This is the first study that provides patient perspective on the burden of living with PK deficiency and lays the foundation for future studies to examine the effect of pharmacologic interventions on overall HRQoL for patients living with PK deficiency.

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OBJECTIVES: This study aimed to examine the validity of the modified Reflux Symptom Questionnaire–electronic Diary (mRESQ-eD) through patient input and psychometric testing of the questionnaire to support use in clinical trials in patients with persistent gastroesophageal reflux disease (GERD) and in accordance with Food and Drug Administration guidance on patient-reported outcome instruments. METHODS: Cognitive interviews were conducted with patients (n = 30) to evaluate the interpretability and content validity of draft mRESQ-eD items. Patient data from a phase 2b clinical study (ClinicalTrials.gov identifier: NCT02637557) on persistent GERD served to aid in the construction of weekly scores for heartburn severity, regurgitation severity, and total GERD severity. These scores' psychometric properties were also evaluated. RESULTS: Minor modifications were made to the draft mRESQ-eD based on patient feedback to improve interpretability and clarity of the instrument. Psychometric analysis suggested that an 8-item version of the mRESQ-eD was best suited to the clinical data. The internal consistency was found to be high (Coefficient ω = 0.95). Retest reliability and convergent validity were strong for a heartburn weekly severity score, regurgitation weekly severity score, and total GERD severity score. DISCUSSION: The final 8-item mRESQ-eD is a reliable and valid instrument with good psychometric properties for use in clinical trials in patients with persistent GERD. The mRESQ-eD may be considered for inclusion in clinical trials for persistent GERD and potentially positioned, in consultation with Food and Drug Administration, as endpoints to characterize treatment benefit.

Background: Research on women's perspective of uterine fibroids (UF) experiences using their own words is limited. This study aimed to provide new insights on the symptoms experienced and their impacts on daily life. Methods: Interview substudy in 30 US women with heavy menstrual bleeding (HMB) associated with UF who completed one of two phase 3, randomized, double-blind, placebo-controlled trials (LIBERTY 1 and 2; identifiers: NCT03049735, NCT03103087). Women who consented to participate in this substudy were interviewed after their last clinical trial study visit. Concepts ( i.e. , symptoms and impacts) of importance to women were determined via open-ended questions, and the frequency of symptoms and their impacts, including the relationship between pain and menstruation, were assessed. Data were analyzed using established qualitative research methods, including grounded theory and constant comparative methods, and concept saturation was assessed. Results: Fifteen unique symptoms of UF emerged: the most commonly reported were HMB ( n = 30, 100.0%), pelvic pain ( n = 28, 93.3%), and passing of blood clots ( n = 24, 80.0%). In total, 25 unique impacts were identified across eight concepts: physical impacts, activities of daily living, sleep, emotional impacts, sex life, social impacts, work and school, and financial impacts. Concept saturation was achieved for both symptoms and impacts. Conclusion: This study provides data on the symptoms experienced by women with HMB associated with UF, as well as the negative impacts of these symptoms as reported using their own words. The study findings confirm the significant burden associated with symptomatic UF.

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