The role of ineffective erythropoiesis in non-transfusion-dependent thalassemia

Blood Reviews

Volumn 26, Issue SUPPL.1, 2012, Pages

  Rivella, Stefano ^a  

^a WEILL CORNELL MEDICAL COLLEGE   (United States)

Author keywords

Beta thalassemia; Erythropoiesis; Iron absorption; Jak inhibitor; Jak2; Janus kinase 2; Thalassemia

Indexed keywords

ALPHA GLOBIN; BETA GLOBIN; ERYTHROPOIETIN; HEPCIDIN; JANUS KINASE 2; JANUS KINASE 2 INHIBITOR; RUXOLITINIB; TRANSFERRIN;

ARTICLE; BETA THALASSEMIA; CELL DIFFERENTIATION; CELL PROLIFERATION; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; DRUG TREATMENT FAILURE; ERYTHROCYTE; ERYTHROID PRECURSOR CELL; ERYTHROPOIESIS; HUMAN; IRON ABSORPTION; IRON METABOLISM; MEMBRANE DAMAGE; NON TRANSFUSIONAL DEPENDENT THALASSEMIA; NONHUMAN; OXIDATIVE STRESS; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN FUNCTION; PROTEIN PHOSPHORYLATION; PROTEIN SYNTHESIS; SPLENOMEGALY; THALASSEMIA; UPREGULATION;

ANIMALS; BETA-THALASSEMIA; BLOOD TRANSFUSION; ERYTHROPOIESIS; HUMANS; IRON;

EID: 84861357425     PISSN: 0268960X     EISSN: 15321681     Source Type: Journal    
DOI: 10.1016/S0268-960X(12)70005-X     Document Type: Article

References (19)

1. Olivieri NF The beta-thalassemias. N Engl J Med 1999, 341(2):99-109.
2. Ginzburg Y, Rivella S (beta)-thalassemia: a model for elucidating the dynamic regulation of ineffective erythropoiesis and iron metabolism. Blood 2011, 118(16):4321-4330.
3. Rivella S Ineffective erythropoiesis and thalassemias. Curr Opin Hematol 2009, 16(3):187-194.
4. Gardenghi S, Grady RW, Rivella S Anemia, ineffective erythropoiesis, and hepcidin: interacting factors in abnormal iron metabolism leading to iron overload in beta-thalassemia. Hematol Oncol Clin North Am 2010, 24(6):1089-1107.
5. Libani IV, Guy EC, Melchiori L, et al. Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in beta-thalassemia. Blood 2008, 112(3):875-885.
6. Melchiori L, Gardenghi S, Rivella S beta-Thalassemia: HiJAKing Ineffective Erythropoiesis and Iron Overload. Adv Hematol 2010, 2010:938640.
7. Wood AD, Chen E, Donaldson IJ, et al. ID1 promotes expansion and survival of primary erythroid cells and is a target of JAK2V617F-STAT5 signaling. Blood 2009, 114(9):1820-1830.
8. Tanno T, Miller JL Iron Loading and Overloading due to Ineffective Erythropoiesis. Adv Hematol 2010, 2010:358283.
9. Tanno T, Bhanu NV, Oneal PA, et al. High levels of GDF15 in thalassemia suppress expression of the iron regulatory protein hepcidin. Nat Med 2007, 13(9):1096-1101.
10. Rivella S, Rachmilewitz E Future alternative therapies for beta-thalassemia. Expert Rev Hematol 2009, 2(6):685.
11. De Domenico I, Lo E, Ward DM, Kaplan J Hepcidin-induced internalization of ferroportin requires binding and cooperative interaction with Jak2. Proc Natl Acad Sci 2009, 106(10):3800-3805.
12. Kerenyi MA, Grebien F, Gehart H, et al. Stat5 regulates cellular iron uptake of erythroid cells via IRP-2 and TfR-1. Blood 2008, 112(9):3878-3888.
13. Zhu BM, McLaughlin SK, Na R, et al. Hematopoietic-specific Stat5-null mice display microcytic hypochromic anemia associated with reduced transferrin receptor gene expression. Blood 2008, 112(5):2071-2080.
14. Tanno T, Noel P, Miller JL Growth differentiation factor 15 in erythroid health and disease. Curr Opin Hematol 2010, 17(3):184-190.
15. Musallam KM, Taher AT, Duca L, Cesaretti C, Halawi R, Cappellini MD Levels of growth differentiation factor-15 are high and correlate with clinical severity in transfusion-independent patients with beta thalassemia intermedia. Blood Cells Mol Dis 2011, 47(4):232-234.
16. Verstovsek S, Kantarjian H, Mesa RA, et al. Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N Engl J Med 2010, 363(12):1117-1127.
17. Gardenghi S, Ramos P, Marongiu MF, et al. Hepcidin as a therapeutic tool to limit iron overload and improve anemia in beta-thalassemic mice. J Clin Invest 2010, 120(12):4466-4477.
18. Parrow NL, Gardenghi S, Rivella S Prospects for a hepcidin mimic to treat beta-thalassemia and hemochromatosis. Expert Rev Hematol 2011, 4(3):233-235.
19. Li H, Rybicki AC, Suzuka SM, et al. Transferrin therapy ameliorates disease in beta-thalassemic mice. Nat Med 2010, 16(2):177-182.