Abstract
In patients with very severe aplastic anemia (VSAA), neutropenia is prolonged and persistent, resulting in refractory overwhelming infections. Hematopoiesis recovery is urgently needed. Six patients with de novo VSAA lacking HLA-identical sibling donors and those who experienced refractory infections underwent haploidentical related donor (HRD) hematopoietic stem cell transplantation (HSCT) as a first-line therapy. The conditioning regimen consisted of busulfan, cyclophosphamide, and rabbit antithymocyte globulin. Culture-expanded allogeneic bone marrow-derived mesenchymal stromal cells were infused on day 0 and day +14. From diagnosis to HSCT, 6 patients experienced a total of 28 episodes of persistent fever, and the median number was 4 (range, 3-7). All cases developed major bacterial infections and invasive pulmonary fungal infection pre-HSCT. The median time from diagnosis to HSCT was 2months (range, 1-3.5months). All patients achieved sustained, full donor chimerism, and the median time of myeloid recovery and platelet engraftment was 13days (range, 9-19days) and 15.5days (range, 10-23days), respectively. One patient died of aGVHD, and 5 patients are alive after a median follow-up of 21months (range 17-40.5). Upfront HRD-HSCT may be a safe and promising choice for patients with VSAA in critical situations without suitably matched donors.
Published Version
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