Hematopoietic stem cell transplantation for inherited bone marrow failure syndromes: alternative donor and disease-specific conditioning regimen with unmanipulated grafts

References

• Macmillan ML, Wagner JE. Haematopoeitic cell transplantation for Fanconi anaemia – when and how? Br J Haematol. 2010;149(1):14–21.
   PubMed Web of Science ®Google Scholar
• Alter BP. Inherited bone marrow failure syndromes: considerations pre- and posttransplant. Hematology Am Soc Hematol Educ Program. 2017;1:88–95.
   Google Scholar
• Sieff CA. Introduction to acquired and inherited bone marrow failure. Hematol Oncol Clin North Am. 2018;32(4):569–558.
   PubMed Web of Science ®Google Scholar
• Mehta PA, Davies SM, Leemhuis T, et al. Radiation-free, alternative-donor HCT for Fanconi anemia patients: results from a prospective multi-institutional study. Blood. 2017;129(16):2308–2315.
   PubMed Web of Science ®Google Scholar
• Dalle J-H, de Latour RP. Allogeneic hematopoietic stem cell transplantation for inherited bone marrow failure syndromes. Int J Hematol. 2016;103(4):373–379.
   PubMed Web of Science ®Google Scholar
• Wegman-Ostrosky T, Savage SA. The genomics of inherited bone marrow failure: from mechanism to the clinic. Br J Haematol. 2017;177(4):526–542.
   PubMed Web of Science ®Google Scholar
• Alter BP, Giri N, Savage SA, et al. Cancer in the National Cancer Institute inherited bone marrow failure syndrome cohort after fifteen years of follow-up. Haematologica. 2018;103(1):30–39.
   PubMed Web of Science ®Google Scholar
• Li Q, Luo C, Luo C, et al. Disease-specific hematopoietic stem cell transplantation in children with inherited bone marrow failure syndromes. Ann Hematol. 2017;96(8):1389–1397.
   PubMed Web of Science ®Google Scholar
• Peffault de Latour R, Peters C, Gibson B, et al. Recommendations on hematopoietic stem cell transplantation for inherited bone marrow failure syndromes. Bone Marrow Transplant. 2015;50(9):1168–1172.
   PubMed Web of Science ®Google Scholar
• Harris AC, Young R, Devine S, et al. International, multicenter standardization of acute graft-versus-host disease clinical data collection: a report from the Mount Sinai acute GVHD international consortium. Biol Blood Marrow Transplant. 2016;22(1):4–10.
   PubMed Web of Science ®Google Scholar
• Jagasia MH, Greinix HT, Arora M, et al. National institutes of health consensus development project on criteria for clinical trials in chronic graft-versus-host disease. I. The 2014 diagnosis and staging working group report. Biol Blood Marrow Transplant. 2015;21(3):389–401.e1.
   PubMed Web of Science ®Google Scholar
• Clark JR, Scott SD, Jack AL, et al. Monitoring of chimerism following allogeneic haematopoietic stem cell transplantation (HSCT): technical recommendations for the use of short tandem repeat (STR) based techniques, on behalf of the United Kingdom National External Quality assessment service. Br J Haematol. 2015;168(1):26–37.
   PubMed Web of Science ®Google Scholar
• Zecca M, Strocchio L, Pagliara D, et al. HLA-Haploidentical T cell-depleted allogeneic hematopoietic stem cell transplantation in children with Fanconi anemia. Biol Blood Marrow Transplant. 2014;20:571–576.
   PubMed Web of Science ®Google Scholar
• Kojima S, Ehlert K. Reconsidering the indication of haematopoietic stem cell transplantation for dyskeratosis congenita. Br J Haematol. 2018;183(1):11–12.
   PubMed Web of Science ®Google Scholar
• Fioredda F, Iacobelli S, Biezen A, et al. Stem cell transplantation in severe congenital neutropenia: an analysis from the European society for blood and marrow transplantation. Blood. 2015;126(16):1885–1892.
   PubMed Web of Science ®Google Scholar
• Ebens CL, MacMillan ML, Wagner JE. Hematopoietic cell transplantation in Fanconi anemia: current evidence, challenges and recommendations. Expert Rev Hematol. 2017;10(1):81–97.
   PubMed Web of Science ®Google Scholar
• Simon E, Zuccolo S, Dufort G, et al. Feasibility and outcome of haploidentical SCT in pediatric high-risk hematologic malignancies and Fanconi anemia in Uruguay. Bone Marrow Transplant. 2012;47(5):663–668.
   PubMed Web of Science ®Google Scholar
• Smetsers SE, Smiers FJ, Bresters D, et al. Four decades of stem cell transplantation for Fanconi anaemia in the Netherlands. Br J Haematol. 2016;174(6):952–961.
   PubMed Web of Science ®Google Scholar
• Maury S, Prete A, Locasciulli A, et al. Fludarabine, cyclophosphamide and anti-thymocyte globulin for alternative donor transplants in acquired severe aplastic anemia: a report from the EBMT-SAA Working Party. Bone Marrow Transplant. 2005;36(11):947–950.
   PubMed Web of Science ®Google Scholar
• Macmillan ML, Defor TE, Young JH, et al. Alternative donor hematopoietic cell transplantation for Fanconi anemia. Blood. 2015;125(24):3798–3804.
   PubMed Web of Science ®Google Scholar
• Ebens CL, Defor TE, Tryon R, et al. Comparable outcomes after HLA-matched sibling and alternative donor hematopoietic cell transplantation for children with Fanconi anemia and severe aplastic anemia. Biol Blood Marrow Transplant. 2018;24:765–771.
   PubMed Web of Science ®Google Scholar
• Dulley FL, Vigorito AC, Aranha FJP, et al. Addition of low-dose busulfan to cyclophosphamide in aplastic anemia patients prior to allogeneic bone marrow transplantation to reduce rejection. Bone Marrow Transplant. 2004;33(1):9–13.
   PubMed Web of Science ®Google Scholar
• Auerbach AD, Rogatko A, Schroeder-Kurth TM. International Fanconi anemia registry: Relation of clinical symptoms to diepoxybutane sensitivity. Blood. 1989;73(2):391–396.
   PubMed Web of Science ®Google Scholar
• De Latour P, Porcher R, Dalle J, et al. Allogeneic hematopoietic stem cell transplantation in Fanconi anemia: the European group for blood and marrow Transplantation experience. Blood. 2013;122(26):4279–4286.
   PubMed Web of Science ®Google Scholar
• Guardiola P, Pasquini R, Dokal I, et al. Outcome of 69 allogeneic stem cell transplantations for Fanconi anemia using HLA-matched unrelated donors: a study on behalf of the European group for blood and marrow transplantation. Blood. 2010;95(2):422–429.
   Google Scholar
• Peffault de Latour R, Soulier J. How I treat MDS and AML in Fanconi anemia. Blood. 2016;127(24):2971–2979.
   PubMed Web of Science ®Google Scholar
• Dufour C. How I manage patients with Fanconi anaemia. Br J Haematol. 2017;178(1):32–47.
   PubMed Web of Science ®Google Scholar
• West AH, Churpek JE. Old and new tools in the clinical diagnosis of inherited bone marrow failure syndromes. Hematology Am Soc Hematol Educ Program. 2017;1:79–87.
   Google Scholar
• Ebens CL, MacMillan ML, Wagner JE. Hematopoietic cell transplantation in Fanconi anemia: current evidence, challenges and recommendations. Expert Rev Hematol. 2017;10(1):81–97.
   PubMed Web of Science ®Google Scholar
• Wynn RF, Cross MA, Hatton C, et al. Accelerated telomere shortening in young recipients of allogeneic bone-marrow transplants. Lancet. 1998;351(9097):178–181.
   PubMed Web of Science ®Google Scholar
• Amarasinghe K, Dalley C, Dokal I, et al. Late death after unrelated-BMT for dyskeratosis congenita following conditioning with alemtuzumab, fludarabine and melphalan. Bone Marrow Transplant. 2007;40(9):913–914.
   PubMed Web of Science ®Google Scholar
• Fioredda F, Korthof ET, Van Biezen A, et al. Outcome of haematopoietic stem cell transplantation in dyskeratosis congenita. Br J Haematol. 2018;183(1):110–118.
   PubMed Web of Science ®Google Scholar
• Barbaro P, Vedi A. Survival after hematopoietic stem cell transplant in patients with dyskeratosis congenita: systematic review of the literature. Biol Blood Marrow Transplant. 2016;22(7):1152–1158.
   PubMed Web of Science ®Google Scholar
• Dietz AC, Orchard PJ, Baker KS, et al. Disease-specific hematopoietic cell transplantation: nonmyeloablative conditioning regimen for dyskeratosis congenita. Bone Marrow Transplant. 2011;46(1):98–104.
   PubMed Web of Science ®Google Scholar
• Otrock ZK, Bacigalupo A, Mahfouz RA, et al. A reduced intensity conditioning regimen of fludarabine, cyclophosphamide, antithymocyte globulin, plus 2 Gy TBI facilitates successful hematopoietic cell engraftment in an adult with dyskeratosis congenita. Bone Marrow Transplant. 2012;47(9):1254–1255.
   PubMed Web of Science ®Google Scholar
• Oshima K, Hanada R, Kobayashi R, et al. Hematopoietic stem cell transplantation in patients with severe congenital neutropenia: an analysis of 18 Japanese cases. Pediatr Transplant. 2010;14(5):657–663.
   PubMed Web of Science ®Google Scholar
• Ferry C, Ouachée M, Leblanc T, et al. Hematopoietic stem cell transplantation in severe congenital neutropenia: experience of the French SCN register. Bone Marrow Transplant. 2005;35(1):45–50.
   PubMed Web of Science ®Google Scholar
• Fioredda F, Iacobelli S, van Biezen A, et al. Stem cell transplantation in severe congenital neutropenia: an analysis from the European society for blood and marrow transplantation. Blood. 2015;126(16):1885–1892.
   PubMed Web of Science ®Google Scholar