Axi-Cel Delivery Feasibility in Outpatient Setting – Phase 2 Study
- This text details the results of the ZUMA-24 trial, which investigated outpatient management of axi-cel (a CAR T-cell therapy) and the ongoing discussion surrounding expanding access to these...
- * Study Design: 30 patients received axi-cel (2 x 106 CAR T-cells/kg) with prophylactic dexamethasone. Daily monitoring was conducted at a healthcare facility for the first 7 days,with...
- * Access Issues: Only 20% of eligible patients currently access CAR T-cell and bispecific therapies.
Summary of the ZUMA-24 Trial & Outpatient CAR T-cell Therapy Discussion
This text details the results of the ZUMA-24 trial, which investigated outpatient management of axi-cel (a CAR T-cell therapy) and the ongoing discussion surrounding expanding access to these therapies in community settings. Here’s a breakdown of the key points:
ZUMA-24 Trial Results:
* Study Design: 30 patients received axi-cel (2 x 106 CAR T-cells/kg) with prophylactic dexamethasone. Daily monitoring was conducted at a healthcare facility for the first 7 days,with optional telemedicine follow-up. Patients remained within 2 hours of the clinic for at least 4 weeks.
* Primary Endpoints: Incidence and severity of cytokine Release Syndrome (CRS) and Neurologic Events (NEs).
* Key Findings:
* CRS: 90% experienced grade 1-2 CRS; no grade ≥3 CRS. Median onset: 4 days, duration: 5 days.
* NEs: 80% experienced NEs of any grade, 23% with grade 3 or higher. No deaths due to NEs. Median onset: 7 days, duration: 6 days.
* Adverse Events: All patients experienced AEs, 83% with grade 3 or higher.
* Hospitalization: 93% (28 patients) were hospitalized (median time to hospitalization: 4 days, median stay: 8 days). most hospitalizations (n=21) were for grade 1 events. 4 patients (13%) required ICU admission for 2-7 days.
* Quality of Life (QOL): QOL scores initially dropped at the time of infusion but improved significantly by months 3 and 6 post-infusion.
* Follow-up: Median follow-up was 13 months.
Discussion on Outpatient CAR T-cell Therapy & Access:
* Access Issues: Only 20% of eligible patients currently access CAR T-cell and bispecific therapies. Community settings offer lower costs of care.
* FDA Changes: The FDA recently eased REMS requirements, potentially facilitating wider access in community practices.
* Payer Concerns: Payers (like Humana) are questioning weather the removal of dual certification requirements for community practices will impact the safety and effectiveness of outpatient delivery.
* Trial Setting: The ZUMA-24 trial included both academic and community centers, including OneOncology’s Greco-Hainsworth Tennessee Oncology Centers.
In essence, the ZUMA-24 trial suggests outpatient axi-cel administration is feasible, though not without significant toxicity requiring hospitalization. The broader conversation centers on how to safely and effectively expand access to these potentially life-saving therapies beyond major academic centers, while addressing payer concerns about safety and cost.