OBJECTIVES: We determined the proportion of PIOs in neonatal RCTs included in Cochrane Neonatal reviews.
METHODS: We extracted up to 5 outcomes from each RCT included in Cochrane Neonatal reviews published until January 2018, with independent determination of PIOs among authors followed by a discussion leading to a consensus. We defined PIOs as outcomes that matter to patient care, such as clinical events or physiological or laboratory parameters that are widely used to guide management.
RESULTS: Among 6,832 outcomes extracted from 1,874 RCTs included in 276 reviews, 5,349 (78.3%) were considered PIOs; 461 studies (24.5%) included 5 or more PIOs, 1,278 (68.2%) included 1-4 PIOs, while 135 (7.2%) had no PIO included. PIOs were observed more often among dichotomous than among continuous outcomes (94.9 vs. 61.5%; RR: 1.54; 95% CI: 1.50-1.58), and more among subjective than among objective outcomes (95.9 vs. 76.8%; RR: 1.25; 95% CI: 1.22-1.28). Newer studies were more likely to have a greater number of PIOs (adjusted OR: 1.033 [95% CI: 1.025-1.041] with each publication year).
CONCLUSIONS: The large and increasing representation of PIOs over the years suggests an improving awareness by neonatal trialists of the need to incorporate important outcomes in order to justify the utilization of resources. Further research should explore the reasons for non-inclusion or non-reporting of PIOs in a small proportion of RCTs.
DESIGN: Systematic review and regression analysis.
ELIGIBILITY: Medication adherence levels studied at primary, secondary and tertiary care settings. Self-reported measures with scoring methods were included. Studies without proxy measures were excluded.
DATA SOURCES: Using detailed searches with key concepts including questionnaires, reliability and validity, and restricted to English, MEDLINE, EMBASE, CINAHL, International Pharmaceutical Abstracts, and Cochrane Library were searched until 01 March 2022. Preferred Reporting Items for Systematic Reviews and Meta-Analyses 2020 (PRISMA-2020) checklist was used.
DATA ANALYSIS: Risk of bias was assessed via COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN-2018) guidelines. Narrative synthesis aided by graphical figures and statistical analyses.
OUTCOME MEASURES: Process domains [behaviour (e.g., self-efficacy), barrier (e.g., impaired dexterity) or belief (e.g., perception)], and overall outcome domains of either intentional (I), unintentional (UI), or mixed non-adherence.
RESULTS: Paper summarises evidence from 59 studies of PROMs, validated among patients aged 18-88 years in America, the United Kingdom, Europe, Middle East, and Australasia. PROMs detected outcome domains: intentional non-adherence, n=44 (I=491 criterion items), mixed intentionality, n=13 (I=79/UI=50), and unintentional, n=2 (UI=5). Process domains detected include belief (383 criterion items), barrier (192) and behaviour (165). Criterion validity assessment used proxy measures (biomarkers, e-monitors), and scoring was ordinal, dichotomised, or used Visual Analogue Scale. Heterogeneity was revealed across psychometric properties (consistency, construct, reliability, discrimination ability). Intentionality correlated positively with negative beliefs (r(57)=0.88) and barriers (r(57)=0.59). For every belief or barrier criterion-item, PROMs' aptitude to detect intentional non-adherence increased by β=0.79 and β=0.34 units, respectively (R2=0.94). Primary care versus specialised care predicted intentional non-adherence (OR 1.9; CI 1.01 to 2.66).
CONCLUSIONS: Ten PROMs had adequate psychometric properties. Of the ten, eight PROMs were able to detect total, and two PROMs were able to detect partial intentionality to medication default. Fortification of patients' knowledge and illness perception, as opposed to daily reminders alone, is most imperative at primary care levels.
METHODS: Narrative review of the literature, identifying and describing outcome measures that may be used in the evaluation of CRSwNP and for assessment of treatment responses.
RESULTS: In this review, we identify many different outcome measures for CRSwNP that fall under the categories of PROM, objective test, psychophysical test or biomarker. We describe the history of each - including seminal studies - and demonstrate the formal validation, psychometric performance, and limitations of each.
CONCLUSIONS: PROMs, objective tests, psychophysical tests and biomarkers represent different classes of outcome measures that are complementary means of assessing CRSwNP disease status and treatment efficacy. The choice or interpretation of a CRSwNP outcome measure should be undertaken with full knowledge of its formal validation, psychometric performance, and limitations.
METHODS: Adult patients (≥18 years) tested positive for COVID-19 via reverse transcription-polymerase chain reaction (RT-PCR) and admitted in Hospital Tuanku Ja'afar Seremban, Malaysia, were recruited in this study. Patients completed a questionnaire via telephone interview comprising the following details: age, sex, ethnicity, comorbidities, general and otorhinolaryngological symptoms, onset and duration of olfactory and gustatory dysfunction. Patients with persistent olfactory and gustatory dysfunction at the time of the initial interview were followed-up every 3 to 5 days till resolution.
RESULTS: A total of 145 patients were included in our study. The mean age of patients was 43.0 ± 17.7 (range: 18-86). Fever (44.1%) and cough (39.3%) were the most prevalent general symptoms. Thirty-one patients (21.4%) reported olfactory dysfunction and 34 (23.4%) reported dysgeusia. There was a significant association between both olfactory and gustatory dysfunction (P patients (13.6%) reported isolated sudden-onset anosmia. The median duration of olfactory and gustatory dysfunctions was 7 days. Complete recovery was achieved for 70.5% of the patients within 7 days of symptom onset. Only 6 (19.4%) of the 31 patients with olfactory dysfunction experienced nasal obstruction or rhinorrhea. Olfactory dysfunction was not significantly associated with nasal obstruction or rhinorrhea. Olfactory dysfunction was significantly associated with younger age (P = .002), female (P = .011), and hyperlipidemia (P = .012). Gustatory dysfunction was significantly associated with fever (P = .019) and cough (P = .039).
CONCLUSION: Olfactory and gustatory dysfunction is a pertinent manifestation of COVID-19. Most of the affected patients achieve rapid and complete recovery. Sudden onset of olfactory and gustatory dysfunction should be recognized as a major symptom of COVID-19 as we implore to contain this pandemic.
Objective: This study assessed feasibility of using quality improvement (QI) tools to improve management of perioperative pain in hospitals in multiple developing countries.
Methods: The International Pain Registry and Developing Countries working groups, from the International Association for the Study of Pain (IASP), sponsored the project and PAIN OUT, a QI and research network, coordinated it, and provided the research tools. The IASP published a call about the project on its website. Principal investigators (PIs) were responsible for implementing a preintervention and postintervention study in 1 to 2 surgical wards in their hospitals, and they were free to choose the QI intervention. Trained surveyors used standardized and validated web-based tools for collecting findings about perioperative pain management and patient reported outcomes (PROs). Four processes and PROs, independent of surgery type, assessed effectiveness of the interventions.
Results: Forty-three providers responded to the call; 13 applications were selected; and PIs from 8 hospitals, in 14 wards, in 7 countries, completed the study. Interventions focused on teaching providers about pain management. Processes improved in 35% and PROs in 37.5% of wards.
Conclusions: The project proved useful on multiple levels. It offered PIs a framework and tools to perform QI work and findings to present to colleagues and administration. Management practices and PROs improved on some wards. Interpretation of change proved complex, site-dependent, and related to multiple factors. PAIN OUT gained experience coordinating a multicentre, international QI project. The IASP promoted research, education, and QI work.
PATIENTS AND METHODS: Materials and methods: The study included 165 patients admitted with STEMI within 12 hours of the onset of symptoms be¬tween January 2020 and August 2021. All patients underwent primary PCI according to the guidelines, followed by standard examination and treatment at the hospital. Blood samples for biomarker analysis (MMP-9, cTnI) and other routine tests were taken on admission. At six months after the event, all patients underwent clinical follow-up. Patients were contacted either by phone, through family members or their physicians 1 year after the event.
RESULTS: Results: The composite endpoint reached 9% of patients at one-year follow-up. ROC analysis of MMP-9 with the one-year com¬posite endpoint showed an AUC=0.711, with 91.7% sensitivity, and 47.4% specificity, 95% CI - 0.604 to 0.802, p=0.0037. ROC analysis of EQ-5D questionnaire with the one-year composite endpoint showed AUC = 0.73, the 95% CI - 0.624 to 0.820, p< 0.0195, with sensitivity 54.5% and specificity 94.7%. A logistic regression model showed a statistical association with the com¬posite endpoint at one year after STEMI in both EQ-5D (OR=0.89, 95% CI: 0.8313- 0.9725, p=0.0079) and MMP-9 (OR=1.0151, 95% CI:1.0001-1.0304, p=0.0481).
CONCLUSION: Conclusions: The level of MMP-9 more than 194 ng/ml and <55 points in EQ-5D predicts major adverse cardiovascular events, in¬cluding cardiovascular mortality and progressive heart failure, as well as other elements of composite endpoints, during a 1-year follow-up in patients with STEMI after primary PCI. Future studies are needed to clarify this result.
OBJECTIVE: To establish consensus on a core set of clinician- and patient-reported outcome measures recommended for use in clinical practice and to establish the appropriate interval within which these measures should be applied.
EVIDENCE REVIEW: Clinician- and patient-reported HS measures and studies describing their psychometric properties were identified through literature reviews. Identified measures comprised an item reduction survey and subsequent electronic Delphi (e-Delphi) consensus rounds. In each consensus round, a summary of outcome measure components and scoring methods was provided to participants. Experts were provided with feasibility characteristics of clinician measures to aid selection. Consensus was achieved if at least 67% of respondents agreed with use of a measure in clinical practice.
FINDINGS: Among HS experts, response rates for item reduction, e-Delphi round 1, and e-Delphi round 2 surveys were 76.4% (42 of 55), 90.5% (38 of 42), and 92.9% (39 of 42), respectively; among patient research partners (PRPs), response rates were 70.8% (17 of 24), 100% (17 of 17), and 82.4% (14 of 17), respectively. The majority of experts across rounds were practicing dermatologists with 18 to 19 years of clinical experience. In the final e-Delphi round, most PRPs were female (12 [85.7%] vs 2 males [11.8%]) and aged 30 to 49 years. In the final e-Delphi round, HS experts and PRPs agreed with the use of the HS Investigator Global Assessment (28 [71.8%]) and HS Quality of Life score (13 [92.9%]), respectively. The most expert-preferred assessment interval in which to apply these measures was 3 months (27 [69.2%]).
CONCLUSIONS AND RELEVANCE: An international group of HS experts and PRPs achieved consensus on a core set of HS measures suitable for use in clinical practice. Consistent use of these measures may lead to more accurate assessments of HS disease activity and life outcomes, facilitating shared treatment decision-making in the practice setting.
METHODS: Patients were randomly assigned 2:1 to pembrolizumab 200 mg or placebo every 3 weeks for up to 35 cycles plus investigator's choice chemotherapy (nab-paclitaxel, paclitaxel, or gemcitabine plus carboplatin). The European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (QLQ-C30), Breast Cancer-Specific Quality of Life Questionnaire, and EuroQol 5-Dimension questionnaire visual analog scale were prespecified. Patient-reported outcomes were analyzed for patients who received at least 1 dose of study treatment and completed at least 1 patient-reported outcome assessment. Changes in patient-reported outcome scores from baseline were assessed at week 15 (latest time point at which completion and compliance rates were at least 60% and at least 80%, respectively). Time to deterioration in patient-reported outcomes was defined as time to first onset of at least a 10-point worsening in score from baseline.
RESULTS: Patient-reported outcome analyses included 317 patients with tumor PD-L1 combined positive score of at least 10 (pembrolizumab plus chemotherapy: n = 217; placebo plus chemotherapy: n = 100). There were no between-group differences in change from baseline to week 15 in QLQ-C30 global health status/quality of life (QOL; least-squares mean difference = -1.81, 95% confidence interval [CI] = -6.92 to 3.30), emotional functioning (least-squares mean difference = -1.43, 95% CI = -7.03 to 4.16), physical functioning (least-squares mean difference = -1.05, 95% CI = -6.59 to 4.50), or EuroQol 5-Dimension questionnaire visual analog scale (least-squares mean difference = 0.18, 95% CI = -5.04 to 5.39) and no between-group difference in time to deterioration in QLQ-C30 global health status/QOL, emotional functioning, or physical functioning.
CONCLUSIONS: Together with the efficacy and safety findings, patient-reported outcome results from KEYNOTE-355 support pembrolizumab plus chemotherapy as a standard of care for patients with advanced triple-negative breast cancer with tumor PD-L1 expression (combined positive score ≥10).
OBJECTIVES: This study aimed to (i) assess the reliability and validity of the Arabic version of HIT-6 in Arabic-speaking patients experiencing migraine, and (ii) evaluate the responsiveness of HIT-6 following migraine preventive therapy.
METHODS: In this prospective study, patients with migraine (n = 145) were requested to fill out a headache diary, the Arabic version of HIT-6, and Migraine Disability Assessment Scale (MIDAS) at two time points (baseline and 3 months after initiation of prophylactic treatment). Some respondents (n = 73) were requested to fill out HIT-6 again 1 week from the baseline for test-retest reliability. The intensity of migraine headache attacks was evaluated using the Visual Analogue Scale (VAS). An anchor-based method was used to establish the minimal important change (MIC) value and responsiveness of HIT-6.
RESULTS: The total scores of HIT-6 were significantly correlated to a fair degree with MIDAS (r = 0.41), as well as VAS (r = 0.53), and monthly migraine days (r = 0.38) at the baseline while at the follow-up (after 3 months), the correlations were of moderate degree with MIDAS scores (r = 0.62) and monthly migraine days (r = 0.60; convergent validity). Reliability estimates of the Arabic HIT-6 were excellent (Cronbach's α = 0.91 at baseline and 0.89 at follow-up). The average measure interclass correlation coefficient (ICC) value for the test-retest reliability was 0.96 (95% confidence interval = 0.94-0.98, p
PATIENTS AND METHODS: Patients were part of a prospective multicentre observational study recruiting people with bladder cancer for a urine biomarker study (DETECT II; ClinicalTrials.gov: NCT02781428). A mixed-methods approach comprising (1) a questionnaire to assess patients' experience with cystoscopy and patients' preference for cystoscopy vs urinary biomarker, and (2) semi-structured interviews to understand patient views, choice and reasons for their preference.
RESULTS: A urine biomarker with an MAS of 90% would be accepted by 75.8% of patients. This was despite a high self-reported prevalence of haematuria (51.0%), dysuria/lower urinary tract symptoms (69.1%) and urinary tract infection requiring antibiotics (25.8%). There was no association between MAS with patient demographics, adverse events experienced, cancer characteristics or distance of patients' home to hospital. The qualitative analysis suggested that patients acknowledge that cystoscopy is invasive, embarrassing and associated with adverse events but are willing to tolerate the procedure because of its high sensitivity. Patients have confidence in cystoscopy and appreciate the visual diagnosis of cancer. Both low- and high-risk patients would consider a biomarker with a reported sensitivity similar to that of cystoscopy.
CONCLUSION: Patients value the high sensitivity of cystoscopy despite the reported discomfort and adverse events experienced after it. The sensitivity of a urinary biomarker must be close to cystoscopy to gain patients' acceptance.
METHODS: Clinical, laboratory, and PRO data (Chronic Liver Disease Questionnaire-nonalcoholic steatohepatitis [NASH], Functional Assessment of Chronic Illness Therapy-Fatigue, and the Work Productivity and Activity Index) were collected from NAFLD patients seen in real-world practices and enrolled in the Global NAFLD/NASH Registry encompassing 18 countries in 6 global burden of disease super-regions.
RESULTS: Across the global burden of disease super-regions, NAFLD patients (n = 5691) were oldest in Latin America and Eastern Europe and youngest in South Asia. Most men were enrolled at the Southeast and South Asia sites. Latin America and South Asia had the highest employment rates (>60%). Rates of cirrhosis varied (12%-21%), and were highest in North Africa/Middle East and Eastern Europe. Rates of metabolic syndrome components varied: 20% to 25% in South Asia and 60% to 80% in Eastern Europe. Chronic Liver Disease Questionnaire-NASH and Functional Assessment of Chronic Illness Therapy-Fatigue PRO scores were lower in NAFLD patients than general population norms (all P < .001). Across the super-regions, the lowest PRO scores were seen in Eastern Europe and North Africa/Middle East. In multivariate analysis adjusted for enrollment region, independent predictors of lower PRO scores included younger age, women, and nonhepatic comorbidities including fatigue (P < .01). Patients whose fatigue scores improved over time experienced a substantial PRO improvement. Nearly 8% of Global NAFLD/NASH Registry patients had a lean body mass index, with fewer metabolic syndrome components, fewer comorbidities, less cirrhosis, and significantly better PRO scores (P < .01).
CONCLUSIONS: NAFLD patients seen in real-world practices in different countries experience a high comorbidity burden and impaired quality of life. Future research using global data will enable more precise management and treatment strategies for these patients.
METHODS: The study encompassed a comprehensive three-stage approach to the development and validation of the PROM. Initially, during the preliminary design stage, the necessity for a new PROM was recognized, an expert panel was formed, and semi-structured qualitative interviews were carried out with GSM patients. In the second stage, the study used the five-step pre-validation methodology established by Prior et al. to generate and refine the PROM items. The third and final stage encompassed the determination of scale and item content validity indexes to ensure validity. Additionally, the reliability of each construct was evaluated using Cronbach's α.
RESULTS: The resulting PROM was named GSM-SVTAQ (GSM-symptoms and vaginal treatments acceptability questionnaire). It demonstrated excellent validity in assessing symptoms burden, health-related and sexual quality of life, and vaginal treatment acceptability, with high content validity indices and strong internal consistency. The scale content validity indices and Cronbach's α coefficients for the three domains were (0.926, 0.939), (0.875, 0.947), and (0.824, 0.855), respectively.
CONCLUSION: The GSM-SVTAQ stands as the first GSM-specific, valid, and reliable PROM capable of comprehensively measuring the three components of GSM and the acceptability of vaginal treatments. Its implementation has the potential to significantly enhance patient care and outcomes in GSM management.
MATERIAL AND METHODS: Thirty-four patients (mean age 60.70 ± 8.7 years) received telescopic crown or locator attachments for ISOD and completed OHIP-14 (Malaysian version) and DS questionnaires, at baseline (T0 ) with new conventional complete dentures (CCD) and 3 months (T1 ) and 3 years (T2 ) after ISOD conversion. Mandibular bone volume was calculated from cone beam computed tomography (CBCT) datasets using Mimics software. Mean changes (MC) in OHIP-14 and DS at intervals were analyzed using the Wilcoxon signed-rank test and effect size (ES). The association of bone volume, implant attachment type, and other patient variables with the change in OHIP-14 and DS were determined using multivariate linear regression analysis.
RESULTS: The MC in OHIP-14 and DS scores from T0 to T1 and T2 showed significant improvement with moderate and large ES, respectively. Regression analyses for the change in OHIP-14 score from T0 to T2 showed significant association with implant attachment type (P = 0.043), bone volume (P = 0.004), and baseline OHIP-14 (P = 0.001), while for DS, the association was only significant with baseline DS score (P = 0.001).
CONCLUSION: Improvement in patients' OHRQoL and satisfaction with ISOD was associated with their baseline ratings. Mandibular bone volume had a stronger association for improvement in OHRQoL compared to type of attachment.