Abstract
Severe aplastic anemia (SAA) is a bone marrow failure syndrome that can be treated with hematopoietic cell transplantation (HCT) or immunosuppressive (IS) therapy. A retrospective cohort of 56 children with SAA undergoing transplantation with fludarabine–cyclophosphamide–ATG-based conditioning (FluCyATG) was analyzed. The endpoints were overall survival (OS), event-free survival (EFS), cumulative incidence (CI) of graft versus host disease (GVHD) and CI of viral replication. Engraftment was achieved in 53/56 patients, and four patients died (two due to fungal infection, and two of neuroinfection). The median time to neutrophil engraftment was 14 days and to platelet engraftment was 16 days, and median donor chimerism was above 98%. The overall incidence of acute GVHD was 41.5%, and that of grade III-IV acute GVHD was 14.3%. Chronic GVHD was diagnosed in 14.2% of children. The probability of 2-year GVHD-free survival was 76.1%. In the univariate analysis, a higher dose of cyclophosphamide and previous IS therapy were significant risk factors for worse overall survival. Episodes of viral replication occurred in 33/56 (58.9%) patients, but did not influence OS. The main advantages of FluCyATG include early engraftment with a very high level of donor chimerism, high overall survival and a low risk of viral replication after HCT.
Highlights
Three patients died before engraftment on days +1, +5 and +17
The hematopoietic cell transplantation (HCT) results in patients with Severe aplastic anemia (SAA) after FluCyATG conditioning were unquestionably good in terms of neutrophil engraftment, predominant donor chimerism and overall survival
The benefits of FluCyATG are associated with high overall survival probability, early engraftment with a very high level of donor chimerism and minimal impact of posttransplantation opportunistic infections
Summary
Severe aplastic anemia (SAA) is a rare but life-threatening hematological disorder with an extremely high risk of fatal infectious complications. The hallmark of SAA is pancytopenia caused by bone marrow (BM) hypoplasia or aplasia as a consequence of direct damage by chemical or physical factors or constitutional or acquired genetic defects, e.g., Fanconi anemia, or telomere biology disorders [1,2,3]. In the majority of SAA patients, the cause cannot be directly identified, but immune-mediated destruction of BM hematopoiesis is the most likely culprit. Autoimmunity can be triggered by alterations in antigens modified by drugs, chemical agents or viral infections and, can lead to
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