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

¹ Academish Ziekenhuis, Leiden, The Netherlands
² Albert Ludwig Universität, Freiburg, Germany
³ Carlos Haya Hospital, Malaga, Spain
⁴ Charité Hospital, Berlin, Germany
⁵ Hôpital Erasme, Brussels, Belgium
⁶ Hospital del SAS, Cadiz Jerez de la Frontera, Spain
⁷ Hospital G.U. Gregorio Maranon, Madrid, Spain
⁸ Hospital Infantil La Paz, Madrid, Spain
⁹ Institut Paoli Calmettes, Marseille, France
¹⁰ IRCCS G. Gaslini, Genova, Italy
¹¹ Kantonsspital Basel, Switzerland
¹² Medizinische Hochschule, Hannover, Germany
¹³ Ospedale San Martino, Genova, Italy
¹⁴ Royal Free Hospital, London, UK
¹⁵ San Matteo University Hospital, Pavia, Italy
¹⁶ San Raffaele Hospital, Milano, Italy
¹⁷ St Anna University Hospital, Ferrara, Italy
¹⁸ St Louis Hospital, Paris, France
¹⁹ Universitätsklinikum Tübingen, Germany
²⁰ University Hospital, Leeds, UK
²¹ University Hospital, Birmingham, UK
²² University Hospital, Nijmegen, The Netherlands
²³ University Hospital, Kuopio, Finland

Correspondence to:
Correspondence to:
Professor D Farge
Service de Médecine Interne, hôpital Saint-Louis, 1 avenue Claude Vellefaux 75010 Paris, France; dominique.farge-bancel{at}chu-stlouis.fr

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<0.005). After 22.9 (4.5–81.1) months, partial (n = 32) or complete response (n = 14) was seen in 92% and non-response in 8% (n = 4) of 50 observed cases. 35% of the patients with initial partial (n = 13/32) or complete response (n = 3/14) relapsed within 10 (2.2–48.7) months after HSCT. The TRM was 8.7% (n = 5/57). Deaths related to progression accounted for 14% (n = 8/57) of the 23% (n = 13/57) total mortality rate. At 5 years, progression probability was 48% (95% CI 28 to 68) and the projected survival was 72% (95% CI 59 to 75).

Conclusion: This EBMT/EULAR report showed that response in two thirds of the patients after HSCT was durable with an acceptable TRM. Based on these results prospective, randomised trials are proceeding.

Abbreviations: AD, autoimmune disease; ATG, antithymocyte globulins; EBMT, European Group for Blood and Marrow Transplantation; G-CSF, granulocyte-colony stimulating factor; HSCT, haematopoietic stem cell transplantation; LVEF, left ventricular ejection fraction; PAP, pulmonary artery pressure; PBSC, peripheral blood stem cells; SSc, systemic sclerosis; TBI, total body irradiation; TLCO, carbon monoxide transfer factor; TLI, total lymphoid irradiation; TRM, transplant related mortality; VC, vital capacity

Keywords: systemic sclerosis; autoimmune diseases; autologous bone marrow transplantation; haematopoietic stem cell transplantation; Rodnan skin score

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