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Autologous hematopoietic stem cell transplantation (HSCT) for autoimmune diseases: an observational study on 12 years of experience from the European Group for Blood and Marrow Transplantation (EBMT) Working Party on Autoimmune Diseases

¹ Internal Medicine, Saint Louis Hospital, Paris, France
² Hôpital Saint Antoine, service d’hématologie et thérapie cellulaire, AP-HP, Paris, France
³ Rheumatology, Rheumatologische Universtätsklinik, Basel, Switzerland
⁴ Neurology and haematology, The George Papanicolau Hospital, Thessaloniki, Greece
⁵ Neurosciences, Oftalmology and Genetics, University of Genova, Genova, Italy
⁶ Institute of Cellular Medicine, Newcastle University, Newcastle Upon Tyne, United Kingdom
⁷ Hematology, Hospital of Nanjing, Nanjing, China
⁸ Clinical Haematology, Charles University Prague, Prague, Czech Republic
⁹ Hematology Department, St Vincents Hospital, Sydney, Australia
¹⁰ Immunology, Rheumatology and Autoimmune Diseases, Universitätsklinikum Tübingen, Tübingen, Germany
¹¹ INSERM UMR-S 938, UPMC, Paris, France
¹² Haematology, Ospedale di San Martino, Genova, Italy
¹³ Haematology, Kantonspital Basel, Basel, Switzerland
¹⁴ Bone Marrow Transplant Unit, UO Ematologia, Policlinico Careggi, Florence, Italy

Correspondence: Dominique Farge, Service de Médecine Interne et Unité INSERM U 976, Hôpital Saint-Louis, Assistance-Publique Hôpitaux de Paris, Paris-7 Université Denis Diderot, 1 avenue Claude Vellefaux, 75 010 Paris France. E-mail :dominique.farge-bancel{at}sls.ap-hop-paris.fr

ABSTRACT

Background: Since autologous hematopoietic stem cell transplantation (HSCT) has been used since 1996 for treating severe autoimmune diseases (AD) refractory to approved treatments, we aimed to evaluate long term outcomes and to identify potential prognostic factors.

Design and Methods: This observational study (1996–2007) analysed all first AHSCT for AD reported to the European Group for Blood and Marrow Transplants (EBMT) registry. Primary end-points were overall survival (OS), progression-free survival (PFS) and the 100 days transplant related mortality (TRM).

Results: 900 AD patients (64 % female, median 35 years) with first autologous HSCT were included, mainly: 345 multiple sclerosis, 175 systemic sclerosis, 85 lupus erythematosus, 89 rheumatoid arthritis, 65 juvenile arthritis, 37 hematological immune cytopenia. Overall, the 5 years OS was 85 % and PFS 43 %, varying widely according to AD type. By multivariate analysis, the day 100 TRM was associated with centre experience (p=0.003) and AD type (p=0.03). No significant influence of transplant technique could be identified. Age < 35 yrs (HR 1.37, 95%CI (1.1–1.7), p=0.004), autologous HSCT after 2000 (HR 1.47, 95%CI (1.16–1.86), p=0.0015) and diagnosis (p=0.0007) were associated with PFS.

Conclusions: This largest cohort studied worldwide shows that autologous HSCT can induce sustained remissions for more than 5 years in patients with severe AD refractory to conventional therapy. AD disease type, rather than transplant techniques, was the most relevant determinant of outcome. Results improved with time and were associated with centre experience. These data support ongoing and planned phase III trials to evaluate the place of autologous HSCT in the treatment strategy for severe AD.

Key words: autologous hematopoietic stem cell transplantation, systemic lupus erythematosus, rheumatoid arthritis, juvenile idiopathic arthritis, total body irradiation, antithymocyte globulins, cyclophosphamide.

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