Results 1 to 10 of about 2,626,879 (142)

Content validity of a newly developed observer-reported measure for pediatric asthma in children aged 2–5 years

Journal of Patient-Reported Outcomes, 2022
Background An observer-reported outcome (ObsRO) measure assessing both symptom control and health-related quality of life (HRQoL) in children with asthma younger than 6 years is lacking. The objective of this study was to evaluate the content validity of
Yi Zhang, Jennifer L. Clegg, Shannon Keith, Shehan McFadden, Tara Symonds, Rajesh Kumar, Asif H. Khan, Siddhesh Kamat, Jingdong Chao   +8 more
doaj   +1 more source

Polypharmacy and clinical outcomes [PDF]

Canadian Medical Association Journal, 2015
We very much appreciate Shen’s[1][1] interest in our paper,[2][2] however, Shen’s comments are misleading. First, Shen’s recalculation of the number of patients who had a death event in Table 3, using data from Table 1, was incorrect. Our study is longitudinal, with up to 10 years of follow-
Wan-Hsuan, Lu, Yu-Wen, Wen, Liang-Kung, Chen, Fei-Yuan, Hsiao   +3 more
openaire   +5 more sources

A qualitative study to explore symptoms and impacts of pediatric and adolescent Crohn’s disease from patient and caregiver perspective

Journal of Patient-Reported Outcomes, 2021
Background Crohn’s disease (CD) is a chronic inflammatory condition of the gastrointestinal tract that affects people across the age spectrum but often starts in childhood or early adulthood.
Louise Newton, Laure Delbecque, Ufuk Coşkun, Tara Symonds, Jennifer Clegg, Theresa Hunter   +5 more
doaj   +1 more source

Understanding the patient experience of Classic Galactosemia in pediatric and adult patients: increased disease burden, challenges with daily living, and how they evolve over time

Journal of Patient-Reported Outcomes, 2023
Background Classic Galactosemia (CG) is a rare, autosomal recessive condition. Newborn screening and a timely galactose-restricted diet can resolve acute symptoms and decrease fatalities, but significant chronic, progressive morbidities remain and ...
Jason A. Randall, Carolyn Sutter, Lydia Raither, Stella Wang, Evan Bailey, Riccardo Perfetti, Shoshana Shendelman, Claire Burbridge   +7 more
doaj   +1 more source

Qualitative interviews with adults with Classic Galactosemia and their caregivers: disease burden and challenges with daily living

Orphanet Journal of Rare Diseases, 2022
Background Classic Galactosemia is a rare, autosomal recessive disease in which galactose is not metabolized properly due to severe deficiency/absence of the galactose-1-phosphate uridylyltransferase (GALT) enzyme, converting to an aberrant and toxic ...
Jason A. Randall, Carolyn Sutter, Stella Wang, Evan Bailey, Lydia Raither, Riccardo Perfetti, Shoshana Shendelman, Claire Burbridge   +7 more
doaj   +1 more source

Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale

Orphanet Journal of Rare Diseases, 2021
Background Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, genetic disease leading to impaired lysosomal function and neurodegeneration causing serious morbidity and shortened life expectancy.
Marc C. Patterson, Lucy Lloyd-Price, Christina Guldberg, Helen Doll, Claire Burbridge, Michael Chladek, Christine íDali, Eugen Mengel, Tara Symonds   +8 more
doaj   +1 more source

Qualitative research to explore the symptoms and impacts experienced by children with ulcerative colitis

Journal of Patient-Reported Outcomes, 2020
Background Ulcerative Colitis (UC) is a chronic gastrointestinal disease that often presents during one’s most productive years and is characterized by colon inflammation.
Jason A. Randall, Aiste Guobyte, Laure Delbecque, Louise Newton, Tara Symonds, Theresa Hunter   +5 more
doaj   +1 more source

Key measurement concepts and appropriate clinical outcome assessments in pediatric achondroplasia clinical trials

Orphanet Journal of Rare Diseases, 2022
Background This study aimed to identify fit-for-purpose clinical outcome assessments (COAs) to evaluate physical function, as well as social and emotional well-being in clinical trials enrolling a pediatric population with achondroplasia.
Natalie V. J. Aldhouse, Helen Kitchen, Chloe Johnson, Chris Marshall, Hannah Pegram, Sheryl Pease, Sam Collins, Christine L. Baker, Katherine Beaverson, Chandler Crews, Jill Massey, Kathleen W. Wyrwich   +11 more
doaj   +1 more source

Willingness of Greek general population to get a COVID-19 vaccine

Global Health Research and Policy, 2021
Background Epidemiological data indicate that a large part of population needs to be vaccinated to achieve herd immunity. Hence, it is of high importance for public health officials to know whether people are going to get vaccinated for COVID-19.
Georgia Kourlaba, Eleni Kourkouni, Stefania Maistreli, Christina-Grammatiki Tsopela, Nafsika-Maria Molocha, Christos Triantafyllou, Markela Koniordou, Ioannis Kopsidas, Evangelia Chorianopoulou, Stefania Maroudi-Manta, Dimitrios Filippou, Theoklis E. Zaoutis   +11 more
doaj   +1 more source

Development and validation of a psoriasis treatment acceptability measure through group concept mapping

Health and Quality of Life Outcomes, 2023
Background Psoriasis is a common autoimmune dermatologic condition which has a pronounced negative impact on patient quality of life and disease burden.
Stacie Hudgens, Amy Howerter, Shannon Keith, Colby Evans, Corey Pelletier   +4 more
doaj   +1 more source