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
Uppungunduri, C. R. S.
AffiliationPaediatric Onco-Haematology Unit, Geneva University Hospital, Geneva, Switzerland
MetadataShow full item record
AbstractHaematopoietic 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.
CitationBernard 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.
JournalBritish Journal of Haematology
- Outcome of Fludarabine-Based Conditioning in High-Risk Aplastic Anemia Patients Undergoing Matched Related Donor Transplantation: A Single-Center Study from Pakistan.
- Authors: Chaudhry QUN, Iftikhar R, Satti TM, Mahmood SK, Ghafoor T, Shamshad GU, Farhan M, Shahbaz N, Khan MA, Khattak TA, Rehman J, Humayun S, Satti HS, Anwer F, Ahmed P
- Issue date: 2019 Dec
- Better Failure-Free Survival and Graft-versus-Host Disease-Free/Failure Free Survival with Fludarabine-Based Conditioning in Stem Cell Transplantation for Aplastic Anemia in Children.
- Authors: Im SH, Kim BR, Park SM, Yoon BA, Hwang TJ, Baek HJ, Kook H
- Issue date: 2020 Feb 24
- Post-transplant cyclophosphamide for graft-versus-host disease prophylaxis in HLA matched sibling or matched unrelated donor transplant for patients with acute leukemia, on behalf of ALWP-EBMT.
- Authors: Ruggeri A, Labopin M, Bacigalupo A, Afanasyev B, Cornelissen JJ, Elmaagacli A, Itälä-Remes M, Blaise D, Meijer E, Koc Y, Milpied N, Schouten HC, Kroeger N, Mohty M, Nagler A
- Issue date: 2018 Mar 15
- Use of matched unrelated donors compared with matched related donors is associated with lower relapse and superior progression-free survival after reduced-intensity conditioning hematopoietic stem cell transplantation.
- Authors: Ho VT, Kim HT, Aldridge J, Liney D, Kao G, Armand P, Koreth J, Cutler C, Ritz J, Antin JH, Soiffer RJ, Alyea EP
- Issue date: 2011 Aug
- Impact of the source of hematopoietic stem cell in unrelated transplants: comparison between 10/10, 9/10-HLA matched donors and cord blood.
- Authors: Granier C, Biard L, Masson E, Porcher R, Peffault de Latour R, Robin M, Boissel N, Xhaard A, Ribaud P, Lengline E, Larghero J, Charron D, Loiseau P, Socié G, Dhédin N
- Issue date: 2015 Oct