Haploidentical transplantation in patients with acquired aplastic anemia

Abstract

on behalf of the SAA-WP EBMT Haploidentical SCT (haplo-SCT) has been considered a therapeutic option in patients with acquired severe aplastic anemia (SAA) failing at least one course of immune suppressive therapy with antithymocyte globulin and lacking an HLA-matched related or unrelated donor. The platforms of both ex vivo T-cell-depleted and unmanipulated grafts have been explored in children and adults. Overall, the primary objective of a stable haploidentical hematopoietic engraftment with a low rate of GVHD is unmet in a significant proportion of patients undergoing haplo-SCT for SAA. Haploidentical transplants for refractory SAA should be performed in a specialist center with major experience in hematopoietic SCT procedures and preferably performed within the framework of a local clinical protocol designed specifically to address the prevention of graft rejection and GVHD.

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

“…3,[6][7][8][9][10] Higher risk HSCT for those who lack a histocompatible donor often include umbilical cord blood and haploidentical donor as a source of HSCs (HSCs). [11][12][13][14][15][16][17][18][19] The use of haploidentical donors has some advantages compared with matched unrelated donors (MUD) and cord blood units, as a donor is more widely and readily available usually within the family, abbreviating the time for stem cell collection and infusion into the recipient. 18 However, past reports of haploidentical HSCT as therapy in SAA have encountered important limitations with graft failure and risk of severe graft-versus host disease (GVHD).…”

“…[11][12][13][14][15][16][17][18][19] The use of haploidentical donors has some advantages compared with matched unrelated donors (MUD) and cord blood units, as a donor is more widely and readily available usually within the family, abbreviating the time for stem cell collection and infusion into the recipient. 18 However, past reports of haploidentical HSCT as therapy in SAA have encountered important limitations with graft failure and risk of severe graft-versus host disease (GVHD). [11][12][13][14][15][16][17][18]20,21 Recently, the use of post-transplant cyclophosphamide (Cy) has permitted transplantation across major HLA barriers for patients receiving BM from a haploidentical donor.…”

“…18 However, past reports of haploidentical HSCT as therapy in SAA have encountered important limitations with graft failure and risk of severe graft-versus host disease (GVHD). [11][12][13][14][15][16][17][18]20,21 Recently, the use of post-transplant cyclophosphamide (Cy) has permitted transplantation across major HLA barriers for patients receiving BM from a haploidentical donor. [22][23][24][25][26] The use of posttransplant Cy has several advantages compared with traditional methods of ex vivo T-cell depletion, which include lower complexity and cost, and improved immune reconstitution permitting a more widespread adoption of this transplant procedure.…”

Haploidentical hematopoietic SCT for acquired severe aplastic anemia using post-transplant high-dose CY

“…When using an unmanipulated BM as a graft source, failure rates of 25% and an OS of 30% was reported by EBMT. 9 Recently Castagna et al 10 reported 100% engraftment rates after GCSF primed BM or PBSC haploidentical transplants, but a high incidence of chronic GVHD was seen with an OS of 64%. There are few reports of patients transplanted with purified CD34+ cells or recently with selective CD3/CD3 and CD19 depletion.…”

Outcome of patients activating an unrelated donor search for severe acquired aplastic anemia

“…As illustrated in around one third of patients in our cohort, the search for an HLA-matched VUD may be unsuccessful. Thus, the usefulness of alternative HSCT procedures using haplo-identical donors [30][31][32] or cordblood cells [33] in these patients also needs to be further explored in the near future. research article…”