2018
Burosumab Therapy in Children with X-Linked Hypophosphatemia
Carpenter TO, Whyte MP, Imel EA, Boot AM, Högler W, Linglart A, Padidela R, Van't Hoff W, Mao M, Chen CY, Skrinar A, Kakkis E, San Martin J, Portale AA. Burosumab Therapy in Children with X-Linked Hypophosphatemia. New England Journal Of Medicine 2018, 378: 1987-1998. PMID: 29791829, DOI: 10.1056/nejmoa1714641.Peer-Reviewed Original ResearchMeSH KeywordsAlkaline PhosphataseAntibodies, MonoclonalAntibodies, Monoclonal, HumanizedChildChild, PreschoolFamilial Hypophosphatemic RicketsFemaleFibroblast Growth Factor-23Fibroblast Growth FactorsGenetic Diseases, X-LinkedGrowthHumansKidney TubulesKnee JointMalePain ManagementPhosphorusRadiographySeverity of Illness IndexConceptsSerum phosphorus levelsRenal tubular phosphate reabsorptionTubular phosphate reabsorptionWeek 40Week 64Adverse eventsPhosphate reabsorptionNormal rangeMean serum phosphorus levelFibroblast growth factor 23End pointMean serum alkaline phosphatase levelSerum alkaline phosphatase levelsTotal scorePrimary end pointPhase 2 trialGrowth factor 23Additional end pointsPatient-reported outcomesAlkaline phosphatase levelsRadiographic Global ImpressionPhosphorus levelsOverall mean increaseBurosumab therapySubcutaneous burosumab
2014
Randomized trial of the anti-FGF23 antibody KRN23 in X-linked hypophosphatemia
Carpenter TO, Imel EA, Ruppe MD, Weber TJ, Klausner MA, Wooddell MM, Kawakami T, Ito T, Zhang X, Humphrey J, Insogna KL, Peacock M. Randomized trial of the anti-FGF23 antibody KRN23 in X-linked hypophosphatemia. Journal Of Clinical Investigation 2014, 124: 1587-1597. PMID: 24569459, PMCID: PMC3973088, DOI: 10.1172/jci72829.Peer-Reviewed Original ResearchMeSH KeywordsAdultAgedAntibodies, MonoclonalAntibodies, Monoclonal, HumanizedCalciumFamilial Hypophosphatemic RicketsFemaleFibroblast Growth Factor-23Fibroblast Growth FactorsGlomerular Filtration RateHalf-LifeHumansInjections, IntravenousInjections, SubcutaneousKidney TubulesMaleMiddle AgedPhosphatesVitamin DYoung AdultConceptsTmP/GFRSerum PiParathyroid hormonePhosphate reabsorptionXLH patientsRenal tubular thresholdSerum parathyroid hormoneFavorable safety profileElevated serum FGF23Renal phosphate reabsorptionLow serum concentrationsPhosphate-regulating endopeptidaseSerum Pi concentrationFGF23 antibodySerum FGF23Dihydroxyvitamin DSafety profileTubular thresholdSingle doseSerum concentrationsKRN23Mean t1/2Potential treatmentPatientsEffect duration