Produção Nacional
Revisado por pares
Outcome of patients with Fanconi anemia developing myelodysplasia and acute leukemia who received allogeneic hematopoietic stem cell transplantation: A retrospective analysis on behalf of EBMT group
2020; Wiley; Volume: 95; Issue: 7 Linguagem: Inglês
10.1002/ajh.25810
ISSN1096-8652
AutoresStefano Giardino, Régis Peffault de Latour, Mahmoud Aljurf, Dirk‐Jan Eikema, Paul Bosman, Yves Bertrand, Abdelghani Tbakhi, Wolfgang Holter, Martin Bornhäuser, Claudia Rössig, Birgit Burkhardt, Marco Zecca, Boris Afanasyev, Gérard Michel, Arnold Ganser, Amal Al-Seraihy, Mouhab Ayas, Duygu Uçkan, Bénédicte Bruno, Katharine Patrick, Peter Bader, Maija Itälä‐Remes, Vanderson Rocha, Charlotte Jubert, Miguel Ángel Díaz, Peter J. Shaw, Luiz Guilherme Darrigo, Franco Locatelli, Nicolaus Kröger, Maura Faraci, Filomena Pierri, Edoardo Lanino, Maurizio Miano, Antonio M. Risitano, Marie Robin, Carlo Dufour,
Tópico(s)Acute Myeloid Leukemia Research
ResumoAllogeneic hematopoietic stem cell transplantation (allo-HSCT) is curative for bone marrow failure in patients with Fanconi anemia (FA), but the presence of a malignant transformation is associated with a poor prognosis and the management of these patients is still challenging. We analyzed outcome of 74 FA patients with a diagnosis of myelodysplastic syndrome (n = 35), acute leukemia (n = 35) or with cytogenetic abnormalities (n = 4), who underwent allo-HSCT from 1999 to 2016 in EBMT network. Type of diagnosis, pre-HSCT cytoreductive therapies and related toxicities, disease status pre-HSCT, donor type, and conditioning regimen were considered as main variables potentially influencing outcome. The 5-year OS and EFS were 42% (30-53%) and 39% (27-51%), respectively. Patients transplanted in CR showed better OS compared with those transplanted in presence of an active malignant disease (OS:71%[48-95] vs 37% [24-50],P = .04), while none of the other variables considered had an impact. Twenty-two patients received pre-HSCT cytoreduction and 9/22 showed a grade 3-4 toxicity, without any lethal event or negative influence on survival after HSCT(OS:toxicity pre-HSCT 48% [20-75%] vs no-toxicity 51% [25-78%],P = .98). The cumulative incidence of day-100 grade II-IV a-GvHD and of 5-year c-GvHD were 38% (26-50%) and 40% (28-52%). Non-relapse-related mortality and incidence of relapse at 5-years were 40% (29-52%) and 21% (11-30%) respectively, without any significant impact of the tested variables. Causes of death were transplant-related events in most patients (34 out of the 42 deaths, 81%). This analysis confirms the poor outcome of transformed FA patients and identifies the importance of achieving CR pre-HSCT, suggesting that, in a newly diagnosed transformed FA patient, a cytoreductive approach pre-HSCT should be considered if a donor have been secured.
Referência(s)