Which medications are used in the treatment of hypophosphatemic rickets?

Updated: May 02, 2018
  • Author: James CM Chan, MD; Chief Editor: Sasigarn A Bowden, MD  more...
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Answer

Burosumab (Crysvita) is the first drug approved in the U.S. for X-linked hypophosphatemia (XLH). It is a monoclonal IgG1 antibody that binds excess fibroblast growth factor 23 (FGF23). This action normalizes phosphorus levels, improves bone mineralization, improves rickets in children, and helps to heal fractures in adults. [8, 9, 10]

Other treatment options include calcitriol, GH, phosphates, and anticalciurics to promote healthy bone growth and diminish mineral loss associated with hypophosphatemic rickets. As previously stated, acute hypercalcemia (with resulting irritability, confusion, and potential seizures) can occur during treatment. Nephrocalcinosis, the long-term result of overaggressive therapy, [11] may be more damaging.

Unless a concomitant GH deficiency is observed, administration of biosynthetic GH for growth promotion has not been approved. Only preliminary evidence of improved final height with GH therapy has been reported. [31]


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