Abstract

BackgroundCompared with HLA-matched sibling donor (MSD) transplant, the outcomes of haploidentical donor (HID) transplant for refractory acute leukemia need to be further explored. In this study, we compared the outcomes of HID with MSD for refractory acute leukemia.Patients and methodsThis study population came from two prospective multicenter trials (NCT01883180, NCT02673008). Two hundred and seventy-eight patients with refractory acute leukemia were enrolled in this study, including 119 in HID group and 132 in MSD group. Sequential intensified conditioning was employed in all patients, and donor lymphocyte infusion (DLI) was administered in patients in the absence of active GVHD and according to minimal residual disease (MRD) from day + 60 post-transplantation for preventing relapse.ResultsThe complete remission of leukemia by day + 30 post-transplant were 94% and 93%, respectively, in HID and MSD groups (p = .802). The 1-year incidence of grades II–IV acute GVHD was 62% and 54% (p = .025), and 3-year incidence of chronic GVHD was 55% and 55% (p = .789), respectively, in two groups. HID transplant had lower incidence of first episode of MRD positivity and relapse than MSD transplant (28% vs 45%, p = .006; 26% vs 38%, p = .034). There was higher infection-related mortality in HID than MSD (8% vs 2%, p = .049) within the first 100 days’ post-transplant. The 5-year overall survival was 46% and 42% (p = .832), respectively; the 5-year disease-free survival was 43% and 39% (p = .665), in HID and MSD groups, respectively.ConclusionsHID transplant has lower relapse, but higher infection-related mortality and similar survival rates in refractory acute leukemia by the strategy of sequential intensified conditioning followed by DLI compared with MSD transplant.

Highlights

  • Allogeneic hematopoietic stem cell transplantation(allo-HSCT)remains the most effective way to cure refractory leukemia [1,2,3,4] and undergoing allo-HSCT promptly is essential for them

  • haploidentical donor (HID) transplant has lower relapse, but higher infection-related mortality and similar survival rates in refractory acute leukemia by the strategy of sequential intensified conditioning followed by donor lymphocyte infusion (DLI) compared with matched sibling donor (MSD) transplant

  • With improvements having been made in haploidentical donor (HID) transplant strategies, some studies showed that transplant outcomes of HID were similar to MSD in acute leukemia [11,12,13,14], but data comparing HID with MSD for refractory leukemia are quite limited [15, 16]

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Summary

Introduction

Allogeneic hematopoietic stem cell transplantation(allo-HSCT)remains the most effective way to cure refractory leukemia [1,2,3,4] and undergoing allo-HSCT promptly is essential for them. Quick access to appropriate donors is one of the key elements to the success of transplantation for refractory leukemia. As it is known, only 25–30% patients can get a HLA-matched sibling donor (MSD), and most patients cannot wait to search for a suitably matched unrelated donor if it is not already available. With improvements having been made in haploidentical donor (HID) transplant strategies, some studies showed that transplant outcomes of HID were similar to MSD in acute leukemia [11,12,13,14], but data comparing HID with MSD for refractory leukemia are quite limited [15, 16]. Compared with HLA-matched sibling donor (MSD) transplant, the outcomes of haploidentical donor (HID) transplant for refractory acute leukemia need to be further explored. We compared the outcomes of HID with MSD for refractory acute leukemia

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