Since the publication of the last Cellular Therapy and Stem Cell Transplant blueprint in 2013, Children’s Oncology Group cellular therapy-based trials for 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 pre-transplant 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.

Certain patients with inborn errors of immunity have defects in DNA damage response, predisposing them to malignancy. Subsequent cancer therapy may require substantial attenuation given defective DNA repair; however, this carries risk of incomplete disease control. We describe a 5-year-old boy with peripheral T-Cell lymphoma with ataxia-telangiectasia (A-T). After incomplete chemotherapeutic response, he underwent allogenic hematopoietic cell transplantation (allo-HCT) with an attenuated preparative regimen, but developed graft rejection and relapse. Following remission with salvage chemotherapy, second allo-HCT with reduced intensity conditioning (RIC) resulted in minimal toxicity and short-term disease control. HCT with RIC can be considered in patients with A-T.