Fanconi anaemia: New strategies

Bone Marrow Transplantation

Volumn 41, Issue SUPPL. 2, 2008, Pages

  Dufour, C ^a   Svahn, J ^a  

^a G GASLINI INSTITUTE   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

ANDROGEN; CORTICOSTEROID; CYCLOPHOSPHAMIDE; CYTOKINE; ERYTHROPOIETIN; ETANERCEPT; FLUDARABINE; GRANULOCYTE COLONY STIMULATING FACTOR; GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR; GROWTH FACTOR; INTERLEUKIN 3; LENTIVIRUS VECTOR; OXYMETHOLONE; PREDNISOLONE; RETROVIRUS VECTOR;

ACUTE GRANULOCYTIC LEUKEMIA; ALGORITHM; BONE MARROW APLASIA; BONE MARROW DEPRESSION; CANCER SUSCEPTIBILITY; CHROMOSOMAL INSTABILITY; CLINICAL DECISION MAKING; COHORT ANALYSIS; COMBINATION CHEMOTHERAPY; CORD BLOOD STEM CELL TRANSPLANTATION; DISEASE COURSE; DISEASE SEVERITY; DONOR SELECTION; DRUG DOSE INCREASE; DRUG DOSE REDUCTION; DRUG MECHANISM; DRUG MEGADOSE; ENGRAFTMENT; FANCONI ANEMIA; GENETIC DISORDER; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; LIVER ADENOMA; LIVER CELL CARCINOMA; LOW DRUG DOSE; MONOTHERAPY; MORBIDITY; MORTALITY; MYELOABLATIVE CONDITIONING; MYELODYSPLASTIC SYNDROME; NEUTROPENIA; NONHUMAN; PELIOSIS HEPATIS; PRENATAL DIAGNOSIS; PRIORITY JOURNAL; RECOMMENDED DRUG DOSE; REVIEW; SHORT STATURE; SIBLING; VIRAL GENE THERAPY; VIRILIZATION; WHOLE BODY RADIATION;

ADRENAL CORTEX HORMONES; ANDROGENS; CHILD; CHILD, PRESCHOOL; FANCONI ANEMIA; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; TRANSPLANTATION CONDITIONING;

EID: 45749085809     PISSN: 02683369     EISSN: 14765365     Source Type: Journal    
DOI: 10.1038/bmt.2008.63     Document Type: Review

References (23)

1. Faivre L, Guardiola P, Lewis C, Dokal I, Ebell W, Zatterale A et al Association of complementation group and mutation type with clinical outcome in Fanconi anemia. European Fanconi Anemia Research Group. Blood 2000; 96: 4064-4070.
2. Levitus M, Rooimans MA, Steltenpool J, Cool NF, Oostra AB, Mathew CG et al. Heterogeneity in Fanconi anemia: Evidence for 2 new genetic subtypes. Blood 2004; 103: 2498-2503.
3. Bagby GC, Alter BP. Fanconi anemia. Semin Hematol 2006; 43: 147-156.
4. Dufour C, Corcione A, Svahn J, Haupt R, Poggi V, Beka'ssy AN et al TNF-alplia and IFN-gamma are overexpressed in the bone marrow of Fanconi anemia patients and TNF-alpha suppresses erythropoiesis in vitro. Blood 2003; 102: 2053-2059.
5. Sejas DP, Rani R, Qiu Y, Zhang X, Fagerlie SR, Nakano H et al. Inflammatory reactive oxygen species-mediated hemopoietic suppression in Fance-deficient mice. J Immunol 2007; 178: 5277-5287.
6. Zhang X, Sejas DP, Qiu Y, Williams DA, Pang Q. Inflammatory ROS promote and cooperate with the Fanconi anemia mutation for hematopoietic senescence. J Cell Sci 2007; 120: 1572-1583.
7. Alter BP. Inherited bone marrow failure syndromes. In: Nathan DG, Orkin SH, Ginsburg D, Look AT (eds). Haematology of Infancy and Childhood. 6th edn. WB Saunders Company: Philadelphia, PA, 2003, pp 280-365.
8. Guardiola P, Pasquini R, Dokal I, Ortega JJ, van Weel-Sipman M, Marsh JC 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 2000; 95: 422-429.
9. de Medeiros CR, Bitencourt MA, Zanis-Neto J, Maluf EC, Carvalho DS, Bonfim CS et al. Allogeneic hematopoietic stem cell transplantation from an alternative stem cell source in Fanconi anemia patients: Analysis of 47 patients from a single institution. Braz J Med Biol Res 2006; 39: 1297-1304.
10. Gluckman E. Auerbach AD. Horowitz MM. Sobocinski KA. Ash RC, Bortin MM et al. Bone marrow transplantation for Fanconi anemia. Blood 1995; 86: 2856-2862.
11. Zanis-Neto J, Flowers ME, Medeiros CR, Bitencourt MA, Bonfim CM. Setubal DC et al. Low-dose cyclophosphamide conditioning for haematopoietic cell transplantation from HLA-matched related donors in patients with Fanconi anaemia. Br J Haematol 2005; 130: 99-106.
12. Dufour C. Rondelli R. Locatelli F. Miano M. Di Girolamo G, Bacigalupo A 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.
13. Farzin A, Davies SM, Smith FO. Filipovich A. Hansen M. Auerbach AD et al. Matched sibling donor haematopoietic stem cell transplantation in Fanconi anaemia: An update of the Cincinnati Children's experience. Br J Haematol 2007; 136: 633-640.
14. 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: 630-636.
15. Socie G, Devergie A, Girinski T, Piel G, Ribaud P, Esperou H et al Transplantation for Fanconi's anaemia: Long-term follow-up of tifty patients transplanted from a sibling donor after low-dose cyclophosphamide and thoraco-abdominal irradiation for conditioning. Br J Haematol 1998; 103: 249-255.
16. Rosenberg PS, Socie 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: 67-73.
17. Wagner JE, Eapen N, Mc Milian ML, Harris RE, Pasquini R, Boulad F et al. Unrelated donor bone marrow transplantation for the treatment of Fanconi anemia. Blood 2007; 109: 2256-2262.
18. Yabe H, Inoue H, Matsumoto M, Hamanoue S, Koike T, Ishiguro H et al Allogeneic haematopoietic cell transplantation from alternative donors with a conditioning regimen of low-dose irradiation, fludarabine and cyclophosphamide in Fanconi anaemia. Br J Haematol 2006; 134: 208-212.
19. Deeg HJ, Socie G. Schoch G. Henry-Amar M. Witherspoon RP. Devergie A et al. Malignancies after marrow transplantation For aplastic anemia and Fanconi anemia: A joint Seattle and Paris analysis of results in 700 patients. Blood 1996; 87: 380-392.
20. Kelly PF. Radtke S. Kalle C, Balcik B. Bohn K. Mueller R et al. Stem cell collection and gene transfer in Fanconi anemia. Mol Ther 2007; 15: 211-219.
21. Jacome A. Navarro S, Casado JA, Rio P, Madero L, Estella J et al. A simplified approach to improve the efficiency and safety of ex vivo hematopoietic gene therapy in Fanconi anemia patients. Hum Gene Ther 2006; 17: 245-250.
22. Yamada K, Ramezani A, Hawley RG, Ebell W. Arwert F, Arnold LW et al Phenotype correction of Fanconi anemia group A hematopoietic stem cells using lentiviral vector. Mol Ther 2003; 8: 600-610.
23. Grewall SS, Kahn JP, Mc Millan ML. Ramsay NK. Wagner JE. Successful hematopoietic stem cell transplantation for Fanconi anemia from an unaffected HLA-genotype-identical sibling selected using preimplantation genetic diagnosis. Blood 2004; 103: 1147-1151.