Defining success with cellular therapeutics: the current landscape for clinical end point and toxicity analysis
- PMID: 29728399
- PMCID: PMC6032897
- DOI: 10.1182/blood-2018-02-785881
Defining success with cellular therapeutics: the current landscape for clinical end point and toxicity analysis
Abstract
Cellular therapies play a major and expanding role in the treatment of hematologic diseases. For each of these therapies, a narrow therapeutic window exists, where efficacy is maximized and toxicities minimized. This review focuses on one of the most established cellular therapies, hematopoietic stem cell transplant, and one of the newest cellular therapies, chimeric antigen receptor-T cells. In this review, I will discuss the current state of the field for clinical end point analysis with each of these therapeutics, including their critical toxicities, and focus on the major elements of success for each of these complex treatments for hematologic disease.
© 2018 by The American Society of Hematology.
Conflict of interest statement
Conflict-of-interest disclosure: L.S.K. has received research funding from Juno Therapeutics, Bristol-Myers Squibb, Kymab Ltd, and Regeneron, and is a consultant for Bristol-Myers Squibb, Kymab, Regeneron, and Equilium.
References
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- Vaughn JE, Storer BE, Armand P, et al. . Design and validation of an augmented hematopoietic cell transplantation-comorbidity index comprising pretransplant ferritin, albumin, and platelet count for prediction of outcomes after allogeneic transplantation. Biol Blood Marrow Transplant. 2015;21(8):1418-1424. - PMC - PubMed
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