The Thalassemias

Goldman's Cecil Medicine: Twenty Fourth Edition

Volumn 1, Issue , 2012, Pages 1060-1066

  Cappellini, Maria Domenica ^a  

^a FONDAZIONE IRCCS CA GRANDA OSPEDALE MAGGIORE POLICLINICO   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 84896729941     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1016/B978-1-4377-1604-7.00165-2     Document Type: Chapter

References (13)

1. Pennell DJ, Berdoukas V, Karagiorga M, et al. Randomized controlled trial of deferiprone or deferoxamine in beta-thalassemia major patients with asymptomatic myocardial siderosis. Blood 2006, 107:3738-3744.
2. Tanner MA, Galanello R, Dessi C, et al. A randomized, placebo-controlled, double-blind trial of the effect of combined therapy with deferoxamine and deferiprone on myocardial iron in thalassemia major using cardiovascular magnetic resonance. Circulation 2007, 115:1876-1884.
3. Maggio A, Vitrano A, Capra M, et al. Long-term sequential deferiprone-deferoxamine versus deferiprone alone for thalassemia major patients: a randomized clinical trial. Br J Haematol 2009, 145:245-254.
4. Cao A, Galanello R Beta thalassemia. Genet Med 2010, 12:61-74. Review.
5. Pennell DJ, Porter JB, Cappellini MD, et al. Continued improvement in myocardial T2* over two years of deferasirox therapy in β-thalassemia major patients with cardiac iron overload. Haematologica 2011, 96:48-54. Continuous treatment with deferasirox (40 mg/kg/d) continued to remove iron from the heart.
6. Pennell DJ, Porter JB, Cappellini MD, et al. Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia. Blood 2010, 115:2364-2371. Deferasirox is effective in removing and preventing myocardial iron accumulation.
7. Serjeant GR, Serjeant BE, Fraser RA, et al. Hb S-β-thalassemia: molecular, hematological and clinical comparisons. Hemoglobin 2011, 35:1-12. Clinically, Hb S-β(0)-thal and Hb S-β(+)-thal type I were generally severe.
8. Smiers FJ, Krishnamurti L, Lucarelli G Hematopoietic stem cell transplantation for hemoglobinopathies: current practice and emerging trends. Pediatr Clin North Am 2010, 57:181-205. Improved results are possible with new conditioning regimens and expanded donor pools.
9. Taher AT, Musallam KM, Karimi M, et al. Overview on practices in thalassemia intermedia management aiming for lowering complication-rates across a region of endemicity: the OPTIMAL CARE study. Blood 2010, 115:1886-1892. Study of 584 thalassemia intermedia patients, searching for associations among disease characteristics, treatment received, and rate of complications.
10. Testa U Fetal hemoglobin chemical inducers for treatment of hemoglobinopathies. Review. Ann Hematol 2009, 88:505-528. The increase in HbF in response to these drugs varies among patients with thalassemia and sickle cell disease owing to individual genetic determinants.
11. Weatherall DJ The inherited diseases of hemoglobin are an emerging global health burden. Blood 2010, 115:4331-4336. Strong argument for recognition of inherited hemoglobinopathies as a major, growing global health burden.
12. Higgs DR, Weatherall DJ The alpha thalassemias. Cell Mol Life Sci 2009, 66:1154-1162. Examines the mechanisms involved in the very high frequency of the different forms of α-thalassemia and their phenotypic heterogeneity.
13. Shander A, Cappellini MD, Goodnough LT Iron overload and toxicity: the hidden risk of multiple blood transfusions. Vox Sang 2009, 97:185-197. Patients receiving chronic transfusion therapy should be monitored for iron overload and, once identified, should be treated to reduce the risk of morbidity and mortality.