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Background: The total burden of migraine includes not only the episodes with headache pain but extends throughout the interictal periods. Interictal symptoms and associated psychological responses may profoundly impact well-being and drive treatment-seeking behavior. Methods: A cross-sectional online survey was conducted with participants aged ≥ 18 years, 250 with episodic migraine (EM) and 250 with chronic migraine (CM), having ≥ 4 monthly migraine headache days. All were naïve to galcanezumab or began ≤ 6 months before survey completion. The study evaluated factors associated with the Migraine Interictal Burden Scale (MIBS-4), including social determinants of health and well-being. Multiple linear regression, logistic regression, and random forests (RF) were used to explore predictors of MIBS-4. Results: The majority of participants (90%) were female with a mean (standard deviation) age of 40.6 (± 12.0) years and 18.1 (± 12.7) years since the first migraine episode. Sociodemographically, the EM and CM groups were similar. Common comorbidities were anxiety disorder (45%) and depression (44%). Migraine family history was reported in 59% of participants. MIBS-4 was correlated with a number of diverse variables, including well-being, anxiety sensitivity, income, aura symptoms, and the worst migraine pain in the year before starting galcanezumab. Linear and logistic regression identified years since the first symptom, worst migraine attack pain, premonitory symptoms, and income as significant predictors. RF explained more of the variance than multiple linear regression and introduced additional concepts to the prediction of MIBS, identifying well-being (WHO-5 total score), the WHO-5 item "cheerful and in good spirits," worry about exercise, and fear of missing social obligations as significant predictors. Socioeconomic status and income were also critical explanatory variables for interictal burden (IIB) based on regression modeling and RF. Still, income was the only variable significantly associated with IIB across regression and RF methods. Conclusions: Interictal burden should be considered in the medical care of people with migraine. This additional burden is holistic, with psychosocial and socioeconomic elements in addition to residual symptoms. It is essential to consider this when assessing the impact of IIB. [ABSTRACT FROM AUTHOR]

Objective: Tolerability and safety of treatments are important in oncology trials and should be informed by patient assessments. We identified the most relevant patient‐reported symptomatic adverse events (AEs) to measure in patients with non‐small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations. Methods: This study selected relevant symptomatic AEs from 78 AEs available in the Patient‐Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO‐CTCAE) measurement system. Initially, symptomatic AEs were selected based on literature and product labeling reviews, and then core sets of symptomatic AEs were identified by patient and clinician interviews. Qualitative and descriptive analyses were performed using the data collected from three iterative rounds of patient interviews. Results: During concept elicitation interviews involving 29 patients, 12 symptomatic AEs were identified and were then adapted into a 25‐item PRO‐CTCAE form for use in future clinical trials along with commonly used PRO measures. Cognitive interviews showed that the PRO‐CTCAE items were easy to answer and appropriate for assessing the patients' experience with symptomatic AEs. This study also assessed disease symptoms, impacts, and overall patient experience. Conclusions: The 25‐item PRO‐CTCAE form captures the most relevant symptomatic AEs in this patient population, and it is available for future studies. Baseline characterization of AEs associated with this distinct patient group contributes to our broader knowledge about NSCLC and through platforms like Project Patient Voice will expand our understanding of treatment tolerability and safety for NSCLC. Ultimately, this data collection will help inform decision‐making for patients, caregivers, healthcare providers, and regulators. [ABSTRACT FROM AUTHOR]

Objective:The objective of this study was to evaluate the validity of the Heartburn Reflux Dyspepsia Questionnaire (HRDQ), a newly developed measure of gastro-oesophageal reflux disease (GORD) symptoms. Specifically, the HRDQ was developed for patients, who still experience symptoms with proton pump inhibitor (PPI) treatment. Material and methods:The psychometric properties of HRDQ were evaluated based on data from two clinical trials of patients with GORD with a partial response to PPIs, one from the UK and one from Denmark and Germany. Results:The HRDQ had good internal consistency (Cronbach’s alpha range .83–.88) and test–retest reliability (intraclass correlation coefficient range .71–.90). Convergent and discriminant validity were supported by high correlations with ReQuest™ and ability to differentiate between groups based on ReQuest™ cut-off values. Responsiveness of HRDQ was demonstrated by moderate to high correlations with ReQuest™ change scores and time with symptoms. An HRDQ cut-off value of 0.70 for definition of ‘bad day’ was also evaluated. Conclusions:Based on existing evidence, the HRDQ is a valid and reliable measure of GORD symptoms that can be used as a study outcome in clinical trials. [ABSTRACT FROM PUBLISHER]

ABSTRACT Background and objectives: Although it is accepted that reflux of stomach acid causes gastroesophageal reflux disease (GERD), it is less well understood that it also contributes to the clinical signs and symptoms of laryngitis in the form of laryngopharyngeal reflux (LPR). Study objectives were to identify what is known about the impact of LPR on health-related quality of life (HRQL) compared with the impact of GERD on HRQL and to assess whether currently available HRQL instruments adequately measure this impact or if a new disease-specific instrument should be recommended. Methods: The authors combined a systematic literature review with prospective patient evaluation via focus groups. The review, using MEDLINE, focused on clinical characteristics and HRQL measurement and impact. Focus groups involving a total of 30 patients with LPR provided input on clinical manifestations of the disease and its HRQL impact. Results: Information gleaned from the literature indicates that less than 40% of patients presenting with symptoms of laryngitis directly attributable to reflux also report experiencing the classic symptoms of heartburn and acid regurgitation associated with GERD. Reflux laryngitis is thus a distinct clinical entity from GERD and may have a unique impact on HRQL. Although multiple instruments are available to assess the impact of GERD on HRQL, no specific instruments are available for LPR. Focus group discussions identified voice problems, chronic cough, throat clearing, and swallowing difficulties to be key concerns of patients with LPR. These manifestations negatively impact HRQL as described by the focus group participants, notably in role functioning, physical well-being, and emotional well-being. Conclusions: A disease-specific instrument to assess the impact of LPR on HRQL would contribute to clinical care and the evaluation of new therapies. This instrument would ideally be sensitive to the variety of LPR's symptomatic presentations. [ABSTRACT FROM AUTHOR]

Background: Incorporating patient reporting of symptomatic adverse events (AEs) is important in evaluating safety and tolerability in cancer clinical trials. The Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) was developed to assess the frequency, severity, and/or interference of patient-reported symptomatic AEs. The objective of this study was to identify, based on oncologist and patient interviews, a relevant subset of symptomatic AEs from the PRO-CTCAE that can be used to optimize patient reporting of symptomatic AEs in hepatocellular carcinoma (HCC) clinical trials. Methods: Qualitative and quantitative data on HCC diagnosis, treatment, symptoms, and side effects were collected from patients. Using a numerical rating scale, medical oncologists specializing in HCC rated the importance of (1) 34 symptomatic AEs (Grade ≥2) identified in past sponsor HCC clinical trials, (2) each PRO-CTCAE symptomatic AE item to the patient, and (3) each PRO-CTCAE symptomatic AE item with respect to patient safety or tolerability. Patients completed the PRO-CTCAE items and were debriefed on the importance of each PRO-CTCAE symptomatic AE to them. Results: Five medical oncologists from the United States, Spain, Taiwan, Korea, and Hong Kong with 14 to 30 years of experience, and 17 patients with HCC and Child-Pugh class A or B cirrhosis status completed interviews. Medical oncologists rated the following symptomatic AEs from prior trials as being highly important to patients (mean rating of ≥7 on a scale from 0 to 10): hand-foot syndrome, diarrhea, fatigue, decreased appetite, rash, vomiting, and weight loss. PRO-CTCAE symptomatic AEs rated by medical oncologists as being highly important to patients included diarrhea, vomiting, shivering or shaking chills, hand-foot syndrome, rash, fatigue, difficulty swallowing, decreased appetite, and loss of control of bowel movements. Patients rated the following PRO-CTCAE symptomatic AEs as being highly important: loss of appetite/lack of interest in food, pain/tenderness at injection/insertion site, fatigue/lack of energy/tiredness, nausea, and hair loss. Conclusions: This study identified a preliminary list of clinically relevant symptomatic AEs from interviews with both medical oncologists and patients that can be used to support assessments of treatment safety and tolerability in HCC clinical trials. [ABSTRACT FROM AUTHOR]