The Safety and Feasibility of a Pediatric Ambulatory CAR-T Program

Topic Significance & Study Purpose/Background/Rationale

Chimeric antigen receptor T-cells (CAR-T cells) are a novel immunotherapy involving genetically modified T-lymphocytes designed to target tumor-specific antigens. At Texas Children's Hospital, an accredited Good Manufacturing Practice facility, we have established the infrastructure to support both commercial and investigational CAR-T-cell therapies in an outpatient setting, enhancing patient access and convenience.

Methods, Intervention, & Analysis

Our CAR-T nurse coordinator serves as the primary liaison, facilitating pre-workup testing and patient education at home or in-house. This role includes coordinating home healthcare for intravenous hydration prior to chemotherapy. The outpatient treatment regimen consists of 3-5 days of lymphodepleting chemotherapy, followed by cell infusion and a 28-day monitoring period with weekly clinic visits. Caregivers receive a patient-specific safety card on day zero, outlining fever-monitoring protocols (temperature checks twice daily), emphasizing not masking fevers, and providing direct communication channels with the care team. Comprehensive training for nursing staff is supported by standard practice guidelines and educational pathways tailored for critical care. Educating caregivers about potential side effects, particularly cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), is essential for ensuring patient safety and promoting timely follow-up.

Findings & Interpretation

Since November 2017-May 2024, TCH has successfully conducted 109 research infusions and 79 commercial CAR-T infusions. For commercial infusions, 65% were performed in the outpatient setting. We tracked admission data for these patients, revealing that 25 out of 52 patients required hospitalization post-infusion for CRS. Among those admitted, 17 presented with grade 1 CRS, while 8 experienced grades 2 and 3; 28% of these patients were transferred to the Pediatric Intensive Care Unit (PICU). Importantly, there were no grade 4 CRS admissions.

Discussion & Implications

Our findings demonstrate that CAR-T therapy can be safely administered in an ambulatory setting, offering cost-saving benefits and increased patient satisfaction. The success of this program underscores the critical role of a skilled nursing team in managing outpatient CAR-T therapy and ensuring patient safety.