Defining success with cellular therapeutics: the current landscape for clinical endpoint 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 critical 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 (HCT), and one of the newest cellular therapies, CAR-T cells. In this review I will discuss the current state-of-the field for analysis of their key clinical endpoints and critical toxicities, and focus on the major elements of success for each of these complex therapies.

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