2022
The efficacy and safety of burosumab in two patients with cutaneous skeletal hypophosphatemia syndrome
Sugarman J, Maruri A, Hamilton D, Tabatabai L, Luca D, Cimms T, Krolczyk S, Roberts M, Carpenter T. The efficacy and safety of burosumab in two patients with cutaneous skeletal hypophosphatemia syndrome. Bone 2022, 166: 116598. PMID: 36341949, DOI: 10.1016/j.bone.2022.116598.Peer-Reviewed Original ResearchConceptsCutaneous skeletal hypophosphatemia syndromeTumor-induced osteomalaciaBurosumab therapyDaily dosesMild injection site reactionsActive vitamin D analoguesExtra-cutaneous manifestationsMore daily dosesMultiple daily dosesSafety of burosumabTreatment of XLHDihydroxyvitamin D levelsInjection site reactionsCurrent treatment optionsLow serum phosphorusPromising therapeutic optionVitamin D analogsHuman monoclonal antibodyOral phosphorusAdult patientsAdverse eventsBone healthSerum phosphorusTherapeutic optionsD levels
2019
Sustained efficacy and safety of burosumab, a fully human anti-FGF23 monoclonal antibody, in children and early adolescents with X-linked hypophosphatemia
Hogler W, Carpenter T, Imel E, Portale A, Boot A, Linglart A, Padidela R, Hoff W, Mao M, Skrinar A, Martin J, Whyte M. Sustained efficacy and safety of burosumab, a fully human anti-FGF23 monoclonal antibody, in children and early adolescents with X-linked hypophosphatemia. Bone Abstracts 2019 DOI: 10.1530/boneabs.7.oc15.Peer-Reviewed Original ResearchContinued 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 periodBurosumabEfficacy 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 Phase 3 randomized, double-blind, placebo-controlled study investigating the efficacy and safety of Burosumab, an anti-FGF23 antibody, in adult X-Linked Hypophosphatemia (XLH)
Kamenicky P, Lachmann R, Carpenter T, Cohen-Solal M, Eastell R, Brandi M, Crowley R, Ralston S, Javaid M, Keen R, Briot K, Il C, Imanishi Y, Ito N, Tanaka H, Zhang L, Theodore-Oklota C, Mealiffe M, Martin J, Insogna K. A Phase 3 randomized, double-blind, placebo-controlled study investigating the efficacy and safety of Burosumab, an anti-FGF23 antibody, in adult X-Linked Hypophosphatemia (XLH). Endocrine Abstracts 2018 DOI: 10.1530/endoabs.56.oc3.1.Peer-Reviewed Original Research