Some CAR T therapies are approved and some are being investigated in clinical trials.

Considerations for Appropriate Patient Selection

CAR T cell therapies have been approved for adult, pediatric, and young adult patients with certain relapsed or refractory cancers.1-3

Patient identification

CAR T cell therapy is generally an autologous cell therapy that may have different patient selection considerations than autologous stem cell transplant (ASCT); some examples include prior lines of therapy, upper age limit, patient fitness, severity of comorbidities, and chemoresistance status.4-6

Patient selection

General eligibility requirements for CAR T cell therapy7-9

  • Tumor expressing a CAR target (eg, BCMA, CD19, CD20, CD22, CD23, ROR1, and the kappa light chain)
  • Adequate number of T cells for collection
  • No active, uncontrolled infections, including hepatitis B, hepatitis C, or HIV
  • Adequate performance status and organ function
  • Absence of clinically relevant comorbidities (eg, select cardiovascular, neurologic, or immune disorders)

BCMA, B-cell maturation antigen; CD19/CD20/CD22/CD23, B-lymphocyte antigen CD19/CD20/CD22/CD23.

Precise criteria for eligibility vary by7:

  • Malignancy
  • Treatment regimen or protocol
  • CAR T cell product

Eligibility for chimeric antigen receptor (CAR) T cell therapy may differ from eligibility for stem cell transplantation (SCT)5,6

Patient selection considerations for CAR T cell therapy may differ from stem cell transplants—some CAR T clinical trials have included transplant-ineligible patients.10 Active clinical trials are also studying CAR T cell therapy in earlier lines of treatment and for both transplant-eligible and -ineligible patients.11,12

A Potentially Larger Pool of Patients May Be Eligible for CAR T Cell Therapy Compared With SCT5,6,8,10-13

Figure illustrating population that may be eligible for CAR T cell therapy vs ASCT Figure illustrating population that may be eligible for CAR T cell therapy vs ASCT

Considerations for Patients Treated With CAR T Cell Therapy

Performance status

Eastern Cooperative Oncology Group performance status (ECOG PS) is often used to determine patient eligibility for CAR T cell therapy. Some investigational trials have included patients with ECOG PS Grade 2.14 Patients generally range between ECOG PS Grade 0 (fully active) and Grade 1 (physically restricted and limited to light activity).15-17

Organ function

Patient selection may be based on adequate organ function and physiological reserve. A patient’s ability to tolerate fever and other potentially severe adverse reactions, such as cytokine release syndrome (CRS) and neurological toxicity (NT) associated with CAR T cell therapy, should be determined.1,3,4

T cell health

Patients who are earlier in their disease tend to have healthier T cell populations, which may help improve the chances of manufacturing success of CAR T cell products and also help drive CAR T cell expansion and persistence following infusion. Multiple lymphotoxic treatments, such as cyclophosphamide, venetoclax, bendamustine, and dexamethasone, may compromise T cell health and limit the ability to generate viable CAR T cells.4,18-20

Accessibility and support

Insurance coverage and ability to travel to an authorized treatment center, as well as having an adequate patient support network, are key considerations in patient selection. CAR T cell therapy is an involved process, making it necessary for patients to have competent and committed caregiver support.8 Patients will be required to remain within close proximity to the center throughout the process and for a period of time after receiving CAR T cell therapy to monitor for adverse reactions.8

These are not all of the patient selection considerations for CAR T cell therapy, and the considerations will differ from product to product.

References: 1. News release. Silver Spring, MD: Food and Drug Administration; August 30, 2017. Accessed November 6, 2019. 2. News release. Silver Spring, MD: Food and Drug Administration; May 1, 2018. Accessed November 6, 2019. 3. News release. Silver Spring, MD: Food and Drug Administration; October 18, 2017. Accessed November 6, 2019. 4. Maus MV, Levine BL. Oncologist. 2016;21:608-617. 5. Hayden PJ, Sirait T, Koster L, Snowden JA, Yakoub-Agha I. Curr Res Transl Med. 2019;67:79-88. 6. Okamoto S. Hematol Oncol Stem Cell Ther. 2017;10:178-183. 7. Leukemia and Lymphoma Society. Facts about chimeric antigen receptor (CAR) T-cell therapy. 2020:1-10. 8. Beaupierre A, Lundberg R, Marrero L, Jain M, Wang T, Alencar MC. Clin J Oncol Nurs. 2019;23:27-34. 9. D’Aloia MM, Zizzari IG, Sacchetti B, et al. Cell Death Dis. 2018;9:282. doi:10.1038/s41419-018-0278-6. 10. Gisselbrecht C, Glass B, Mounier N, et al. J Clin Oncol. 2010;28(27):4184-4190. 11. Gisselbrecht C, Van Den Neste E. Br J Haematol. 2018;182(5):633-643. 12. Nastoupil LJ, Jain MD, Feng L, et al. J Clin Oncol. 2020;38:1-19. 13. Chavez JC, Bachmeier C, Kharfan-Dabaja MA. Ther Adv Hematol. 2018;1-20. 14. NCT02348216. Accessed November 6, 2018. 15. NCT02445248. Accessed November 6, 2018. 16. NCT03602612. Accessed November 6, 2018. 17. Accessed November 6, 2019. 18. Garfall AL, Dancy EK, Cohen AD, et al. Blood Adv. 2019;3(19):2812-2815. 19. Brudno JN, Maric I, Hartman SD, et al. J Clin Oncol. 2018;36(22):2267-2280. 20. de Weerdt I, Hofland T, de Boer R, et al. Blood Adv. 2019;3(17):2642-2652.