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Peter L. Greenberg, Cynthia K. Rigsby, Richard M. Stone, H. Joachim Deeg, Steven D. Gore, Michael M. Millenson, Stephen D. Nimer, Margaret R. O'Donnell, Paul J. Shami and Rashmi Kumar

; 2 : 27 – 30 . 4 Hershko CM Link GM Konijn AM Cabantchik ZI . Iron chelation therapy . Curr Hematol Rep 2005 ; 4 : 110 – 116 . 5 Olivieri NF Brittenham GM . Iron-chelating therapy and the treatment of thalassemia . Blood

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Peter L. Greenberg

. Blood 1996 ; 88 : 4275 – 4287 . 16. Olivieri NF Brittenham GM . Iron-chelating therapy and the treatment of thalassemia . Blood 1997 ; 89 : 739 – 761 . 17. Brittenham GM Griffith PM Nienhuis AW . Efficacy of deferoxamine in

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Steven D. Gore

-globin synthesis in a patient with beta+ thalassemia . N Engl J Med 1982 ; 307 : 1469 – 1475 . 14. Silverman LR Holland JF Weinberg RS . Effects of treatment with 5-azacytidine on the in vivo and in vitro hematopoiesis in patients with

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Peter L. Greenberg, Leon E. Cosler, Salvatore A. Ferro and Gary H. Lyman

myelodysplastic syndromes and other transfusion-dependent anaemias to deferasirox (ICL670): a 1-yr prospective study . Eur J Haematol 2008 ; 80 : 168 – 176 . 17. Olivieri NF Brittenham GM . Iron-chelating therapy and the treatment of thalassemia

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Presenter : Aaron T. Gerds

express written permission of NCCN. To view the most recent version, visit NCCN.org . “We're always trying to move things forward, and on the horizon is luspatercept, which is already approved for the treatment of anemia in patients with thalassemia, as

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David P. Steensma

, exacerbated by RBC transfusions, has proven to be a leading cause of death in several congenital forms of anemia, such as severe β-thalassemia. 4 The risk of iron overload in chronic acquired anemic states, such as MDS, could be clinically relevant, because

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Prithviraj Bose and Srdan Verstovsek

activin receptor IIA ligand traps have been shown to correct ineffective erythropoiesis and improve anemia in mouse models of α-thalassemia through inactivation of GDF11, a cytokine that blocks terminal erythroid maturation. 62 These drugs have shown

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Mark D. Anderson and Mark R. Gilbert

mutation. This latter group has a worse prognosis than the 1p19q-codeleted subpopulation, but an improved prognosis compared with the IDH wild-type group. 25 Additionally, inactivating alterations in alpha thalassemia/mental retardation syndrome X

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Peter L. Greenberg, Eyal Attar, John M. Bennett, Clara D. Bloomfield, Carlos M. De Castro, H. Joachim Deeg, James M. Foran, Karin Gaensler, Guillermo Garcia-Manero, Steven D. Gore, David Head, Rami Komrokji, Lori J. Maness, Michael Millenson, Stephen D. Nimer, Margaret R. O'Donnell, Mark A. Schroeder, Paul J. Shami, Richard M. Stone, James E. Thompson and Peter Westervelt

overload and its consequences. 45 Thus, effective treatment of transfusional siderosis in patients with MDS is germane. Studies in patients requiring relatively large numbers of RBC transfusions (e.g., those with thalassemia and MDS) have shown the

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Jeffrey Crawford, Pamela Sue Becker, James O. Armitage, Douglas W. Blayney, Julio Chavez, Peter Curtin, Shira Dinner, Thomas Fynan, Ivana Gojo, Elizabeth A. Griffiths, Shannon Hough, Dwight D. Kloth, David J. Kuter, Gary H. Lyman, Mary Mably, Sudipto Mukherjee, Shiven Patel, Lia E. Perez, Adam Poust, Raajit Rampal, Vivek Roy, Hope S. Rugo, Ayman A. Saad, Lee S. Schwartzberg, Sepideh Shayani, Mahsa Talbott, Saroj Vadhan-Raj, Sumithira Vasu, Martha Wadleigh, Peter Westervelt, Jennifer L. Burns and Lenora Pluchino

. 98. Grigg AP . Granulocyte colony-stimulating factor-induced sickle cell crisis and multiorgan dysfunction in a patient with compound heterozygous sickle cell/beta+ thalassemia . Blood 2001 ; 97 : 3998 – 3999 . 99. Kang EM Areman EM