Allogeneic bone marrow transplantation for severe aplastic anemia patients with risk factors for poor prognosis: is fludarabine a requirement? Transplante alogênico de medula óssea em portadores de anemia aplásica severa com fatores de mau prognóstico: é necessário fludarabina?

Hematopoietic progenitor cell transplantation from HLA-identical sibling donors cures 70-90% of Severe Aplastic Anemia (sAA) patients. Older age, heavy exposure to transfusions, immunosuppression treatment (IST) with a long interval from diagnosis to transplant and infection at procedure are associated with poor outcomes. We transplanted 18 patients with sAA and at least one risk factor (RF) for poor prognosis (age >35 years, >50 transfusions prior to transplant, unresponsiveness to previous IST and bacterial or fungal infection at transplant) from 2001 to 2005, using cyclophosphamide (CY - 5 patients) or busulfan plus CY (13 patients). Sixteen patients engrafted, two died with no engraftment, three patients had evidence of graft failure at days +67, +524 and +638 (two died and one was rescued with IST). Grade III/IV mucositis occurred in 39% but neither aGVHD nor cGVHD were observed. The Kaplan-Meier probability of survival was 75% at 2.14 years, with a trend favoring survival by number of RF (1 versus =2 RF) (P = 0.06). These results are comparable to recent data reported with fludarabine-based conditioning in patients with poor prognosis sAA. Due to the small sample size, prospective clinical trials with larger cohorts of patients are needed to confirm the real benefits of fludarabine-based conditioning, and also to define the best agent(s) to be associated with Fludarabine as preparative regimen for sAA patients with poor prognosis.Transplante de medula óssea de doador irmão HLA-idêntico pode curar 70%-90% dos portadores de anemia aplásica severa (AAs). Pacientes mais idosos, muito transfundidos, longamente tratados com imunossupressão (IS) e com infecções ao tempo do transplante têm pior evolução. Nós transplantamos 18 pacientes com AAs e pelo menos um dos fatores associados a pior prognóstico (idade >35 anos, >50 transfusões antes do transplante, falta de resposta à imunossupressão prévia e infecção bacteriana ou fúngica ao transplante) de 2001 a 2005, com ciclofosfamida (Cy - 5 pacientes) ou bussulfan mais Cy (13 pacientes). Dezesseis pacientes apresentaram pega do enxerto, dois morreram sem pega, três tiveram rejeição aos dias +67, +524 e +638 (dois morreram e um foi resgatado com IS). Mucosite grau III/IV ocorreu em 39% e não observamos DECH aguda ou crônica. A probabilidade de sobrevida pelo método de Kaplan-Meier foi de 75% aos 2,14 anos, e uma tendência a melhor sobrevida foi encontrada entre os portadores de apenas um fator de risco ao transpl