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Randomized Controlled Trial
. 2022 Oct;57(10):1573-1580.
doi: 10.1038/s41409-022-01754-y. Epub 2022 Jul 15.

Low-dose post-transplant cyclophosphamide with low-dose antithymocyte globulin for prevention of graft-versus-host disease in first complete remission undergoing 10/10 HLA-matched unrelated donor peripheral blood stem cell transplants: a multicentre, randomized controlled trial

Yingling Zu  1   2 , Zhen Li  1 , Ruirui Gui  1 , Yanyan Liu  1 , Yanli Zhang  1 , Fengkuan Yu  1 , Huifang Zhao  1 , Yuewen Fu  1 , Xinrong Zhan  3 , Zhongliang Wang  3 , Pengtao Xing  3 , Xianjing Wang  4 , Huili Wang  4 , Jian Zhou  5 , Yongping Song  6   7
Affiliations
Randomized Controlled Trial

Low-dose post-transplant cyclophosphamide with low-dose antithymocyte globulin for prevention of graft-versus-host disease in first complete remission undergoing 10/10 HLA-matched unrelated donor peripheral blood stem cell transplants: a multicentre, randomized controlled trial

Yingling Zu et al. Bone Marrow Transplant. 2022 Oct.

Abstract

The most widely used regimens of graft-versus-host disease (GVHD) prophylaxis in HLA-matched unrelated donor peripheral blood stem cell transplantation (MUD-PBSCT) are based on anti-thymocyte globulin (ATG) or post-transplant cyclophosphamide (PTCy). To improve the efficiency of GVHD prophylaxis, a novel regimen, composed of low-dose PTCy (20 mg/kg on day +3 and +4) and low-dose ATG (6 mg/kg), was evaluted in patients with hematological malignancies ungoing 10/10 HLA MUD-PBSCT in first remission (CR1). In our prospective, multicenter study, 104 patients were randomly assigned one-to-one to low-dose PTCy-ATG (n = 53) or standard-dose ATG (10 mg/kg, n = 51). Both the cumulative incidences (CIs) of grade II-IV acute GVHD (aGVHD) and chronic GVHD (cGVHD) at 2 years in low-dose PTCy-ATG cohort were significantly reduced (24.5% vs. 47.1%; P = 0.017; 14.1% vs. 33.3%; P = 0.013). The CI of non-relapse-mortality (NRM) was much lower (13.2% vs. 34.5%; P = 0.049) and GVHD-free, relapse-free survival (GRFS) was significantly improved at 2 years in low-dose PTCy-ATG arm (67.3% vs 42.3%; P = 0.032). The low-dose PTCy-ATG based GVHD prophylaxis is a promising strategy for patients in CR1 after 10/10 HLA MUD-PBSCT.

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Conflict of interest statement

The authors declare no competing interests.

Figures

Fig. 1
Fig. 1. Flowchart of the study participants.
PTCy post-transplant cyclophosphamide, ATG anti-thymocyte globulin.
Fig. 2
Fig. 2. Cumulative incidences (CIs) of graft-versus-host-disease (GVHD) between low-dose post-transplant cyclophosphamide (PTCy) combined with low-dose anti-thymocyte globulin (ATG) and standard-dose ATG cohorts.
a The CI of grade II-IV acute GVHD (aGVHD); b The CI of grade III-IV aGVHD; c The 2-year CI of chronic GVHD (cGVHD); d The 2-year CI of moderate to severe cGVHD.
Fig. 3
Fig. 3. Clinical outcomes after matched unrelated donor peripheral blood stem cell transplantation (MUD-PBSCT) between low-dose post-transplant cyclophosphamide (PTCy) combined with low-dose anti-thymocyte globulin (ATG) and standard-dose ATG cohorts.
a The 2-year CI of relapse; b The 2-year CI of non-relapse-mortality (NRM); c The 2-year probability of overall survival (OS); d The 2-year probability of disease-free survival (DFS); e The 2-year CI of GVHD-free, relapse-free survival (GRFS).
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