Unrelated donor transplantation for relapsed refractory aplastic anemia, with quadruple GVHD prophylaxis

The outcome of unrelated donor (UD) transplants in patients with acquired severe aplastic anemia (SAA) has improved over time [1, 2], such that survival is now comparable to transplants from matched sibling donors (MSD) [3]. Despite high-resolution HLA matching [1], improved supportive care and GvHD prophylaxis, a longer interval from diagnosis to transplant, and older patient age remain negative predictors [4, 5]. The Baltimore group has developed a transplant platform for haploidentical (HAPLO) transplants in SAA, with four drugs for GvHD prophylaxis, namely anti-thymocyte globulin ATG, post-transplant cyclophosphamide (PTCY), a calcineurin inhibitor (CNI), and mycophenolate (MMF) [6]: results have been very good in terms of engraftment and control of GvHD, such that the Baltimore group has moved from HAPLO transplants in relapse/refractory (R/R) patients [6] to upfront transplant at diagnosis of SAA [7]. A small number of patients have been reported in Brazil, using the Baltimore regimen for UD transplants in SAA with encouraging early result