2024
Item Response Theory Quantifies the Relationship Between Improvements in Serum Phosphate and Patient‐Reported Outcomes in Adults With X‐Linked Hypophosphatemia
Mehta K, Gosselin N, Insogna K, Barriere O, Quattrocchi E, Hruska M, Marsteller D. Item Response Theory Quantifies the Relationship Between Improvements in Serum Phosphate and Patient‐Reported Outcomes in Adults With X‐Linked Hypophosphatemia. Clinical Pharmacology & Therapeutics 2024, 116: 1343-1351. PMID: 39129452, DOI: 10.1002/cpt.3406.Peer-Reviewed Original ResearchPatient-reported outcomesPatient-reported outcome dataX-linked hypophosphatemiaItem response theory parametersBrief Fatigue Inventory scoreSerum phosphateWestern Ontario and McMaster Universities Osteoarthritis IndexTreatment-induced improvementMcMaster Universities Osteoarthritis IndexTreatment response biomarkersBrief Pain InventoryInventory scoresAssociated with improvementsPain InventoryOsteoarthritis IndexTreatment optimization strategiesDisability scoresPopulation pharmacokinetic-pharmacodynamic modelPhase III studySerum phosphate levelsRare bone diseasesExposure metricsPharmacokinetic-pharmacodynamic modelLongitudinal dataIII studies
2021
Burosumab treatment in adults with X-linked hypophosphataemia: 96-week patient-reported outcomes and ambulatory function from a randomised phase 3 trial and open-label extension
Briot K, Portale AA, Brandi ML, Carpenter TO, Cheong HI, Cohen-Solal M, Crowley RK, Eastell R, Imanishi Y, Ing S, Insogna K, Ito N, de Beur S, Javaid MK, Kamenicky P, Keen R, Kubota T, Lachmann RH, Perwad F, Pitukcheewanont P, Ralston SH, Takeuchi Y, Tanaka H, Weber TJ, Yoo HW, Nixon A, Nixon M, Sun W, Williams A, Imel EA. Burosumab treatment in adults with X-linked hypophosphataemia: 96-week patient-reported outcomes and ambulatory function from a randomised phase 3 trial and open-label extension. RMD Open 2021, 7: e001714. PMID: 34548383, PMCID: PMC8458321, DOI: 10.1136/rmdopen-2021-001714.Peer-Reviewed Original ResearchConceptsBrief Pain Inventory-Short FormPatient-reported outcomesBrief Fatigue InventoryBPI-SF scoresAmbulatory functionWeek 96Burosumab treatmentMcMaster Universities Osteoarthritis IndexOpen-label extensionDouble-blinded trialPhase 3 trialMin walk testSignificant improvementWOMAC stiffnessOsteoarthritis IndexWeek 48Worse fatigueWeek 24Walk testFatigue InventoryWestern OntarioBFI scoresSubstantial burdenWOMACBurosumab
2016
Effect of four monthly doses of a human monoclonal anti-FGF23 antibody (KRN23) on quality of life in X-linked hypophosphatemia
Ruppe MD, Zhang X, Imel EA, Weber TJ, Klausner MA, Ito T, Vergeire M, Humphrey JS, Glorieux FH, Portale AA, Insogna K, Peacock M, Carpenter TO. Effect of four monthly doses of a human monoclonal anti-FGF23 antibody (KRN23) on quality of life in X-linked hypophosphatemia. Bone Reports 2016, 5: 158-162. PMID: 28326356, PMCID: PMC4926842, DOI: 10.1016/j.bonr.2016.05.004.Peer-Reviewed Original ResearchSF-36v2FGF23 antibodyPatients' perceptionsPhysical functioningSF-36v2 Health SurveyFibroblast growth factor 23Physical healthMcMaster Osteoarthritis IndexDose-escalation trialAssessment of HRQoLHealth-related qualityGrowth factor 23Decreased renal tubular reabsorptionRenal tubular reabsorptionLower extremity deformitiesPoor physical healthMean scoreQuality of lifeChronic functional impairmentOsteoarthritis IndexGeneral HRQOLJoint painSecondary outcomesPhase 1/2Stiffness scores