Merli P, Algeri M, Galaverna F, et al. TCRαß/CD19 Cell-Depleted HLA-Haploidentical Transplantation to Treat Pediatric Acute Leukemia: Updated Final Analysis. Blood. 2023; (doi: 10.1182/blood.2023021336).
A clinical update of TCRαß/CD19-cell depleted haploidentical hematopoietic stem cell transplantation (HSCT) enabled the identification of pediatric patients with acute leukemia who were at higher risk of treatment failure and required more personalized therapy. An earlier Phase I-II clinical trial of the treatment involving 80 children yielded encouraging results. The updated analysis involved 213 children with a longer follow-up, a median of 47.6 months for surviving patients. The 5-year cumulative incidence of non-relapse mortality in this cohort was 5.2%, with a 22.7% cumulative incidence of relapse. The projected rate of 10-year overall survival was 75.4%, while the rate of disease-free survival was 71.6%. The cumulative incidence of grade II-IV acute graft-versus-host disease (GVHD) was 14.7% and the cumulative incidence of chronic GVHD was 8.1%. Factors independently associated with outcome in multivariable analysis included disease type, use of total body irradiation in the conditioning regimen, disease status at HSCT, and increased levels of pre-HSCT minimal residual disease.