2025
Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Pediatric Patients With X-Linked Hypophosphatemia
Ali D, Mirza R, Hussein S, Alsarraf F, Alexander R, Abu Alrob H, Appelman-Dijkstra N, Biosse-Duplan M, Brandi M, Carpenter T, Chaussain C, Dandurand K, Filler G, Florenzano P, Fukumoto S, Grasemann C, Imel E, de Beur S, Morgante E, Ward L, Khan A, Guyatt G. Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Pediatric Patients With X-Linked Hypophosphatemia. The Journal Of Clinical Endocrinology & Metabolism 2025, 110: 1205-1217. PMID: 39787354, PMCID: PMC12012687, DOI: 10.1210/clinem/dgaf011.Peer-Reviewed Original ResearchX-linked hypophosphatemiaConventional therapyLower limb deformitiesNo treatmentMedical therapyAdverse eventsManagement of "X-linked hypophosphatemiaTreatment-emergent adverse eventsObservational study of individualsObservational studyEfficacy of medical therapyPhysical health QoLAssessed risk of biasBurden of symptomsEffects of medical therapyOpen-label RCTRisk of biasCertainty of evidenceIncreased adverse eventsLimb deformitiesPost hoc studyWeb of ScienceHealth QoLStudy of individualsDental abscess
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 lists
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