Sideroblastic Anemias Medication

Updated: Feb 01, 2021
  • Author: Nandakumar Mohan, DO; Chief Editor: Emmanuel C Besa, MD  more...
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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.



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).



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.


Specifically useful in the syndromic form of SA called Thiamine Responsive Megaloblastic Anemia, where the SLC19A2 gene which aids in the transport of Thiamine is dysfunctional. Supplementing Thiamine at high doses anywhere from 25mg - 75mg/day will help replenish the thiamine that these patients will be missing.