Kutler, DI, Singh, B, Satagopan, J, et al. Auerbach AD A 20-year perspective on the International Fanconi Anemia Registry (IFAR). Blood. 2003;101(4):1249–56.CrossRefGoogle ScholarPubMed

Berger, R, Bernheim, A, Gluckman, E, Gisselbrecht, C. In vitro effect of cyclophosphamide metabolites on chromosomes of Fanconi. anaemia patients. Br J Haematol. 1980;45, 565–8.CrossRefGoogle ScholarPubMed

Gluckman, E, Berger, R, Dutreix, J. Bone marrow transplantation for Fanconi anemia. Sem Hematol. 1984;21, 20–6.Google ScholarPubMed

Socié, G, Devergie, A, Girinski, T, et al. Transplantation for Fanconi’s anaemia: long-term follow-up of fifty patients transplanted from a sibling donor after low-dose cyclophosphamide and thoraco-abdominal irradiation for conditioning. Br J Haematol. 1998;103(1):249–55.CrossRefGoogle ScholarPubMed

Peffault de Latour, R, Porcher, R, Dalle, JH, et al; FA Committee of the Severe Aplastic Anemia Working Party; Pediatric Working Party of the European Group for Blood and Marrow Transplantation. Allogeneic hematopoietic stem cell transplantation in Fanconi anemia: the European Group for Blood and Marrow Transplantation experience. Blood. 2013;122(26):4279–86. doi:10.1182/blood-2013-01-479733. Epub 2013 Oct 21.CrossRefGoogle Scholar

Ayas, M, Saber, W, Davies, SM, et al. Allogeneic hematopoietic cell transplantation for fanconi anemia in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome, or acute leukemia. J Clin Oncol. 2013;31(13):1669–76. doi: 10.1200/JCO.2012.45.9719. Epub 2013 Apr 1.CrossRefGoogle ScholarPubMed

Kumar, AR, Wagner, JE, Auerbach, AD, et al. Fatal hemorrhage from androgen-related hepatic adenoma after hematopoietic cell transplantation. J Pediatr Hematol/Oncol. 2004;26:16–18.CrossRefGoogle ScholarPubMed

Pasquini, R, Carreras, J, Pasquini, MC, et al. HLA-matched sibling hematopoietic stem cell transplantation for fanconi anemia: comparison of irradiation and non irradiation containing conditioning regimens. Biol Blood Marrow Transplant. 2008;14(10):1141–7.CrossRefGoogle ScholarPubMed

Svahn, J, Onofrillo, D, Cappelli, E, et al. Long-term hematologic follow-up in Fanconi anemia and impact on survival in 97 patients in a national data base. The 26th Fanconi Anemia Research Fund Scientific Symposium, 18–21 Sept, 2014. Abstract, pp. 117–118.Google Scholar

MacMillan, ML, Wagner, JE. Haematopoietic cell transplantation for Fanconi anaemia – when and how? Br J Haematol. 2010;149(1):14–21. Review.CrossRefGoogle Scholar

Dufour, C, Rondelli, R, Locatelli, F, et al. Stem cell transplantation from HLA-matched related donor for Fanconi’s anaemia: a retrospective review of the multicentric Italian experience on behalf of AIEOP-GITMO. Br J Haematol. 2001;112:796–805.CrossRefGoogle Scholar

Bonfim, CM, de Medeiros, CR, Bitencourt, MA, et al. HLA-matched related donor hematopoietic cell transplantation in 43 patients with Fanconi anemia conditioned with 60 mg/kg of cyclophosphamide. Biol Blood Marrow Transplant. 2007;13, 1455–60.CrossRefGoogle ScholarPubMed

Locatelli, F, Zecca, M, Pession, A, et al.; Italian Pediatric Group. The outcome of children with Fanconi anemia given hematopoietic stem cell transplantation and the influence of fludarabine in the conditioning regimen: a report from the Italian pediatric group. Haematologica. 2007;92(10):1381–8.CrossRefGoogle ScholarPubMed

Tan, PL, Wagner, JE, Auerbach, AD, Defor, TE, Slungaard, A, MacMillan, ML. Successful engraftment without radiation after fludarabine-based regimen in Fanconi anemia patients undergoing genotypically identical donor hematopoietic cell transplantation. Pediatr Blood Cancer. 2006;46:630–6.CrossRefGoogle ScholarPubMed

Ertem, M, Ileri, T, Azik, F, Uysal, Z, Gozdasoglu, S. Related donor hematopoietic stem cell transplantation for Fanconi anemia without radiation: a single center experience in Turkey. Pediatr Transplant. 2009;13(1):88–95. doi: 10.1111/j.1399–3046.2008.00952.x. Epub 2008 Apr 22.CrossRefGoogle ScholarPubMed

Torjemane, L, Ladeb, S, Ben Othman, T, Abdelkefi, A, Lakhal, A, Ben Abdeladhim, A. Bone marrow transplantation from matched related donors for patients with Fanconi anemia using low dose busulfan and cyclophosphamide as conditioning. Pediatr Blood Cancer. 2006;46:496–500.CrossRefGoogle ScholarPubMed

Wagner, JE, Eapen, M, MacMillan, ML, et al. Unrelated donor bone marrow transplantation for the treatment of Fanconi anemia. Blood. 2007;109:2256–62.CrossRefGoogle ScholarPubMed

MacMillan, ML, Blazar, Br, DeFor Te, Ma L, Tolar, J, Zierhut, H, Wagner, E. Alternate donor HCT for Fanconi Anemia (FA): results of a total body irradiation (TBI) dose de-escalation study. Biol Blood Marrow Transplant. 2009;15:2–4.CrossRefGoogle Scholar

MacMillan, ML, Hughes, MR, Agarwal, S, Daley, GQ. Cellular therapy for Fanconi anemia: the past, present, and future. Biol Blood Marrow Transplant. 2011;17(1 Suppl):S109–14. doi: 10.1016/j.bbmt.2010.11.027. Review.CrossRefGoogle ScholarPubMed

Chaudhury, S, Auerbach, AD, Kernan, NA, et al. Fludarabine-based cytoreductive regimen and T-cell-depleted grafts from alternative donors for the treatment of high-risk patients with Fanconi anaemia. Br J Haematol. 2008;140(6):644–55. doi: 10.1111/j.1365–2141.2007.06975. Review.CrossRefGoogle ScholarPubMed

Gluckman, E, Rocha, V, Boyer-Chammard, A, et al; Eurocord Transplant Group and the European Blood and Marrow Transplantation Group. Outcome of cord-blood transplantation from related and unrelated donors. N Engl J Med. 1997;337(6):373–381.CrossRefGoogle ScholarPubMed

Gluckman, E, Rocha, V, Ionescu, I, Alternate donor HCT for Fanconi Anemia (FA): results of a total body irradiation (TBI) dose de-escalation study; Eurocord-Netcord and EBMT. Results of unrelated cord blood transplant in fanconi anemia patients: risk factor analysis for engraftment and survival. Biol Blood Marrow Transplant. 2007;13(9):1073–82. Epub 2007 Jul 20.CrossRefGoogle Scholar

Motwani, J, Lawson, SE, Darbyshire, PJ. Successful HSCT using nonradiotherapy-based conditioning regimens and alternative donors in patients with Fanconi anaemia – experience in a single UK centre. Bone Marrow Transplant. 2005;36:405–10. doi:10.1038/sj.bmt.1705071.CrossRefGoogle Scholar

Yabe, H, Inoue, H, Matsumoto, M, et al. Unmanipulated HLA-haploidentical bone marrow transplantation for the treatment of fatal, nonmalignant diseases in children and adolescents. Int J Hematology. 2004, 80(1):78–82.CrossRefGoogle ScholarPubMed

Rihani, R, Lataifeh, I, Halalsheh, H, et al. Haploidentical stem cell transplantation as a salvage therapy for cord blood engraftment failure in a patient with Fanconi anemia. Pediatr Blood Cancer. 2010;55:580–2. doi: 10.1002/pbc.22584.CrossRefGoogle Scholar

Locatelli, F, Zecca, M, Pession, A, et al. The outcome of children with Fanconi anemia given hematopoietic stem cell transplantation and the influence of fludarabine in the conditioning regimen: a report from the Italian Pediatric Group. Haematologica. 2007;92:1381–8. doi:10.3324/haematol.11436.CrossRefGoogle ScholarPubMed

Dufort, G, Pisano, S, Incoronato, A, et al. Feasibility and outcome of haploidentical SCT in pediatric high-risk hematologic malignancies and Fanconi anemia in Uruguay. Bone Marrow Transplant. 2012;47:663–8. doi:10.1038/bmt.2011.148; published online 18 July 2011.CrossRefGoogle ScholarPubMed

Thakar, MS, Bonfim, C, Sandmaier, BM, et al. Cyclophosphamide-based in vivo T-cell depletion for HLA-haploidentical transplantation in Fanconi anemia. Pediatr Hematol Oncol. 2012;29(6):568–78. doi: 10.3109/08880018.2012.708708. Epub 2012 Jul 27.CrossRefGoogle ScholarPubMed

Butturini, A, Gale, RP, Verlander, PC, et al. Hematologic abnormalities in Fanconi anemia: An International Fanconi Anemia Registry study. Blood. 1994;84:1650–5.CrossRefGoogle ScholarPubMed

Tonnies, H, Huber, S, Kuhl, JS, et al. Clonal chromosomal aberrations in bone marrow cells of Fanconi anemia patients: Gains of the chromosomal segment 3q26q29 as an adverse risk factor. Blood. 2003;101:3872–4.CrossRefGoogle ScholarPubMed

Mehta, PA, Harris, RE, Davies, SM, et al. Numerical chromosomal changes and risk of developmentof myelodysplastic syndrome: acute myeloid leukemia in patients with Fanconi anemia. Cancer Genet Cytogenet. 2010;203:180–6.CrossRefGoogle Scholar

Cioc, AM, Wagner, JE, MacMillan, ML, DeFor, T, Hirsch, B. Diagnosis of myelodysplastic syndrome among a cohort of 119 patients with fanconi anemia: morphologic and cytogenetic characteristics. Am J Clin Pathol. 2010;133(1):92–100.CrossRefGoogle ScholarPubMed

Mehta, PA, Ileri, T, Harris, RE, et al .Chemotherapy for myeloid malignancy in children with Fanconi anemia. Pediatr Blood Cancer, 48 (2007), pp. 668–672.CrossRefGoogle ScholarPubMed

Talbot, A, Peffault de Latour, R, Buchbinder, N, et al. Sequential treatment with chemotherapy and reduced-intensity conditioning for allogeneic hematopoietic stem cell transplantation in Fanconi anemia patients with acute myeloid leukemia or myelodysplastic syndrome. Blood: ASH Annual Meeting Abstracts. 2012;120: 2363.CrossRefGoogle Scholar

Mitchell, R, Wagner, JE, Hirsch, B, DeFor, TE, Zierhut, H, MacMillan, ML. Haematopoietic cell transplantation for acute leukaemia and advanced myelodysplastic syndrome in Fanconi anaemia. Br J Haematol. 2014;164(3):384–95. doi: 10.1111/bjh.12634. Epub 2013 Oct 30.CrossRefGoogle ScholarPubMed

Rosenberg, PS, Socié, G, Alter, BP, Gluckman, E. Risk of head and neck squamous cell cancer and death in patients with Fanconi anemia who did and did not receive transplants. Blood. 2005;105(1):67–73. Epub 2004 Aug 26.CrossRefGoogle Scholar

Curtis, RE, Metayer, C, Rizzo, JD, et al. Impact of chronic GVHD therapy on the development of squamous-cell cancers after hematopoietic stem-cell transplantation: an international case-control study. Blood. 2005;105(10):3802–11. Epub 2005 Feb 1.CrossRefGoogle Scholar