期刊
HAEMATOLOGICA-THE HEMATOLOGY JOURNAL
卷 95, 期 2, 页码 284-292出版社
FERRATA STORTI FOUNDATION
DOI: 10.3324/haematol.2009.013458
关键词
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
类别
资金
- Amgen Europe
- Hoffmann-La Roche Ltd.
- Gilead Sciences
- Baxter Oncology
- Pharmacia Corporation
- Chugai-Aventis
- Fresenius HemoCare
- SangStat
- Schering AG
- Gambro BCT
- Elan Pharmaceuticals
- Miltenyl Biotec GmbH
- Therakos
- Wyeth-Lederle
- Astra
- Cobe International
- Nextar
- Liposome Co
- Imtix
- Direction de la Recherche Clinique Assistance Publique - Hopitaux de Paris (DRC AP-HP)
- INSERM U 976
- Groupe Francais de la recherche sur la Sclerodermie
- Fondation de l'Avenir pour la Recherche Medicate Appliquee [ET7-457]
- Scleroderma Association
- Swiss National Research Foundation
- Horton Foundation
- Italian MS Society (AISM)
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 trans plants 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 trans plant-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=0.003). 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.
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