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    Excellent overall and chronic graft-versus-host-disease-free event-free survival in Fanconi anaemia patients undergoing matched related- and unrelated-donor bone marrow transplantation using alemtuzumab-Flu-Cy: the UK experience

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    Authors
    Bernard, F.
    Uppungunduri, C. R. S.
    Meyer, Stefan
    Cummins, M.
    Patrick, K.
    James, B.
    Skinner, R.
    Tewari, S.
    Carpenter, B.
    Wynn, R.
    Veys, P.
    Amrolia, P.
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    Affiliation
    Paediatric Onco-Haematology Unit, Geneva University Hospital, Geneva, Switzerland
    Issue Date
    2021
    
    Metadata
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    Abstract
    Haematopoietic stem cell transplantation (HSCT) remains the only curative option in Fanconi anaemia (FA). We analysed the outcome of children transplanted for FA between 1999 and 2018 in the UK. A total of 94 transplants were performed in 82 patients. Among the donors, 51·2% were matched related donors (MRD) while the remainder were alternative donors. Most patients received a fludarabine-cyclophosphamide (Flu-Cy)-based conditioning regimen (86·6%) and in vivo T-cell depletion with alemtuzumab (69·5%). Five-year overall survival (OS) was 85·4% [70·4-93.2] with MRD, 95·7% [72·9-99.4] with matched unrelated donors (MUD), 44·4% [6·6-78.5] with mismatched unrelated donors (MMUD) and 44·4% [13·6-71.9] with mismatched related donors (MMRD) (P < 0·001). Other factors significantly impacting OS were pre-transplant bone marrow status, source of stem cells, cytomegalovirus (CMV) serostatus, preparation with Flu-Cy, use of total body irradiation (TBI) and alemtuzumab as serotherapy. In multivariate analysis, absence of myelodysplastic syndrome (MDS) or leukaemia, bone marrow as source of stem cells, cytomegalovirus (CMV) other than +/- (Recipient/Donor) and Flu-Cy were protective factors for five-year OS. Five-year chronic graft-versus-host-disease (cGVHD)-free event-free survival was 75·4% with the same risk factors except for CMV serostatus. Five-year non-relapse mortality was 13·8% [7·3-22.3]. Only five patients (6·1%) developed grade II-IV acute GVHD and two patients chronic GVHD. These data confirm the excellent outcome of matched related or unrelated HSCT in children with FA.
    Citation
    Bernard F, Uppungunduri CRS, Meyer S, Cummins M, Patrick K, James B, et al. Excellent overall and chronic graft‐ versus ‐host‐disease‐free event‐free survival in Fanconi anaemia patients undergoing matched related‐ and unrelated‐donor bone marrow transplantation using alemtuzumab–Flu–Cy: the UK experience. Br J Haematol. 2021 Apr 14;193(4):804–13.
    Journal
    British Journal of Haematology
    URI
    http://hdl.handle.net/10541/624014
    DOI
    10.1111/bjh.17418
    PubMed ID
    33855694
    Additional Links
    https://dx.doi.org/10.1111/bjh.17418
    Type
    Article
    Language
    en
    ae974a485f413a2113503eed53cd6c53
    10.1111/bjh.17418
    Scopus Count
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