Autologous stem cell transplantation in the treatment of systemic sclerosis: report from the EBMT/EULAR Registry

• Published in: Annals of the rheumatic diseases 2004-08, Vol.63 (8), p.974-981
• Main Authors: Farge, D, Passweg, J, van Laar, J M, Marjanovic, Z, Besenthal, C, Finke, J, Peter, H H, Breedveld, F C, Fibbe, W E, Black, C, Denton, C, Koetter, I, Locatelli, F, Martini, A, Schattenberg, A V N, van den Hoogen, F, van de Putte, L, Lanza, F, Arnold, R, Bacon, P A, Bingham, S, Ciceri, F, Didier, B, Diez-Martin, J L, Emery, P, Feremans, W, Hertenstein, B, Hiepe, F, Luosujärvi, R, Leon Lara, A, Marmont, A, Martinez, A M, Pascual Cascon, H, Bocelli-Tyndall, C, Gluckman, E, Gratwohl, A, Tyndall, A
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Aged; antithymocyte globulins; ATG; autoimmune disease; autoimmune diseases; autologous bone marrow transplantation; Biological and medical sciences; carbon monoxide transfer factor; Child; Clinical Trials, Phase I as Topic; Clinical Trials, Phase II as Topic; Diseases of the osteoarticular system; EBMT; Europe - epidemiology; European Group for Blood and Marrow Transplantation; Extended Report; Female; G-CSF; granulocyte-colony stimulating factor; haematopoietic stem cell transplantation; Hematopoietic Stem Cell Transplantation - methods; Hematopoietic Stem Cell Transplantation - mortality; HSCT; Humans; left ventricular ejection fraction; LVEF; Male; Medical sciences; Middle Aged; PAP; Patients; PBSC; peripheral blood stem cells; Pulmonary arteries; pulmonary artery pressure; Registries; Rheumatism; Rodnan skin score; Sarcoidosis. Granulomatous diseases of unproved etiology. Connective tissue diseases. Elastic tissue diseases. Vasculitis; Scleroderma; Scleroderma, Systemic - mortality; Scleroderma, Systemic - physiopathology; Scleroderma, Systemic - therapy; Severity of Illness Index; Skin; Skin - pathology; SSc; Stem cell transplantation; Survival Analysis; systemic sclerosis; TBI; Tlco; TLI; total body irradiation; total lymphoid irradiation; transplant related mortality; Treatment Outcome; TRM; Ventricular Function, Left; Vital Capacity
• ISSN: 0003-4967; 1468-2060
• DOI: 10.1136/ard.2003.011205

Objective: To analyse the durability of the responses after haematopoietic stem cell transplantation (HSCT) for severe systemic sclerosis (SSc) and determine whether the high transplant related mortality (TRM) improved with experience. This EBMT/EULAR report describes the longer outcome of patients originally described in addition to newly recruited cases. Methods: Only patients with SSc, treated by HSCT in European phase I–II studies from 1996 up to 2002, with more than 6 months of follow up were included. Transplant regimens were according to the international consensus statements. Repeated evaluations analysed complete, partial, or non-response and the probability of disease progression and survival after HSCT (Kaplan-Meier). Results: Given as median (range). Among 57 patients aged 40 (9.1–68.7) years the skin scores improved at 6 (n = 37 patients), 12 (n = 30), 24 (n = 19), and 36 (n = 10) months after HSCT (p