Journal of Patient-Reported Outcomes最新文献

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.

Background: Older people with severe frailty (OPWSF) have palliative needs but typically do not receive specialist palliative care (SPC). Patient-Reported Outcome Measures (PROMs) may offer valuable means to capture these needs. There is a limited understanding of what to include and how to implement PROMs in settings where this group receive most care. The study aimed to: (1) Critically examine how existing PROMs are currently implemented with OPWSF within a SPC setting. (2) Understand how the items with the PROMs are used (3) Develop a programme theory to determine how PROMs can be optimally designed and implemented to effectively capture the needs and priorities of OPWSF in the care setting where they receive most care.

Methodology: Mixed methods study in SPC community service in an urban area in the UK including: • Healthcare professionals (HCPs) providing care to OPWSF with a minimum of 6 months experience in a patient-facing role were purposively sampled: semi-structured interviews (n11); non-participatory observations (n10) - thematically analysed. • One-year retrospective case-note review of 357 episodes of care involving service-users identified with frailty at referral. Exploratory descriptive statistics were used to analyse the use of the Integrated Palliative Outcome Scale (IPOS) alongside additional clinical-led outcome measures. • Date integration using the Consolidation Framework for Implementation Research (CFIR) to develop a programme theory.

Results: PROMs can be effectively used with OPWSF, yet existing PROMs require adapting to ensure they capture the needs that matter most. • Completion of PROMs for this group largely depends on the assistance of others. • HCPs' use of PROMs may be driven by immediate care and priorities of the care system rather than determining changes over time, with the psycho-social aspect of the PROMs being more challenging to complete.

Conclusion: By utilising the CFIR, the study highlights the complexities and variabilities of using PROMs with OPWSF. Future research should focus on adapting and validating existing PROMs to ensure they are fit for purpose with this population, with the involvement of older people with frailty and unpaid carers. Providers should extend support and training for professionals in the use and value of PROMs and psychosocial-spiritual care.

Background: The Wound-QoL-17 and its short version, the Wound-QoL-14, measure health-related quality of life in patients with chronic wounds. This study assessed the content validity of this questionnaire.

Methodology: We recruited adult patients with chronic wounds in outpatient and inpatient settings in Germany. We conducted semi-structured interviews, which were audio-recorded, transcribed verbatim and analysed using qualitative content analysis.

Results: Almost all of the 21 patients (mean age 63 years, n = 16 male) had leg ulcers (n = 11) or diabetic foot ulcers (n = 8). The analysis resulted in six main categories: items; relevance; comprehensibility; comprehensiveness; version Wound-QoL-17 vs. Wound-QoL-14; further aspects. Participants mostly understood the distinct items well and found them easy to answer and relevant to their situation. The overall questionnaire was mostly rated relevant, comprehensible and comprehensive, including instructions, response scale, and recall period.

Conclusions: This study confirms the content validity of the Wound-QoL for patients with leg ulcers or diabetic foot ulcers and shows that it adequately reflects the construct of wound-specific quality of life. The Wound-QoL-17 should be used in clinical settings where differentiated assessment is appropriate. In research contexts where the calculation of scores is paramount, the Wound-QoL-14 should be used.