Optimization of Therapy for Severe Aplastic Anemia Based on Clinical, Biologic, and Treatment Response Parameters: Conclusions of an International Working Group on Severe Aplastic Anemia Convened by the Blood and Marrow Transplant Clinical Trials Network, March 2010

Michael A. Pulsipher, Neal S. Young, Jakub Tolar, Antonio M. Risitano, H. Joachim Deeg, Paolo Anderlini, Rodrigo Calado, Seiji Kojima, Mary Eapen, Richard Harris, Phillip Scheinberg, Sharon Savage, Jaroslaw P. Maciejewski, Ramon V. Tiu, Nancy DiFronzo, Mary M. Horowitz, Joseph H. Antin

Research output: Contribution to journalReview articlepeer-review

29 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)291-299
Number of pages9
JournalBiology of Blood and Marrow Transplantation
Volume17
Issue number3
DOIs
StatePublished - Mar 2011

Bibliographical note

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.

Keywords

  • Blood and marrow transplantation
  • Immunosuppressive therapy
  • Severe aplastic anemia
  • Telomeres

Fingerprint Dive into the research topics of 'Optimization of Therapy for Severe Aplastic Anemia Based on Clinical, Biologic, and Treatment Response Parameters: Conclusions of an International Working Group on Severe Aplastic Anemia Convened by the Blood and Marrow Transplant Clinical Trials Network, March 2010'. Together they form a unique fingerprint.

Cite this