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

被引：269

作者：

Farge, Dominique ^{[1
]}

Labopin, Myriam ^{[2
]}

Tyndall, Alan ^{[3
]}

Fassas, Athanasios ^{[4
]}

Mancardi, Gian Luigi ^{[5
]}

Van Laar, Jaap ^{[6
]}

Jian Ouyang ^{[7
]}

Kozak, Tomas ^{[8
]}

Moore, John ^{[9
]}

Koetter, Ina

Chesnel, Virginie ^{[10
,11
]}

Marmont, Alberto ^{[12
]}

Gratwohl, Alois ^{[13
]}

Saccardi, Riccardo ^{[14
]}

机构：

[1] Univ Paris 07, Hop St Louis, AP HP, Serv Med Interne, F-75010 Paris, France

[2] Univ Paris 06, Hop St Antoine, AP HP,UMR S 938, Serv Hematol & Therapie Cellulaire,CEREST TC EBMT, Paris, France

[3] Univ Klin, Basel, Switzerland

[4] George Papanicolau Hosp, Thessaloniki, Greece

[5] Univ Genoa, Genoa, Italy

[6] Newcastle Univ, Inst Cellular Med, Newcastle Upon Tyne NE1 7RU, Tyne & Wear, England

[7] Hosp Nanjing, Nanjing, Peoples R China

[8] Charles Univ Prague, Prague, Czech Republic

[9] St Vincents Hosp, Dept Hematol, Sydney, NSW 2010, Australia

[10] Univ Tubingen, Tubingen, Germany

[11] Univ Paris 06, INSERM, UMR S 938, Paris, France

[12] Osped San Martino Genova, Genoa, Italy

[13] Kantonspital Basel, Basel, Switzerland

[14] Policlin Careggi, UO Ematol, BMT Unit, Florence, Italy

来源：

HAEMATOLOGICA-THE HEMATOLOGY JOURNAL | 2010年 / 95卷 / 02期

基金：

新加坡国家研究基金会;

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; TERM-FOLLOW-UP; SYSTEMIC-SCLEROSIS; MULTIPLE-SCLEROSIS; THERAPY; CYCLOPHOSPHAMIDE; MORTALITY; JUVENILE;

D O I：

10.3324/haematol.2009.013458

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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.

引用

页码：284 / 292

页数：9