Published online 22 September 2009
Haematologica, Vol 95, Issue 2, 284-292 doi:10.3324/haematol.2009.013458
Copyright © 2010 by Ferrata Storti Foundation

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Stem Cell Transplantation

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

Dominique Farge¹, Myriam Labopin², Alan Tyndall³, Athanasios Fassas⁴, Gian Luigi Mancardi⁵, Jaap Van Laar⁶, Jian Ouyang⁷, Tomas Kozak⁸, John Moore⁹, Ina Kötter¹⁰, Virginie Chesnel¹¹, Alberto Marmont¹², Alois Gratwohl¹³, Riccardo Saccardi¹⁴

¹ Internal Medicine, Saint Louis Hospital, Paris, France
² Hôpital Saint Antoine, Service d’hématologie et Thérapie Cellulaire, AP-HP, UPMC Univ Paris 06, UMR-S 938, CEREST-TC EBMT, Paris, France
³ Rheumatology, Rheumatologische Universtätsklinik, Basel, Switzerland
⁴ Neurology and Haematology, The George Papanicolau Hospital, Thessaloniki, Greece
⁵ Neurosciences, Ophthalmology and Genetics, Univ. of Genova, Genova, Italy
⁶ Institute of Cellular Medicine, Newcastle Univ., Newcastle Upon Tyne, United Kingdom
⁷ Hematology, Hospital of Nanjing, Nanjing, China;
⁸ Clinical Haematology, Charles Univ. Prague, Prague, Czech Republic;
⁹ Hematology Dept., St Vincents Hospital, Sydney, Australia;
¹⁰ Immunology, Rheumatology and Autoimmune Diseases, Univ. of Tübingen, Tübingen, Germany;
¹¹ INSERM UMR-S 938, UPMC Univ Paris 06, Paris, France;
¹² Haematology, Ospedale di San Martino, Genova, Italy;
¹³ Haematology, Kantonspital Basel, Basel, Switzerland and
¹⁴ BMT 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

Background: Autologous hematopoietic stem cell transplantation has been used since 1996 for the treatment of severe autoimmune diseases refractory to approved therapies. We evaluated the long-term outcomes of these transplants and aimed to identify potential prognostic factors.

Design and Methods: In this observational study we analyzed all first autologous hematopoietic stem cell transplants for autoimmune diseases reported to the European Group for Blood and Marrow Transplantation (EBMT) registry between 1996–2007. The primary end-points for analysis were overall survival, progression-free survival and transplant-related mortality at 100 days.

Results: Nine hundred patients with autoimmune diseases (64% female; median age, 35 years) who underwent a first autologous hematopoietic stem cell transplant were included. The main diseases were multiple sclerosis (n=345), systemic sclerosis (n=175), systemic lupus erythematosus (n=85), rheumatoid arthritis (n=89), juvenile arthritis (n=65), and hematologic immune cytopenia (n=37). Among all patients, the 5-year survival was 85% and the progression-free survival 43%, although the rates varied widely according to the type of autoimmune disease. By multivariate analysis, the 100-day transplant-related mortality was associated with the transplant centers’ experience (P=0.003) and type of autoimmune disease (P=0.03). No significant influence of transplant technique was identified. Age less than 35 years (P=0.004), transplantation after 2000 (P=0.0015) and diagnosis (P=0.0007) were associated with progression-free survival.

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

Key words: autologous hematopoietic stem cell transplantation, autoimmune diseases, multiple sclerosis, systemic sclerosis, systemic lupus erythematosus, rheumatoid arthritis, juvenile idiopathic arthritis, hematologic immune cytopenia, total body irradiation, antithymocyte globulins, cyclophosphamide.

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