Children’s Oncology Group’s 2023 Blueprint for Research: Cellular
Therapy and Stem Cell Transplantation
Abstract
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.