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Sideroblastic Anemias Medication

  • Author: Muhammad A Mir, MD, FACP; Chief Editor: Emmanuel C Besa, MD  more...
 
Updated: Nov 18, 2015
 

Medication Summary

In cases of sideroblastic anemia, the goals of pharmacotherapy are to reduce morbidity and prevent complications. Medications used include vitamins and antidotes to toxic metal ions.

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Antidotes

Class Summary

Antidotes are used to decrease toxic blood levels of metal ions such as iron.

Deferoxamine mesylate (Desferal)

 

Deferoxamine (desferrioxamine) is usually administered as a slow subcutaneous infusion through a portable pump. It is freely soluble in water. Approximately 8 mg of iron is bound by 100 mg of deferoxamine. Deferoxamine promotes renal and hepatic excretion in urine and bile in feces. It gives urine a red discoloration.

Deferoxamine readily chelates iron from ferritin and hemosiderin but not transferrin. It does not affect iron in cytochromes or hemoglobin. It is most effective when provided to the circulation continuously by infusion. It helps prevent damage to the liver and bone marrow from iron deposition.

Deferoxamine may be administered either by intramuscular (IM) injection or by slow intravenous (IV) infusion. It does not effectively chelate other trace metals of nutritional importance. It is provided in vials containing 500 mg of lyophilized sterile drug. Two mL of sterile water for injection should be added to each vial, bringing the concentration to 250 mg/mL. For IV use, this may be diluted in 0.9% sterile saline, 5% dextrose solution, or Ringer solution.

The IM route is the preferred route of administration, except in the presence of hypotension and cardiovascular collapse, when the IV route should be considered.

Deferasirox (Exjade)

 

Deferasirox is supplied as a tab for oral suspension. It is an oral iron chelation agent demonstrated to reduce liver iron concentration in adults and children who receive repeated red blood cell (RBC) transfusions. It binds iron with high affinity in a 2:1 ratio.

Deferasirox is approved for treatment of chronic iron overload due to multiple blood transfusions. Treatment initiation is recommended with evidence of chronic iron overload (ie, transfusion of about 100 mL/kg packed RBCs [about 20 U for a 40-kg person] and a serum ferritin level consistently higher than 1000 µg/L).

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Vitamins

Class Summary

Vitamins are used to meet necessary dietary requirements and are used in metabolic pathways, as well as DNA and protein synthesis.

Folic acid

 

Folic acid is a water-soluble vitamin used in nucleic acid synthesis. It is required for normal erythropoiesis.

Pyridoxine (Aminoxin)

 

Pyridoxine is necessary for normal metabolism of proteins, carbohydrates, and fats. It is also involved in the synthesis of gamma-aminobutyric acid (GABA) within the central nervous system.

Thiamine

 

Thiamine (vitamin B-1) works by an incompletely understood mechanism to correct sideroblastic anemia in DIDMOAD (diabetes insipidus, diabetes mellitus, optic atrophy, deafness) syndrome.

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Contributor Information and Disclosures
Author

Muhammad A Mir, MD, FACP Assistant Professor of Medicine (Hematology, Blood/Marrow Transplant) Milton S Hershey Medical Center, Pennsylvania State University College of Medicine

Muhammad A Mir, MD, FACP is a member of the following medical societies: American College of Physicians, American Society of Hematology, American Society for Blood and Marrow Transplantation, American Society of Clinical Oncology

Disclosure: Nothing to disclose.

Coauthor(s)

Gerald L Logue, MD Professor of Medicine, Head of the Division of Hematology, Vice Chairman for Education, Department of Medicine, University of Buffalo State University of New York School of Medicine and Biomedical Sciences

Gerald L Logue, MD is a member of the following medical societies: Alpha Omega Alpha, American Association for the Advancement of Science, American College of Physicians, American Society of Hematology, American Federation for Clinical Research

Disclosure: Nothing to disclose.

Specialty Editor Board

Francisco Talavera, PharmD, PhD Adjunct Assistant Professor, University of Nebraska Medical Center College of Pharmacy; Editor-in-Chief, Medscape Drug Reference

Disclosure: Received salary from Medscape for employment. for: Medscape.

Chief Editor

Emmanuel C Besa, MD Professor Emeritus, Department of Medicine, Division of Hematologic Malignancies and Hematopoietic Stem Cell Transplantation, Kimmel Cancer Center, Jefferson Medical College of Thomas Jefferson University

Emmanuel C Besa, MD is a member of the following medical societies: American Association for Cancer Education, American Society of Clinical Oncology, American College of Clinical Pharmacology, American Federation for Medical Research, American Society of Hematology, New York Academy of Sciences

Disclosure: Nothing to disclose.

Additional Contributors

Clarence Sarkodee Adoo, MD, FACP Consulting Staff, Department of Bone Marrow Transplantation, City of Hope Samaritan BMT Program

Clarence Sarkodee Adoo, MD, FACP is a member of the following medical societies: American College of Physicians-American Society of Internal Medicine, American Society of Hematology, American Society of Clinical Oncology

Disclosure: Nothing to disclose.

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Ring sideroblast.
Heme synthesis.
Sideroblastic anemias: etiologic classification. DIDMOAD = diabetes insipidus, diabetes mellitus, optic atrophy, deafness.
Iron and total iron-binding capacity in physiology and pathology.
 
 
 
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