Pediatric Megaloblastic Anemia Treatment & Management

Updated: Dec 16, 2021
  • Author: James L Harper, MD; Chief Editor: Max J Coppes, MD, PhD, MBA  more...
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Approach Considerations

Treatment of megaloblastic anemia depends on the underlying cause. Folate deficiency due to dietary deficiency or increased demands is best treated with folate supplements. In addition, a diet rich in green, leafy vegetables is essential for normal intake of folic acid.

Folate deficiency caused by the use of sulfa drugs or other antifolate medications may be addressed by folate supplementation or by reducing or eliminating the drug. Folate deficiency due to celiac sprue requires treatment of the underlying disorder and folate supplements.

Management of vitamin B-12 deficiency is often more complex, because of the nature of B-12 deficiency in childhood. Data from small trials suggest that oral B-12 supplementation is as effective as parenteral supplementation in patients with nutritional deficiency. [5]  A report by Sezer et al supported the idea that either parenteral or oral therapy with vitamin B-12 can effectively treat B-12 deficiency in children, with the investigators finding that after 1 month of treatment in the parenteral and oral arms of the study, 14 out of 41 (34.1%) and 8 out of 41 (19.5%) pediatric patients, respectively, still had anemia. [17]

Indeed, since many children and adults have a dietary B-12 deficiency, oral B-12 treatment should be attempted first unless clinically contraindicated. Such contraindication could be due to previous treatment failure or another clinical factor that would argue for initial parenteral therapy.

Even in patients with intrinsic factor (IF) deficiency, oral supplements may be effective, using higher doses, although often this is not the case. In many instances, high-dose oral B-12 supplements are unsuccessful in patients with IF deficiency or in those who have undergone intestinal surgery. These patients may require parenteral supplementation because of impaired secretion or absorption of IF.

Because vitamin B-12 is contained exclusively in animal products (meat), vitamin supplementation is the only means of appropriate vitamin B-12 intake in individuals choosing vegetarian diets.

For children with congenital disorders that lead to B-12 deficiency, supplementation is a lifelong necessity; therefore, establishing a regimen that is tolerable over time is essential to maintaining compliance.

A study by Canadian researchers reported that in patients with transcobalamin deficiency, treatment with intramuscular injections of hydroxycobalamin or cyanocobalamin is favored in current practice. [18]

Go to Pediatric Chronic Anemia, Anemia of Prematurity, Donath-Landsteiner Hemolytic Anemia, Pediatric Acute Anemia, and Fanconi Anemia for complete information on these topics.



Consultation with a pediatric gastroenterologist to evaluate for inflammation of the ileum or jejunum and assist in treatment planning is often helpful for patients with newly diagnosed Crohn disease or celiac sprue. A gastroenterologist may also be needed to evaluate the extent of liver disease, which may manifest with macrocytic erythrocytes.

Consider consulting a hematologist to evaluate the bone marrow for evidence of other marrow diseases that can manifest with macrocytic anemia and thrombocytopenia.