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Pediatric Megaloblastic Anemia Medication

  • Author: James L Harper, MD; Chief Editor: Max J Coppes, MD, PhD, MBA  more...
Updated: Oct 14, 2015

Medication Summary

The 2 most common causes of megaloblastic anemia are vitamin B-12 (cobalamin) deficiency and folinic acid deficiency. Treatment may require supplemental administration of these vitamins.



Class Summary

Vitamins are organic substances required by the body in small amounts for various metabolic processes. Vitamins may be synthesized in small or insufficient amounts in the body or not synthesized at all, thus requiring supplementation. Use folic acid and vitamin B-12 supplements as indicated.

Folic acid (Folacin-800)


A member of the vitamin B group, folic acid is reduced in the body to 5 methyl tetrahydrofolate (THF), which is a coenzyme for various metabolic processes including purine and pyrimidine nucleotides synthesis essential for DNA. Folic acid is an important cofactor for enzymes used in production of red blood cells.

Cyanocobalamin (CaloMist, Nascobal, Ener-B)


Deoxyadenosylcobalamin and hydroxocobalamin are active forms of vitamin B-12 in humans. Vitamin B-12 is synthesized by microbes but not humans or plants. Vitamin B-12 deficiency may result from inadequate dietary intake, intrinsic factor deficiency (pernicious anemia), partial or total gastrectomy, or diseases of the distal ileum.

Contributor Information and Disclosures

James L Harper, MD Associate Professor, Department of Pediatrics, Division of Hematology/Oncology and Bone Marrow Transplantation, Associate Chairman for Education, Department of Pediatrics, University of Nebraska Medical Center; Associate Clinical Professor, Department of Pediatrics, Creighton University School of Medicine; Director, Continuing Medical Education, Children's Memorial Hospital; Pediatric Director, Nebraska Regional Hemophilia Treatment Center

James L Harper, MD is a member of the following medical societies: American Society of Pediatric Hematology/Oncology, American Federation for Clinical Research, Council on Medical Student Education in Pediatrics, Hemophilia and Thrombosis Research Society, American Academy of Pediatrics, American Association for Cancer Research, American Society of Hematology

Disclosure: Nothing to disclose.

Specialty Editor Board

Mary L Windle, PharmD Adjunct Associate Professor, University of Nebraska Medical Center College of Pharmacy; Editor-in-Chief, Medscape Drug Reference

Disclosure: Nothing to disclose.

Steven K Bergstrom, MD Department of Pediatrics, Division of Hematology-Oncology, Kaiser Permanente Medical Center of Oakland

Steven K Bergstrom, MD is a member of the following medical societies: Alpha Omega Alpha, Children's Oncology Group, American Society of Clinical Oncology, International Society for Experimental Hematology, American Society of Hematology, American Society of Pediatric Hematology/Oncology

Disclosure: Nothing to disclose.

Chief Editor

Max J Coppes, MD, PhD, MBA Executive Vice President, Chief Medical and Academic Officer, Renown Heath

Max J Coppes, MD, PhD, MBA is a member of the following medical societies: American College of Healthcare Executives, American Society of Pediatric Hematology/Oncology, Society for Pediatric Research

Disclosure: Nothing to disclose.

Additional Contributors

Sharada A Sarnaik, MBBS Professor of Pediatrics, Wayne State University School of Medicine; Director, Sickle Cell Center, Associate Hematologist/Oncologist, Children's Hospital of Michigan

Sharada A Sarnaik, MBBS is a member of the following medical societies: American Society of Hematology, American Society of Pediatric Hematology/Oncology, New York Academy of Sciences, Society for Pediatric Research, Children's Oncology Group, American Academy of Pediatrics, Midwest Society for Pediatric Research

Disclosure: Nothing to disclose.

  1. Lorber A, Gazit AZ, Khoury A, Schwartz Y, Mandel H. Cardiac manifestations in thiamine-responsive megaloblastic anemia syndrome. Pediatr Cardiol. 2003 Sep-Oct. 24(5):476-81. [Medline].

  2. Olsen BS, Hahnemann JM, Schwartz M, Østergaard E. Thiamine-responsive megaloblastic anaemia: a cause of syndromic diabetes in childhood. Pediatr Diabetes. 2007 Aug. 8(4):239-41. [Medline].

  3. Whitehead VM. Acquired and inherited disorders of cobalamin and folate in children. Br J Haematol. 2006 Jul. 134(2):125-36. [Medline].

  4. Duma A, Cartmill C, Blood J, Sharma A, Kharasch ED, Nagele P. The hematological effects of nitrous oxide anesthesia in pediatric patients. Anesth Analg. 2015 Jun. 120 (6):1325-30. [Medline].

  5. Korenke GC, Hunneman DH, Eber S, Hanefeld F. Severe encephalopathy with epilepsy in an infant caused by subclinical maternal pernicious anaemia: case report and review of the literature. Eur J Pediatr. 2004 Apr. 163(4-5):196-201. [Medline].

  6. Molloy AM, Kirke PN, Brody LC, Scott JM, Mills JL. Effects of folate and vitamin B12 deficiencies during pregnancy on fetal, infant, and child development. Food Nutr Bull. 2008 Jun. 29(2 Suppl):S101-11; discussion S112-5. [Medline].

  7. Erkurt MA, Aydogdu I, Dikilitas M, Kuku I, Kaya E, Bayraktar N, et al. Effects of cyanocobalamin on immunity in patients with pernicious anemia. Med Princ Pract. 2008. 17(2):131-5. [Medline].

  8. Pawlak R, Parrott SJ, Raj S, Cullum-Dugan D, Lucus D. How prevalent is vitamin B(12) deficiency among vegetarians?. Nutr Rev. 2013 Feb. 71(2):110-7. [Medline].

  9. Dror DK, Allen LH. Effect of vitamin B12 deficiency on neurodevelopment in infants: current knowledge and possible mechanisms. Nutr Rev. 2008 May. 66(5):250-5. [Medline].

  10. Ozkale M, Sipahi T. Hematologic and Bone Marrow Changes in Children with Protein-Energy Malnutrition. Pediatr Hematol Oncol. 2013 Aug 29. [Medline].

  11. Dugué B, Ismail E, Sequeira F, Thakkar J, Gräsbeck R. Urinary excretion of intrinsic factor and the receptor for its cobalamin complex in Gräsbeck-Imerslund patients: the disease may have subsets. J Pediatr Gastroenterol Nutr. 1999 Aug. 29(2):227-30. [Medline].

  12. Baumgartner MR. Vitamin-responsive disorders: cobalamin, folate, biotin, vitamins B1 and E. Handb Clin Neurol. 2013. 113:1799-810. [Medline].

  13. Trakadis YJ, Alfares A, Bodamer OA, et al. Update on transcobalamin deficiency: clinical presentation, treatment and outcome. J Inherit Metab Dis. 2014 May. 37 (3):461-73. [Medline].

Bone marrow aspirate from a patient with untreated pernicious anemia. Megaloblastic maturation of erythroid precursors is shown. Two megaloblasts occupy the center of the slide with a megaloblastic normoblast above. Photo courtesy of Marcel E Conrad, MD.
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