSCT, outcomes varied significantly between diseases. There was chronological improvement in progression-free survival (PFS, P < 10-5), relapse/progression (P < 10-5), and nonrelapse mortality (P = .01). Health care expenditure was associated with improved outcomes in systemic sclerosis and multiple sclerosis (MS). On multivariate analysis selecting adults for MS, systemic sclerosis, and Crohn disease, better PFS was associated with experience (≥23 transplants for AD, P = .001), learning (time from first HSCT for AD ≥6 years, P = .01), and Joint Accreditation Committee of the International Society for Cellular Therapy and European Society for Blood and Marrow Transplantation accreditation status (P = .02). Despite improved survival over time (P = .02), allogeneic HSCT use remained low and largely restricted to pediatric practice. Autologous HSCT has evolved into a treatment modality to be considered alongside other modern therapies in severe AD. Center experience, accreditation, interspecialty networking, and national socioeconomic factors are relevant for health service delivery of HSCT in AD.

Centre de Référence des Maladies auto immunes systémiques Rares d'Ile de France Filière FAI2R Paris France

Department of Bone Marrow Transplantation and Onco Hematology Maria Sklodowska Curie Institute Oncology Center Gliwice Branch Gliwice Poland

Department of Haematology Hospital Clinic Barcelona Spain

Department of Haematology Saint Antoine Hospital INSERM Unité Mixte de Recherche 938 Université Pierre et Marie Curie Paris France

Department of Haematology Sheffield Teaching Hospitals National Health Service Foundation Trust Sheffield United Kingdom

Department of Hematology Oncology and Internal Diseases Medical University of Warsaw Warsaw Poland

Department of Neuroscience Uppsala University Uppsala Sweden

Department of Scientific Studies and Clinical Technologies Dmitry Rogachev National Research Center of Pediatric Hematology Oncology and Immunology Moscow Russia

Divisie Kinderen Cluster Immunologie Reumatologie Hematologie en Infectiologie Wilhelmina Kinderziekenhuis Utrecht The Netherlands

European Society for Blood and Marrow Transplantation Paris Study Office and

Haematology Department Careggi University Hospital Florence Italy

Haematology Department St Vincent's Health Network Darlinghurst NSW Australia

Hematology and Bone Marrow Transplantation Unit Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele Scientific Institute Milan Italy

Joint Accreditation Committee of the International Society for Cellular Therapy and EBMT Office Barcelona Spain

Kings Health Partners Department of Haematology Guys Hospital London United Kingdom

Klinik für Rheumatologie und Klinische Immunologie Charité Universitätsmedizin Berlin Germany

Přednosta Interní hematologické kliniky Univerzity Karlovy a Fakultní nemocnice Královské Vinohrady Prague Czech Republic

Service d'Hematologie clinique et Therapie Cellulaire Hopital Saint Antoine Assistance Publique Hôpitaux de Paris Paris France

Stammzelltransplantation und Immunologie Klinik für Kinder und Jugendmedizin Frankfurt am Main Germany

Unité Clinique de Médecine Interne Maladies Auto immunes et Pathologie Vasculaire Unité Fonctionnelle 04 Hôpital Saint Louis AP HP INSERM Unité Mixte de Recherche Scientifique 1160 Paris Denis Diderot University Paris France; and

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