Children's Oncology Group's 2023 blueprint for research: Cellular therapy and stem cell transplantation

Since the publication of the last Cellular Therapy and Stem Cell Transplant blueprint in 2013, Children's Oncology Group cellular therapy-based trials advanced the field and created new standards of care across a wide spectrum of pediatric cancer diagnoses. Key findings include that tandem autologous transplant improved survival for patients with neuroblastoma and atypical teratoid/rhabdoid brain tumors, one umbilical cord blood (UCB) donor was safer than two UCB donors, killer immunoglobulin receptor (KIR) mismatched donors did not improve survival for pediatric acute myeloid leukemia when in vivo T-cell depletion is used, and the depth of remission as measured by next-generation sequencing-based minimal residual disease assessment pretransplant was the best predictor of relapse for acute lymphoblastic leukemia. Plans for the next decade include optimizing donor selection for transplants for acute leukemia/myelodysplastic syndrome, using novel engineered cellular therapies to target a wide array of malignancies, and developing better treatments for cellular therapy toxicities such as viral infections and graft-vs-host disease.

Automated summary

Since 2013, the Children's Oncology Group cellular therapy-based trials have advanced the field and set new standards of care for pediatric cancer. Key findings include improved survival with tandem autologous transplant for neuroblastoma and atypical teratoid/rhabdoid brain tumors, safer single umbilical cord blood donor compared to two donors, lack of survival improvement with killer immunoglobulin receptor (KIR) mismatched donors for pediatric acute myeloid leukemia, and the importance of minimal residual disease assessment for predicting relapse in acute lymphoblastic leukemia. Future plans include optimizing donor selection, using engineered cellular therapies, and developing better treatments for therapy toxicities.