Thalassemia, Beta: Multimedia

References

1. Lucarelli G, Galimberti M, Polchi P. Marrow transplantation in patients with thalassemia responsive to iron chelation therapy. N Engl J Med. Sep 16 1993;329(12):840-4. [Medline]. [Full Text].

2. Olivieri NF, Brittenham GM, McLaren CE, et al. Long-term safety and effectiveness of iron-chelation therapy with deferiprone for thalassemia major. N Engl J Med. Aug 13 1998;339(7):417-23. [Medline]. [Full Text].

3. Cohen AR, Galanello R, Pennell DJ, et al. Thalassemia. In: Hematology (Am Soc Hematol Educ Program). 2004:14-34.

4. Forget BG. Thalassemia Syndromes. In: Hematology: Basic Principles and Practice. 2000:485-509.

5. Fucharoen S, Winichagoon P. Clinical and hematologic aspects of hemoglobin E beta-thalassemia. Current Opinion in Hematology. 2000;7:106-112. [Medline].

6. Hoffbrand AV, AL-Refaie F, Davis B. Long-term trial of deferiprone in 51 transfusion-dependent iron overloaded patients. Blood. 1998;91:295-300. [Medline].

7. Ikuta T, Atweh G, Boosalis V. Cellular and molecular effects of a pulse butyrate regimen and new inducers of globin gene expression and hematopoiesis. Ann N Y Acad Sci. 1998;850:87-99. [Medline].

8. Koshy M, Dorn L, Bressler L. 2-deoxy 5-azacytidine and fetal hemoglobin induction in sickle cell anemia. Blood. 2000;96:2379-2384. [Medline].

9. Malik P, Arumugam PI. Gene Therapy for {beta}-Thalassemia. In: Hematology (Am Soc Hematol Educ Program). 2005:45-50.

10. May C, Rivella S, Callegari J. Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin. Nature. 2000;406:82-86. [Medline].

11. Perrine SP. Fetal Globin Induction--Can It Cure {beta} Thalassemia?. In: Hematology (Am Soc Hematol Educ Program). 2005:38-44.

12. Rund D, Rachmilewitz E. Beta-thalassemia. N Engl J Med. Sep 15 2005;353(11):1135-46. [Medline].

13. Schrier SL, Angelucci E. New strategies in the treatment of the thalassemias. Annu Rev Med. 2005;56:157-71. [Medline].

14. Thein SL. Pathophysiology of {beta} Thalassemia--A Guide to Molecular Therapies. In: Hematology (Am Soc Hematol Educ Program). 2005:31-7.

15. Weatherall DJ, Clegg JB. Genetic disorders of hemoglobin. Semin Hematol. Oct 1999;36(4 Suppl 7):24-37. [Medline].

16. Italia KY, Jijina FJ, Merchant R, et al. Response to hydroxyurea in beta thalassemia major and intermedia: experience in western India. Clin Chim Acta. Sep 2009;407(1-2):10-5. [Medline].

17. Tan JA, Tan KL, Omar KZ, et al. Interaction of Hb South Florida (codon 1; GTG-->ATG) and HbE, with beta-thalassemia (IVS1-1; G-->A): expression of different clinical phenotypes. Eur J Pediatr. Sep 2009;168(9):1049-54. [Medline].

18. Galanello R, Sanna S, Perseu L, et al. Amelioration of Sardinian beta-zero thalassemia by genetic modifiers. Blood. Aug 20 2009;epub ahead of print. [Medline].

Further Reading

Related eMedicine Topics

• Cooley Anemia
• Hematologic Disease and Pregnancy [in the Obstetrics and Gynecology section]
• Hemoglobin H Disease [in the Pediatrics: General Medicine section]
• Iron Deficiency Anemia
• Thalassemia [in the Pediatrics: General Medicine section]
• Thalassemia [in the Radiology section]
• Thalassemia, Alpha 
• Thalassemia Intermedia [in the Pediatrics: General Medicine section]

Clinical Trials

• Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major
• Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-Thalassaemia Patients) With Chronic Iron Overload
• Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia Major
• Genetic Factors Affecting the Severity of Beta Thalassemia
• Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia
• Stem Cell Transplant in Sickle Cell Disease and Thalassemia

National Guideline Clearinghouse

• Carrier screening for thalassemia and hemoglobinopathies in Canada. Society of Obstetricians and Gynaecologists of Canada - Medical Specialty Society. 2008 Oct. 10 pages. NGC:006773
• Hemoglobinopathies in pregnancy. American College of Obstetricians and Gynecologists - Medical Specialty Society. 2007 Jan. 9 pages. NGC:005700
• Screening for iron deficiency anemia - including iron supplementation for children and pregnant women. United States Preventive Services Task Force - Independent Expert Panel. 1996 (revised 2006). 12 pages. NGC:004965
• Surgical treatment of disease and injuries of the spleen. Society for Surgery of the Alimentary Tract, Inc - Medical Specialty Society. 2004 Feb 21. 3 pages. NGC:003836 

Keywords

beta-thalassemia, beta thalassemia syndromes, Cooley anemia, Mediterranean anemia, thalassemia major, thalassemia intermedia, thalassemia minor, erythroblastic anemia, thalassemia trait, hemoglobin E, hereditary disorder

Contributor Information and Disclosures

Author

Kenichi Takeshita, MD, Adjunct Associate Professor, Department of Medicine, Division of Hematology, New York University School of Medicine; Medical Director, Clinical Research and Development, Celgene
Kenichi Takeshita, MD is a member of the following medical societies: American Society of Hematology
Disclosure: Nothing to disclose.

Medical Editor

Wadie F Bahou, MD, Chief, Division of Hematology, Hematology/Oncology Fellowship Director, Professor, Department of Internal Medicine, State University of New York at Stony Brook
Wadie F Bahou, MD is a member of the following medical societies: American Society of Hematology
Disclosure: Nothing to disclose.

Pharmacy Editor

Francisco Talavera, PharmD, PhD, Senior Pharmacy Editor, eMedicine
Disclosure: eMedicine Salary Employment

Managing Editor

Marcel E Conrad, MD, (Retired) Distinguished Professor of Medicine, University of South Alabama
Marcel E Conrad, MD is a member of the following medical societies: Alpha Omega Alpha, American Association for the Advancement of Science, American Association of Blood Banks, American Chemical Society, American College of Physicians, American Physiological Society, American Society for Clinical Investigation, American Society of Hematology, Association of American Physicians, Association of Military Surgeons of the US, International Society of Hematology, Society for Experimental Biology and Medicine, and Southwest Oncology Group
Disclosure: No financial interests None None

CME Editor

Rajalaxmi McKenna, MD, FACP, Consulting Staff, Department of Medicine, Southwest Medical Consultants, SC, Good Samaritan Hospital, Advocate Health Systems
Rajalaxmi McKenna, MD, FACP is a member of the following medical societies: American Society of Clinical Oncology, American Society of Hematology, and International Society on Thrombosis and Haemostasis
Disclosure: Nothing to disclose.

Chief Editor

Emmanuel C Besa, MD, Professor, Department of Medicine, Division of Hematologic Malignancies, Kimmel Cancer Center, Thomas Jefferson University
Emmanuel C Besa, MD is a member of the following medical societies: American Association for Cancer Education, American College of Clinical Pharmacology, American Federation for Medical Research, American Society of Hematology, and New York Academy of Sciences
Disclosure: Nothing to disclose.