2022
OR13-2 Characterizing the Impact of Burosumab on Bone Health in Children with X-Linked Hypophosphatemia: Results from Year 1 of the Disease Monitoring Program
Carpenter T, Cassinelli H, Glorieux F, Hetzer J, Merritt J, Moreira C, Portale A, Ward L, Woo C, Imel E. OR13-2 Characterizing the Impact of Burosumab on Bone Health in Children with X-Linked Hypophosphatemia: Results from Year 1 of the Disease Monitoring Program. Journal Of The Endocrine Society 2022, 6: a191-a192. PMCID: PMC9624613, DOI: 10.1210/jendso/bvac150.395.Peer-Reviewed Original ResearchSerum alkaline phosphatase levelsNew safety concernsAlkaline phosphatase levelsLower limb deformitiesGroup 1Serum phosphorusGroup 2Group 3Burosumab therapyBone healthPhosphatase levelsHeight z-score changeLimb deformitiesHuman IgG1 monoclonal antibodyYear 1 visitRecombinant human IgG1 monoclonal antibodySerum phosphorus levelsZ-score changeYears of treatmentRadiographic Global ImpressionYear 1Safety concernsIgG1 monoclonal antibodyBone painDMP enrolment
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