Engaging family members in patient care in the intensive care unit (ICU) is a recommended practice by critical care societies. However, there are currently no validated tools to measure family engagement in the ICU setting. The objective of this study was to validate the FAMily Engagement (FAME) tool in the ICU.

The FAME study was a multicenter prospective cohort study of family members of ICU patients in 8 Canadian ICUs. Family members completed the FAME questionnaire during the ICU stay. The FAME questionnaire comprised 12 items that assessed various domains of family engagement behavior. FAME scores were reported in a 0-100 scoring system with higher scores indicating increased care engagement. Following hospital discharge, we assessed associations between the FAME score and family satisfaction with care and mental health (anxiety and depression). The internal consistency (reliability), convergent validity, and predictive validity of the FAME tool were evaluated.

There were 269 family members (age 56.8 ± 15.1; 68.4 % women) included in the analysis. The most common relationships to the patient were spouse/partner (40.5 %) and daughter/son (33.8 %). The overall mean FAME score was 77.7 ± 14.8. The FAME score had high internal consistency (Cronbach's α = 0.83) and the tool demonstrated convergent and predictive validity. The FAME score was associated with family satisfaction, but not with mental health outcomes.

The FAME tool demonstrated reliability, convergent, and predictive validity in this multicenter ICU cohort. The FAME tool could be used to evaluate the effectiveness of family engagement interventions.

Mixed urinary incontinence, which includes both stress and urgency urinary incontinence, adversely affects quality of life and can be difficult to manage. Studies comparing procedural-based treatments for mixed urinary incontinence are lacking.

To determine whether intradetrusor onabotulinumtoxinA is more effective than midurethral sling for the treatment of mixed urinary incontinence in females.

Randomized, superiority trial involving females (aged ≥21 years) with moderate to severe bother from both stress and urgency urinary incontinence who had unsuccessful conservative treatments and oral medications. The study was conducted at 7 US sites with enrollment between July 2020 and September 2022; the last date of follow-up was December 29, 2023.

Intradetrusor injection of onabotulinumtoxinA, 100 U (treatment focused on the urgency component), vs surgical synthetic mesh midurethral sling (treatment focused on the stress component). Recipients of onabotulinumtoxinA could receive an additional injection between 3 and 6 months. All participants could receive additional treatment (including crossover to the alternative treatment) between 6 and 12 months.

The primary outcome was change at 6 months in mixed incontinence symptoms as measured by the Urogenital Distress Inventory (UDI) total score (0-300 points; higher scores indicate worse symptoms; minimal clinically important difference, 26.1). Secondary outcomes included stress and irritative UDI subscores.

Among 150 females randomized, 137 were treated, had postbaseline outcome data, and were included in the primary analysis (mean [SD] age, 59.0 [11.5] years). Both groups demonstrated mean improvement in UDI total score at 6 months with no significant difference between groups (onabotulinumtoxinA: -66.8 points [95% CI, -84.9 to -48.8]; sling: -84.9 [95% CI, -100.5 to -69.3]; mean difference, 18.1 points [95% CI, -4.6 to 40.7]; P = .12). For secondary outcomes, greater UDI stress score improvement was seen with the sling (-45.2 [95% CI, -53.7 to -36.8]) compared with onabotulinumtoxinA (-25.1 [95% CI, -34.1 to -16.1]) (P < .001); however, no significant difference was seen between groups in UDI irritative score (onabotulinumtoxinA: -32.9 [95% CI, -40.3 to -25.6] vs sling: -27.4 [95% CI, -34.6 to -20.3]; P = .27). In the onabotulinumtoxinA group, 12.7% and 28.2% received a second injection by 6 and 12 months, respectively. By 12 months, 30.3% in the sling group received onabotulinumtoxinA, and 15.5% in the onabotulinumtoxinA group received a sling. Overall, adverse events were not different between groups.

There was no observed difference in UDI total score improvement at 6 months between the onabotulinumtoxinA and midurethral sling groups in females with moderate to severe mixed urinary incontinence who previously did not respond to conservative treatments. These findings may help inform treatment decisions based on patient preference in partnership with clinician recommendations.

ClinicalTrials.gov Identifier: NCT04171531.

Persons with inflammatory bowel disease (IBD) have an increased risk of suffering from psychological problems. The association is assumed to be bi-directional. Psychological treatment is expected to improve quality of life (QoL), psychological issues and, possibly, disease activity. Many trials have tested various psychotherapy approaches, often in combination with educational modules or relaxation techniques, with inconsistent results.

To assess the effects of psychological interventions on quality of life, emotional state and disease activity in persons of any age with IBD.

We searched Web of Science Core Collection, KCI-Korean Journal Database, Russian Science Citation Index, MEDLINE, Psyndex, PsycINFO, Embase, Cochrane Central Register of Controlled Trials, and LILACS from inception to May 2023. We also searched trial registries and major gastroenterological and selected other IBD-related conferences from 2019 until 2023.

Randomized controlled trials of psychological interventions in children or adults with IBD compared to no therapy, sham (i.e. simulated intervention), or other active treatment, with a minimum follow-up time of two months, were eligible for inclusion, irrespective of publication status and language of publication. Interventions included psychotherapy and other non-pharmacological interventions addressing cognitive or emotional processing, patient education, or relaxation techniques to improve individual health status.

Two raters independently extracted data and assessed the study quality using the Risk of Bias 2 Tool. Pooled standardized mean differences (SMD) for continuous outcomes and relative risks (RR) for event data were calculated with 95% confidence intervals (CI), based on separate random-effects models by age group, type of therapy and type of control. An SMD of 0.2 was considered a minimally relevant difference. SMD ≥ 0.4 was considered a moderate effect. Group analyses were planned to examine differential effects by type of IBD, disease activity, psychological comorbidity, therapy subtype, and treatment intensity. Statistical heterogeneity was determined by calculating the I2 statistic. Publication bias was assessed by presenting a funnel plot and calculating the Eggers Test. GRADE Profiling was used to describe the certainty of the evidence for relevant results.

Sixty-eight studies were eligible. Of these, 48 had results reported in sufficient detail for inclusion in the meta-analyses (6111 adults, 294 children and adolescents). Two trials were excluded from the meta-analysis following sensitivity analysis and tests for asymmetry because of implausible results. Most studies used multimodular approaches. The risk of bias was moderate for most outcomes, and high for some. The most common problems in individual trials were the inability to blind participants and investigators and outcome measures susceptible to measurement bias. The main issues leading to downgrading of the certainty of the evidence were heterogeneity of results, low precision and high or moderate risk of bias in the included trials. Publication bias could not be shown for any of the inspected analyses. In adults, psychotherapy was slightly more effective than care-as-usual (CAU) in improving short-term QoL (SMD 0.23, 95% CI 0.12 to 0.34; I2 = 13%; 20 trials, 1572 participants; moderate-certainty), depression (SMD -0.27, 95% CI -0.39 to -0.16; I2 = 0%; 16 trials, 1232 participants; moderate-certainty), and anxiety (SMD -0.29, 95% CI -0.40 to -0.17; I2 = 1%; 15 studies, 1135 participants; moderate-certainty). The results for disease activity were not pooled due to high heterogeneity (I2 = 72%). Interventions which used patient education may also have small positive short-term effects on QoL (SMD 0.19, 95% CI 0.06 to 0.32; I2 = 11%; 12 trials, 1058 participants; moderate-certainty), depression (SMD -0.22, 95% CI -0.37 to -0.07; I2 = 11%; 7 studies, 765 participants; moderate-certainty) and anxiety (SMD -0.16, 95% CI -0.32 to 0.00; I2 = 10%; 6 studies, 668 participants; moderate-certainty). We did not find an effect of education on disease activity (SMD -0.09, 95% CI -0.28 to 0.10; I2 = 38%; 7 studies, 755 participants; low-certainty). Pooled results on the effects of relaxation techniques showed small effects on QoL (SMD 0.25, 95% CI 0.08 to 0.41; I2 = 30%; 12 studies, 916 participants; moderate-certainty), depression (SMD -0.18, 95% CI -0.35 to -0.02; I2 = 0%; 7 studies, 576 participants; moderate-certainty), and anxiety (SMD -0.26, 95% CI -0.43 to -0.09; I2 = 13%; 8 studies, 627 participants; moderate-certainty). Results for disease activity were not pooled due to high heterogeneity (I2 = 72%). In children and adolescents, multimodular psychotherapy increased quality of life (SMD 0.54, 95% CI 0.06 to 1.02; I2 = 19%; 3 studies, 91 participants; moderate-certainty). The results for anxiety were inconclusive (SMD -0.09; 95% CI 0.-64 to 0.46; 2 trials, 51 patients, very low-certainty). Pooled effects were not calculated for depressive symptoms. Disease activity was not assessed in any of the trials compared to CAU. In education, based on one study, there might be a positive effect of the intervention on quality of life (MD 7.1, 95% CI 2.18 to 12.02; 40 patients; low-certainty evidence) but possibly not on depression (MD -6, 95% CI -12.01 to 0.01; 41 patients; very low-certainty). Anxiety and disease activity were not assessed for this comparison. Regarding the effects of relaxation techniques on children and adolescents, all results were inconclusive (very low-certainty).

Psychological interventions in adults are likely to improve the quality of life, depression and anxiety slightly. Psychotherapy is probably also effective for improving the quality of life in children and adolescents. The evidence suggests that psychological interventions may have little to no effect on disease activity. The interpretation of these results presents a challenge due to the clinical heterogeneity of the included trials, particularly concerning the type and various components of the common multimodular interventions. This complexity underscores the need for further research and exploration in this area.

Minimum important changes (MICs) represent thresholds for clinically meaningful change. Multi-attribute utility instruments (MAUIs) generate health state utilities (holistic measures of health-related quality of life). No systematic review of MICs specifically for MAUIs has been conducted. In addition, no guidelines for estimating MICs for MAUIs have been proposed. We aimed to correct these evidence gaps by producing guidelines contextualised by a systematic review.

We searched ten databases for relevant records using various search terms. Extracted data were analysed narratively and descriptively. The presence of key reporting items (relating to precision, sensitivity, and concurrent validity) was also evaluated. Guidelines for MIC estimation were informed by the broader MIC literature and contextualised using study results.

The review identified 5035 non-duplicate records, with 68 entering the study. 282 unique, anchor-based MICs were extracted. Of these MICs, 119 (42.20%) pertained to the EQ-5D-3L, 82 (29.08%) to the EQ-5D-5L, and 50 (17.73%) to the SF-6D.v1. The most common anchor-based method used to estimate MICs (107, 37.94%) involved taking the mean change score for a group considered to have experienced a MIC. Distribution-based methods were also common, appearing in 31 (45.59%) of the included studies. The inclusion of key reporting items was generally deficient.

Deficiencies in reporting and diverse estimation methods raise concerns regarding the extant MAUI MIC literature. Researchers should exercise caution when using existing MAUI MICs. Recommendations presented in our study may assist researchers in effectively estimating MICs for use in health economics.

To evaluate outcomes of patients who underwent arthroscopic repair for partial-thickness rotator cuff tears (PTRCTs) with a minimum 10-year follow-up.

Patients who underwent arthroscopic surgery for PTRCTs between 2006 and 2014 were retrospectively analyzed. Articular-sided tears were repaired by converting to full-thickness, bursal-sided tears were repaired on the tear side, and intratendinous tears were repaired by converting to bursal-sided tears. Assessment included American Shoulder and Elbow Surgeons (ASES) score, Subjective Shoulder Value (SSV), and visual analog scale (VAS). The proportion of patients who met the minimal clinically important difference (MCID), substantial clinical benefit, and patient acceptable symptom state thresholds for ASES, SSV, and VAS was determined. Patients were classified into 3 subgroups on the basis of tear type, and demographics and patient-reported outcome measures (PROMs) were compared.

Among 89 eligible patients, 72 with complete data were included. The mean age was 51.1 ± 10.8 years, and the mean follow-up period was 12 ± 2.2 years (10-19 years). Patients had significant improvements in ASES (28.6 to 87.2), SSV (35.6 to 90.3), and VAS (8.2 to 1.5) (P < .001 for all PROMs). The rates of patients achieving MCIDanchor, MCIDdistribution, patient acceptable symptom state, and substantial clinical benefit were determined, respectively, ASES (89%, 98%, 92%, 90%), SSV (92%, 92%, 92%, 89%), and VAS (92%, 94%, 88%, 88%). Subgroup (bursal-sided [53%], intratendinous [19%], articular-sided [28%]) analysis revealed significant improvements in all PROMs (for bursal- and articular-sided tears, P < .001 for all PROMs; for intratendinous tears, PASES= .001, PSSV= 0.002, and PVAS= .001).

At a minimum 10-year follow-up, arthroscopic double-row repair for articular (converted to full thickness and repaired), bursal (tear-side repair), and intratendinous (converted to bursal-side and repaired accordingly) PTRCTs provides significant improvements in PROMs and when considered as a single PTRCT cohort, these patients achieve satisfactory clinically meaningful outcomes.

Level IV, retrospective case series.

Increasing reliance on patient-reported outcome measures (PROMs) following reverse total shoulder arthroplasty (rTSA) has resulted in variability in the thresholds for minimum clinically important difference (MCID) and substantial clinical benefit (SCB) reported in the literature. In this study, we aimed to identify the best threshold values for MCID and SCB for common rTSA PROMs.

The PubMed, Embase, MEDLINE, Cochrane Library, and Google Scholar databases were queried for studies published from January 1, 2000 to March 1, 2024 that identified clinically significant thresholds for PROMs following rTSA. The threshold calculation method, anchor questions/response options, follow-up duration, and confidence intervals were extracted. A novel hierarchical methodology evaluated various threshold calculation characteristics to identify the best MCID and SCB values following rTSA.

We included 37 studies for analysis comprising 11,960 patients. Using our methodology, the MCID values from the literature we recommend for American Shoulder and Elbow Surgeons (ASES), Simple Shoulder Test (SST), Constant-Murley Shoulder Outcome Score (Constant), University of California at Los Angeles Shoulder Score (UCLA), Shoulder Pain and Disability Index (SPADI) score, and Visual Analog Scale (VAS) following rTSA are 8.9, 3.7, 9.3, 2.9, 20, and 1.4, respectively. The recommended MCID threshold values for ASES and SST were from Werner and McLaughlin, respectively. The recommended thresholds for Constant and UCLA were from Zhou, while the thresholds for SPADI and VAS were from Simovitch. Using our methodology, the SCB threshold values from the literature we recommend for ASES following rTSA are 28.3 for glenohumeral osteoarthritis and 23.1 for rotator cuff arthropathy from Puzzitiello. For SST, Constant, UCLA, SPADI, and VAS, the SCB values we recommend are 3.2, 13.6, 10.4, 42.7, and 2.6, respectively. The recommended SCB thresholds for SST, Constant, UCLA, SPADI, and VAS were all from Simovitch.

We present recommended MCID and SCB values identified through our comprehensive methodology and recommend how these thresholds should be calculated for common rTSA PROMs moving forward. With increasing use and dependence on MCID and SCB values for PROMs, these recommended threshold values for MCID and SCB will help standardize assessment of clinically significant improvement for patients undergoing rTSA.

This review evaluates the effectiveness of nutritional supplementation combined with exercise training on frailty characteristics, physical function, and health-related quality of life in patients with chronic kidney disease (CKD). A systematic search of PubMed, Embase, Web of Science, and Scopus was conducted, identifying 7 articles (9 trials, 324 patients). Meta-analysis showed that combined interventions improved frailty characteristics, such as walking speed (mean difference: 0.09 m/s, 95% confidence interval (CI): 0.02 to 0.16) and physical functioning, including cardiorespiratory fitness (standardized mean difference: 0.56, 95% CI: 0.20 to 0.93) and lower extremity mobility (Timed Up and Go test: -1.11 s, 95% CI: -1.79 to -0.43). However, effects on body weight, fatigue, and health-related quality of life remain uncertain. Due to study heterogeneity and small sample sizes, findings should be interpreted cautiously. Larger, long-term studies are needed to confirm these results and explore additional health outcomes.

Calculating the threshold for meaningful within-individual change (MWIC) is essential for interpreting patient-reported outcomes (PRO). However, traditional methods of determining MWIC threshold yield varying estimates and lack a standardized approach. We aim to propose a novel method for more accurate MWIC threshold estimation.

We developed a weighted predictive modeling method. The weighting involved using the rank difference between PRO score change and the anchor of each individual. A Monte Carlo simulation was conducted to compare the performance of the new method and that of existing state-of-the-art methods. Simulation parameters included distributions of PRO score changes, sample sizes, improvement proportions, and correlation strengths. Statistical performance was assessed using relative bias (rbias), coefficient of variation (CV), and relative root mean squared error (rRMSE).

Distribution-based methods had the largest rbias and rRMSE among all methods. Existing anchor-based methods except for the Terluin 2022 method were biased when the correlation strength was weak or when the improvement proportion was not 50%. The Terluin 2022 method requires estimating an important reliability parameter, and this method had highest CV compared to other predictive modeling methods. The new weighted method demonstrated the smallest rRMSE across most simulation settings. It also maintained relatively high accuracy under weak correlation strength or imbalanced improvement proportion. Similar results were presented under normal or skewed distributions of PRO score changes.

This novel method offers a simple and feasible alternative to existing predictive modeling methods for estimating MWIC threshold, which can facilitate the application of PRO.

To identify distinct morning and evening fatigue profiles in patients with gynecologic cancers and evaluate for differences in demographic and clinical characteristics, common symptoms, and quality-of-life outcomes.

Outpatients with gynecologic cancers (N = 233) were recruited before their second or third cycles of chemotherapy at four cancer centers in San Francisco Bay and New York.

The Lee Fatigue Scale was completed six times over two cycles of chemotherapy in the morning and in the evening. Latent profile analysis was used to identify distinct morning and evening fatigue profiles.

Four distinct morning and two distinct evening fatigue classes were identified. Common risk factors for morning and evening fatigue included younger age, higher body mass index, lower functional status, and higher comorbidity burden. Patients in the worst morning and evening fatigue classes reported higher levels of anxiety, depression, and sleep disturbance; lower levels of energy and cognitive function; and poorer quality of life.

Clinicians can use this information to identify higher-risk patients and develop individualized interventions for morning and evening fatigue.

This study aimed to enhance the interpretability and clinical utility of the strength and stressors in parenting (SSF) questionnaire, a parent-reported questionnaire designed to assess strength, stress and associated risks of mental ill-health in parents of children with developmental disabilities. Responses to the SSF and a demographic questionnaire were collected from 576 parents of children with (n = 203) and without (n = 373) developmental disabilities. To enhance the interpretability of the SSF, a subset of 129 parents were invited to complete an additional questionnaire consisting of three free-text questions regarding recent help-seeking behavior, experiences of mental ill-health and experiences of parenthood. Parents' responses to the free-text questions were then categorized as indicative of higher or lower degrees of stress and compared to their SSF score distribution to derive empirical cut-offs for strength, stress and risk of mental ill-health as measured by the SSF. The credibility of these cut-offs was evaluated by comparing the cut-offs with SSF scores collected from the other 447 parents. Finally, SSF scores from parents of children without developmental disabilities (n = 373) were used to generate percentile values for the SSF to enable a standardized interpretation of SSF scores. To increase the utility of the SSF, we examined a recurring pattern of missing answers to items 23 and 33-38, noted in previous studies of the SSF and repeated in the present study. These items were excluded from further analysis since our examination revealed that they were not missing at random but rather constituted real differences in parental experiences, such as receiving a healthcare allowance, or caring for more than one child. The proposed empirical cut-offs performed well in discriminating between the two groups and yielded a specificity of 77-89% and a sensitivity of 68-76% for the strength, stress and risk of mental ill-health subscales of the SSF. This study also presents a conversion chart associating each SSF score with a corresponding percentile value. We propose modifications to the SSF, whereby items 23 and 33-38 are excluded, which will enable a more reliable assessment of parental experiences. This will, together with the empirical cut-offs and percentile values, enhance the interpretability and clinical utility of the SSF.

The Singing Voice Handicap Index-10 (SVHI-10) is a validated patient-reported outcome measure (PROM) that assesses patients' perception of handicap related to singing voice. A normative value has been established with a score ≥20 being abnormal. However, there is no defined minimal clinically important difference (MCID). This study prospectively determines the MCID of SVHI-10 among a diverse group of singers.

103 adult singers with and without voice complaints completed SVHI-10 twice, 30 days apart. MCID for the SVHI-10 was determined using distribution-based receiver-operating characteristic (ROC) curve analysis.

Twenty-two men (1 transgender), 75 women (1 transgender), and 6 nonbinary individuals participated. The most frequently reported singing genres were classical (44.7%), musical theater (17.5%), and pop (10.7%). Mean initial SVHI-10 score was 13.05 (standard deviation 7.397), and mean follow-up SVHI-10 was 13.13 (7.994). There was a significant positive correlation between initial and follow-up SVHI-10 scores (r = 0.879, p < 0.001). SVHI-10 scores were significantly higher among participants who reported voice changes in the past year (p < 0.001) or sought voice treatment (p = 0.001) compared with participants who did not. SVHI-10 scores varied significantly based on singing type. The area under the ROC curve for SVHI-10 was 0.700 (p = 0.003). The SVHI-10 MCID was determined to be 9.5.

An SVHI-10 score change ≥10 should be considered clinically meaningful. This definition has been missing from the literature and will improve understanding of patients' responses to treatment, which will help advance clinical care and track research outcomes.

4 Laryngoscope, 135:752-757, 2025.

Ablative fractional carbon dioxide laser (AFCO2L) is widely used as a treatment for hypertrophic burn scars. This study aimed to evaluate clinician and patient-reported outcomes after AFCO2L treatment, safety, and identify factors influencing outcomes.

This longitudinal study recruited adult patients with hypertrophic burn scars treated with AFCO2L at a single Australian burn unit. Patients received up to four AFCO2L treatments over approximately 12 months. Outcomes were ultrasound scar thickness, the Patient and Observer Scar Assessment Scale (POSAS), and the Brisbane Burn Scar Impact Scale (BBSIP), measured at baseline and 3, 6, and 12 months after the first AFCO2L treatment. Analysis used mixed effects linear models.

47 patients were included with median age 32 years (IQR: 24, 53) and median burn TBSA of 35 % (IQR: 7.5 %, 48 %). Statistically significant improvements between baseline and 12-month follow-up occurred in scar thickness, and all POSAS and BBSIP subscores. Most improvements remained when accounting for TBSA, Fitzpatrick skin type, scar maturity, and body area treated. Patients reported transient symptoms after 61 of 89 (69 %) AFCO2L treatments, but infection or delayed wound healing occurred after only 4 of 89 (4 %) treatments.

This study supports safety and improved clinician and patient-reported outcomes in patients undergoing AFCO2L for hypertrophic burn scars.

Immunoglobulin replacement therapy (IgRT) is the current standard of care for primary antibody deficiency patients (majority of all primary immunodeficiency (PID) diseases), with growing real-world evidence supporting use for secondary immunodeficiency (SID) patients. Infusion methods and practices can affect patients' satisfaction with their treatment and perception of their health-related quality of life.

An online survey of US patients with PID and SID was conducted. This research investigates primarily the impact of two IgRT infusion methods, intravenous immunoglobulin therapy (IVIG) and subcutaneous immunoglobulin (SCIG), on the patient reported outcome (PRO) Life Quality Index (LQI) tool. Patient reported infusion time efficiency, physical and mental health (PROMIS GPH-2 and PROMIS GMH-2 respectively), patient acceptability of their symptom state (PASS), upper extremity disability (Quick DASH) and general health perception (via the GHP) are also investigated.

Responses of 990 patients (391 IVIG and 598 SCIG) were analyzed. The median total LQI score amongst SCIG patients (84.7) was higher than IVIG patients (81.9) (p < 0.001), and was significantly higher on 3 out of 4 sub-domains of the LQI. SCIG patients scored higher on items that are related to convenience and reported less interference with everyday life: "Are convenient", "Are scheduled according to my convenience", "Do not interfere with my work/school" and "Require very little time and cost". However, there was no significant difference between the two patient cohorts on other, non-IG specific PROs (PASS, PROMIS GPH-2 and GMH-2 and Quick DASH). Patient reported time per infusion was lower for SCIG infusions than IVIG infusions (pre-infusion time; 22 min vs. 63 min, p < 0.001, infusion time; 120 min vs. 240 min, p < 0.001, post-infusion time; 9 min vs. 31 min, p < 0.001). IVIG patients also reported more interference with everyday life than SCIG patients (82 vs. 86, p < 0.001).

The significantly higher LQI scores for patients receiving SCIG than those receiving IVIG confirms existing evidence that substitution of SCIG for IVIG may favorably impact immunoglobulin specific perceptions of quality of life and treatment satisfaction for appropriately selected patients. Our evidence on infusion times indicates similar improvement may be possible on infusion time efficiency.

Novel clinical measures assessed by a digital health technology tool require thresholds to interpret change over time, such as the minimal clinically important difference. Establishing such thresholds is a key component of clinical validation, facilitating understanding of relevant treatment effects.

Many of the approaches to derive interpretative thresholds for patient-reported outcomes can be applied to digital clinical measures. We present theoretical background to the use of interpretative thresholds, including the distinction between thresholds based on perceived importance versus measurement error, and thresholds for group- versus individual-level interpretations. We then review methods to estimate such thresholds, including anchor-based approaches. We illustrate the methods using data on cough frequency counts as measured by a wearable device in a clinical trial.

This paper provides an overview of statistical methodologies to estimate thresholds for the interpretation of change.

The number of clinical trials in facioscapulohumeral muscular dystrophy (FSHD) is expected to increase in the near future. There is a need for clinical outcome assessments (COAs) that can capture disease progression over the relatively short time span of a clinical trial. In this study, we report the natural progression of FSHD and determine the feasibility of COAs for clinical trials.

Genetically confirmed FSHD patients underwent various COAs at baseline and after 5 years. COAs consisted of the Motor Function Measure (MFM), manual muscle testing using the Medical Research Council score, six-minute walk test, quantitative muscle strength assessment of the quadriceps muscle, clinical severity score, and FSHD evaluation score (FES). Statistical significance and the minimal clinically important difference (MCID) were calculated and power calculations were performed.

One hundred fifty-four symptomatic FSHD patients were included, with a mean (SD) age of 51.4 (14.6) years old. All COAs showed a minimal, yet statistically significant progression after 5 years. MCID was reached for the MFM Domain 1, MFM total score, and FES. These three COAs showed the lowest sample size requirements for clinical trials (185, 156, and 201 participants per group, respectively, for a trial duration of 2 years).

The captured FSHD disease progression rate in 5 years was generally minimal. The COAs in this study are not feasible for clinical trials with a duration of 2 years. Extended trial durations or novel outcome assessments might be necessary to improve trial feasibility in FSHD.

Alzheimer's disease and Alzheimer's disease related dementias (AD/ADRD) have a significant impact on an individual's functional cognitive abilities, highlighting the need to prioritize measures of function in evaluating minimally clinically important difference (MCID) thresholds in AD/ADRD research. Input directly from individuals living with AD/ADRD on measures of function are lacking in MCID discussions, including what it means to live with AD/ADRD and what type and degree of improvements are most meaningful across the disease continuum. Most measures for assessing function in AD/ADRD trials are largely focused on basic and instrumental activities of daily living (BADL, IADL), which lack aspects of everyday function that matter most to individuals living with AD/ADRD. Expanding outcome evaluation to other dimensions of everyday function and diversifying measurement approaches is essential for optimizing inclusion of personally meaningful aspects of everyday function prioritized by individuals living with AD/ADRD and improving detection of potentially more sensitive changes in functioning. This perspective outlines four directions to expand and integrate what matters most to individuals living with AD/ADRD into trial outcome evaluation, including (1) consideration of how what matters most to individuals living with AD/ADRD may change across the disease continuum from mild to advanced dementia, (2) identification and evaluation of goals around strengths-based domains such as social participation rather than solely emphasizing deficits and losses, (3) utilization of goal-attainment scaling to more specifically match individually-specific functional goals, and (4) strengthening the inclusion and use of self-report and performance-based measures of function and triangulating these measures with informant-report measures.

Alzheimer's disease and Alzheimer's disease related dementias (AD/ADRD) have a significant impact on an individual' functional cognitive abilities, of which changes in these abilities are measured through detection of minimally clinically important difference (MCID) thresholds to determine the effectiveness of AD/ADRD clinical trials.Widely used measures for assessing MCID thresholds in AD/ADRD trials focus on basic and instrumental activities of daily living, presenting opportunities to expand measurement of MCID to account for other dimensions of everyday function that are prioritized by individuals living with AD/ADRD.To expand outcome evaluation and improve integration of aspects of functioning that matter most to people living with AD/ADRD, we identify opportunities to incorporate more diverse strategies via goal-attainment scaling, self-report, and performance-based measures as appropriate. We also highlight the importance of incorporating strengths-based domains such as social participation, moving beyond deficit-focused assessment of functioning.

"Successful aging" as defined in Rowe & Kahn's model aims to include the major domains of aging under a single overarching concept. From here the question arises how "successful aging" itself can be implemented as an endpoint in clinical research in a way that it is compliant to methodological regulatory framework and recommendations as formulated, for example, by the United States Food and Drug Admnistration (FDA). This article discusses from an applied perspective approaches and examples of how "successful aging" as a multidomain concept can be put into measurement practice.

Narrative literature review.

Existing methods of merging insights from, for example, functional, social, and cognitive outcomes can fail to perform under conditions of extreme heterogeneity, as often present in samples of older adults. In research on rare diseases, the methodological approach of a Multidomain Responder Index (MDRI) has been proposed to handle heterogeneity. MDRIs may also provide a solution to combine aging outcomes from different domains into a single "successful aging" endpoint. However, strict measurement criteria will need to be fulfilled to find acceptance in decision making and validated meaningful change thresholds are a critical prerequisite.

Gerontology can spearhead methodological approaches of handling multidimensional measurement under heterogeneity by validating a "successful aging" MDRI suitable for use as an endpoint in clinical research.

Falls pose a significant public health concern, with increasing occurrence due to the aging population, and they are associated with high mortality rates and risks such as multimorbidity and frailty. Falls not only lead to physical injuries but also have detrimental psychological and social consequences, negatively impacting quality of life. Identifying individuals at high risk for falls is crucial, particularly for those aged ≥60 years and living in residential care settings; current professional guidelines favor personalized, multifactorial fall risk assessment approaches for effective fall prevention.

This study aimed to explore the prognostic validity of the Fall Risk Score (FRS), a multifactorial-based metric to assess fall risk (using longitudinal real-world data), and establish the clinical relevance of the FRS by identifying threshold values and the minimum clinically important differences.

This retrospective cohort study involved 617 older adults (857 observations: 615 of women, 242 of men; mean age 83.3, SD 8.7 years; mean gait speed 0.49, SD 0.19 m/s; 622 using walking aids) residing in German residential care facilities and used the LINDERA mobile health app for fall risk assessment. The study focused on the association between FRS at the initial assessment (T1) and the normalized number of falls at follow-up (T2). A quadratic regression model and Spearman correlation analysis were utilized to analyze the data, supported by descriptive statistics and subgroup analyses.

The quadratic model exhibited the lowest root mean square error (0.015), and Spearman correlation analysis revealed that a higher FRS at T1 was linked to an increased number of falls at T2 (ρ=0.960, P<.001). Subgroups revealed significant strong correlations between FRS at T1 and falls at T2, particularly for older adults with slower gait speeds (ρ=0.954, P<.001) and those using walking aids (ρ=0.955, P<.001). Threshold values revealed that an FRS of 45%, 32%, and 24% corresponded to the expectation of a fall within 6, 12, and 24 months, respectively. Distribution-based minimum clinically important difference values were established, providing ranges for small, medium, and large effect sizes for FRS changes.

The FRS exhibits good prognostic validity for predicting future falls, particularly in specific subgroups. The findings support a stratified fall risk assessment approach and emphasize the significance of early and personalized intervention. This study contributes to the knowledge base on fall risk, despite limitations such as demographic focus and potential assessment interval variability.

Alzheimer's disease is the most prevalent form of dementia in the elderly, which is clinically characterized by a gradual and progressive deterioration of cognitive functions. The central and early role of β-amyloid in the pathogenesis of Alzheimer's disease is supported by a plethora of studies including genetic analyses, biomarker research and genome-wide association studies in both familial (early-onset) and sporadic (late-onset) forms of Alzheimer's. Monoclonal antibodies directed against β-amyloid demonstrate slowing of the clinical deterioration of patients with early Alzheimer's disease. Aducanumab, lecanemab and donanemab clinical trials showed slowing of Alzheimer's disease progression on composite scores by 25-40% based on the measure used. Anti-β-amyloid antibodies can cause side effects of bleeding and swelling in the brain, called amyloid-related imaging abnormalities. Amyloid-related imaging abnormalities typically occur early in treatment and are often asymptomatic, and though in rare cases, they can lead to serious or life-threatening events. The aim of this review is to evaluate the clinical meaningfulness of anti-β-amyloid therapies amidst amyloid-related imaging abnormalities concern in Alzheimer's disease.

To derive estimates of clinically meaningful change (improvement) on the 25-item National Eye Institute Visual Function Questionnaire (NEI VFQ-25) in patients with diabetic macular edema (DME) using anchor- and distribution-based methods.

In this exploratory post hoc analysis of data from the RIDE/RISE (NCT00473382/NCT00473330) clinical trials of ranibizumab for DME, the NEI VFQ-25 was completed at baseline and six, 12, 18, and 24 months. Anchor-based (≥5-, ≥10-, and ≥15-letter gain in best-corrected visual acuity [BCVA]) and distribution-based estimates were calculated. Subgroup analyses included outcomes when the study eye was the better- or worse-seeing eye.

Baseline characteristics were balanced between the trials (RIDE, N = 382; RISE, N = 377). Anchor-based estimates of clinically meaningful improvement in composite scores (for ≥15-letter gain in BCVA) were 3.78 and 2.23 for RIDE and RISE, respectively. Estimates appeared similar for most subscales: near activities (4.11 and 3.31), distance activities (3.53 and 3.74), driving difficulties (5.15 and 3.15), and vision-specific dependency (4.70 and 1.83). Supportive distribution-based meaningful change composite score estimates also were similar between RIDE and RISE for values based on 0.5 standard deviation (9.85 and 9.70, respectively) or standard error of the mean (5.10 and 4.82, respectively).

These analyses suggest improvement of three to five points on the NEI VFQ-25 composite score and four individual subscales as clinically meaningful in patients with DME.

This analysis supports considering these thresholds when assessing the clinical risk-benefit of DME treatment from the patient perspective using the NEI VFQ-25.

The objective of the study was to demonstrate whether athletes, players, boxers and military personnel can really be victims of Chronic traumatic encephalopathy (CTE), and to elucidate this pathology. In 53 articles, 14 were selected for qualitative synthesis in the results table that addresses CTE in football, soccer and rugby players, boxers and the military. Neuropathologically, CTE shows cerebral atrophy, a pelvic septum cavity with fenestrations, dense diffuse immunoreactive inclusions and a TDP-43 proteinopathy. Microscopically, there are extensive neurofibrillary tangles and spindle-shaped neurites throughout the brain. Thus, CTE is characterized by being a distinct tauopathy and with a clear environmental etiology. American football players, boxers and the military are more likely to trigger CET, due to the constant mechanical shocks from their heads. The most frequent clinical manifestations were: headache, aggressiveness, dementia, executive dysfunction and suicide. CET is definitely diagnosed only at autopsy, there is no specific treatment for it, but support and safety measures can help the patient. Advances to definitively diagnose CTE in living people and specific treatment for this disease are needed.

Patients with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) experience a wide range of symptoms due both to their underlying disease and the effects of treatment. Designing early phase trials to explore effective therapies in these patients should not only examine anti-tumor activity, but also consider the effects of treatments on how patients feel and function. Assessing symptomatic toxicities associated with new therapies in early phase trials from the patient perspective is best measured using patient-reported outcomes (PROs) and offers valuable insight and complementary information to the traditional adverse event reporting in cancer clinical trials. This review describes PROs, highlights their importance in MDS drug development, and outlines the key psychometric properties and practical considerations that make PROs essential and desirable in evaluating the impact of new therapies. We will provide a general overview of PROs and follow with application of PROs in MDS/AML including strategies to be considered in early phase trials. Finally, we describe the creation of the Office of Patient-Centered Outcomes Research at the US National Institutes of Health which has developed a standardized PROs methodology for early phase trials conducted in the Center for Cancer Research at the US National Cancer Institute.

To estimate the minimum clinically important difference (MCID) for the parent-reported 55-item Quality of Life in Childhood Epilepsy Questionnaire (QOLCE-55) and its shortened 16-item version, QOLCE-16.

Data came from 74 children with epilepsy (CWE) (ages 4-10, mean age = 8 [SD = 1.8]) enrolled in the Making Mindfulness Matter in Epilepsy (M3-E) trial, a pilot, parallel randomized-controlled trial of a mindfulness-based intervention. Both anchor-based and distribution-based methods were used to estimate MCID values for the QOLCE-55 and QOLCE-16. For the anchor-based approach, the Patient Centered Global Ratings of Change (PCGRC) scale and linear regression analysis were used to estimate the MCID. For the distribution-based approach, .5 SD of the health-related quality of life (HRQOL) change score distribution was used to estimate the MCID.

For the QOLCE-55, the MCID obtained using an anchor-based approach was 10 points and using a distribution-based method was 6 points. For the QOLCE-16, the MCID obtained using an anchor-based method was 13 points and using a distribution-based method was 7 points.

This is the first study to estimate MCID values for the QOLCE-55 and the QOLCE-16. It has been well documented that CWE are at risk of experiencing psychological, behavioral, and cognitive impairments, which can negatively impact their HRQOL. Reporting MCID values for the QOLCE-55 and QOLCE-16 is important in determining whether changes in HRQOL observed are meaningful to CWE themselves, as a key factor in shaping the nature of epilepsy care delivered.

The BODY-Q is a widely used patient-reported outcome measure for comprehensive assessment of treatment outcomes specific to patients undergoing body contouring surgery (BCS). However, for the BODY-Q to be meaningfully interpreted and used in clinical practice, minimal important difference (MID) scores are needed. A MID is defined as the smallest change in outcome measure score that patients perceive as important.

The aim of this study was to determine BODY-Q MID estimates for patients undergoing BCS to enhance the interpretability of the BODY-Q.

Data from an international, prospective cohort from Denmark, Finland, Germany, Italy, the Netherlands, and Poland were included. Two distribution-based methods were used to estimate MID: 0.2 standard deviations of mean baseline scores and the mean standardized response change of BODY-Q scores from baseline to 3 years postoperatively.

A total of 12,554 assessments from 3237 participants (mean age 42.5 ± 9.3 years; BMI 28.9 ± 4.9 kg/m2) were included. Baseline MID scores ranged from 1 to 5 on the health-related quality of life (HRQL) scales and 3 to 6 on the appearance scales. The estimated MID scores from baseline to 3-year follow-up ranged from 4 to 5 for HRQL and from 4 to 8 on the appearance scales.

The BODY-Q MID estimates from before BCS to 3 years postoperatively ranged from 4 to 8 and are recommended for interpretation of patients' BODY-Q scores, evaluation of treatment effects of different BCS procedures, and calculation of sample size for future studies.

Decrements in energy are a significant problem associated with chemotherapy. To date, no study examined the variability of energy in patients with gynecologic cancers.

To identify distinct morning and evening energy profiles in patients with gynecologic cancers and evaluate for differences in demographic and clinical characteristics, other common symptoms, and quality-of-life (QOL) outcomes.

A sample of 232 patients with gynecologic cancers completed questionnaires 6 times over 2 cycles of chemotherapy. Latent profile analysis was used to identify distinct morning and evening energy profiles. Differences in demographic and clinical characteristics, other common symptoms, and QOL outcomes were evaluated using parametric and nonparametric tests.

Three distinct morning (ie, high [9.2%], low [63.1%], very low [27.1%]) and 2 distinct evening (moderate [30.6%], very low [69.4%]) energy classes were identified. Clinical risk factors associated with the worst morning energy profiles included lower functional status and a higher comorbidity burden. Less likely to exercise on a regular basis was the only characteristic associated with the worst evening energy profile. For both symptoms, the worst profiles were associated with higher levels of depression and sleep disturbance, lower levels of cognitive function, and poorer QOL.

Approximately 70% of patients with gynecologic cancers experienced decrements in morning and evening energy. The study identified modifiable risk factors associated with more decrements in morning and evening energy.

Clinicians can use these findings to identify higher-risk patients and develop individualized energy conservation interventions for these vulnerable patients.

Purpose: The purpose of this study is to determine the magnitude of change that is clinically meaningful in the EORTC QLQ-C30 and EORTC QLQ-HN35 instruments in head and neck cancer patients. Methods: Two hundred and twenty-two patients completed the EORTC QLQ-C30 and EORTC QLQ-HN35 at baseline and follow-up one to two months later. Minimal clinically important differences (MCIDs) were calculated through anchor- and distribution-based methods for improvement and deterioration. Karnofsky Performance status (KPS) was used as the anchor to determine meaningful change. Results: In the group of patients who deteriorated, more scales and symptoms demonstrated statistically significant meaningful change. EORTC QLQ-C30 meaningful change values for deterioration with KPS anchor ranged from 7.2 (physical functioning) to 16.7 units (Global Health Status), and for improvement ranged from 10.0 (role functioning) to 16.7 units (Global Health Status). Conclusions: We report-for the first time, to the best of our knowledge-MCID for EORTC QLQ-C30 and QLQ-HN35 in head and neck cancer patients. Knowledge of meaningful change in these questionnaires allows physicians to assess patient change over time, along with evaluating the impact of treatment on quality of life.

Patients with fibrotic interstitial lung disease (FILD) have impaired health status. The simple questionnaire in the COPD assessment test (CAT) has been validated for idiopathic pulmonary fibrosis (IPF) and connective tissue disease-related interstitial lung disease (CTD-ILD), but no or limited data exist for patients with FILD as a whole. The aim of this study was to evaluate the reliability, repeatability and responsiveness of the CAT, and estimate the minimal clinically important difference (MCID) in patients with FILD.

This study was a retrospective chart review of 358 consecutive patients with FILD including 131 with IPF, who underwent clinical assessment over 6-12 month intervals. We assessed the cross-sectional and longitudinal validity of the CAT. MCID was estimated using distribution methods and anchor methods with mean change and regression models.

Internal consistency (Cronbach's alpha = 0.898) and repeatability (intraclass correlation coefficient [ICC] = 0.865) for the CAT score was acceptable. A cross-sectional study showed constructive validity. Changes in the CAT over 6-12 months were significantly associated with change in anchors including physiological function, exercise capacity, and dyspnea regardless of IPF diagnosis. The estimated MCIDs of the CAT for the deterioration and improvement directions were at least +5 and at least -3 points, respectively.

The CAT is a reliable, responsive and clinically relevant instrument for assessing health status in patients with FILD.

Administrative data are invaluable for assessing outcomes at the population level. However, there are few validated patient-centered outcome measures that capture morbidity following traumatic brain injury (TBI) using these data. We sought to characterize and validate days at home (DAH) as a measure to quantify population-level outcomes after moderate to severe TBI. We additionally assessed the earliest feasible outcome assessment period for patients with TBI using this outcome measure.

This multicenter retrospective cohort study used linked health administrative data sources to identify adults with moderate to severe TBI presenting to trauma centers in Ontario, Canada, between 2009 and 2021. DAH at 180 days (DAH180 days) reflects the total number of days spent alive and at home excluding the days spent institutionalized across care settings. Construct validity was determined using hierarchical quantile regression to assess the associations between clinical and injury covariates with DAH180 days. Predictive validity was assessed using Spearman rank correlation. We estimated minimally important difference (MID) in DAH180 days to aid with outcome measure interpretability.

There were 6,340 patients who met inclusion criteria. Median DAH180 days was 70 days (interquartile range 0-144). Mortality occurred in 2,162 (34.1%) patients within 90 days following injury. Patients in the lower DAH180 days group were more commonly older (absolute standardized difference [ASD] = 0.68) with higher preinjury health resource utilization (ASD = 0.36) and greater injury severity (ASD = 0.81). Increased baseline health resource utilization (-10.1 days, 95% CI -17.4 to -2.8, p = 0.0041), older age (-4.6 days, 95% CI -5.7 to -3.4, p < 0.001), higher cranial injury severity (-84.6 days, 95% CI -98.3 to -71.0, p < 0.001), and major extracranial injuries (-14.2 days, 95% CI -19.5 to -8.93, p < 0.001) were significantly associated with fewer DAH180 days. DAH180 days was positively correlated with DAH at up to 3 years (r = 0.91, 95% CI 0.90-0.92) and negatively correlated with direct health care expenditure (rs = -0.89, 95% CI -0.88 to -0.90). The average MID estimated from anchor-based and distribution-based methods was 18 days.

We validate DAH180 days as a potentially useful outcome measure with construct, predictive, and face validity in a population with moderate to severe TBI. Given the intensity of acute care requirements for patients with TBI, our work highlights DAH180 days as a feasible and sufficiently responsive outcome measure.

Evidence suggests that lower levels of morning energy are associated with higher levels of stress and lower levels of resilience in patients receiving chemotherapy. Study purposes were to identify subgroups of patients with distinct morning energy profiles; evaluate for differences among the profiles in demographic and clinical characteristics, as well as measures of stress, resilience, and coping.

A total of 1,343 outpatients receiving chemotherapy completed a demographic questionnaire and measures of global, cancer-related, and cumulative life stress, and resilience at study enrollment. Morning energy was assessed using the Lee Fatigue Scale at six time points over two cycles of chemotherapy. Latent profile analysis was used to identify subgroups of patients with distinct morning energy profiles. Differences among the subgroups were evaluated using parametric and nonparametric tests.

Three morning energy profiles were identified (i.e., High (17.3%), Low (60.3%), Very Low (22.4%)). Compared to High class, the other two morning energy classes were less likely to be employed; had a lower functional status and a higher comorbidity burden; and were more likely to self-report depression and back pain. For all three types of stress, significant differences were found among the three classes with scores that demonstrated a dose response effect (i.e., High < Low < Very Low; as decrements in morning energy increased, stress scores increased). Compared to High class, Very Low class reported higher rates of physical and sexual abuse. The resilience scores exhibited a dose response effect as well (i.e., High > Low > Very Low). Patients with the two worst energy profiles reported a higher use of disengagement coping strategies.

Findings highlight the complex relationships among decrements in morning energy, various types of stress, resilience, and coping in patients undergoing chemotherapy.

Clinicians need to assess for stress and adverse childhood experiences to develop individualized management plans to increase patients' energy levels.

It's more important to measure the subjective perception of change after undergoing any behavioral or psychological intervention. Global rating of change method-anchor item-based is a method by which the change can be quantified and "minimum subjective perceived effect" (MSPE) can be calculated.

The current study was planned to quantify the effects of heartfulness meditation intervention in healthcare professionals by anchor-based method and compare the result with the pre-post-difference.

The current study was done using multimethod study design among the healthcare professionals. These were 1. Cross-sectional study design. 2. Quasi-experimental-Solomon four-nonequivalent control group study design for intervention.

Cohens' d for perceived stress scale (PSS) in "Much less negative" category was -.96.

The study highlights the use of anchor-based method to estimate the minimal subjective perceived difference, and it can also be compared with the groups pre-post-difference for academic purposes.

The Gonds tribe, indigenous to the Sonbhadra region in Uttar Pradesh, India, faces unique challenges and disparities in women's health, particularly in the domains of menstrual health, pregnancy, and postpregnancy care. This study aims to shed light on the demographic patterns of these critical aspects of women's health among the Gonds tribe, providing valuable insights for healthcare interventions and policy development.

This study explores the demographic and socioeconomic factors affecting Gond women's health, focusing on menstrual hygiene and life stages before, during, and after pregnancy.

We collected data on menstruation wellness behaviors, pregnancy experiences, and postpregnancy care by conducting an extensive survey among Gonds women in the Sonbhadra area. In addition, focus groups and qualitative interviews were used to acquire greater knowledge of the socioeconomic and social factors impacting these health-distinctive characteristics.

The Chi-square test shows no significant link between age group and menstrual hygiene product use. Young women (under 18) mostly use cotton cloth (56%). Women aged 18-49 prefer cotton cloth (53.2%). Women 50+ use cotton cloth (43.2%), cotton (29.7%), and sanitary napkins (27%). Marital status affects product use and cycle regularity. Cultural beliefs and education influence healthcare decisions. Menstrual hygiene mainly involves cotton cloth (45%) and cotton (26.1%).

In conclusion, our analysis uncovers key insights: Marital status, healthcare access, education, cultural beliefs, and reproductive health practices influence community healthcare decisions. These findings inform tailored interventions to enhance health outcomes and equity.

BODY-Q is a patient-reported outcome measure for comprehensive assessment of outcomes specific to patients undergoing bariatric surgery. The clinical utility of BODY-Q is hampered by the lack of guidance on score interpretation. This study aimed to determine minimal important difference (MID) for assessment of BODY-Q. Prospective BODY-Q data from Denmark and the Netherlands pre- and post-bariatric surgery were collected. Two distribution-based methods were used to estimate MID by 0.2 standard deviations of baseline scores and the mean standardized response change of scores from baseline to 3-years postoperatively. In total, 5476 assessments from 2253 participants were included of which 1628 (72.3%) underwent Roux-en-Y gastric bypass, 586 (26.0%) sleeve gastrectomy, 33 (1.5%) gastric banding, and 6 (0.03%) other surgeries. The mean age was 45.1 ± 10.9 with a mean BMI of 46.6 ± 9.6. Baseline MID ranged from 1 to 4 in health-related quality of life (HRQL) and from 2 to 8 in appearance scales. The mean change of scores ranged from 4 to 5 in HRQL and from 4 to 7 in the appearance scales. The estimated MID for the change in BODY-Q HRQL and appearance scales ranged from 3 to 8 and is recommended for use to interpret BODY-Q scores and assess treatment effects in bariatric surgery.

Many studies have highlighted the use of oncolytic viruses as a new class of therapeutic agents for central nervous system (CNS) tumors, especially glioblastomas (GMB). Zika Virus (ZIKV) proteins targeted to specific stem cells have been studied in vitro and animal models with promising results.

A systematic review was evaluated the efficacy and safety of the ZIKV use for CNS tumors treatment. Data were extracted and the in vivo studies were evaluated using the Robins-I tool. We assessed bias in each study using criteria such as selection bias, performance bias, detection bias, attrition bias, reporting bias, and others. According to Cochrane guidelines, bias was classified as high, low, or uncertain. High bias occurred when studies did not meet the criteria. Low bias was assigned when criteria were clearly met. Uncertain bias reflected insufficient information for a clear classification.

The 14 included studies shown that ZIKV reduced cell viability or inhibited the growth, proliferation of glioma stem cells (GSCs), and Bcl2 expression - which could potentially enhance the effect of chemotherapy/radiotherapy; caused cytopathic effects, induced tumor cell damage, manifested oncolytic properties, and even selectively safely killed GSCs; ultimately, it led to significant tumor remission and enhanced long-term survival through enhanced T-cell response.

Although current evidence suggests ZIKV as a promising treatment for CNS tumors and may improve survival when combined with surgery and radiotherapy. Despite limited human evidence, it shows potential benefits. Further research is needed to confirm safety, efficacy, and optimize treatment in humans.

The minimal important difference (MID) is a useful tool to interpret changes in patients' health-related quality of life. This study aims to estimate MIDs for interpreting within-patient change for both components of the EQ-5D-5L questionnaire [EQ-Visual Analogue Scale (EQ-VAS) and utility index] and domains of the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) for cancer patients.

Data were obtained from the Cancer 2015 dataset, a longitudinal cohort of Australian cancer patients. Anchor-based approaches were used to estimate MIDs for the EQ-5D-5L index-based utility index [Australia and the United States (US) tariff sets], EQ-VAS scores, and the EORTC QLQ-C30. Clinical [Eastern Cooperative Oncology Group (ECOG) performance status] and patient-reported (items 29 and 30 of the EORTC QLQ-C30 and the EQ-VAS) anchors were assessed for appropriateness by their correlation strength. Clinical change groups (CCGs) were defined a priori for improvement and deterioration based on estimates used in previous literature. MIDs were estimated via linear regression and distribution-based methods.

For the index-based utility scores in Australia, the anchor-defined MID estimates were 0.01 to 0.06 for improvement and - 0.04 to -0.03 for deterioration, with a weighted value of 0.03 for improvement and deterioration. The EQ-VAS MID estimate was 5 points for both improvement and deterioration. For the EORTC QLQ-C30, changes of at least 3.64 (improvement) and - 4.28 (deterioration) units on the physical functioning scale, 6.31 (improvement) and - 7.11 (deterioration) units on the role functioning scale, 4.65 (improvement) and - 3.41 (deterioration) units on the emotional functioning scale, and 5.41 (improvement) and - 5.56 (deterioration) units on the social functioning scale were estimated to be meaningful.

This study identified lower MIDs for the EQ-5D-5L utility index, EQ-VAS, and EORTC QLQ-C30 domain scores, than those reported previously. The use of a real-world cancer-specific panel dataset may reflect smaller MID estimates that are more applicable to cancer patients in the clinical practice, rather than using MIDs that have been estimated from clinical trials.

The minimal clinically important difference (MCID) is an important concept with big appeal in a field struggling to interpret quality of life (QOL) and other patient-reported outcomes (PRO), is also a bridge between statistics and clinical medicine. This study uses the ROC curve to formulate the MCID value of the Quality of Life Instruments for Chronic Diseases of Systemic lupus erythematosus (QLICD-SLE V2.0) scale. Using the representative item "In general, would you say your health is" of the MOS item short form health survey(SF-36) as an anchor, the questionnaire of QLICD-SLE V2.0 and the anchor item were used to investigate the patients on the first day of hospitalization, and the day before the patient was discharged. 279 patients with lupus erythematosus were participated in this longitudinal follow-up study. The ROC curve was constructed by using the classification based on the anchor item as the gold standard and the difference score of the scale as the test variable. The cut-off point corresponding to the maximum value of the Youden index in the ROC curve is taken as the minimum clinical importance difference (MCID) value of the QLICD-SLE (V2.0) scale. The Results showed that the MCID of physical domain, psychological domain, social domain, general module, specific module and QLICD-SLE (V2.0) total scale are 8.3, 2.3, 2.5, 2.7, 9.2 and 3.2, respectively. Area under the ROC curve of QLICD-SLE (V2.0) is 0.898, P (Area = 0.5) < 0.001, the sensitivity is 100%, the specificity is 66.9%. It concluded that if the total scores after treatments changes at least 3.2 points positively, the treatment intervention can be considered as clinically significant. It is more convincing to use the corresponding cut-off point as the MCID for ROC curve method can visualize the sensitivity and specificity.

Human papillomavirus (HPV) infection and menopause entail a considerable impairment in health-related quality of life (HRQoL). The objective of the present study was to analyze the impact of the menopause status on HRQoL in women with HPV infection.

A cross-sectional, nationwide, multicenter sample of women with HPV infection was conducted throughout clinics of gynecology representative of the Spanish population with regard to age, geographic density, and autonomous regions. Demographic and clinical characteristics and the specific HPV-QoL questionnaire score with its domains were compared according to reproductive status: premenopausal and peri-/postmenopausal. Correlation with other validated patient-reported outcomes measurements was also tested, including General Health Questionnaire-12 (GHQ-12), Female Sexual Function Index (FSFI), and Hospital Anxiety and Depression Scale (HADS).

A sample of 1,016 noninstitutionalized women, aged 18-80 y, was recorded, 191 (18.8%) peri-/postmenopausal and 825 (81.2%) premenopausal. Total HPV-QoL scoring was significantly lower in peri-/postmenopausal (38.8, 95% CI [35.2-42.4]) compared to premenopausal (46.4, 95% CI [45.0-47.8]) women, and also in every domain of the scale (P < 0.05), except in social well-being and health domains, with a small effect size of 0.39. In women with sexual dysfunction according to FSFI, adjusted total scoring and domains sexuality, general well-being, and psychological well-being scored significantly higher in premenopause women (P < 0.01), although the magnitude of differences were of small to moderate size.

HRQoL was impaired during menopause in women with HPV infection according to HPV-QoL questionnaire. The sexuality domain was the most differentiating dimension between these populations.

Minimal clinically important difference (MCID) is important to establish as a meaningful outcome in research when using patient reported outcome measures (PROMs). We determined the MCID using the distribution-based approach for three measurements used as part of the GALAXY study, which is an observational prospective study on gastrointestinal (GI) symptoms in cystic fibrosis (CF).

Four hundred and two persons with cystic fibrosis (PwCF) participated in the GALAXY study, all with baseline values available for all questionnaires. Mean age was 20.9 years (2.1- 61.1) with 75 females and 94 males under the age of 18 (42.04 %) and 118 females and 115 males aged 18 or older (57.99 %). MCID was measured for Patient Assessment of Constipation Symptoms (PAC-SYM), Patient Assessment of Upper Gastrointestinal Symptoms (PAGI-SYM), Patient Assessment of Constipation-Quality of Life (PAC-QOL) and their subscales. Two distribution-based approaches, defined as multiplications of the standard deviation (SD) or standard error of the mean (SEM), were used to approximate the MCID.

The two distribution-based approaches for determining the MCID estimates produced comparable results in trends in MCIDs across the subscales and total scores. In general, MCID estimates of subscales for all three measurements were higher than their total score MCIDs. The one-half SD- and SEM-based MCID estimates for total scores of each questionnaire are as follows: PAC-SYM: 0.26 and 0.14; PAGI-SYM: 0.32 and 0.15; PAC-QOL: 0.27 and 0.18, respectively.

This paper establishes initial MCIDs estimated by the distribution-based approach for the PAC-SYM, PAGI-SYM and PAC-QOL that can now be used to evaluate interventional studies that may impact gastrointestinal symptoms in PwCF.

Estimates of minimally important differences (MID) can assist interpretation of data collected using patient-reported outcomes (PRO), but variability exists in the emphasis placed on MIDs in health technology assessment (HTA) guidelines. This study aimed to identify to what extent information on the MID of a commonly used PRO, the EQ-5D, is required and utilised by selected HTA agencies.

Technology appraisal (TA) documents from HTA agencies in England, France, Germany, and the US between 2019 and 2021 were reviewed to identify documents which discussed MID of EQ-5D data as a clinical outcome assessment (COA) endpoint.

Of 151 TAs utilising EQ-5D as a COA endpoint, 58 (38%) discussed MID of EQ-5D data. Discussion of MID was most frequent in Germany, in 75% (n = 12/16) of Gemeinsamer Bundesausschuss (G-BA) and 44% (n = 34/78) of Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, (IQWiG) TAs. MID was predominantly applied to the EQ-VAS (n = 50), most frequently using a threshold of > 7 or > 10 points (n = 13). G-BA and IQWiG frequently criticised MID analyses, particularly the sources of MID thresholds for the EQ-VAS, as they were perceived as being unsuitable for assessing the validity of MID.

MID of the EQ-5D was not frequently discussed outside of Germany, and this did not appear to negatively impact decision-making of these HTA agencies. While MID thresholds were often applied to EQ-VAS data in German TAs, analyses were frequently rejected in benefit assessments due to concerns with their validity. Companies should pre-specify analyses of continuous data in statistical analysis plans to be considered for treatment benefit assessment in Germany.

The test-retest (TRT) reliability of FACE-Q Aesthetic scales is yet to be assessed. The aim of this study was to establish the TRT reliability of 17 FACE-Q Aesthetic scales and determine the smallest detectable change (SDC) that can be identified using these scales.

Data were collected from an online international sample platform (Prolific). Participants ≥20 years old, who had been to a dermatologist or plastic surgeon for a facial aesthetic treatment within the past 12 months were asked to provide demographic and clinical information and complete an online REDcap survey consisting of 17 FACE-Q Aesthetic scales. Participants were asked if they would be willing to complete the survey again in 7 days. Only the participants who reported no important change in the scale construct and completed the retest within 14 days were included.

A total of 342 unique participants completed the TRT survey. The mean age of the sample was 36.6 (±11.5) years, and 82.4% were female. With outlier data removed, all FACE-Q scales demonstrated an intraclass correlation coefficient >0.70 indicating "good" TRT reliability. The standard error of measurement for the included scales ranged from 3.37 to 11.87, corresponding to a range of SDCgroup from 0.95 to 3.23 and SDCind from 9.34 to 32.91.

All included FACE-Q scales demonstrated sufficient TRT reliability and stability overall after the outlier data were removed. Moreover, the authors calculated the values for the SDC for these scales.