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Hypophosphatemic rickets linked to the X (XLH) chromosome – 
KRN 23 (burosumab) therapy: expert opinion of the Society for Metabolic Bone Diseases within the Czech Medical Association 
of J.E. Purkyně


Authors: Palička Vladimír;  Bayer Milan;  Rosa Jan;  Kutílek Štěpán
Published in the journal: Clinical Osteology 2018; 23(1): 38-41
Category:

Summary

Received 5. 9. 2018


Zdroje
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  2. Imel EA, Econs MJ. Fibroblast growth factor 23: roles in health and disease. J Am Soc Nephrol 2005; 16(9): 2565–2575.

  3. Beck-Nielsen SS, Brock-Jacobsen B, Gram Jet al. Incidence and prevalence of nutritional and hereditary rickets in southern Denmark. Eur J Endocrinol 2009; 160(3): 491–497.

  4. Rafaelsen SH, Johansson S, Raeder H et al. Hereditary hypophosphatemia in Norway: a retrospective population-based study of genotypes, phenotypes and treatment complications. Eur J Endocrinol 2015; 174(2): 125–136.

  5. Endo I, Fukumoto S, Ozono K et al. Nationwide survey of fibroblast growth factor 23 (FGF23)-related hypophosphatemic diseases in Japan: prevalence, biochemical data and treatment. Endocr J 2015; 62(9):811–816.

  6. Carpenter TO. New perspectives on the biology and treatment of X-linked hypophosphatemic rickets. Pediatr Clin North Am 1997; 44(2): 443–466.

  7. de Paula FJ, Rosen CJ. Bone Remodeling and Energy Metabolism: New Perspectives. Bone Res 2013; 1(1): 72–84.

  8. Knochel JP. The pathophysiology and clinical characteristics of severe hypophosphatemia. Arch Intern Med 1977; 137(2): 203–220.

  9. Glorieux FH, Marie PJ, Pettifor JM et al. Bone response to phosphate salts, ergocalciferol, and calcitriol in hypophosphatemic vitamin D-resistant rickets. N Engl J Med 1980; 303(18): 1023–1031.

  10. Imel EA, DiMeglio LA, Hui SLet al. Treatment of X-linked hypophosphatemia with calcitriol and phosphate increases circulating fibroblast growth factor 23 concentrations. J Clin Endocrinol Metab 2010; 95(4): 1846–1850.

  11. Carpenter TO, Imel EA, Holm IA et al. A clinician’s guide to X-linked hypophosphatemia. J Bone Miner Res 2011; 26(7): 1381–1388.

  12. Beck-Nielsen H. The metabolic syndrome in the daily clinic. Ugeskr Laeger 2010; 172(23): 1746–1751.

  13. Berndt M, Ehrich JH, Lazovic D et al. Clinical course of hypophosphatemic rickets in 23 adults. Clin Nephrol 1996; 45(1): 33–41.

  14. Hardy DC, Murphy WA, Siegel BA et al. X-linked hypophosphatemia in adults: prevalence of skeletal radiographic and scintigraphic features. Radiology 1989; 171(2): 403–414.

  15. Reid IR, Hardy DC, Murphy WA et al. X-linked hypophosphatemia: a clinical, biochemical, and histopathologic assessment of morbidity in adults. Medicine (Baltimore) 1989; 68(6): 336–352.

  16. Whyte M et al. Burosumab (KRN23), a Fully Human Anti-FGF23 Monoclonal Antibody for X-linked Hypophosphatemia (XLH): Final 64-Week Results of a Randomized, Open-label Phase 2. Study of 52 Children. Oral presentation 1154. The Annual American Society for Bone and Mineral Research Meeting 2017; Denver, CO, USA.

  17. Carpenter TO et al. Effects of Burosumab (KRN23), a Fully Human Anti-FGF23 Monoclonal Antibody, on Functional Outcomes in Children with X-linked Hypophosphatemia (XLH): Final Results from a Randomized, 64-week, Open-label Phase 2 Study Oral presentation 331. The Annual American Society for Bone and Mineral Research Meeting 2017; Denver, CO, USA.

  18. Imel EA et al. Oral presentation at the ICCBH 2017; Würzburg, Germany.

  19. Imel EA et al. The Effects of Burosumab (KRN23), a Fully Human Anti-FGF23 Monoclonal Antibody, on Phosphate Metabolism and Rickets in 1 to 4-Year-Old Children with X-linked Hypophosphatemia (XLH). Oral presentation 695. The Annual American Society for Bone and Mineral Research Meeting 2017; Denver, CO, USA.

  20. Makitie O, Doria A, Kooh SW et al. Early treatment improves growth and biochemical and radiographic outcome in X-linked hypophosphatemic rickets. J Clin Endocrinol Metab 2003; 88(8): 3591–3597.

  21. Quinlan C, Guegan K, Offiah A et al. Growth in PHEX-associated X-linked hypophosphatemic rickets: the importance of early treatment. Pediatr Nephrol 2012; 27(4): 581–588.

  22. Informace dostupné z WWW: <https://clinicaltrials.gov/ct2/show/NCT02526160>.

  23. Informace dostupné z WWW: <https://clinicaltrials.gov/ct2/show/NCT02163577>.

  24. 24. Barth JH, Jones RG, Payne RB. Calculation of renal tubular reabsorption of phosphate: the algorithm performs better than the nomogram. Ann Clin Biochem 2000; 37(Pt 1): 79–81.

Štítky
Clinical biochemistry Paediatric gynaecology Paediatric radiology Paediatric rheumatology Endocrinology Gynaecology and obstetrics Internal medicine Orthopaedics General practitioner for adults Radiodiagnostics Rehabilitation Rheumatology Traumatology Osteology

Článok vyšiel v časopise

Clinical Osteology

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2018 Číslo 1
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