Yes, CAR T-cell Therapy is Performed in the United States
CAR T-cell therapy is actively performed throughout the United States, with multiple FDA-approved products available for treating various hematologic malignancies. 1
FDA-Approved Products Available in the US
The United States currently has several approved CAR T-cell therapies:
CD19-Targeted Therapies
- Tisagenlecleucel (Kymriah)
- Axicabtagene ciloleucel (Yescarta)
- Brexucabtagene autoleucel
- Lisocabtagene maraleucel
These target B-cell malignancies including B-cell acute lymphoblastic leukemia (ALL), chronic lymphocytic leukemia, and B-cell lymphomas 1.
BCMA-Targeted Therapy
- Idecabtagene vicleucel - approved in 2021 for multiple myeloma treatment 1
Utilization Trends in the US
CAR T-cell therapy adoption has increased dramatically in the United States. From 2017 to 2022, utilization grew from just 70 cases to 4,335-4,725 cases annually, representing an annual growth rate of approximately 32.6% 2, 3. This rapid expansion demonstrates that CAR T-cell therapy has become an established treatment modality across the country.
Where Treatment Occurs
Most CAR T-cell therapies are administered in large private hospitals (approximately 75% of cases), with the majority of patients receiving initial care in the inpatient setting due to the need for continuous toxicity monitoring 4, 3. However, outpatient management is permitted on the label of at least one FDA-approved CAR T therapy for lymphoma, though many institutions prefer inpatient monitoring 4.
Treatment Process in the US
The multistep CAR T-cell therapy process in the United States includes:
- Leukapheresis - collecting white blood cells from the patient
- Manufacturing - T cells are isolated, activated, and transduced with CAR transgene (typically 2-4 weeks)
- Lymphodepletion chemotherapy - fludarabine and cyclophosphamide given prior to infusion
- CAR T-cell infusion - administered at specialized centers 1
Regulatory Requirements
The FDA mandates long-term safety monitoring for patients receiving CAR T-cell therapy. Patients receiving tisagenlecleucel and axicabtagene ciloleucel must participate in 15 years of postmarketing observational studies to monitor long-term safety and risk of secondary malignancies 4, 5.
Access Considerations
CAR T-cell therapy requires patients to remain within proximity of the treating institution for at least 4 weeks following infusion according to Risk Evaluation and Mitigation Strategies (REMS) programs 4. This requirement, combined with the need for a 24-hour caretaker, creates logistical challenges but ensures appropriate monitoring for complications like cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome.
Clinical Outcomes
Recent real-world data from 2017-2022 shows:
- Overall in-hospital mortality rate: 3.4%
- Mean length of stay: 18.3 days (decreasing by 1.16 days annually)
- Cytokine release syndrome occurred in 61-62% of patients
- Immune effector cell-associated neurotoxicity syndrome observed in 24.2% 2
CAR T-cell therapy has demonstrated improved survival outcomes, including 4-year overall survival of 54.6% in large B-cell lymphoma compared to 46.0% with standard chemotherapy and stem cell transplant 6.
Important Caveats
While CAR T-cell therapy is widely available in the US, persistent sociodemographic disparities exist. White patients comprise 57-74% of recipients, and individuals in the highest income quartile consistently represent the largest proportion (28-37%) 3. Geographic access limitations and substantial financial burden (costs ranging from $373,000-$475,000 per treatment, increasing to $378,507 in 2022) remain significant barriers 4, 2, 7.