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
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