2024
12605 Two Systematic Reviews Of Treatment Efficacy On Patient Important Outcomes In Children X-linked Hypophosphatemia
Ali D, Mirza R, Hussein S, Alsarraf F, Alexander R, AbuAlrob H, Brandi M, Carpenter T, Dandurand K, Filler G, Florenzano P, Fukumoto S, Grasemann C, Imel E, De Beur S, Morgante E, Ward L, Khan A, Guyatt G. 12605 Two Systematic Reviews Of Treatment Efficacy On Patient Important Outcomes In Children X-linked Hypophosphatemia. Journal Of The Endocrine Society 2024, 8: bvae163.528. PMCID: PMC11453601, DOI: 10.1210/jendso/bvae163.528.Peer-Reviewed Original ResearchRandomized controlled trialsBurden of symptomsCertainty of evidenceLower limb deformitiesPhysical health quality of lifePhysical health QoLSystematic reviewKyowa KirinOpen-label designModerate certaintyTreatment-emergent adverse eventsHealth quality of lifeObservational studyReviewed reference listsHealth QoLPatient-important outcomesRisk of biasHigh-certainty evidenceQuality of lifeLimb deformitiesWeb of ScienceX-linked hypophosphatemiaPost hoc studyCertainty evidenceReference listsImpact of burosumab on lower limb alignment in children with X-linked hypophosphatemia
Frumberg D, Merritt J, Chen A, Carpenter T. Impact of burosumab on lower limb alignment in children with X-linked hypophosphatemia. Journal Of The Pediatric Orthopaedic Society Of North America 2024, 6: 100012. DOI: 10.1016/j.jposna.2024.100012.Peer-Reviewed Original ResearchMechanical femoral-tibial angleLower limbsNeutral alignmentLower limb malalignmentLower limb alignmentLevel of Evidence IIIX-linked hypophosphatemiaLower limb deformitiesFemoral-tibial angleLower limb radiographsValgus limbsLimb malalignmentImpact of burosumabLimb alignmentLimb radiographsCrossover armAngular deformityNormal limbsLimb deformitiesLimbVarusMalalignmentClinically normal limbsHemiepiphysiodesisBaseline
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