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Haematopoietic cell transplantation for acute leukaemia and advanced myelodysplastic syndrome in F anconi anaemia
Author(s) -
Mitchell Richard,
Wagner John E.,
Hirsch Betsy,
DeFor Todd E.,
Zierhut Heather,
MacMillan Margaret L.
Publication year - 2014
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.12634
Subject(s) - medicine , transplantation , myelodysplastic syndromes , chemotherapy , acute leukemia , haematopoiesis , oncology , population , immunology , leukemia , gastroenterology , stem cell , bone marrow , environmental health , biology , genetics
Summary Acute leukaemia or advanced myelodysplastic syndrome ( MDS ≥ 5% blasts) in F anconi a naemia ( FA ) patients is associated with a poor prognosis. We report 21 FA patients with acute leukaemia or advanced MDS who underwent haematopoietic cell transplantation ( HCT ) at the University of Minnesota between 1988 and 2011. Six patients had biallelic BRCA 2 mutations. Eight patients received pre‐transplant cytoreduction, with 3 achieving complete remission. HCT donor source included human leucocyte antigen‐matched sibling ( n = 2) or alternative donors ( n = 19). Neutrophil engraftment was 95% for the entire cohort, and the incidence of acute graft‐ versus ‐host disease was 19%. 5‐year overall survival ( OS ) was 33%, with a relapse rate of 24%, with similar OS in patients with biallelic BRCA 2 mutations. Our study supports the use of HCT in the treatment of FA patients with acute leukaemia or advanced MDS , however, the role of chemotherapy prior to HCT remains unclear for this population. FA patients with biallelic BRCA 2 are unique and may benefit from higher dose chemotherapy relative to other complementation groups.