Results of a multicenter phase I/II trial of TCR and CD19-depleted haploidentical hematopoietic stem cell transplantation for adult and pediatric…

Hematopoietic stem cell transplantation (HSCT) from haploidentical donors is a viable option for patients lacking HLA-matched donors. Here we report the results of a prospective multicenter phase I/II trial of transplantation of TCR and CD19-depleted peripheral blood stem cells from haploidentical family donors after a reduced-intensity conditioning with fludarabine, thiotepa, and melphalan. Thirty pediatric and 30 adult patients with acute leukemia (n=43), myelodysplastic or myeloproliferative syndrome (n=6), multiple myeloma (n=1), solid tumors (n=6), and non-malignant disorders (n=4) were enrolled. TCR /CD19-depleted grafts prepared decentrally at six manufacturing sites contained a median of 12.1106CD34+cells/kg and 14.2103TCR+T-cells/kg. None of the patients developed grade lll/IV acute graft-versus-host disease (GVHD) and only six patients (10%) had grade II acute GVHD. With a median follow-up of 733 days 36/60 patients are alive. The cumulative incidence of non-relapse mortality at day 100, 1 and 2 years after HSCT was 5%, 15%, and 17% for all patients, respectively. Estimated probabilities of overall and disease-free survival at 2 years were 63% and 50%, respectively. Based on these promising results in a high-risk patient cohort, haploidentical HSCT using TCR/CD19-depleted grafts represents a viable treatment option.

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Results of a multicenter phase I/II trial of TCR and CD19-depleted haploidentical hematopoietic stem cell transplantation for adult and pediatric...