Defining success with cellular therapeutics: the current landscape for clinical end point and toxicity analysis

Leslie S. Kean


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.

  • Submitted February 21, 2018.
  • Accepted April 11, 2018.
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