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In Vivo Osteo-organoid Approach for Harvesting Therapeutic Hematopoietic Stem/Progenitor Cells
Published on: February 16, 2024
Children's Oncology Group's 2023 blueprint for research: Cellular therapy and stem cell transplantation
Carrie L Kitko1, Catherine M Bollard2,3,4, Mitchell S Cairo5
1Pediatric Stem Cell Transplant Program, Vanderbilt University Medical Center, Nashville, Tennessee, USA.
Abstract:
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.
Insights
Pediatric cancer trials show tandem transplants improve survival for some tumors. Umbilical cord blood transplants are safer with one donor, and minimal residual disease predicts relapse risk in acute lymphoblastic leukemia.
Area of Science:
- Pediatric Oncology
- Hematology
- Immunology
Background:
- Children's Oncology Group trials have significantly advanced cellular therapy and stem cell transplantation since 2013.
- New standards of care have been established for various pediatric cancers based on recent research.
Purpose of the Study:
- To review key advancements in pediatric cellular therapy and stem cell transplantation.
- To highlight findings that have shaped current treatment protocols.
- To outline future directions in the field.
Main Methods:
- Analysis of data from Children's Oncology Group cellular therapy-based clinical trials.
- Review of survival outcomes, safety data, and predictive markers in pediatric cancer patients.
Main Results:
- Tandem autologous transplants improved survival in neuroblastoma and atypical teratoid/rhabdoid brain tumors.
- Single umbilical cord blood (UCB) donor transplants demonstrated improved safety compared to double UCB donor transplants.
- Minimal residual disease (MRD) assessment pretransplant is a strong predictor of relapse in acute lymphoblastic leukemia (ALL).
- Killer immunoglobulin receptor (KIR) mismatched donors did not enhance survival in pediatric acute myeloid leukemia (AML) with in vivo T-cell depletion.
Conclusions:
- Cellular therapy and stem cell transplantation have evolved, setting new benchmarks in pediatric cancer care.
- Future research will focus on optimizing donor selection, engineered cellular therapies, and managing treatment toxicities.
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