Osteopetrosis Medication

Updated: Sep 11, 2017
  • Author: Robert Blank, MD, PhD; Chief Editor: George T Griffing, MD  more...
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Medication Summary

Medications administered in osteopetrosis include the following:

  • Vitamin-D supplements - Appear to help by stimulating dormant osteoclasts, thus stimulating bone resorption

  • Corticosteroids - Have also been used to stimulate bone resorption and to treat anemia

  • Erythropoietin - Another agent that can be used against anemia

  • Gamma interferon - Improves white blood cell function, greatly decreasing the incidence of new infections


Vitamin D Analogs

Class Summary

These supplements increase serum calcium levels by increasing calcium absorption from the gastrointestinal tract.

Calcitriol (Rocaltrol, Calcijex, Vectical)

In large doses, with restricted calcium intake, calcitriol sometimes improves osteopetrosis dramatically. It can be used to treat infantile osteopetrosis and appears to help by stimulating dormant osteoclasts and, thus, bone resorption. Markers of bone turnover (eg, serum osteocalcin, bone-specific alkaline phosphatase, urine hydroxyproline levels) increase during therapy. However, calcitriol usually produces only modest clinical improvement, which is not sustained after discontinuation.



Class Summary

These agents have anti-inflammatory properties and cause profound and varied metabolic effects. Corticosteroids modify the body's immune response to diverse stimuli.


Prednisone is an immunosuppressant used for the treatment of autoimmune disorders. It may decrease inflammation by reversing increased capillary permeability and suppressing polymorphonuclear leukocyte activity. The drug stabilizes lysosomal membranes and suppresses lymphocytes and antibody production.


Hematopoietic Growth Factors

Class Summary

These agents are used to manage anemia related to chronic renal failure, rheumatoid arthritis, and AIDS.

Epoetin alfa (Procrit, Epogen)

Epoetin alfa is a purified glycoprotein produced from mammalian cells modified with gene coding for human erythropoietin (EPO). The amino acid sequence is identical to that of endogenous EPO. Biological activity mimics human urinary EPO, which stimulates division and differentiation of committed erythroid progenitor cells and induces the release of reticulocytes from bone marrow into the blood stream.

Darbepoetin (Aranesp)

Darbepoetin is an erythropoiesis-stimulating protein closely related to erythropoietin, a primary growth factor produced in kidney that stimulates development of erythroid progenitor cells. Its mechanism of action is similar to that of endogenous erythropoietin, which interacts with stem cells to increase red cell production.

Darbepoetin contains 5 N-linked oligosaccharide chains, whereas epoetin alfa contains 3 such chains. Darbepoetin has longer a half-life than epoetin alfa and may be administered weekly or biweekly.



Class Summary

These agents delay disease progression in severe, malignant osteopetrosis. [24] Combined with calcitriol, interferons are substantially more effective than calcitriol alone. The combination reduces the incidence of severe infections, the number of transfusions needed, and the patient’s bone mass considerably more than calcitriol alone. The FDA approved interferon in 2000 for use in children with osteopetrosis.

Gamma-1b interferon (Actimmune)

Gamma-1b interferon is synthesized by eukaryotic cells in response to viruses and a variety of natural and synthetic stimuli. It possesses antiviral, immunomodulatory, and antiproliferative activity. Gamma interferon has potent phagocyte-activating effects not seen with other interferon preparations. It works by stimulating osteoclast activity.