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
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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. / Giardino, Stefano; de Latour, Regis P; Aljurf, Mahmoud; Eikema, Dirk-Jan; Bosman, Paul; Bertrand, Yves; Tbakhi, Abdelghani; Holter, Wolfgang; Bornhäuser, Martin; Rössig, Claudia; Burkhardt, Birgit; Zecca, Marco; Afanasyev, Boris; Michel, Gerard; Ganser, Arnold; Alseraihy, Amal; Ayas, Mouhab; Uckan-Cetinkaya, Duygu; Bruno, Benedicte; Patrick, Katharine; Bader, Peter; Itälä-Remes, Maija; Rocha, Vanderson; Jubert, Charlotte; Diaz, Miguel A; Shaw, Peter J; Junior, Luiz G D; Locatelli, Franco; Kröger, Nicolaus; Faraci, Maura; Pierri, Filomena; Lanino, Edoardo; Miano, Maurizio; Risitano, Antonio; Robin, Marie; Dufour, Carlo; Severe Aplastic Anemia and Chronic Malignancies Working Parties of the European Group for Blood and Marrow Transplantation.
In: AM J HEMATOL, Vol. 95, No. 7, 07.2020, p. 809-816.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - 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
AU - Giardino, Stefano
AU - de Latour, Regis P
AU - Aljurf, Mahmoud
AU - Eikema, Dirk-Jan
AU - Bosman, Paul
AU - Bertrand, Yves
AU - Tbakhi, Abdelghani
AU - Holter, Wolfgang
AU - Bornhäuser, Martin
AU - Rössig, Claudia
AU - Burkhardt, Birgit
AU - Zecca, Marco
AU - Afanasyev, Boris
AU - Michel, Gerard
AU - Ganser, Arnold
AU - Alseraihy, Amal
AU - Ayas, Mouhab
AU - Uckan-Cetinkaya, Duygu
AU - Bruno, Benedicte
AU - Patrick, Katharine
AU - Bader, Peter
AU - Itälä-Remes, Maija
AU - Rocha, Vanderson
AU - Jubert, Charlotte
AU - Diaz, Miguel A
AU - Shaw, Peter J
AU - Junior, Luiz G D
AU - Locatelli, Franco
AU - Kröger, Nicolaus
AU - Faraci, Maura
AU - Pierri, Filomena
AU - Lanino, Edoardo
AU - Miano, Maurizio
AU - Risitano, Antonio
AU - Robin, Marie
AU - Dufour, Carlo
AU - Severe Aplastic Anemia and Chronic Malignancies Working Parties of the European Group for Blood and Marrow Transplantation
N1 - © 2020 Wiley Periodicals, Inc.
PY - 2020/7
Y1 - 2020/7
N2 - Allogeneic 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.
AB - Allogeneic 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.
KW - Acute Disease
KW - Allografts
KW - Disease-Free Survival
KW - Fanconi Anemia/complications
KW - Female
KW - Follow-Up Studies
KW - Hematopoietic Stem Cell Transplantation
KW - Humans
KW - Leukemia/etiology
KW - Male
KW - Myelodysplastic Syndromes/etiology
KW - Retrospective Studies
KW - Survival Rate
U2 - 10.1002/ajh.25810
DO - 10.1002/ajh.25810
M3 - SCORING: Journal article
C2 - 32267023
VL - 95
SP - 809
EP - 816
JO - AM J HEMATOL
JF - AM J HEMATOL
SN - 0361-8609
IS - 7
ER -