αβT- and B-cell-depleted HLA-haploidentical hematopoietic stem cell transplantation in children with myelodysplastic syndromes

Abstract

Not available.

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

“…ASCT2031 : Allogeneic HCT is the best option for cure for many high‐risk malignancies, but acute and chronic GVHD cause substantial short‐ and long‐term morbidity and mortality. Preliminary data from phase 2 studies in both children and adults showed that transplants from haploidentical donors using GVHD prophylaxis that included either T‐cell receptor (TCR)αβ + T‐cell/CD19 + B‐cell depletion or post‐HCT cyclophosphamide with tacrolimus and mycophenolate prophylaxis (PTCy/Tac/MMF) reduced severe acute and chronic GVHD without increasing relapse or TRM 41,42 . ASCT2031 is a phase 3 randomized trial that hypothesizes that recipients of a haploidentical donor HCT using either TCRαβ + T‐cell/CD19 + B‐cell depletion or PTCy/Tac/MMF prophylaxis will have less severe GVHD without more relapses than recipients of HLA‐matched unrelated donor (MUD) HCT using standard tacrolimus and methotrexate prophylaxis.…”

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

“…Preliminary data from phase 2 studies in both children and adults showed that transplants from haploidentical donors using GVHD prophylaxis that included either T-cell receptor (TCR)αβ+ T cell/CD19+ B cell depletion or post-HCT cyclophosphamide with tacrolimus and mycophenolate prophylaxis (PTCy/Tac/MMF) reduced severe acute and chronic GVHD without increasing relapse or TRM. 41,42 ASCT2031 is a phase 3 randomized trial that hypothesizes that recipients of a haploidentical donor HCT using either TCRαβ+ T cell/CD19+ B cell depletion or PTCy/Tac/MMF prophylaxis will have less severe GVHD without more relapses than recipients of HLA-matched unrelated donor (MUD) HCT using standard tacrolimus and methotrexate prophylaxis. Eligible patients must have either acute leukemia or myelodysplastic syndrome (MDS) and access to both donor sources for randomization.…”

Disease burden at time of transplant is a primary predictor of outcomes in pediatric MDS: A single center experience

“…(9,10) Single institution trials have reported similar or more favorable OS, NRM and disease recurrence results across stem cell sources, with the exception of lower relapse rates reported more recently in a single center pediatric cohort who received umbilical cord blood transplantation or αβT-and B-cell depleted HLA-haploidentical transplantation. (11)(12)(13)(14)(15) While HCT outcomes for pediatric MDS are similar across the largest registry and single center trials, factors identi ed as contributing to inferior outcomes vary from study to study. HLA-mismatched unrelated donor (MMUD) HCT, excluding cord blood and haploidentical donor sources, resulted in inferior outcomes related to increased incidence of relapse or NRM.…”

“…Two recent single center analyses showed clear survival improvement for more recently transplanted children with MDS. (12,13) Therefore, we performed an updated analysis to provide more clarity on the prognostic implications of disease characteristics including blast burden and cytogenetic abnormalities in the current era. (16,17)The analysis includes results of 36 consecutive children transplanted for MDS between 2000 and 2019 at the Fred Hutchinson Cancer Center with standardized supportive care and GVHD management guidelines.…”