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Importance of Establishing Definitions to Increase Survival After Blood/Marrow Transplant

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Key Points

  • Reported mortalities due to leukemia had almost perfect agreement between transplant centers and the consensus panel.
  • There was less agreement for transplant-related mortality, and the level of agreement/discordance varied depending on the specific cause of death.
  • Future solutions may include determining better matches between patients and donors based on testing genes in addition to human leukocyte antigen.

Blood and marrow transplantation is a potentially curative treatment for patients with leukemia or other life-threating blood diseases. With a goal of increasing survival rates, a research team led by Roswell Park Cancer Institute (RPCI) investigators verified patient outcome data submitted by more than 150 U.S. transplant centers over an 11-year period to the Center for International Blood and Marrow Transplant Research (CIBMTR). The detailed investigation, published by Hahn et al in Biology of Blood and Marrow Transplantation, offers insight into different causes of death.

Background of Analysis

Theresa Hahn, PhD, of the Department of Medicine, and Lara Sucheston-Campbell, PhD, of the Department of Cancer Prevention and Population Sciences, were the coprincipal investigators of this study.

“This work is part of the personalized medicine effort…of the National Institutes of Health Personalized Medicine Initiative, aimed at modifying treatment based on a patient’s genetic information,” said Dr. Sucheston-Campbell. “It is important that we accurately define outcomes in these types of genomic studies as precisely as possible. Our work is a critical first step toward the ultimate goal of finding a better match for patients receiving an unrelated-donor blood or marrow transplant.”

The investigators convened a consensus panel to review specific causes of death to reduce misclassification and to determine the impact of genetics on blood and marrow transplantation outcomes. The panel evaluated patient outcomes data for 1,484 patients who died within 1 year after an allogeneic blood and marrow transplantation.

Findings

In the cases where the transplant center reported mortality due to leukemia, Drs. Hahn and Sucheston-Campbell found almost perfect agreement between the consensus panel and transplant center, in terms of how those deaths were classified. There was less agreement for transplant-related mortality, and the level of agreement/discordance varied depending on the specific cause of death. These results indicate that transplant-related mortality needs to be better defined. This study provides a mechanism for prioritizing those blood and marrow transplantation cases that should be reviewed.

“We need to make sure that patients who experienced similar clinical events after a transplant were consistently defined, regardless of where they were treated. This is a difficult topic to consider, but we can’t make progress to improve transplant outcomes without these discussions,” added Dr. Hahn.

Researchers conducting clinical trials commonly use committees to review and define endpoints. Those conducting genome-wide association studies rarely do, instead relying on center-reported outcomes, which are variable, according to Drs. Hahn and Sucheston-Campbell.

Blood and marrow transplantation patients are matched to the best possible unrelated donors through human leukocyte antigen (HLA) typing, also called “tissue typing.” HLA matching is important, because a close match improves the chances of a successful transplant. This research may open doors to determining better matches between patients and donors based on testing genes in addition to HLA of both the donor and the patient.

Dr. Hahn is the corresponding author of the Biology of Blood and Marrow Transplantation article.

This work was supported by the National Institutes of Health.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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