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Published in Benign Hematology

Expert Opinion / Commentary · November 19, 2018

2018 Top Stories in Benign Hematology: Severe Thalassemias

Written by
Michael H. Kroll MD


Additional Info

  1. Thompson AA, Walters MC, Kwiatkowski J, et al. Gene therapy in patients with transfusion-dependent β-thalassemia. N Engl J Med. 2018;378(16):1479-1493.
  2. Borgna-Pignatti C, Rugolotto S, De Stefano P, et al. Survival and complications in patients with thalassemia major treated with transfusion and deferoxamine. Haematologica. 2004;89(10):1187-1193.
  3. Modell B, Darlison M. Global epidemiology of haemoglobin disorders and derived service indicators. Bull World Health Organ. 2008;86(6):417-496
  4. Paulson RF. Targeting a new regulator of erythropoiesis to alleviate anemia. Nat Med. 2014;20(4):334-335.
  5. Komrokji R, Garcia-Manero G, Ades L, et al. Sotatercept with long-term extension for the treatment of anaemia in patients with lower-risk myelodysplastic syndromes: a phase 2, dose-ranging trial. Lancet Haematol. 2018;5(2):e63-e72.
  6. Cappellini MD, Porter J, Origa R, et al. Sotatercept, a novel transforming growth factor beta ligand trap, improves anemia in beta-thalassemia: a phase 2, open-label, dose-finding study. Haematologica. 2018 Oct 18. doi: 10.3324/haematol.2018.198887. [Epub ahead of print.]
  7. Taher AT, Cappellini MD. How I manage medical complications of β-thalassemia in adults. Blood. 2018;132(17):1781-1791.
  8. Casu C, Nemeth E, Rivella S. Hepcidin agonists as therapeutic tools. Blood. 2018;131(16):1790-1794.
  9. Machogu EM, Machado RF. How I treat hypoxia in adults with hemoglobinopathies and hemolytic disorders. Blood. 2018;132(17):1770-1780.
  10. Ballas SK, Zeidan AM, Duong VH, et al. The effect of iron chelation therapy on overall survival in sickle cell disease and β-thalassemia: a systematic review. Am J Hematol. 2018;93(7):943-952.

Disclosure statements are available on the authors' profiles:

Further Reading