Haploidentical Donor Bone Marrow Transplantation for Acquired Severe Aplastic Anemia
摘要
Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) has emerged as a viable and increasingly utilized option for patients with acquired severe aplastic anemia (SAA) lacking a matched sibling or unrelated donor. Recent innovations, particularly post-transplant cyclophosphamide (PTCy)–based regimens, have improved outcomes by reducing graft-versus-host disease (GVHD) and enhancing engraftment. Multiple retrospective and prospective studies demonstrate encouraging survival rates (up to 90%), low rates of chronic GVHD, and successful hematopoietic reconstitution, especially when bone marrow (BM) is used as the graft source. Conditioning regimens incorporating fludarabine (Flu), cyclophosphamide, and low-dose total body irradiation (TBI) or antithymocyte globulin (ATG) have shown favorable safety and efficacy profiles. Factors such as donor-specific antibodies (DSAs), graft source, and CD34+ cell dose are key determinants of success. Haplo-HSCT is now considered both as a salvage and a frontline therapy in selected patients, supported by emerging data and ongoing clinical trials. This strategy significantly expands donor availability and offers curative potential for high-risk or refractory SAA patients.