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

Overview

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 T-cell 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 T-cell 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. Study Population and Source of Data: Subjects who were less than \< 25 years of age at the time of diagnosis and received a CAR T-cell product for 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.