Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2018 Jun 14;131(24):2630-2639.
doi: 10.1182/blood-2018-02-785881. Epub 2018 May 4.

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

Affiliations
Review

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

Leslie S Kean. Blood. .

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.

PubMed Disclaimer

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

    1. Sorror ML, Maris MB, Storb R, et al. . Hematopoietic cell transplantation (HCT)-specific comorbidity index: a new tool for risk assessment before allogeneic HCT. Blood. 2005;106(8):2912-2919. - PMC - PubMed
    1. 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
    1. Orozco JJ, Kenoyer A, Balkin ER, et al. . Anti-CD45 radioimmunotherapy without TBI before transplantation facilitates persistent haploidentical donor engraftment. Blood. 2016;127(3):352-359. - PMC - PubMed
    1. Mawad R, Gooley TA, Rajendran JG, et al. . Radiolabeled anti-CD45 antibody with reduced-intensity conditioning and allogeneic transplantation for younger patients with advanced acute myeloid leukemia or myelodysplastic syndrome. Biol Blood Marrow Transplant. 2014;20(9):1363-1368. - PMC - PubMed
    1. Nakamae H, Wilbur DS, Hamlin DK, et al. . Biodistributions, myelosuppression, and toxicities in mice treated with an anti-CD45 antibody labeled with the alpha-emitting radionuclides bismuth-213 or astatine-211. Cancer Res. 2009;69(6):2408-2415. - PMC - PubMed

Publication types

MeSH terms