Sideroblastic Anemias Treatment & Management

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

Treatment of sideroblastic anemia may include the following:

  • Removal of toxic agents
  • Administration of pyridoxine, thiamine, or folic acid
  • Transfusion (along with antidotes if iron overload develops from transfusion)
  • Other medical measures
  • Bone marrow or liver transplantation

Admission and inpatient care may be needed for patients with sideroblastic anemia and cirrhosis, as well as those who have need of repeated blood transfusions. Regular follow-up is essential, even for patients with sideroblastic anemia whose condition responds to therapy.

Acute idiopathic sideroblastic anemia, refractory anemia with ring sideroblasts (RARS) or myelodysplastic syndrome (MDS) must be monitored for potential transformation into acute leukemias.

Go to Anemia, Iron Deficiency Anemia, and Chronic Anemia for complete information on these topics.


Removal of Toxic Agents

Toxic agents such as zinc, lead, and drugs such as penicillamine should be removed whenever possible; however, this may not always be easy. For example, isoniazid is the mainstay of treatment of active tuberculosis, and a risk-benefit analysis of treatment discontinuation is essential for each patient.


Vitamin B Therapy


Pyridoxine (vitamin B-6) deserves a trial in all cases of sideroblastic anemia as many acquired and certain congenital forms of sideroblastic anemia respond to this relatively safe agent. The response will be evident in a few weeks, with reticulocytosis and improving hemoglobin levels.

The dose should be tailored to the patient’s tolerance. Dosages up to 1 g/day have been used, but the goal is to find a dosage of pyridoxine (usually 50-200 mg/d) that will maintain the hemoglobin level and yet prevent toxicity (peripheral neuropathy). For patients whose condition responds, treatment is lifelong.

Pyridoxal 5′ phosphate (PLP) is an active form of pyridoxine that has been successfully used in the treatment of sideroblastic anemias in some patients who do not respond to pyridoxine. [73]

Folic acid

Folic acid by itself has been reported to reverse sideroblastic changes in some patients. [74] It is advisable to supplement folate in pyridoxine-responsive cases to ensure an adequate supply during a period of increased hemoglobin synthesis.


As would be expected from its name, thiamine-responsive megaloblastic anemia is treated with thiamine. In this acquired form of sideroblastic anemia, supraphysiologic doses of thiamine (25 to 75 mg daily) have been shown to improve not only the anemia component, but also the associated diabetes and deafness. [49] However, some studies indicate that these doses may be ineffective during puberty. [75]



Transfusion is the mainstay of treatment for patients whose sideroblastic anemia does not respond to pyridoxine therapy. [76] It is problematic and should be avoided if the anemia is mild to moderate and the patient asymptomatic.

Even without transfusions, patients with sideroblastic anemia are prone to develop iron overload. [77] Transfusion in sideroblastic anemia has been known to worsen iron overload and lead to secondary hemochromatosis and cirrhosis, which can be fatal. [78]


Other Agents

Other agents studied for the treatment of sideroblastic anemias include the following:

  • Heme arginate as an infusion has been used with mixed results. It is not a first-line drug. [79]
  • Erythropoietin (EPO) has been tried and does not appear to reverse sideroblastic anemia [80] ; it has also been reported to cause neutropenia in this setting. [81] EPO in combination with granulocyte colony-stimulating factor (G-CSF) appears to have a better response rate than EPO alone (50%). [82, 83]
  • Lenalidomide may reduce transfusion needs in some patients with refractory anemia with ring sideroblasts (RARS). [84]
  • Prednisone and danazol have not been effective, [85] except for some patients with active connective tissue disease (eg, systemic lupus erythematosus (SLE), [86] ) in whom sideroblastic changes disappear with prednisone when the SLE flare subsides.
  • Cytotoxic therapies such as cyclophosphamide have been tried with some success in patients with erythropoietin inhibitors that result in ineffective erythropoiesis. [87]
  • Chloroquine has been successfully used to treat pyridoxine-resistant sideroblastic anemia, but no large study has been done, and thus only limited data are available. [88] The drug affects heme metabolism and is also used in certain porphyrias.
  • Ubidecarenone (coenzyme Q10) has been used with mixed results in the treatment of sideroblastic anemia and is not recommended as a standard of care. [89, 90]
  • Azacitidine and other newer agents being used for myelodysplastic syndrome (MDS) have not been specifically studied or approved for sideroblastic anemias in general.


Bone marrow transplantation is a treatment of last resort and is best saved for young patients whose conditions are pyridoxine resistant [91] and transfusion dependent [92] and who have a human leukocyte antigen (HLA)-matched sibling. Even then, the response ranges from cure to death within weeks. [62] Severe graft versus host disease has been reported even with HLA-identical sibling donors. [63] The evidence is limited to a few case reports in the literature. [93, 94, 95]

Liver transplantation has been used in cases of sideroblastic anemia with cirrhosis and iron overload that is not responsive to chelation and phlebotomy.


Surgical Care

While surgical management is often not needed in patients with sideroblastic anemias, untreated patients can build up an excess amount of iron in their organs, in particular the liver. As noted above, this hepatic iron overload can act very similarly to congenital hemochromatosis, and eventually lead tocan build up in such excess and cause profound liver failure. At that point the only clinical management would be to go for a Liver Transplantation. 

Patients who being considered for liver transplantation will need to undergo a workup to assess their degree of illness and overall suitability for the procedure, to help determine their eligibility for listing (see Liver Transplantation). In the rare cases where iron accumulates to toxic levels in the heart, heart transplantation  may eventually be needed if all other treatments fail. 




See Anemia for more information on the general managment of anemia-related complications. For iron overload–related complications, the primary goal is to eliminate excess iron 

Management of iron overload

Deferoxamine (desferrioxamine; Desferal) can be used if iron overload develops from repeated blood transfusions. [96]  Although effective, it must be administered by a subcutaneous pump for several hours a day.

Deferasirox (Exjade) is a newer oral iron chelator that has been used instead of deferoxamine and appears to be effective. First used in Europe, it has been introduced in United States and is a once-daily pill. Renal toxicity and allergic reactions are a concern.

Phlebotomy can be performed for iron overload. [97]  In some patients who do not tolerate deferoxamine therapy, this procedure is an option, but the limiting factor may be anemia.

Refractory iron overload

In some cases, chelation or phlebotomy may be unable to prevent the accumulation of iron to toxic levels, most often in the liver and more rarely the heart. Treatment options for liver toxicity are slim; liver transplantation may eventually prove necessary. For iron accumulation in the heart, management focuses on treating heart failure secondary to restrictive cardiomyopathy. However, continuing iron buildup may eventually result in such profound heart failure that heart transplantation becomes the only option.



Transfer to the care of a hematologist is recommended, especially for cases of pyridoxine-resistant sideroblastic anemia and for patients who become transfusion dependent. Consider consultations with a neurologist for mitochondrial myopathy-anemia syndromes.

If surgical treatment is ever needed in the form of a liver or heart transplant, it is key to get hepatologists, cardiologists, and transplant surgeons all invovled in the care of the patient.