Favorable Results After GCSF-Primed Allogeneic BMT for Children with Severe Aplastic Anemia Using Busulfan, Cyclophosphamide and ATG as Conditioning Regimen and Cyclosporine-A and Micofenolate Mofetil as Graft-Versus-Host-Disease Prophylaxys: Rapid Engraf

Mauricio Ostronoff, MD1, Fabiana Ostronoff, MD2*, Rodolfo Calixto3*, Alexandre Sucupira4*, Manuela Gomes5*, Rodrigo Florencio, MD6*, Monique Martins1* and ANA Patricia Souto Maior1*

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1BONE MARROW TRANSPLANTATION, Real Hosp. Portugues - Real TMO, Recife, Brazil 2BMT, Real Hospital Portuguës - Real TMO, Recife, Brazil 3BONE MARROW TRANSPLANTATION, REAL HOSPITAL PORTUGUES, Recife, Brazil 4BMT, REAL HOSPITAL PORTUGUES, Recife 5Re


Patients with SAA in Brazil, in general, receive multiple transfusions and prolonged immunosuppresion with steroids and/ or cyclosporine (CSA) prior to undergoing allo-SCT. Therefore the rate of early and late graft rejection and infectious complications are high. GCSF-primed allo-BMT has shown to accelerate neutrophil recovery after allo-BMT from HLA-matched related donor (Ostronoff M. et al. Biol Blood Marrow Transplant 2006; 12:729-33). Conditioning regimen using busulfan (Bu) and cyclophosphamide (Cy) for SAA has been shown to decrease graft rejection after allo-BMT in patients who received multiple transfusions. To faster the neutrophil recovery and reduce the incidence of graft rejection, we used GCSF-primed allo-BMT from matched related donor to treat 22 polytransfused patients with SAA after conditioning with Bu and Cy. In addition, we incorporate ATG to our conditioning regimen to reduce both GVHD and graft rejection. From May 2005, 22 patients with SAA, median age 14 years (range 3-21) mail all heavily transfused (=2 cells) occurred in 9 patients (40%) and one patient developed CT scan findings suggestive of CMV pneumonia. All patients were successfully treated with gancyclovir. Grade II-IV acute GVHD extensiv chronic occurred in 6 patients (27%) and grade III-IV occurred in 2 patients (9%). Extensive GVHD occurred in 1 patient (5%). Two patients died: 1 due to complications of acute GVHD and one from aspergillosis which was probably acquired prior to allo-BMT as the patient was leucopenic under immunosuppression with steroids for 4 years prior to transplant. The overall survival was 91%, with median follow up of 36 months (range 6 to 60 months). The neutrophil engraftment was fast, the incidence of chronic GVHD and graft reject was low. The immunosuppression was significant, requiring constant surveillance for infectious process and treatment with antivirals. The overall survival and quality of life in this patient population was notable.

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Full conference title: 

52nd American Society of Haematologists Annual Meeting
    • ASH 52nd (2010)