TY - JOUR
T1 - Optimization of Therapy for Severe Aplastic Anemia Based on Clinical, Biologic, and Treatment Response Parameters
T2 - Conclusions of an International Working Group on Severe Aplastic Anemia Convened by the Blood and Marrow Transplant Clinical Trials Network, March 2010
AU - Pulsipher, Michael A.
AU - Young, Neal S.
AU - Tolar, Jakub
AU - Risitano, Antonio M.
AU - Deeg, H. Joachim
AU - Anderlini, Paolo
AU - Calado, Rodrigo
AU - Kojima, Seiji
AU - Eapen, Mary
AU - Harris, Richard
AU - Scheinberg, Phillip
AU - Savage, Sharon
AU - Maciejewski, Jaroslaw P.
AU - Tiu, Ramon V.
AU - DiFronzo, Nancy
AU - Horowitz, Mary M.
AU - Antin, Joseph H.
N1 - Funding Information:
Financial disclosure: This International Working Group was supported by the Blood and Marrow Transplant Clinical Trials Network , the National Heart, Lung, and Blood Institute , the National Cancer Institute , and a grant from Genzyme . The authors gratefully acknowledge the participating centers and co-investigators of the BMT CTN 0301 study.
PY - 2011/3
Y1 - 2011/3
N2 - Although recent advances in therapy offer the promise for improving survival in patients with severe aplastic anemia (SAA), the small size of the patient population, lack of a mechanism in North America for longitudinal follow-up of patients, and inadequate cooperation among hematologists, scientists, and transplant physicians remain obstacles to conducting large studies that would advance the field. To address this issue, the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) convened a group of international experts in March 2010 to define the most important questions in the basic science, immunosuppressive therapy (IST), and bone marrow transplantation (BMT) of SAA and propose initiatives to facilitate clinical and biologic research. Key conclusions of the working group were: (1) new patients should obtain accurate, expert diagnosis and early identification of biologic risk; (2) a population-based SAA outcomes registry should be established in North America to collect data on patients longitudinally from diagnosis through and after treatment; (3) a repository of biologic samples linked to the clinical data in the outcomes registry should be developed; (4) innovative approaches to unrelated donor BMT that decrease graft-versus-host disease are needed; and (5) alternative donor transplantation approaches for patients lacking HLA-matched unrelated donors must be improved. A partnership of BMT, IST, and basic science researchers will develop initiatives and partner with advocacy and funding organizations to address these challenges. Collaboration with similar study groups in Europe and Asia will be pursued.
AB - Although recent advances in therapy offer the promise for improving survival in patients with severe aplastic anemia (SAA), the small size of the patient population, lack of a mechanism in North America for longitudinal follow-up of patients, and inadequate cooperation among hematologists, scientists, and transplant physicians remain obstacles to conducting large studies that would advance the field. To address this issue, the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) convened a group of international experts in March 2010 to define the most important questions in the basic science, immunosuppressive therapy (IST), and bone marrow transplantation (BMT) of SAA and propose initiatives to facilitate clinical and biologic research. Key conclusions of the working group were: (1) new patients should obtain accurate, expert diagnosis and early identification of biologic risk; (2) a population-based SAA outcomes registry should be established in North America to collect data on patients longitudinally from diagnosis through and after treatment; (3) a repository of biologic samples linked to the clinical data in the outcomes registry should be developed; (4) innovative approaches to unrelated donor BMT that decrease graft-versus-host disease are needed; and (5) alternative donor transplantation approaches for patients lacking HLA-matched unrelated donors must be improved. A partnership of BMT, IST, and basic science researchers will develop initiatives and partner with advocacy and funding organizations to address these challenges. Collaboration with similar study groups in Europe and Asia will be pursued.
KW - Blood and marrow transplantation
KW - Immunosuppressive therapy
KW - Severe aplastic anemia
KW - Telomeres
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U2 - 10.1016/j.bbmt.2010.10.028
DO - 10.1016/j.bbmt.2010.10.028
M3 - Review article
C2 - 21034841
AN - SCOPUS:79551684829
SN - 1083-8791
VL - 17
SP - 291
EP - 299
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
IS - 3
ER -