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 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