Abstract
BackgroundThe use of post-transplant cyclophosphamide (PTCy) is highly effective in preventing graft-versus-host disease (GVHD) in the haploidentical (Haplo) transplant setting and is being increasingly used in matched sibling (MSD) and matched unrelated (MUD) transplants. There is no information on the impact of donor types using homogeneous prophylaxis with PTCy.MethodsWe retrospectively compared outcomes of adult patients with acute myeloid leukemia (AML) in first complete remission (CR1) who received a first allogeneic stem cell transplantation (SCT) with PTCy as GVHD prophylaxis from MSD (n = 215), MUD (n = 235), and Haplo (n = 789) donors registered in the EBMT database between 2010 and 2017.ResultsThe median follow-up was 2 years. Haplo-SCT carried a significantly increased risk of acute grade II–IV GVHD (HR 1.6; 95% CI 1.1–2.4) and NRM (HR 2.6; 95% CI 1.5–4.5) but a lower risk of relapse (HR 0.7; 95% CI 0.5–0.9) that translated to no differences in LFS (HR 1.1; 95% CI 0.8–1.4) or GVHD/relapse-free survival (HR 1; 95% CI 0.8–1.3). Interestingly, the use of peripheral blood was associated with an increased risk of acute (HR 1.9; 95% CI 1.4–2.6) and chronic GVHD (HR 1.7; 95% CI 1.2–2.4) but a lower risk of relapse (HR 0.7; 95% CI 0.5–0.9).ConclusionsThe use of PTCy in patients with AML in CR1 receiving SCT from MSD, MUD, and Haplo is safe and effective. Haplo-SCT had increased risk of acute GVHD and NRM and lower relapse incidence but no significant difference in survival.
Highlights
post-transplant cyclophosphamide (PTCy) is being increasingly used in other allogeneic transplant settings, such as stem cell transplant (SCT) from human leukocyte antigen (HLA)-matched sibling donors (MSD) [4,5,6], matched unrelated donors (MUD), and mismatched unrelated donors (MMUD) [1,2,3, 7]
Haplo-SCT recipients received more frequently Myeloablative conditioning (MAC) (p = 0.006) and bone marrow (BM) as stem cell source (p < 0.001), while the proportion of in vivo T cell depletion was higher in MUD transplants (p < 0.001)
The vast majority (93%) of Haplo patients received GvHD prophylaxis with PTCy combined with 2 other immunosuppressive (IS) drugs, only 47% and 26% of MUD and MSD patients received such combination, respectively
Summary
The use of post-transplant cyclophosphamide (PTCy) has proven to be highly effective in preventing graftversus-host (GVHD) and reducing non-relapse mortality (NRM) rates in haploidentical (Haplo) hematopoietic stem cell transplant (SCT) [1,2,3].As a consequence, PTCy is being increasingly used in other allogeneic transplant settings, such as SCT from HLA-matched sibling donors (MSD) [4,5,6], matched unrelated donors (MUD), and mismatched unrelated donors (MMUD) [1,2,3, 7].Since the first reports of Haplo-SCT using PTCy, there has been significant interest in comparing this platform with those using other donor types such as cord blood from unrelated donors [4,5,6, 8] or bone marrow (BM) or peripheral blood (PB) from MSD, MUD, and MMUD transplants [9,10,11,12,13,14,15]. The use of post-transplant cyclophosphamide (PTCy) has proven to be highly effective in preventing graftversus-host (GVHD) and reducing non-relapse mortality (NRM) rates in haploidentical (Haplo) hematopoietic stem cell transplant (SCT) [1,2,3]. Safety and feasibility of PTCy-based GVHD prophylaxis in transplants using different donor types was further evaluated in small single-center prospective non-randomized studies showing comparable outcomes in Haplo-SCT compared to 9/10 MUD [18], as well as in MSD, MUD, and MMUD transplants [19]. The use of post-transplant cyclophosphamide (PTCy) is highly effective in preventing graft-versus-host disease (GVHD) in the haploidentical (Haplo) transplant setting and is being increasingly used in matched sibling (MSD) and matched unrelated (MUD) transplants. There is no information on the impact of donor types using homogeneous prophylaxis with PTCy
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