Patient Related Outcome Measures最新文献

Purpose: Sleep disturbance is common in patients with asthma and can lead to subsequent impacts on health-related quality of life (HRQOL). Fit-for-purpose patient-reported outcome measures (PROMs) assessing asthma-related sleep disturbance and next-day HRQOL impact (next-day impact) are needed to evaluate disease burden and treatment effects.

Patients and methods: Adults (18-65 years) from three US clinics were recruited for semistructured interviews. Concept elicitation (CE) identified how asthma affects participants' sleep and how asthma-related sleep disturbances impact their daily lives, which informed conceptual model development. Cognitive debriefing (CD) of the Asthma Sleep Disturbance Questionnaire (ASDQ), Sleep Diary, and Patient-Reported Outcomes Measurement Information System Sleep-Related Impairment Short Form 8a (PROMIS SRI SF8a) was completed to assess each measure's content validity.

Results: Twelve individuals participated in two interview rounds (6 individuals per round). Participants most frequently reported asthma-related nighttime awakening and decreased sleep quality and duration. Negative impacts of a poor night's sleep due to asthma symptoms included feeling tired/fatigue/lack of energy and subsequent negative impacts on physical functioning, emotions and mood, mental functioning, work or volunteerism, and social functioning. Across both rounds of CD interviews, participants generally found the Sleep Diary and PROMIS SRI SF8a items relevant and easy to complete with no modifications. The ASDQ was modified for clarity and consistency.

Conclusion: As described in the conceptual model, asthma affects multiple aspects of sleep that can cause next-day fatigue and other subsequent negative HRQOL impacts. This study demonstrates that the ASDQ, Sleep Diary, and PROMIS SRI SF8a items are comprehensive, relevant, and appropriate for patients with moderate-to-severe, uncontrolled asthma. Evaluation of psychometric properties for the ASDQ, Sleep Diary, and PROMIS SRI SF8a based on clinical trial data in patients with moderate-to-severe, uncontrolled asthma will further support their use.

Background: The health services delivery system is debilitated in conflict setting areas due to damage of health facilities and attack on health care providers. Armed conflict is a complex phenomenon which causes a large amount of death and disability worldwide. However, there is lack of information towards treatment interruption among chronic disease patients in conflict areas. Hence, this study was conducted to assess the prevalence of armed conflict induced treatment interruption and its outcome among chronic disease patients.

Methods: A mixed cross sectional study design was conducted on a total of 399 sample size at North Wollo and Waghimra zone hospitals. To select study participants a consecutive sampling method was used. Multiple logistic regressions were computed to evaluate the association and thematic analysis was used to analyse qualitative data.

Results: From the total 399 study participants, 264 (64.6%) study participants interrupted their medication. Treatment interruption is significantly associated with poor service availability and perceived high stress.

Conclusion: War has detrimental health effects, both immediate and long term, on populations. Treatment interruptions due to armed conflict were caused by poor health care access, lack of medications and lack of transport and displacement of hospital staff, insecurity and fear. Treatment interruption results in morbidity, mortality, lifelong complications, disability, psychological and economic impact.

Background: One of the key dimensions of healthcare quality is patient-centeredness, which represents how well healthcare is geared towards patients' needs and wishes. Many questionnaires that measure the patient-centeredness are long and complicated, eliciting non-response or careless responding. Moreover, responses to some commonly used questionnaires are difficult to interpret. The Patient-Centeredness Questionnaire-Infertility (PCQ-Infertility) is used to measure the patient's experience of fertility healthcare quality. The aim of this study was to improve the PCQ-Infertility to allow large-scale clinical implementation.

Methods: The study was performed in three parts. First, shortcomings of the original PCQ-Infertility were identified by evaluation of expert opinions. Second, the number of items were reduced, and items were rescaled and rephrased. Third, 844 patients filled in the original PCQ-Infertility and 260 patients filled in the revised PCQ-Infertility and reliability analyses were performed. In addition, a confirmatory factor analysis was performed on the revised PCQ-Infertility.

Results: The number of items in the revised questionnaire was reduced by 24% (from 51 to 39 items), which increased the internal consistency and reliability. The reliability analyses and confirmatory factor analysis indicated high consistency and convergent validity in all seven dimensions (accessibility, information, communication, patient involvement, respect for patient's values, continuity and transition, and competence) of the revised PCQ-Infertility.

Conclusion: The revised PCQ-Infertility is a more valid and reliable instrument than the original PCQ-Infertility, easier to interpret and shorter. Therefore, large-scale clinical implementation and data analysis are now possible, giving the opportunity for fertility care professionals to evaluate and improve their healthcare.

Purpose: To understand pregnant women's experience with midwifery-led antenatal care services using the Respectful Maternity Care charter in primary health centers in Karachi, Pakistan.

Methods: This cross-sectional study was at Rehri Goth and Ibrahim Hyderi, two peri-urban communities in Karachi, Pakistan, where women receive antenatal care services. All pregnant women in their third trimester who consented during the study period were included. The participants were asked about access to care, antenatal care experience, person-centered approach, and general satisfaction with the facility using a pre-designed questionnaire. These themes were mapped onto the universal Respectful Maternity Care charter. Descriptive statistics were used to summarize the findings in each of these themes. Multivariable logistic regression techniques to determine the relationship between the dependent and independent variables.

Results: There were 904 women who agreed to participate in this study during January to December 2021. Majority of the women (94%, n=854) were satisfied with the operating hours and cleanliness. More than 90% of the women reported positive experiences regarding privacy, respectful treatment by midwives, and non-discriminatory care. However, 40% (n=362) of the women reported not receiving adequate information and informed consent before a medical procedure, while 65% (n=587) reported poor counseling for birth preparedness. Maternal age, women's occupation, women's education, and parity were found to be significantly associated with respect provided, satisfaction with counseling and the consent process.

Conclusion: This study reported satisfaction of pregnant women with the facility's ambiance, respect, and care; however, poor communication skills regarding consent and antenatal counseling were reported. The findings suggest the need for more efficient strategies, such as regular respectful maternity care and technical training to strengthen midwife-patient interactions and enhance overall satisfaction, thus improving maternal and newborn outcomes.

Introduction: Decision aids are effective tools in facilitating patient-centered care and patient involvement in the decision-making process. Given unique barriers to providing patient-centered care for Veterans, implementation of decision aids may improve overall quality of care. We aimed to assess the acceptability and feasibility of video-based and pamphlet-based decision aid use in Veterans with knee osteoarthritis.

Materials and methods: Veterans considering treatment for knee osteoarthritis received either an online video-based aid, pamphlet-based aid, or both before their surgical consult. At their visit, patients completed written pre-visit and post-visit questionnaires. The pre-visit questionnaire included questions about the patient's demographics, decision-making preferences, experiences using the assigned decision aids, and the Hip-Knee Decision Quality Instrument. The post-visit questionnaire assessed the patient's overall experience with the decision-making process and how use of the decision aid influenced their discussion with the physician.

Results: All 16 patients who received the pamphlet-based aid reviewed the decision aid before their visit, compared to only five of the 12 patients who received the video-based aid. Thirteen of 20 patients indicated that they preferred to share treatment decision-making with their physician. Seventeen of 20 patients believed they would feel comfortable questioning the treatment recommendation of their surgeon after decision aid use. Most patients reported a positive experience using their decision aid, regardless of modality, and found it easily comprehensible and useful in visit preparation. A preference for a pamphlet-based aid was expressed by the majority of patients.

Conclusion: Veterans considering treatment for knee osteoarthritis are well prepared to engage in a patient-centered care experience. Most patients preferred sharing the decision-making process with their physician and felt comfortable questioning them about treatment recommendations. Decision aids helped Veterans feel more informed about their treatment options and improved engagement and discussion with their physician. Pamphlet-based aids were utilized more reliably than video-based aids.

Purpose: Patients with transthyretin amyloidosis (ATTR) experience a wide variety of symptoms and impacts on health-related quality of life (HRQoL). However, the lack of an ATTR-specific patient-reported outcome (PRO) measure has made consistent measurement of HRQoL in ATTR challenging. This paper describes the development of a conceptual model and subsequent content for the Transthyretin Amyloidosis - Quality of Life Questionnaire (ATTR-QOL), an ATTR-specific PRO measure.

Methods: This was a cross-sectional, non-interventional, US-based study. The study design included three stages: 1) a targeted literature review followed by qualitative data collection with patients and experts; 2) development of a conceptual model and PRO measure; and 3) review of the PRO measure using a modified Delphi method, translatability assessment, and interviews with patients and experts. Revisions were made to the measure after each round of review.

Results: Forty-four patients and 29 experts participated in this study. The conceptual model included two primary concepts of interest: symptoms (cardiac, neuropathic-peripheral, neuropathic-autonomic, and other) and impacts (eg, physical, role, and mental/emotional functioning). Seventy-two items were created (32 symptoms; 40 impacts) to align with the model. A recall period of one month was selected based on participant input.

Conclusion: The ATTR-QOL was created with significant patient involvement and guidance from a multidisciplinary group of experts. The mix of patient and clinical perspectives helped to ensure a balanced representation of all relevant disease experiences and clinical specialties. With further refinement from psychometric testing, the ATTR-QOL will provide a standard, comprehensive measure for all ATTR-specific research including both clinical trials and clinical practice.

Purpose: Disturbance of consciousness is common in patients with severe anti-N-methyl-D-aspartate receptor (NMDAR) encephalitis. However, little is known about it. This study aimed to analyze the clinical manifestations and prognostic factors of anti-NMDAR encephalitis with disturbance of consciousness.

Methods: In this retrospective study, the clinical features, treatment results, and long-term outcomes of anti-NMDAR encephalitis patients with disturbance of consciousness were analyzed, and multivariate logistic regression was used to analyze the factors affecting their prognosis.

Results: In the group with disturbance of consciousness, the incidences of seizures, involuntary movements, pulmonary infection, mechanical ventilation, intensive care unit (ICU) admission, neutrophil-lymphocyte ratio (NLR), abnormal cerebrospinal fluid index, plasma exchange, and immunosuppressive therapy were higher than those in the group without disturbance of consciousness (all P<0.05). During the follow-up period (median: 36 months, range: 12-78 months), the modified Rankin scale (mRS) score, the maximum mRS score during hospitalization, the mRS score at discharge, and the mRS score at 12 months after discharge were higher in the disturbance of consciousness group (all P < 0.001). However, there was no significant difference in long-term outcomes and recurrence between the two groups. Multivariate logistic regression analysis showed that mechanical ventilation, elevated IgG index, and delayed immunotherapy were independent risk factors for poor outcomes in patients with anti-NMDAR encephalitis with disturbance of consciousness at 12 months (odds ratio: 22.591, 39.868, 1.195). The receiver operating characteristics (ROC) curve analysis showed that the area under the curve (AUC) of mechanical ventilation, elevated IgG index, and delayed immunotherapy was 0.971 (95% CI=0.934-1.000, P<0.001).

Conclusion: Mechanical ventilation, elevated IgG index, and delayed immunotherapy may be the influencing factors of poor prognosis of anti-NMDAR encephalitis patients with disturbance of consciousness. Although their condition is relatively serious, most patients with anti-NMDAR encephalitis with disturbance of consciousness will achieve favorable long-term outcomes after long-term treatment.

Reliability and measurement error are measurement properties that quantify the influence of specific sources of variation, such as raters, type of machine, or time, on the score of the individual measurement. Several designs can be chosen to assess reliability and measurement error of a measurement. Differences in design are due to specific choices about which sources of variation are varied over the repeated measurements in stable patients, which potential sources of variation are kept stable (ie, restricted), and about whether or not the entire measurement instrument (or measurement protocol) was repeated or only part of it. We explain how these choices determine how intraclass correlation coefficients and standard errors of measurement formulas are built for different designs by using Venn diagrams. Strategies for improving the measurement are explained, and recommendations for reporting the essentials of these studies are described. We hope that this paper will facilitate the understanding and improve the design, analysis, and reporting of future studies on reliability and measurement error of measurements.

Systemic AL (light chain) amyloidosis is a rare protein misfolding disorder associated with plasma cell dyscrasia affecting various organs leading to organ dysfunction and failure. The Amyloidosis Forum is a public-private partnership between the Amyloidosis Research Consortium and the US Food and Drug Administration Center for Drug Evaluation and Research with the goal of accelerating the development of effective treatments for AL amyloidosis. In recognition of this goal, 6 individual working groups were formed to identify and/or provide recommendations related to various aspects of patient-relevant clinical trial endpoints. This review summarizes the methods, findings, and recommendations of the Health-Related Quality of Life (HRQOL) Working Group. The HRQOL Working Group sought to identify existing patient-reported outcome (PRO) assessments of HRQOL for use in clinical trials and practice deemed relevant across a broad spectrum of patients with AL amyloidosis. A systematic review of the AL amyloidosis literature identified 1) additional signs/symptoms not currently part of an existing conceptual model, and 2) relevant PRO instruments used to measure HRQOL. The Working Group mapped content from each identified instrument to areas of impact in the conceptual model to determine which instrument(s) provide coverage of relevant concepts. The SF-36v2^® Health Survey (SF-36v2; QualityMetric Incorporated, LLC) and Patient-Reported Outcomes Measurement Information System-29 Profile (PROMIS-29; HealthMeasures) were identified as instruments relevant to patients with AL amyloidosis. Existing evidence of reliability and validity was evaluated with a recommendation for future work focused on estimating clinically meaningful within-patient change thresholds for these instruments. For sponsors, the context of use-including specific research objectives, trial population, and investigational product under study-should inherently drive selection of the appropriate PRO instrument and endpoint definitions to detect meaningful change and enable patient-focused drug development.

Objective: This systematic review aimed to assess the adequacy of measurement properties in Patient-Reported Outcome Measures (PROMs) used to quantify psychosocial consequences of colorectal cancer screening among adults at average risk.

Methods: We searched four databases for eligible studies: MEDLINE, CINAHL, PsycINFO, and Embase. Our approach was inclusive and encompassed all empirical studies that quantified aspects of psychosocial consequences of colorectal cancer screening. We assessed the adequacy of PROM development and measurement properties for content validity using The COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) risk of bias checklist.

Results: We included 33 studies that all together used 30 different outcome measures. Two PROMs (6.7%) were developed in a colorectal cancer screening context. COSMIN rating for PROM development was inadequate for 29 out of 30 PROMs (97%). PROMs lacked proper cognitive interviews and pilot studies and therefore had no proven content validity. According to the COSMIN checklist, 27 out of 30 PROMs (90%) had inadequate measurement properties for content validity.

Discussion: The majority of included PROMs had inadequate development and measurement properties. These findings shed light on the trustworthiness of the included studies' findings and call for reevaluation of existing evidence on the psychosocial consequences of colorectal cancer screening. To provide trustworthy evidence about the psychosocial consequences of colorectal cancer screening, editors could require that studies provide evidence of the methodological quality of the PROM. Alternatively, authors should transparently disclose their studies' methodological limitations in measuring psychosocial consequences of screening validly.