2020
OR29-06 Burosumab Improves Biochemical, Skeletal, and Clinical Features of Tumor-Induced Osteomalacia Syndrome
Carpenter T, Miller P, Weber T, Peacock M, Insogna K, Kumar R, Luca D, Cimms T, Roberts M, de Beur S. OR29-06 Burosumab Improves Biochemical, Skeletal, and Clinical Features of Tumor-Induced Osteomalacia Syndrome. Journal Of The Endocrine Society 2020, 4: or29-06. PMCID: PMC7208350, DOI: 10.1210/jendso/bvaa046.403.Peer-Reviewed Original ResearchTumor-induced osteomalaciaCutaneous skeletal hypophosphatemia syndromeSerious adverse eventsAdverse eventsSerum phosphorusBone biopsyPhysical functioningMean physical component summary scoreOpen-label phase 2 studyPhysical component summary scoreOsteoid volume/bone volumeSurface/bone surfaceProximal muscle functionBrief Pain InventoryOsteoid surface/bone surfacePhase 2 studyComponent summary scoresTmP/GFRCrest bone biopsiesRenal phosphate wastingHuman monoclonal antibodyMineralization lag timeQuality of lifeExcess FGF23Bone scan
2019
Continued Beneficial Effects of Burosumab in Adults with X-Linked Hypophosphatemia: Results from a 24-Week Treatment Continuation Period After a 24-Week Double-Blind Placebo-Controlled Period
Portale AA, Carpenter TO, Brandi ML, Briot K, Cheong HI, Cohen-Solal M, Crowley R, Jan De Beur S, Eastell R, Imanishi Y, Imel EA, Ing S, Ito N, Javaid M, Kamenicky P, Keen R, Kubota T, Lachmann R, Perwad F, Pitukcheewanont P, Ralston SH, Takeuchi Y, Tanaka H, Weber TJ, Yoo HW, Zhang L, Theodore-Oklota C, Mealiffe M, San Martin J, Insogna K. Continued Beneficial Effects of Burosumab in Adults with X-Linked Hypophosphatemia: Results from a 24-Week Treatment Continuation Period After a 24-Week Double-Blind Placebo-Controlled Period. Calcified Tissue International 2019, 105: 271-284. PMID: 31165191, DOI: 10.1007/s00223-019-00568-3.Peer-Reviewed Original ResearchConceptsWeek 48Adverse eventsWeek 24Sustained improvementTreatment-related serious adverse eventsOpen-label treatment periodSafety of burosumabDouble-blind placeboFatal adverse eventsSerious adverse eventsSerum phosphorus levelsPatient-reported outcomesSerum phosphorus concentrationRenal phosphate wastingHuman monoclonal antibodyContinued beneficial effectsHealing of fracturesRare genetic disorderMusculoskeletal morbidityPhysical functionContinuation periodMusculoskeletal impairmentsPhosphate wastingTreatment periodBurosumabOR13-1 Burosumab Improves the Biochemical, Skeletal, and Clinical Symptoms of Tumor-Induced Osteomalacia Syndrome
De Beur S, Miller P, Weber T, Peacock M, Insogna K, Kumar R, Luca D, Theodore-Oklota C, Lampl K, San Martin J, Carpenter T. OR13-1 Burosumab Improves the Biochemical, Skeletal, and Clinical Symptoms of Tumor-Induced Osteomalacia Syndrome. Journal Of The Endocrine Society 2019, 3: or13-1. PMCID: PMC6554835, DOI: 10.1210/js.2019-or13-1.Peer-Reviewed Original ResearchMineralization lag timeTumor-induced osteomalaciaSerum phosphorusAdverse eventsOsteoid thicknessTumor progressionBone biopsyDose intervalMean physical component summary scoreOpen-label phase 2 studyPP groupPhysical component summary scoreOsteoid volume/bone volumeSurface/bone surfaceMean serum phosphorusPhase 2 studySerious adverse eventsOsteoid surface/bone surfaceComponent summary scoresCrest bone biopsiesMean percentage changeEpidermal nevus syndromeRenal phosphate wastingHuman monoclonal antibodyOS/BSEfficacy and safety of burosumab in children aged 1–4 years with X-linked hypophosphataemia: a multicentre, open-label, phase 2 trial
Whyte MP, Carpenter TO, Gottesman GS, Mao M, Skrinar A, San Martin J, Imel EA. Efficacy and safety of burosumab in children aged 1–4 years with X-linked hypophosphataemia: a multicentre, open-label, phase 2 trial. The Lancet Diabetes & Endocrinology 2019, 7: 189-199. PMID: 30638856, DOI: 10.1016/s2213-8587(18)30338-3.Peer-Reviewed Original ResearchConceptsSerum phosphorus concentrationPhase 2 trialWeeks of treatmentAdverse eventsWeek 40Week 64Serum phosphorusPhosphatonin fibroblast growth factor 23Treatment-related adverse eventsFibroblast growth factor 23History of toothRickets Severity ScoreSafety of burosumabSerious adverse eventsInjection site reactionsKey secondary outcomesFavorable safety profileGrowth factor 23Severe food allergyHeight z-scoreKey inclusion criteriaRenal phosphate wastingHuman monoclonal antibodyRadiographic Global ImpressionYoung children
2018
A Randomized, Double‐Blind, Placebo‐Controlled, Phase 3 Trial Evaluating the Efficacy of Burosumab, an Anti‐FGF23 Antibody, in Adults With X‐Linked Hypophosphatemia: Week 24 Primary Analysis
Insogna KL, Briot K, Imel EA, Kamenický P, Ruppe MD, Portale AA, Weber T, Pitukcheewanont P, Cheong HI, de Beur S, Imanishi Y, Ito N, Lachmann RH, Tanaka H, Perwad F, Zhang L, Chen C, Theodore‐Oklota C, Mealiffe M, San Martin J, Carpenter TO, Investigators O. A Randomized, Double‐Blind, Placebo‐Controlled, Phase 3 Trial Evaluating the Efficacy of Burosumab, an Anti‐FGF23 Antibody, in Adults With X‐Linked Hypophosphatemia: Week 24 Primary Analysis. Journal Of Bone And Mineral Research 2018, 33: 1383-1393. PMID: 29947083, DOI: 10.1002/jbmr.3475.Peer-Reviewed Original ResearchConceptsTreatment-related serious adverse eventsMean serum phosphate concentrationWOMAC physical function subscaleFibroblast growth factor 23Intact parathyroid hormoneSerious adverse eventsPhase 3 trialPhysical function subscaleChronic musculoskeletal painGrowth factor 23Serum phosphate concentrationRenal phosphate wastingHuman monoclonal antibodyLower limb deformitiesImportant medical needStiffness subscalePlacebo groupUrine calciumWorst painAdverse eventsWeek 24Dental abscessMusculoskeletal painFactor 23Function subscale
2016
Effect of KRN23, a fully human anti-FGF23 monoclonal antibody, on rickets in children with X-linked hypophosphatemia (XLH): 40-week interim results from a randomized, open-label phase 2 study
Linglart A, Carpenter T, Imel E, Boot A, Högler W, Padidela R, Hoff W, Whyte M, Chen C, Skrinar A, Agarwal S, San Martin J, Portale A. Effect of KRN23, a fully human anti-FGF23 monoclonal antibody, on rickets in children with X-linked hypophosphatemia (XLH): 40-week interim results from a randomized, open-label phase 2 study. Annales D Endocrinologie 2016, 77: 440. DOI: 10.1016/j.ando.2016.07.547.Peer-Reviewed Original ResearchRickets Severity ScoreAdverse eventsQ2W dosingXLH childrenSerum PiMost treatment-related adverse eventsOpen-label phase 2 studyTransient injection site reactionsTreatment-related adverse eventsPhase 2 studySerious adverse eventsInjection site reactionsSerum phosphate levelsRadiographic Global ImpressionSerum Pi concentrationSerum iPTHUrine calciumRenal ultrasoundSite reactionsCare treatmentGlobal ImpressionSeverity scoreKRN23Phosphorus homeostasisShort stature