Journal of Patient-Reported Outcomes最新文献

Purpose: To develop a patient-reported screening tool, the Sinus Headache Screener (SHS), to differentiate non-rhinogenic facial pain (NRFP) from chronic rhinosinusitis (CRS) using qualitative research methods.

Methods: We conducted semi-structured interviews with 26 English-speaking adults (15 NRFP, 11 CRS). Interviews included concept elicitation and cognitive interviewing techniques. Content analysis was used to analyze transcripts, and items were iteratively refined over three rounds of interviews.

Results: The final list of potential SHS items consisted of 89 items across 8 sections, including symptoms, episode characteristics, triggers, and treatments.

Conclusions: This study represents the first step in developing a screening questionnaire to identify patients with NRFP in clinical settings. The item list provides a foundation for future quantitative studies to refine the questionnaire, potentially leading to more appropriate diagnosis and treatment of patients presenting with facial pain or pressure.

Objectives: To identify daily activity limitations, including but not limited to impairments in physical function, experienced by persons with rheumatoid arthritis (RA) and identify Patient-Reported Outcomes Measurement Information System (PROMIS) short form (SF) scales that can measure these limitations.

Methodology: A cross-sectional, web-based survey was conducted among a diverse group of adults with RA across the spectrum of disease activity. PROMIS Upper Extremity (UE) SF7a, Physical Function (PF) SF8b, and a Task Difficulty Scale were used to assess daily activity limitations experienced by persons with RA in the United States. An open-ended question asking what other daily activity limitations respondents experienced was also included. The daily activity limitation in the text response and the three scale items were deductively coded using the International Classification of Functioning, Disability, and Health (ICF) based on an established linking rule. Potential PROMIS SF scales were identified and linked to ICF to measure some activity limitations reported by persons with RA. PROMIS PF SF10a was linked to ICF and compared with UE SF7a and PF SF8b.

Results: Eighty-three out of 99 RA patient respondents answered the open-ended question, the majority (> 70%) of whom had severe or moderate physical function limitations. All 9 second-level sub-categories of the Activities and Participation (A&P) in ICF were linked to the text response to the open-ended question. UE SF7a and PF SF8b were linked to four of these second-level sub-categories (e.g., Mobility), while additional four second-level sub-categories (e.g., General tasks and demands, Interpersonal interactions and relationships) linked to PROMIS Fatigue SF7a and Ability to Participate in Social Roles and Activities (APS) SF4a. Moreover, PF SF10a was linked to four of these second-level sub-categories too, but with fewer items than UE SF7a plus the PF SF8b. Some activity limitations, such as driving and using telecommunication devices, were not linked to any items of the five PROMIS SF scales.

Conclusions: Persons with RA reported a variety of activity limitations across multiple domains, including physical function, telecommunication, social interactions, and other aspects of daily living, which could be a focus for goal-setting communication in the clinical setting. PROMIS PF SF10a may be a more effective and efficient scale to measure 4 sub-categories of daily activity limitations. To more comprehensively assess the spectrum of the impact of RA, it appears advisable to also use PROMIS Fatigue SF7a and APS SF4a to examine General tasks and demands, and Interpersonal interactions and relationships limitations.

Background and objectives: Mitochondrial encephalomyopathy with lactic acidosis and stroke-like episodes (MELAS) is a rare genetic syndrome mostly associated with pathogenic variants in mitochondrial DNA. As there is limited research on the life experience of patients with MELAS, this study aimed to develop an understanding of the patient experience of MELAS through qualitative interviews to identify, describe, and substantiate important and relevant signs, symptoms, and health-related quality-of-life (HRQoL) impact (S/S/I) concepts.

Methods: Clinician and patient interviews were conducted virtually using semi-structured interview guides. During 60-minute interviews with five experts in the United States, clinicians were asked for their perspective on S/S/I of patients with MELAS, patient experience of fatigue and cognitive impairment, and whether patients would be able to accurately report and rate their symptoms and complete a 90-minute patient experience interview. During a 45-minute interview conducted with 16 adults with confirmed pathogenic variant and clinical diagnosis of MELAS, patients were asked about S/S/I. Interviews were recorded, transcribed, anonymized, coded, and analyzed for saturation and concept frequency and clarification (e.g., severity, frequency, duration).

Results: Experts reported 44 distinct S/S and 36 HRQoL impact concepts. All five experts confirmed that cognitive impairment would not inhibit a typical patient's ability to report on their own experiences; three reported that patients with MELAS would not be able to complete a 90-minute interview. Sixteen patient interviews (mean age: 42.3 [11.1], n = 10 women) were conducted. Interviews with patients with MELAS achieved saturation of concept and yielded 35 S/S concepts and 68 HRQoL impacts across 15 domains. The most frequently reported S/S concepts were physical fatigue (n = 15, 93.8%), hearing loss (n = 13, 81.3%), mental fatigue (n = 12, 75.0%), and exercise intolerance and memory problems (n = 11, 68.8% each). The most frequently reported impact domains were adaptive behaviors and work impacts (n = 14, 87.5% each) and emotional function (n = 13, 81.3%).

Discussion: Patients with MELAS can self-report on S/S/I. Results from both patient and clinician interviews demonstrate that symptoms related to fatigue and cognitive impairment are frequent, bothersome, and important to improve. Assessments of fatigue and cognitive function should therefore be considered key outcome measures in clinical trials enrolling patients with MELAS.