Journal of Patient-Reported Outcomes最新文献

Background: Dysphagia in a family member may negatively influence caregivers and may disrupt their quality of life. Thus, it is crucial to understand caregiver perspectives as an integral component of holistic dysphagia treatment. Hence, the present study aimed to cross-culturally adapt, translate, and pre-test the Caregiver's Analysis of Reported Experiences with Swallowing Disorders (CARES) into the Kannada language, a structural screening tool for assessing caregiver burden associated with dysphagia.

Methods: The Kannada version of the CARES screening tool was cross-culturally adapted using the ISPOR Principles of Good Practice for Patient-Reported Outcome Measures and was pre-tested on 48 dysphagia caregivers of adult dysphagia cancer survivors. The internal consistency of each item was assessed using Cronbach's alpha.

Results: The results revealed that the Kannada version of the CARES screening tool had good internal consistency for each subscale, Part A-Checklist of Behavioral and Functional Changes", and Part B-Measures of Subjective Caregiver Stress, with α = 0.763 and α = 0.852, respectively. The item-total correlation of each item of the subscale was greater than 0.8, indicating that each item of the tool significantly contributed to the tool.

Conclusions: The Kannada CARES was found to have good internal consistency. Hence, it can be considered a linguistically equivalent tool for identifying caregiver burdens associated with dysphagia care.

Background: Self-swabs and digital patient-reported outcomes (PROs) offer innovative tools for decentralized monitoring of infectious diseases. The DANFLU-2 HomeSwab PRO substudy evaluated the feasibility of using these methods for tracking influenza-like illness (ILI) within a large-scale, pragmatic, randomized trial.

Methods: During the 2023/2024 influenza season, adults aged ≥ 65 years were recruited from the DANFLU-2 trial, which evaluates the relative effectiveness of high-dose influenza vaccine compared to standard-dose. Participants were instructed to self-swab at home upon ILI symptom onset and complete the Respiratory Infection Intensity and Impact Questionnaire (RiiQ™) for 14 days. Swabs were registered via QR code in a webapp and mailed for centralized PCR testing. Compliance was defined as completing all 14 days of RiiQ™ reporting.

Results: Among 1,976 enrolled participants, 208 (10.5%) completed at least one RiiQ™, and 171 (82.2%) met the ILI case definition. Most participants found self-swabbing easy (66.1%) and more practical than visiting a clinic (78.6%). Compliance with daily RiiQ™ symptom tracking was 85.7%. Among those with ILI, 89.4% performed a self-swab within 1 day [IQR: 0; 3] of symptom onset; 65.8% of swabs were correctly registered in the webapp, and 96.5% were RNaseP-positive. Thirty-six participants (1.8%) withdrew, mainly due to weekly reminders; allowing reduced reminder frequency improved retention.

Conclusion: The study confirmed the feasibility of using home-based self-swabs for remote disease diagnosis and digital PRO tracking for symptoms during ILI events in a large-scale, pragmatic randomized trial. While the approach proved viable, the findings also highlighted areas for improvement in participant engagement and data collection efficiency.

Clinicaltrials:

Gov id: NCT05517174.

Background: Cervical cancer remains a significant public health issue in underdeveloped regions like Xinjiang, Western China, where health literacy is low and economic disparities are prominent. While previous studies have focused on preventive measures, there is limited research on the willingness to pay (WTP) for cervical cancer treatments. This study aimed to assess patient preferences and WTP from patient perspective for quality of life improvement, unadjusted life-year extension, and targeted and immunotherapy drugs among cervical cancer patients in Xinjiang.

Methods: A face-to-face survey was conducted using the Contingent Valuation Method (CVM) and Discrete Choice Experiment (DCE) to evaluate WTP for cervical cancer treatments. The CVM assessed patients' WTP for two scenarios: living in perfect health for 5 or 10 years versus unadjusted life-year extension for the same durations. Health related quality of life (HRQoL) was measured using both the EQ-5D-5 L and EQ-VAS instruments. The DCE evaluated patients' preferences and WTP for targeted therapy and immunotherapy drugs.

Results: This study included 106 valid questionnaires (response rate 96.4%), primarily comprising stage III cervical cancer patients (EQ-5D-5 L 0.89, EQ-VAS 0.80) with characteristics of low income and educational attainment. Key findings revealed: (1) Patients prioritized quality of life improvement over lifespan extension, with WTP/QALY in the 10-year perfect health scenario exceeding Xinjiang's 2024 per capita disposable income threshold while other scenarios remained below this value, and WTP/QALY being significantly higher in the 10-year scenario compared to the 5-year scenario; (2) Medication preference analysis demonstrated that quality of life improvement, cost, and incidence of adverse reactions were key decision-making factors, whereas survival extension held relatively lower importance. WTP valuations exhibited significant attribute-specific variations, with the highest WTP corresponding to quality of life improvement attributes and the lowest to survival extension attributes.

Conclusions: Cervical cancer patients in Xinjiang prioritize quality of life improvement over lifespan extension, with their preferences and WTP being influenced by treatment-related factors, disease characteristics, and socioeconomic background. Therefore, when formulating reimbursement policies and resource allocation strategies, priority should be given to interventions that can significantly improve quality of life, while implementing differentiated support policies for patient populations with varying socioeconomic statuses.

Background: Post-acute sequelae of COVID-19 (PASC) or Long COVID is a post-viral complication of SARS-CoV-2 infection, causing ongoing symptoms and impaired function over a prolonged period of time. There is limited understanding of how the signs and symptoms of Long COVID/PASC affect patients' lives and how to measure them, which is essential when developing care strategies. This study aimed to develop and evaluate the content validity for a novel patient-reported outcome (PRO) instrument in Long COVID/PASC through qualitative research, which was informed by the patient experience of Long COVID/PASC and both patient and clinician input.

Methods: A review of literature and PRO instruments developed for Long COVID/PASC identified measurement gaps for the context of use (i.e., weekly assessment of signs/symptoms in clinical trial research). This informed the development of the preliminary Long COVID/PASC PRO instrument, which was tested via patient interviews (combined concept elicitation and cognitive debriefing) to align with regulatory standards, and discussions with clinical experts were conducted to provide clinical insights. The final instrument was modified based on this input to further promote its content validity.

Results: Thirty participants were interviewed about their Long COVID/PASC experiences. Participants most frequently reported experiencing tiredness after physical activity (n = 29/30; 97%), general tiredness (n = 28/30; 93%), shortness of breath (n = 25/30; 83%), cough (n = 23/30; 77%) and muscle/body aches (n = 23/30; 77%). All participants reported that Long COVID/PASC had an impact on their health-related quality of life. Almost all (n = 27/28; 96%) sign/symptom concepts were reported in the first three sets of interviews suggesting conceptual saturation was achieved. Items, response options and the recall period of the preliminary Long COVID/PASC PRO instrument were understood as intended (≥ 90%) and relevant to most participants across both rounds (≥ 47%). Modifications were made to the instrument following patient input, resulting in the 18-item Long COVID/PASC instrument. Clinician input (n = 3) corroborated participant interview results, supporting the content validity of the Long COVID/PASC PRO instrument.

Conclusion: The Long COVID/PASC PRO instrument has been developed in line with regulatory standards and the qualitative evidence demonstrated strong content validity in a Long COVID/PASC population. Research to evaluate psychometric properties will provide further evidence of the instrument's measurement properties.

Our study aims to establish PROMIS Pain Interference score (PI) ranges that clinicians and persons living with chronic pain (PLwCP) associate with none, mild, moderate, and high impact chronic pain. We employed the PRO-Bookmarking technique to identify threshold scores that delineate different levels of chronic pain impact. PROMIS-PI score vignettes or "score stories" were developed to communicate the experience of living with different levels of pain interference. A panel of 10 PLwCP and another of 5 pain clinicians identified pairs of score vignettes they judged to represent the threshold between two levels of pain impact (e.g., ''moderate impact" and ''high impact"). We defined threshold scores as the mean score of the adjacent vignettes. We applied the obtained threshold scores to the distribution of PROMIS-PI scores in a sample of persons (n = 31,090) seen in a tertiary pain clinic. In another sample, we compared the Bookmarking and the revised Graded Chronic Pain Scale (GCPS-R) classifications. Patients and clinicians were in consensus on threshold scores for "no impact" to "mild impact" and "moderate impact" to "high impact," scores of 47 and 69, respectively. However, for the threshold for "mild impact to "moderate impact," the value was 65 for patients and 60 for clinicians. The comparison of classifications in the secondary sample revealed differences, especially for the 3rd level, high impact. The GCPS-R classified 58.2% as having high impact chronic pain; the Bookmarking thresholds classified 23.8% as such. The PRO-Bookmarking approach effectively delineated thresholds for classifying levels of chronic pain impact using PROMIS-PI scores. This method incorporates the perspectives of PLwCP and allows for post-hoc application to diverse patient samples.

Background: Although the group of patients with obstructive sleep apnea (OSA) is very heterogeneous, OSA's severity is mainly expressed by an apnea-hypopnea index (AHI), which does not correlate well with the experienced symptom severity. As a first step to develop a more personalized approach for treatment, the purpose of the current study was to create, through cluster analysis, meaningful OSA phenotypes linked to the Patient Reported Apnea Questionnaire (PRAQ).

Methods: Through a survey, new OSA patients indicated for continuous positive airway pressure (CPAP) treatment completed the Epworth Sleepiness Scale (ESS) and the PRAQ to rate their experienced symptom severity. Clinical data, such as the AHI and comorbidity, were assessed from the patient file. Cluster analysis has been performed to derive OSA phenotypes.

Results: Based on the AHI, comorbidity and experienced symptom severity data of 151 patients, a two-step cluster analysis revealed five OSA phenotypes: "no comorbidity", "hypertension", "high symptom severity", "low symptom severity" and "unclassified". The five phenotypes mainly differ in the experienced level of fatigue, partner-observed snoring severity and symptoms related to performing regular daily activities.

Conclusion: Not only the AHI, but also comorbidity and subjective symptoms should be taken into consideration when diagnosing OSA, assessing its severity and in providing a more patient-oriented treatment, including deciding about CPAP treatment. Not the often-used ESS but the modified PRAQ scales provide relevant information to assess experienced symptom severity. In addition, for an improved prognostication, we propose an evaluation of the CPAP treatment effectiveness for the five reported OSA phenotypes.