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Hypophosphatemic Rickets Medication

  • Author: James CM Chan, MD; Chief Editor: Stephen Kemp, MD, PhD  more...
 
Updated: Dec 02, 2015
 

Medication Summary

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,[20] may be more damaging.

Unless a concomitant GH deficiency is observed, administration of biosynthetic GH for growth promotion has not been approved, unless GH deficiency is observed. Only preliminary evidence of improved final height with GH therapy has been reported.[6]

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Vitamin D

Class Summary

Standard protocol for treatment of familial hypophosphatemic rickets includes the use of 1,25-dihydroxy-vitamin D (calcitriol). The use of calcitriol in place of standard vitamin D obviates near-toxic dosage of the latter, avoids fat storage of parent vitamin D, and diminishes the danger of hypercalcemia.

Calcitriol (Rocaltrol)

 

Calcitriol increases calcium levels by promoting calcium absorption in the intestines and retention in kidneys.

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Phosphate replacement

Class Summary

Massive urinary phosphate loss is a problem intrinsic to the disorder, and the phosphate must be replaced.

Potassium phosphate/sodium acid phosphate (K-Phos Neutral, Phos-Nak)

 

This is a neutralized, buffered, oral phosphate-replacement solution containing 250 mg phosphorus, 280 mg potassium, 160 mg sodium . It is a combination of sodium and potassium phosphate.

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Diuretics

Class Summary

Thiazides are anticalciuric, an effect that can assist in counteracting the tendency for bone calcium loss.

Hydrochlorothiazide (Microzide)

 

Hydrochlorothiazide is a well-known diuretic with antihypertensive action. It inhibits reabsorption of sodium in distal tubules, causing increased excretion of sodium and water, as well as of potassium and hydrogen ions. Hydrochlorothiazide is not metabolized and is rapidly excreted in the urine.

Amiloride

 

Hypokalemia is a hazard when kaliuretic-effect thiazides are used; this danger that can be counteracted with the use of a second diuretic. Amiloride has a well-characterized antikaliuretic effect. Often used together with thiazides for its synergistic antihypertensive effects, amiloride has the benefit of decreasing potassium loss. Thus, it is a useful adjunct in the treatment of patients with familial X-linked hypophosphatemia with thiazides, in whom hypokalemia is a risk.

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Contributor Information and Disclosures
Author

James CM Chan, MD Professor of Pediatrics, Tufts University School of Medicine; Director of Research, The Barbara Bush Children's Hospital, Maine Medical Center

James CM Chan, MD is a member of the following medical societies: American Pediatric Society, Alpha Omega Alpha, American Academy of Pediatrics, American Physiological Society, American Society of Nephrology, American Society of Pediatric Nephrology, International Society of Nephrology

Disclosure: Nothing to disclose.

Coauthor(s)

Karl S Roth, MD Retired Professor and Chair, Department of Pediatrics, Creighton University School of Medicine

Karl S Roth, MD is a member of the following medical societies: Alpha Omega Alpha, American Academy of Pediatrics, American College of Nutrition, American Pediatric Society, American Society for Nutrition, American Society of Nephrology, Association of American Medical Colleges, Medical Society of Virginia, New York Academy of Sciences, Sigma Xi, Society for Pediatric Research, Southern Society for Pediatric Research

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.

George P Chrousos, MD, FAAP, MACP, MACE, FRCP(London) Professor and Chair, First Department of Pediatrics, Athens University Medical School, Aghia Sophia Children's Hospital, Greece; UNESCO Chair on Adolescent Health Care, University of Athens, Greece

George P Chrousos, MD, FAAP, MACP, MACE, FRCP(London) is a member of the following medical societies: American Academy of Pediatrics, American College of Physicians, American Pediatric Society, American Society for Clinical Investigation, Association of American Physicians, Endocrine Society, Pediatric Endocrine Society, Society for Pediatric Research, American College of Endocrinology

Disclosure: Nothing to disclose.

Chief Editor

Stephen Kemp, MD, PhD Former Professor, Department of Pediatrics, Section of Pediatric Endocrinology, University of Arkansas for Medical Sciences College of Medicine, Arkansas Children's Hospital

Stephen Kemp, MD, PhD is a member of the following medical societies: American Academy of Pediatrics, American Association of Clinical Endocrinologists, American Pediatric Society, Endocrine Society, Phi Beta Kappa, Southern Medical Association, Southern Society for Pediatric Research

Disclosure: Nothing to disclose.

Additional Contributors

Arlan L Rosenbloom, MD Adjunct Distinguished Service Professor Emeritus of Pediatrics, University of Florida College of Medicine; Fellow of the American Academy of Pediatrics; Fellow of the American College of Epidemiology

Arlan L Rosenbloom, MD is a member of the following medical societies: American Academy of Pediatrics, American College of Epidemiology, American Pediatric Society, Endocrine Society, Pediatric Endocrine Society, Society for Pediatric Research, Florida Chapter of The American Academy of Pediatrics, Florida Pediatric Society, International Society for Pediatric and Adolescent Diabetes

Disclosure: Nothing to disclose.

References
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  7. Sochett E, Doria AS, Henriques F, et al. Growth and metabolic control during puberty in girls with X-linked hypophosphataemic rickets. Horm Res. 2004. 61(5):252-6. [Medline].

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  13. Bresler D, Bruder J, Mohnike K, et al. Serum MEPE-ASARM-peptides are elevated in X-linked rickets (HYP): implications for phosphaturia and rickets. J Endocrinol. 2004 Dec. 183(3):R1-9. [Medline].

  14. Segawa H, Aranami F, Kaneko I, Tomoe Y, Miyamoto K. The roles of Na/Pi-II transporters in phosphate metabolism. Bone. 2009 Jul. 45 Suppl 1:S2-7. [Medline].

  15. Beck-Nielsen SS, Brock-Jacobsen B, Gram J, Brixen K, Jensen TK. Incidence and prevalence of nutritional and hereditary rickets in southern Denmark. Eur J Endocrinol. 2009 Mar. 160 (3):491-7. [Medline].

  16. Verge CF, Lam A, Simpson JM, Cowell CT, Howard NJ, Silink M. Effects of therapy in X-linked hypophosphatemic rickets. N Engl J Med. 1991 Dec 26. 325(26):1843-8. [Medline].

  17. Alon US, Monzavi R, Lilien M, et al. Hypertension in hypophosphatemic rickets--role of secondary hyperparathyroidism. Pediatr Nephrol. 2003 Feb. 18(2):155-8. [Medline].

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  20. Keskin M, Savaş-Erdeve Ş, Sağsak E, Çetinkaya S, Aycan Z. Risk factors affecting the development of nephrocalcinosis, the most common complication of hypophosphatemic rickets. J Pediatr Endocrinol Metab. 2015 Nov 1. 28 (11-12):1333-7. [Medline].

 
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