The outcome of unrelated donor (UD) transplants for acquired severe aplastic anemia (SAA) has considerably improved over the past few years, such that current results are comparable to matched sibling donor grafts, though still not identical. Commonly a UD transplant is proposed after the patient has received a first course of immunosuppressive therapy (IST), and has failed to recover hematopoiesis at 4 months. However, in selected children, with very severe aplasia, UD transplants have been offered upfront as first line therapy, and have shown a high degree of success.Issues which still require careful evaluation include early and late graft failure (GF), graft versus host disease (GvHD), and infections, all of which are more common in older patients. GF is an issue, especially in sensitized patients, and is not improved with the use of peripheral blood (PB) as a stem cell source: the use of antithymocyte globulin (ATG) or alemtuzumab (Campath), and the combination of cyclophosphamide (CY) and fludarabine (FLU) with low dose total body irradiation (TBI) have been implemented and have reduced this complication. GvHD also remains a problem, often unpredictable: the strong protective effect of posttransplant CY, on acute and chronic GvHD, in haploidentical grafts, may also be applicable to UD transplants. Finally infections, and among these EBV reactivation, are always an issue in severely neutropenic patients: better diagnostic strategies and anti-CD20 antibodies have significantly reduced morbidity and mortality.In the era of high resolution HLA typing, matched UD transplants are the first choice for patients not responding to IST, and may also be considered as first line in the context of a clinical trial or in selected young patients.
|Title of host publication||Congenital and Acquired Bone Marrow Failure|
|Number of pages||10|
|Publication status||Published - Jan 9 2017|
- Aplastic anemia
- Unrelated donors
ASJC Scopus subject areas