scholarly journals Hematopoietic Cell Transplantation for Autoimmune Diseases: A Review of History, Current State, and Future Issues

Author(s):  
Igor B. Resnick ◽  
Krassimir Metodiev ◽  
Paula Lazarova
Hematology ◽  
2003 ◽  
Vol 2003 (1) ◽  
pp. 372-397 ◽  
Author(s):  
Rainer F. Storb ◽  
Guido Lucarelli ◽  
Peter A. McSweeney ◽  
Richard W. Childs

Abstract Allogeneic hematopoietic cell transplantation (HCT) has been successfully used as replacement therapy for patients with aplastic anemia and hemoglobinopathies. Both autologous and allogeneic HCT following high-dose chemotherapy can correct manifestations of autoimmune diseases. The impressive allogeneic graft-versus-tumor effects seen in patients given HCT for hematological malignancies have stimulated trials of allogeneic immunotherapy in patients with otherwise refractory metastatic solid tumors. This session will update the status of HCT in the treatment of benign hematological diseases and solid tumors. In Section I, Dr. Rainer Storb reviews the development of nonmyeloablative conditioning for patients with severe aplastic anemia who have HLA-matched family members. He also describes the results in patients with aplastic anemia given HCT from unrelated donors after failure of responding to immunosuppressive therapy. The importance of leuko-poor and in vitro irradiated blood product transfusions for avoiding graft rejection will be discussed. In Section II, Dr. Guido Lucarelli reviews the status of marrow transplantation for thalassemia major and updates results obtained in children with class I and class II severity of thalassemia. He also describes results of new protocols for class III patients and efforts to extend HCT to thalassemic patients without HLA-matched family members. In Section III, Dr. Peter McSweeney reviews the current status of HCT for severe autoimmune diseases. He summarizes the results of autologous HCT for systemic sclerosis, multiple sclerosis, rheumatoid arthritis, and systemic lupus erythematosus, and reviews the status of planned Phase III studies for autologous HCT for these diseases in North America and Europe. He also discusses a possible role of allogeneic HCT in the treatment of these diseases. In Section IV, Dr. Richard Childs discusses the development and application of nonmyeloablative HCT as allogeneic immunotherapy for treatment-refractory solid tumors. He reviews the results of pilot clinical trials demonstrating graft-versus-solid tumor effects in a variety of metastatic cancers and describes efforts to characterize the immune cell populations mediating these effects, as well as newer methods to target the donor immune system to the tumor.


2016 ◽  
Vol 29 (1) ◽  
pp. 54-66 ◽  
Author(s):  
Mohamed A. Kharfan-Dabaja ◽  
Jessica El-Asmar ◽  
Farrukh T. Awan ◽  
Mehdi Hamadani ◽  
Ernesto Ayala

2021 ◽  
pp. 310-325
Author(s):  
S.H. Lum ◽  
E.P. Buddingh ◽  
M.A. Slatter ◽  
A.C. Lankester

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 3143-3143 ◽  
Author(s):  
Helen Baldomero ◽  
Mahmoud Aljurf ◽  
Luis Fernando Bouzas ◽  
Alois Gratwohl ◽  
Yoshihisa Kodera ◽  
...  

Abstract Abstract 3143 Hematopoietic cell transplantation (HCT) is the treatment of choice for many patients with malignant and non-malignant disorders. Transplant practices have evolved allowing patients previously not otherwise candidates to receive this procedure. Activity surveys and registries play an essential role in the success of HCT, by tracking activities, identifying areas of need and through clinical research to continue improving patient outcome. Because of the need of matched donors, ethnic characteristics and legal requirements HCT has acquired a global dimension. In the current analysis the Worldwide Blood and Marrow Transplantation network (WBMT) reports the global activities, indications and trends for the years 2006 to 2008. The survey consisted of activities reported to the WBMT from international societies members (EBMT, CIBMTR, APBMT, EMBMT, ABMTRR), national registries (SBTMO) and also directly from transplant centers in regions were no societies are established. Annual transplant center specific activities were from 1327 transplant centers in 71 countries in 2006. The number of participating centers increased to 1382 and 1407 in 2007 and 2008, respectively. Annual number of transplants steadily increased from 46,563 in 2006 to 48,709 and 51,536 in 2007 and 2008. When analyzing the median number of transplants/year performed at each center the corresponding annual activity was 38 (range 3–180), 46 (3–421) and 48 (1–389), suggesting that the increment in transplant activity is not only related to higher number of reporting centers. The highest increase in total HCT over two years was observed in the Asia Pacific region (38.6%) followed by the East Mediterranean region (19.4%), Europe (5.6%) and the U.S. (4.5%). An absolute increase of autologous and allogeneic was observed over the three year period with more autologous (55%) than allogeneic (45%) HCT reported, however the increase was less in autologous (+5.0%) when compared to allogeneic (+17.9%). Among indications for allogeneic HCT, acute leukemias (AML +23%; ALL +27%), myelodysplasia (MDS +26%), chronic lymphocytic leukemia (CLL +24.6%) nonmalignant diseases (NMD +23.6%) and bone marrow failure disorders (BMF +21.2%) significantly increased during the study period. Slower increase was reported for lymphoproliferative disorders (LPD +6%) and a clear decrease for chronic myelogenous leukemia (CML -17%) and solid tumors (−13.3%). Among autologous HCT indications autoimmune diseases (+24.5%), PCD (+9.8%) and LPD (+7.5%) increased during the period. A negative trend was seen in autologous HCT for solid tumors (−2.4%), ALL (−22.0%), AML (−9.0%), CLL (−52.0%) and CML (−57.1%). These data show the trend and activity for autologous and allogeneic HSCT worldwide. There is a clear increase in activity especially in acute and chronic (except CML) leukemias for allogeneic HST and for autoimmune diseases, LPD and PCD in autologous HCT. Monitoring global transplant practices is an important activity for WBMT in order for capacity planning of HCT donor pool worldwide and to promote the field by expanding access to transplantation at regions in need. Disclosures: No relevant conflicts of interest to declare.


Hematology ◽  
2003 ◽  
Vol 2003 (1) ◽  
pp. 372-397 ◽  
Author(s):  
Rainer F. Storb ◽  
Guido Lucarelli ◽  
Peter A. McSweeney ◽  
Richard W. Childs

Allogeneic hematopoietic cell transplantation (HCT) has been successfully used as replacement therapy for patients with aplastic anemia and hemoglobinopathies. Both autologous and allogeneic HCT following high-dose chemotherapy can correct manifestations of autoimmune diseases. The impressive allogeneic graft-versus-tumor effects seen in patients given HCT for hematological malignancies have stimulated trials of allogeneic immunotherapy in patients with otherwise refractory metastatic solid tumors. This session will update the status of HCT in the treatment of benign hematological diseases and solid tumors. In Section I, Dr. Rainer Storb reviews the development of nonmyeloablative conditioning for patients with severe aplastic anemia who have HLA-matched family members. He also describes the results in patients with aplastic anemia given HCT from unrelated donors after failure of responding to immunosuppressive therapy. The importance of leuko-poor and in vitro irradiated blood product transfusions for avoiding graft rejection will be discussed. In Section II, Dr. Guido Lucarelli reviews the status of marrow transplantation for thalassemia major and updates results obtained in children with class I and class II severity of thalassemia. He also describes results of new protocols for class III patients and efforts to extend HCT to thalassemic patients without HLA-matched family members. In Section III, Dr. Peter McSweeney reviews the current status of HCT for severe autoimmune diseases. He summarizes the results of autologous HCT for systemic sclerosis, multiple sclerosis, rheumatoid arthritis, and systemic lupus erythematosus, and reviews the status of planned Phase III studies for autologous HCT for these diseases in North America and Europe. He also discusses a possible role of allogeneic HCT in the treatment of these diseases. In Section IV, Dr. Richard Childs discusses the development and application of nonmyeloablative HCT as allogeneic immunotherapy for treatment-refractory solid tumors. He reviews the results of pilot clinical trials demonstrating graft-versus-solid tumor effects in a variety of metastatic cancers and describes efforts to characterize the immune cell populations mediating these effects, as well as newer methods to target the donor immune system to the tumor.


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