Journal of Patient-Reported Outcomes最新文献

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.

Purpose: Quantitative questionnaires can provide a deeper understanding of the health-related physical and psychological well-being in patients who have experienced a pulmonary embolism (PE). This paper describes the development of a questionnaire aiming to assess physical and psychological well-being and the need for rehabilitative strategies among patients diagnosed with PE.

Methods: The International Society for Quality-of-Life Research (ISO-QOL) recommendations for patient-reported outcomes were used. Conceptualization was conducted through literature review and expert interviews, followed by operationalization where items (questions) were constructed. To test content validity, the questionnaire was reviewed by experts and a series of cognitive interviews were performed. Finally, the questionnaire was distributed digitally to 82 randomly selected patients with PE in Denmark.

Results: The questionnaire reached a response rate of 72%. The median age of responders was 71.5 years [inter quartile range: 64.2; 77.0], with 51.9% being female. Most patients did not feel safe about being discharged, with only 9.3% responding that they felt safe to some or a high degree. Approximately 15% were offered physical therapy, 7.9% were offered educational activities addressing psychological reactions, and 3.9% received psychological support.

Conclusion: The questionnaire showed good content validity and a high response rate. The results from the questionnaire have the potential to increase focus and awareness of the potential clinical and social impact of PE. It will facilitate optimized medical interventions and guide physicians in providing appropriate follow-up care for patients with PE.

During a phase 2/3 study of twice-daily nirmatrelvir 300 mg coadministered with ritonavir 100 mg versus placebo for 5 days (NCT05011513), participants reported presence and severity of COVID-19 symptoms using a COVID-19 symptom diary in accordance with FDA guidance. Here, we aimed to evaluate the content validity of the COVID-19 symptom diary and global impression items through qualitative interviews as recommended by the FDA. Study participants kept a daily electronic diary to record the presence and severity of 14 COVID-19 symptoms (stuffy or runny nose, sore throat, shortness of breath, cough, low energy or tiredness, muscle or body aches, headache, chills/shivering, feeling hot or feverish, nausea, frequency of vomiting, frequency of diarrhea, sense of smell, and sense of taste). They also answered questions regarding 3 global impression items (return to health, return to usual activities, and overall symptom severity). A subset of clinical trial participants was interviewed at study completion to assess content validity of the diary. Overall, 25 participants were interviewed across 10 clinical sites. Each of the 14 symptoms included in the diary were reported by ≥ 1 participant, with low energy/tiredness being reported most frequently (88.0%). Twelve participants (48.0%) reported ≥ 1 symptom that was not captured by the diary, with dizziness being the most common (12.0%). Rates of these symptoms were lower (≤ 12.0%) than the 14 prespecified symptoms included in the diary (16.0-88.0%). Based on participant feedback, all 14 diary items were clear and generally easy to answer using the response scales, and all were interpreted as intended. The 3 global items received similar feedback, although a few participants noted that there were challenges in interpreting the items, describing questions as "vague" and "confusing", or that responses were too limited; one-third reported having difficulty calculating overall symptom experience for multiple symptoms. Participants reported that the electronic application for the diary and global items was easy to use and enabled the items to be easily completed. Exit interview results support the content validity of the COVID-19 symptom diary and global impression items, with the 14 symptoms included in the diary reflecting the most commonly experienced COVID-19 symptoms. NCT05011513; https://classic.clinicaltrials.gov/ct2/show/NCT05011513 .

Introduction: Psychometric properties of the Patient-Reported Outcomes Measurement Information System^® Profile 29 (PROMIS-29) Norwegian version has previously been examined in a general population. This multicenter study aimed to examine the internal consistency, construct validity, responsiveness, score distribution and floor/ceiling effects of PROMIS-29 v2.1 in a Norwegian rehabilitation context.

Methods: Patients receiving rehabilitation services participating in a longitudinal cohort study answered PROMIS-29 at baseline and at 3-month follow-up. Internal consistency was assessed by Cronbach's alpha and McDonald's omega. Construct validity was examined through hypothesis testing, using EQ-5D-5L as comparator measure. Hypotheses for correlations of change scores for both questionnaires were tested as an expression of responsiveness. Score distribution and floor/ceiling effects were examined with histograms and descriptive statistics.

Results: A total of 828 patients with a mean age of 54.3 years were included for analysis. The internal consistency for each PROMIS-29 domain was confirmed, with alpha and omega values exceeding the threshold of ≥ 0.70. Regarding correlations between PROMIS-29 and EQ-5D-5L, 34 out of 40 hypotheses were confirmed for construct validity and 19 out of 24 for responsiveness, both meeting our a priori criterion of ≥ 75% confirmed hypotheses. There was no floor effect of any PROMIS-29 domain in our sample, and ceiling effect only for anxiety and depression domain scores. Still, this confirmed the applicability of PROMIS-29 in a rehabilitation context.

Conclusion: The Norwegian PROMIS-29 has sufficient internal consistency, construct validity and responsiveness for use as an outcome measure for health status and health-related quality of life in rehabilitation.

Trial registration number: ClinicalTrials.gov NCT03764982 RehabNytte study, registered 2018-12-04.