Haploidentical BMT and post-transplant Cy for severe aplastic anemia: a multicenter retrospective study

Abstract

Patients with refractory severe aplastic anemia (SAA) who lack a matched sibling or unrelated donor need new therapeutic approaches. Hematopoietic SCT (HSCT) using mismatched or haploidentical related donors has been used in the past, but was associated with a significant risk of GVHD and mortality. Recently, the use of post-transplant cyclophosphamide (Cy) has been shown to be an effective strategy to prevent GVHD in recipients of haploidentical HSCT, but the majority of reports have focused on patients with hematology malignancies. We describe the outcome of 16 patients who underwent haploidentical transplantation using a reduced-intensity conditioning regimen with post-transplant Cy. Stem cell sources were BM (N = 13) or PBSCs (N = 3). The rate of neutrophil engraftment was 94% and of platelet engraftment was 75%. Two patients had secondary graft failure and were successfully salvaged with another transplant. Three patients developed acute GVHD being grades 2-4 in two. Five patients have died and the 1-year OS was 67.1% (95% confidence interval: 36.5-86.4%). In our small series, the use of a reduced-intensity conditioning with post-transplant Cy in haploidentical BMT was associated with high rates of engraftment and low risk of GVHD in patients with relapsed/refractory SAA.

Cyclophosphamide improves engraftment in patients with SCD and severe organ damage who undergo haploidentical PBSCT

“…The addition of PT-Cy has been shown to decrease the incidence of GVHD in the malignant and nonmalignant setting. 37,48,[51][52][53] We acknowledge the fact that higher graft rejection is a competing risk against GVHD but predict that a modest intensification of conditioning will not significantly increase GVHD incidence if PT-Cy remains part of the conditioning.…”

Hematopoietic stem cell transplantation for acquired aplastic anemia

“…Five of the 8 patients survived 3–40 months after transplant, 1 had successfully treated grade II skin GVHD(43)**. Other investigators have reported similar results with this regimen, but patients received 2 to 6 Gy TBI(44). …”

Acquired aplastic anemia in Korean children: treatment guidelines from the Bone Marrow Failure Committee of the Korean Society of Pediatric Hematology Oncology

“…Recent studies on T-cellreplete haploidentical HSCT reported survival rates of 65-85 % after ATG containing regimen [41,42]. A report on haploidentical transplant using RIC with high-dose PTCY showed OS rates of 67.1 % in 16 AA patients [43].…”