Optimal stem cell source for allogeneic stem cell transplantation for hematological malignancies

Abstract

Hematopoietic stem cell transplant (HSCT) is a standard treatment for many hematological malignancies. Three different sources of stem cells, namely bone marrow (BM), peripheral blood stem cells (PBSC) and cord blood (CB) can be used for HSCT, and each has its own advantages and disadvantages. Randomized controlled trials (RCTs) suggest that there is no significant survival advantage of PBSC over BM in Human Leukocyte Antigen-matched sibling transplant for adult patients with hematological malignancies. PBSC transplant probably results in lower risk of relapse and hence better disease-free survival, especially in patients with high risk disease at the expense of higher risks of both severe acute and chronic graft-versus-host disease (GVHD). In the unrelated donor setting, the only RCT available suggests that PBSC and BM result in comparable overall and disease-free survivals in patients with hematological malignancies; and PBSC transplant results in lower risk of graft failure and higher risk of chronic GVHD. High level evidence is not available for CB in comparison to BM or PBSC. The risks and benefits of different sources of stem cells likely change with different conditioning regimen, strategies for prophylaxis and treatment of GVHD and manipulation of grafts. The recent success and rapid advance of double CB transplant and haploidentical BM and PBSC transplants further complicate the selection of stem cell source. Optimal selection requires careful weighing of the risks and benefits of different stem cell source for each individual recipient and donor. Detailed counseling of patient and donor regarding risks and benefits in the specific context of the patient and transplant method is essential for informed decision making.

Keywords: Hematopoietic stem cell transplantation; Bone marrow; Peripheral blood stem cell; Cord blood; Hematological malignancy

Core tip: Randomized controlled trials (RCTs) suggest no difference in survival between peripheral blood stem cell (PBSC) and bone marrow (BM) in matched sibling transplant for patients with hematological malignancies. PBSC may result in fewer relapse in high risk patients but more severe graft-versus-host disease (GVHD). For unrelated donor, the only RCT suggests PBSC and BM result in comparable survivals, with PBSC resulting in fewer graft failure but more chronic GVHD. RCT is not available to compare cord blood with BM or PBSC. The risks and benefits of different sources of stem cells likely change with transplant methods and manipulation of grafts.