The DOI matches its Crossref record; 105 of the 105 references it cites were found in a registry or an index.
Non-transfusion-dependent thalassemias
Open accessAbstract
Non-transfusion-dependent thalassemias include a variety of phenotypes that, unlike patients with beta (β)-thalassemia major, do not require regular transfusion therapy for survival. The most commonly investigated forms are β-thalassemia intermedia, hemoglobin E/β-thalassemia, and α-thalassemia intermedia (hemoglobin H disease). However, transfusion-independence in such patients is not without side effects. Ineffective erythropoiesis and peripheral hemolysis, the hallmarks of disease process, lead to a variety of subsequent pathophysiologies including iron overload and hypercoagulability that ultimately lead to a number of serious clinical morbidities. Thus, prompt and accurate diagnosis of non-transfusion-dependent thalassemia is essential to ensure early intervention. Although several management options are currently available, the need to develop more novel therapeutics is justified by recent advances in our understanding of the mechanisms of disease. Such efforts require wide international collaboration, especially since non-transfusion-dependent thalassemias are no longer bound to low- and middle-income countries but have spread to large multiethnic cities in Europe and the Americas due to continued migration.
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Cites
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The inherited diseases of hemoglobin are an emerging global health burden
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A Hemodynamic Study of Pulmonary Hypertension in Sickle Cell Disease
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The hypercoagulable state in thalassemia
Eldor, A., & Rachmilewitz, E. A. (2002) · Blood · doi:10.1182/blood.v99.1.36 · Cited by 503
Self-citationRachmilewitz, E. A., & Giardina, P. J. (2011) · Blood · doi:10.1182/blood-2010-08-300335 · Cited by 417
Self-citationTaher, A. T., Musallam, K. M., Karimi, M., El-Beshlawy, A., Belhoul, K., Daar, S., Saned, M.-S., El-Chafic, A.-H., Fasulo, M. R., & Cappellini, M. D. (2010) · Blood · doi:10.1182/blood-2009-09-243154 · Cited by 392
Self-citationLiver iron concentrations and urinary hepcidin in -thalassemia
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Taher, A. T., Weatherall, D. J., & Cappellini, M. D. (2018) · The Lancet · doi:10.1016/s0140-6736(17)31822-6 · Cited by 807
Taher, A. T., Musallam, K. M., & Cappellini, M. D. (2021) · New England Journal of Medicine · doi:10.1056/nejmra2021838 · Cited by 477
Self-citationA phase 3 trial of luspatercept in patients with transfusion-dependent β-thalassemia.
Cappellini, M. D., Viprakasit, V., Taher, A. T., Georgiev, P., Kuo, K. H. M., Coates, T., Voskaridou, E., Liew, H.-K., Pazgal-Kobrowski, I., Forni, G. L., et al. (2020) · New England Journal of Medicine · doi:10.1056/nejmoa1910182 · Cited by 332
Minihepcidin peptides as disease modifiers in mice affected by β-thalassemia and polycythemia vera
Casu, C., Oikonomidou, P. R., Chen, H., Nandi, V., Ginzburg, Y., Prasad, P., Fleming, R. E., Shah, Y. M., Valore, E. V., Nemeth, E., et al. (2016) · Blood · doi:10.1182/blood-2015-10-676742 · Cited by 148
Molecular Basis and Genetic Modifiers of Thalassemia
Mettananda, S., & Higgs, D. R. (2018) · Hematology/Oncology Clinics of North America · doi:10.1016/j.hoc.2017.11.003 · Cited by 130
Metadata
- Authors
- Musallam, K. M., Rivella, S., Vichinsky, E., & Rachmilewitz, E. A.
- Venue
- Haematologica
- Published
- 2013-05-31
- Volume
- 98
- Issue
- 6
- Pages
- 833-844
- ISSN
- 0390-6078 (ISSN-L)
- 1592-8721
- OpenAlex
- W2101474649
- Cited by
- 326
- References
- 105
- FWCI
- 9.91
- Type
- journal article
- Language
- en
- Open access
- gold
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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