Cognitive-behavioral therapy and operant behavioral therapy treatments focus on factors that exacerbate or maintain suffering in chronic pain, and should be considered as part of a multidisciplinary treatment paradigm. Self-hypnosis training may also be of benefit, although it appears to be no more (or less) effective than other relaxation strategies that include hypnotic elements.

SIGNIFICANCE: Presbyopia typically occurs around 40 years of age and affects approximately one-quarter of the global population. Up to October 2021, there were no approved pharmacotherapies for presbyopia, and common treatments, such as glasses, can have disadvantages for individuals' health-related quality of life. PURPOSE:This study aimed to document the experience of living with and managing presbyopia, identify perspectives on treatment options, and determine whether there is an unmet need in the treatment landscape.METHODS: Coded transcripts of concept elicitation (CE; n = 20) and cognitive debriefing (n = 20) interviews with presbyopic individuals, originally conducted for development of patient-reported outcome instruments, were reanalyzed to identify salient concepts describing participants' experiences with presbyopia treatments. Qualitative ranking exercises assessed participants' preferences for a potential pharmacotherapy vs. existing treatments.RESULTS: Because most concepts were identified with the CE interviews, data reflect CE findings unless otherwise noted. Average age across CE/cognitive debriefing interviews was 49.4 years; a vast majority of participants used glasses for presbyopia treatment. Four themes related to treatment with glasses were identified with the interviews: inconvenience during daily activities, negative physical sensations around the eyes/head, limitations, and undesirable impacts on daily life (e.g., psychosocial). Most commonly, participants reported inconveniences related to forgetting glasses and psychosocial impacts (e.g., feeling/looking older). Strained/tired eyes and limited ability to see at varying distances were also reported. Among participants with near-vision glasses who provided data, two-thirds expressed interest in alternative treatments. In addition, almost three-quarters of the participants ranked hypothetical eye drops as their first or second preferred option, vs. reading glasses, contact lenses, magnifying glasses, and surgery.CONCLUSIONS: This study explored the experience of living with and managing presbyopia and identified limitations and negative impacts of current treatments. Pharmacological development (e.g., eye drops) may fulfill an unmet need in the presbyopia treatment landscape.

Sickle cell [5+ thalassaemia is regarded as the mildest ofthe sickle cell haemoglobinopathy syndromes with a benign natural course. In contrast to sickle cell disease, severe life threatening complications are not usually associated with this genotype.

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.

Tumor‐induced osteomalacia (TIO) is an ultra‐rare disease caused by tumors that secrete fibroblast growth factor 23, leading to chronic hypophosphatemia, poor skeletal health and impaired physical function. In a phase 2 trial (UX023T‐CL201; NCT02304367; n = 14), 48 weeks' burosumab treatment restored phosphate homeostasis, with improvements in skeletal health, functional mobility, and patient‐reported pain, fatigue, and health‐related quality of life (HRQL) (SF‐36 v2). Here, we report an exploratory mixed‐methods analysis of change from baseline after 144 weeks' burosumab treatment alongside qualitative data from exit interviews with eight of fourteen trial participants to evaluate meaningful treatment effects from a patient perspective. The interview subset (n = 8) reported pain and fatigue and compromised HRQL at baseline. In the interviews, participants reported that compromised HRQL and pain were the most important aspects of the disease to treat; both were considered more bothersome than fatigue, and compromised physical function and activities of daily living. Improvements in pain and fatigue following treatment were reported, some of which achieved statistically and/or clinically meaningful thresholds. Furthermore, improvements in SF‐36 v2 scores were most pronounced in the Physical Component Score and its Physical Function and Bodily Pain domains. Overall, the interview subset provided descriptions of symptomatic improvement and its clinical meaningfulness, including physical function, participation in activities of daily living, and mental wellbeing. Thus, this exploratory mixed‐methods analysis provides deeper understanding of patients' perception of clinical meaningfulness beyond that articulated in validated patient‐reported outcome instruments.This article is protected by copyright. All rights reserved.