Journal of Patient-Reported Outcomes最新文献

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.

Purpose: Warm autoimmune hemolytic anemia (wAIHA) is a rare disorder characterized by premature destruction of red blood cells (hemolysis) and fatigue that affects patients' health-related quality of life. This study aimed to identify fatigue concepts important to patients and evaluate the content validity of the Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue) in the wAIHA patient population.

Methodology: Two observational studies collected data via 60-minute, individual interviews. Eligible participants were English-speaking, US-resident adults diagnosed with wAIHA by a physician. Both studies gathered data regarding fatigue experiences that mattered to patients and comprehension and relevance of the FACIT-Fatigue; one study also gathered data about patient perspectives on meaningful change in item-level response selection.

Results: Each study enrolled 10 individuals with wAIHA (n = 20). All participants described fatigue as the most prominent or most bothersome symptom, with substantial impacts on daily functioning, family and social life, and emotional well-being. The FACIT-Fatigue was well understood, comprehensive, and relevant to participants' experiences with wAIHA. Data analysis indicated that at least a 3-point change in total score likely reflects a meaningful change in fatigue.

Conclusions: Fatigue is the primary patient-reported wAIHA symptom and important to measure from the patient perspective. Findings demonstrated that wAIHA patients found the FACIT-Fatigue understandable, comprehensive, and relevant, and the data indicated that the instrument can detect a meaningful change in fatigue symptoms and impacts. Study findings support the content validity of the FACIT-Fatigue and contribute to the evidence that the FACIT-Fatigue is fit-for-purpose to evaluate fatigue in adults with wAIHA.

Background: Genetic haemochromatosis (GH) is a long-term genetic condition which results in increased iron absorption into the blood and accumulation of iron into certain organs overtime. Increased absorption and accumulation can be fatal. GH can cause many symptoms including arthritis/joint pain, chronic fatigue, and cognitive difficulties. The aim of this study was to measure quality of life (QoL) in people diagnosed with GH (GH-diagnosed) compared to a healthy sample and identify possible explanations for this.

Methodology: QoL was measured in 535 healthy people and 1039 GH-diagnosed, through completion of the World Health Organisation Quality of Life-100 survey (WHOQOL-100). 985 GH-diagnosed respondents completed a GH-focussed survey, which was developed to get further details of the impact of GH.

Results: Comparison of the WHOQOL-100 overall QoL score between GH-diagnosed and the healthy sample found a significantly lower score in the GH-diagnosed. Physical, psychological, level of independence, and spiritual domains were significantly lower in the GH-diagnosed group. The GH-focussed survey found a high incidence of physical and mental symptoms, and some impact on social and work life. Areas in which participants suggest would improve their QoL included: improved healthcare especially with increased understanding of GH in medical professionals, increased access to appointments, in-person appointments, regular checks for organ damage, more nutrition or dietary advice, and local support groups.

Conclusions: Based on the WHOQOL-100 scores and GH-focussed survey, overall QoL is worse in people diagnosed with GH due to worse physical and psychological symptoms. Improved healthcare may aid in reducing the difference in QoL.

Background: The rise in artificial intelligence tools, especially those competent at language interpretation and translation, enables opportunities to enhance patient-centered care. One might be the ability to rapidly and inexpensively create accurate translations of English language patient-reported outcome measures (PROMs) to facilitate global uptake. Currently, it is unclear if machine translation (MT) tools can produce sufficient translation quality for this purpose.

Methodology: We used Generative Pretrained Transformer (GPT)-4, GPT-3.5, and Google Translate to translate the English versions of selected scales from the Breast-Q and Face-Q, two widely used PROMs assessing outcomes following breast and face reconstructive surgery, respectively. We used MT to forward and back translate the scales from English into Arabic, Vietnamese, Italian, Hungarian, Malay, and Dutch. We compared translation quality using the Metrics for Evaluation of Translation with Explicit Ordering (METEOR). We compared the scores between different translation versions using the Kruskal-Wallis test or analysis of variance as appropriate.

Results: In forward translations, the METEOR scores significantly varied depending on target languages for all MT tools (p < 0.001), with GPT-4 having the highest scores in most languages. We detected significantly different scores among translators for all languages (p < .05), except for Italian (p = 0.59). In backward translations, MTs (GPT-4: 0.81 ± 0.10; GPT-3.5: 0.78 ± 0.12; Google Translate: 0.80 ± 0.06) received higher or compatible scores to human translations (0.76 ± 0.11) for all languages. The differences in backward translation scores by different forward translators were significant for all languages (p < 0.01; except for Italian, p = 0.2). The scores between different languages were also significantly different for all translators (p < 0.001).

Conclusions: Our findings suggest that large language models provide high-quality PROM translations to support human translations to reduce costs. However, substituting human translation with MT is not advisable at the current stage.

Short stature in children aged 0-4 years presents unique physical, social, and emotional challenges that significantly impact health-related quality of life (HRQoL). The QoLISSY 0-4 questionnaire was developed as a Patient Reported Outcome measure (PROMS) to address the absence of an age-specific, condition-focused HRQoL assessment tool for this population. A mixed-method approach was employed to adapt the original QoLISSY questionnaire for children aged 5-18. Qualitative interviews were conducted with 24 parents of children diagnosed with short-stature conditions, including achondroplasia, small for gestational age, growth hormone deficiency, and Silver-Russell Syndrome. Cognitive debriefing sessions and iterative feedback guided the development of new items tailored to the needs of children aged 0-4. Pilot testing involved 20 parents, who evaluated the questionnaire's clarity, relevance, and comprehensiveness. The development process yielded an 8-domain, 55-item questionnaire addressing physical health, social interactions, emotions, coping mechanisms, medical care, future concerns, and parental impact. Cognitive debriefing results indicated high item clarity (100%), relevance (93%), and importance (94%), with parents confirming that the questionnaire effectively captured their child's HRQoL experiences. The QoLISSY 0-4 questionnaire provides a tailored, parent-reported tool for assessing HRQoL in children aged 0-4 with short stature. Its development reflects a rigorous, participant-informed process ensuring its relevance and usability. Future validation studies will explore its cross-cultural applicability and psychometric properties to establish its utility in research and clinical settings.