All Articles tagged patient-reported outcomes

• Other Conditions
  February 19, 2025 EDT
  A Vignette Study to Derive Health-Related Quality-of-Life Weights for Individuals with Steroid Refractory Chronic Graft-versus-Host Disease Receiving Third-Line Therapy in the United Kingdom
  Emma WilliamsLuke SkinnerRichard HudsonArunesh SilKatharina EcsyElisheva LewAbdul Jabbar Omar AlsalehElin GruffyddAndrew LloydDaniele Avenoso

  This non-interventional health-related quality of life study involved the development, validation, and valuation of health state vignettes to generate utility values for chronic graft-versus-host disease in the UK.

  Abstract
• Genetic Disorders
  January 02, 2025 EDT
  Exploring Quality of Life in Adults Living With Late-onset Pompe Disease: A Combined Quantitative and Qualitative Analysis of Patient Perceptions from Australia, France, Italy, and the Netherlands
  Holly LumgairLisa BashorumAlasdair MacCullochElizabeth MinasGeorge TimminsDrago BratkovicRichard PerryMedi StoneVasileios BlazosElisabetta ContiRaymond Saich

  This study investigates how self-reported status of late-onset Pompe disease relates to quality of life and characterizes participant experiences, including mobility or ventilation aids, caregivers, symptomology, and daily life impacts.

  Abstract