CAR-Multicenter Analysis (CAR-MA): Retrospective Study to Characterize CAR T-cell Outcomes and Related Toxicities in Children and Young Adults With B-ALL

Study Description:

This retrospective protocol focuses on characterizing clinical outcomes and toxicities following CAR T-cell therapy.

Objectives:

Primary

• To evaluate the Response Free Survival (RFS) at 6 months following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab. COMPLETED.
• To retrospectively evaluate outcomes following CAR T-cell therapy across children and young adults with B-ALL

Secondary

• To evaluate the RFS at 12 months following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab and other immunotherapy. COMPLETED.
• To evaluate the incidence of CD19 negative versus CD19 positive relapse following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab. COMPLETED.
• To evaluate the Complete Response (CR) rate following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab. COMPLETED.
• To evaluate the Minimal Residual Disease (MRD) negative remission rate following CD19 CAR stratified by prior blinatumomab vs no prior blinatumomab. COMPLETED.
• To evaluate the impact of prior blinatumomab, prior inotuzumab, and other immunotherapies on subsequent CAR T cell outcomes.
• To determine the incidence, severity, resolution and risk factors for early and late cytopenias after CAR T-cell therapy.
• To evaluate the response of extramedullary disease following CAR T-cell therapy.
• To evaluate the frequency of subsequent malignant neoplasms after CAR T-cell therapy.
• To describe response, survival, and toxicities after CAR Tcell therapy in children with trisomy 21 and other important subpopulations.
• To determine the impact of next-generation sequencing MRD results and duration of B-cell aplasia on CAR T cell outcomes.
• To evaluate the frequency of infections and describe immune system function after CAR T-cell therapy.
• To describe response, survival, and toxicities after CAR Tcell therapy in children with leukemia containing specific cytogenetic lesions (e.gl. TP53, t(1;19), hypodiploidy).
• To compare toxicities and outcomes across CAR T-cell constructs (e.g., different CD19 CAR constructs, dual-targeted CARs, CD22-targeted CARs, etc.).
• To assess the impact of CAR T cells on other health-related outcomes, including, but not limited to, organ function, immune reconstitution, quality of life, and patient-reported outcomes.

• Subjects will not be recruited for this study; however, up to 210 subjects records will be selected from treatment protocols who received CAR therapy for B-ALL. Subject who opted out of the future use of his/her data will be excluded. The subjects enrolled to a CAR T cell therapy treatment protocol within the Pediatric Oncology Branch unless, are < 25 years of age at the time of diagnosis and must have received prior a CAR T-cell product.