Browsing by Subject "PSYCHOMETRIC PROPERTIES"
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Item Open Access End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain.(Blood advances, 2019-12) Farrell, Ann T; Panepinto, Julie; Carroll, C Patrick; Darbari, Deepika S; Desai, Ankit A; King, Allison A; Adams, Robert J; Barber, Tabitha D; Brandow, Amanda M; DeBaun, Michael R; Donahue, Manus J; Gupta, Kalpna; Hankins, Jane S; Kameka, Michelle; Kirkham, Fenella J; Luksenburg, Harvey; Miller, Shirley; Oneal, Patricia Ann; Rees, David C; Setse, Rosanna; Sheehan, Vivien A; Strouse, John; Stucky, Cheryl L; Werner, Ellen M; Wood, John C; Zempsky, William TTo address the global burden of sickle cell disease (SCD) and the need for novel therapies, the American Society of Hematology partnered with the US Food and Drug Administration to engage the work of 7 panels of clinicians, investigators, and patients to develop consensus recommendations for clinical trial end points. The panels conducted their work through literature reviews, assessment of available evidence, and expert judgment focusing on end points related to: patient-reported outcomes (PROs), pain (non-PROs), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the PROs, pain, and brain panels, as well as relevant findings and recommendations from the biomarkers panel. The panels identify end points, where there were supporting data, to use in clinical trials of SCD. In addition, the panels discuss where further research is needed to support the development and validation of additional clinical trial end points.Item Open Access Key lessons learned from the INDIGO global network on mental health related stigma and discrimination.(World psychiatry : official journal of the World Psychiatric Association (WPA), 2019-06) Thornicroft, Graham; Bakolis, Ioannis; Evans-Lacko, Sara; Gronholm, Petra C; Henderson, Claire; Kohrt, Brandon A; Koschorke, Mirja; Milenova, Maria; Semrau, Maya; Votruba, Nicole; Sartorius, NormanItem Open Access Trajectories of functional ability over the life course: a conceptual model of the interaction of stressor-induced functional loss and resilience.(System dynamics review, 2018-10) Matchar, David B; Ansah, John P; Koh, Victoria; Whitson, Heather EUnderstanding the factors that influence functional ability over the life course is integral to identifying clinical and public health policies to facilitate successful aging. The World Health Organization has advocated a conceptual framework to clarify the policy discussion. We have sought to translate this general framework into an explicit system dynamics model of the interaction of physiological loss, stressors and endogenous responses to produce a familiar variety of trajectories of functional ability over the life courses. Simulation experiments were implemented for both a 30-month duration with only one major stressor; and for the life course with an initial major stressor and subsequent stressors determined by the level of functional ability. For both contexts, variations in the few parameters in the scenarios led to a realistic range of trajectories of function over time.