Remote-Use Applications of the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised Clinical Outcome Assessment Tool: A Scoping Review.

OBJECTIVES: In 2021, the US Congress passed the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act. The law encourages development of "tools, methods, and processes" to improve clinical trial efficiency for neurodegenerative diseases. The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) is an outcome measure administered during in-person clinic visits and used to support investigational studies for persons living with amyotrophic lateral sclerosis. Availability of a standardized, remote-use version of the ALSFRS-R may promote more inclusive, decentralized clinical trials. A scoping literature review was conducted to identify existing remote-use ALSFRS-R tools, synthesize feasibility and comparability of administration modes, and summarize barriers and facilitators to inform development of a standardized remote-use ALSFRS-R tool. METHODS: Included studies reported comparisons between remote and in-person, clinician-reported, ALSFRS-R administration and were published in English (2002-2022). References were identified by searching peer-reviewed and gray literature. Twelve studies met the inclusion criteria and were analyzed to compare findings within and across modes of administration. RESULTS: Remote modes of ALSFRS-R administration were categorized into 4 nonmutually exclusive categories: telephone (n = 6), videoconferencing (n = 3), computer or online platforms (n = 3), mobile applications and wearables (n = 2), and 1 unspecified telemedicine modality (n = 1). Studies comparing in-person to telephone or videoconferencing administration reported high ALSFRS-R rating correlations and nonsignificant between-mode differences. CONCLUSIONS: There is insufficient information in the ALSFRS-R literature to support remote clinician administration for collecting high quality data. Future research should engage persons living with amyotrophic lateral sclerosis, care partners, and providers to develop a standardized remote-use ALSFRS-R version.

Patient-reported outcome, clinician-reported outcome, and patient satisfaction with treatment by crisis resolution teams: a multicenter pre-post study of outcome and associated factors in Norway.

BACKGROUND: Crisis resolution teams (CRTs) have become a part of mental health services in many high-income countries. Many studies have investigated the impact of CRTs on acute admissions to inpatient units, but very few studies have investigated patient-reported and clinician-reported outcomes for CRT service users. Our aims were to study patient-reported and clinician-reported outcomes of CRT treatment, how the outcomes were associated with characteristics of the service user and the treatment, and whether outcomes were different across CRTs. METHODS: The study was a pre-post observational multicenter study of 475 patients receiving treatment from 25 CRTs in urban and rural areas in Norway. There was no control group. Outcomes were change in mental health status reported by service users using CORE-10 and by clinicians using HoNOS. Patient satisfaction was measured using CSQ-8 at the end of the treatment. Components of CRT accessibility and interventions were measured by clinicians reporting details on each session with the service user. CRT model fidelity was measured using the CORE CRT Fidelity Scale version 2. We used paired t-tests to analyze outcomes and linear mixed modeling to analyze associations of the outcomes with the characteristics of service users and the treatment provided. Using independent t-tests, we analyzed differences in outcomes and patient satisfaction between two clusters of CRTs with differences in accessibility. RESULTS: The patient-reported outcomes and the clinician-reported outcomes were significantly positive and with a large effect size. Both were significantly positively associated with practical support and medication management and negatively associated with collaboration with mental health inpatient units. Patient satisfaction was high at the end of the treatment. CRTs with higher accessibility had a significantly better clinician-reported outcome, but no significant differences were reported for patient-reported outcomes or patient satisfaction. CONCLUSIONS: CRT treatment led to improved symptom status as reported by patients and clinicians, as well as high patient satisfaction. Practical support and medication management were the interventions most strongly associated with positive outcomes. Some of the variations in outcomes were at the team level. Patient- and clinician-reported outcomes should be used more in studies on the effect of treatment provided by crisis resolution teams.

Developing a set of patient-centered outcomes for routine use in endometriosis: An international Delphi study.

INTRODUCTION: There is large variation in individual patient care for endometriosis. A uniform approach to measure outcomes could be incorporated into routine clinical practice to personalize and monitor treatments and potentially improve the quality of care. The aim of this study is to identify a group of patient-centered outcomes for use in routine endometriosis care which are relevant to all patient profiles. MATERIAL AND METHODS: By means of a modified two-round Delphi study with international representation including healthcare professionals, researchers and patient representatives (51 participants, 16 countries) we developed a set of patient-centered measurements. The participants evaluated 47 Patient Reported Outcome Measures (PROMs) and 30 Clinician Reported Outcome Measures (CROMs) regarding their feasibility and relevance for their use in routine endometriosis care. After the two rounds of quotation, meetings of the experts were convened to participate in a final discussion to finalize the consensus of the final set of included measures. RESULTS: The final set of patient-centered outcomes includes six PROMs (measuring symptomatic impact, pain, work productivity and quality of life) and 10 CROMs (measuring clinical, imaging and surgical indicators). A supplementary list of outcomes was added to include important dimensions that were considered essential by the expert panel but are not relevant to all patients. In addition the need for development of specific tools (PROMs) measuring the psychological impact and the impact in sexual activity of endometriosis was highlighted. CONCLUSIONS: We have developed a set of patient-centered outcomes measures in endometriosis care. The selected outcomes comprise the common features for all patients suffering from endometriosis. adapted for use in routine practice. The list of outcomes has been adapted for use in routine practice from which clinicians can chose, depending on their needs.

Association between clinician reported outcome and patient reported outcome measures one year after stroke.

OBJECTIVES: Patient reported outcome measures (PROM) are increasingly important in the evaluation of care. In this study we evaluate patient PROM in stroke patients and the association with clinical reported outcomes. MATERIALS AND METHODS: Of 3706 first-ever stroke patients, 1861 were discharged home and invited to fill out PROM at discharge, 90 days and one-year after stroke. PROM include mental and physical health and patients' self-reported functionals status, all available via International Consortium for Health Outcomes Measurement. Clinician reported measures as NIHSS, Barthel index were collected during hospital admission and modified Rankin Scale (mRS) 90 days after stroke. PROM compliance was evaluated. Clinician reported measures were related to PROM. RESULTS: 844 (45%) of the invited stroke patients filled out PROM. In general, those patients were younger and less severely affected (higher Barthel index and lower mRS). Compliance after enrolment is about 75%. Barthel index and mRS correlated with all PROM at 90 days and one-year. In age and gender adjusted multiple regression analysis, mRS was a consistent predictor for all PROM subsets, and Barthel index holds predictive value for physical health and patients' self-reported functional status. CONCLUSIONS: Participation rate to fill out PROM is only 45% among stroke patients who were discharged home, and compliance rate is around 75% up one-year follow-up. PROM associated with the clinician reported functional outcome measures Barthel index, and mRS score. Low mRS score is a consistent predictor of better PROM at one year. We propose to use mRS in stroke care evaluation, until PROM participation improves.

Recommendations for the Electronic Migration and Implementation of Clinician-Reported Outcome Assessments in Clinical Trials.

OBJECTIVES: Although best practices from electronic patient-reported outcome (PRO) measures are transferable, the migration of clinician-reported outcome (ClinRO) assessments to electronic modes requires recommendations that address their unique properties, such as the user (eg, clinician), and complexity associated with programming of clinical content. Faithful migration remains essential to ensuring that the content and psychometric properties of the original scale (ie, validated reference) are preserved, such that clinicians completing the ClinRO assessments interpret and respond to the items the same way regardless of data collection mode. The authors present a framework for how to "faithfully" migrate electronic ClinRO assessments for successful deployment in clinical trials. METHODS: Critical Path Institute's Electronic PRO Consortium and PRO Consortium convened a consensus panel of representatives from member firms to develop recommendations for electronic migration and implementation of ClinRO assessments in clinical trials based on industry standards, regulatory guidelines where available, and relevant literature. The recommendations were reviewed and approved by all member firms from both consortia. CONSENSUS RECOMMENDATIONS: Standard, minimal electronic modifications for ClinRO assessments are described. This article also outlines implementation steps, including planning, startup, electronic clinical outcome assessment system development, training, and deployment. The consensus panel proposes that functional clinical testing by a clinician or clinical outcome assessment expert, as well as copyright holder review of screenshots (if possible) are sufficient to support minimal modifications during migration. Additional evidence generation is proposed for modifications that deviate significantly from the validated reference.

Validation of the Physician's Global Assessment of Fingernail Psoriasis by Rheumatologists Treating Psoriatic Arthritis.

OBJECTIVES: This study aimed to assess the content validity and reliability of the Physician's Global Assessment of Fingernail Psoriasis (PGA-F) by rheumatologists treating patients with psoriatic arthritis. METHODS: There were 3 stages of analyses with 3 clinician cohort groups. Stage 1 (concept confirmation) included rheumatologist qualitative data (cohort 1) to establish content validity, acceptability, utility, and feasibility of the PGA-F in assessing nail severity. Quantitative information regarding the response category utilization in nail abnormalities was assessed by photographs. Stage 2 (inter-rater reliability) involved quantitative analysis of PGA-F data from study investigators, including rheumatologists, involved in a phase III clinical study (cohort 2) and a cohort of newly recruited rheumatologists (cohort 3). Stage 3 included known-groups validity. RESULTS: Qualitative analyses identified consensus that the PGA-F severity levels are comprehensive of real-world patient symptoms and the instrument is simple to use and understand. Psychometric analyses support the PGA-F as a clinical outcome assessment tool. Inter-rater reliability showed rheumatologist agreement across the fingernail psoriasis severity spectrum. They were monotonically ordered by the hypothesized severity structure with excellent fit to the clinicians who evaluated them. Agreement on the rank order of the severity of the photographs in this target rheumatologist population was consistent with previous reports by dermatologists. CONCLUSIONS: The PGA-F was shown to be usable by rheumatologists to measure patients along the full range of the fingernail psoriasis severity spectrum, have a strong relationship with a conceptually similar reference measure, differentiate among patients based on fingernail psoriasis severity, and detect category severity change over a 24-week period.

Comparison of patient- and clinician-reported outcome measures in lower back rehabilitation: introducing a new integrated performance measure (t2D).

PURPOSE: Patient- and clinician-reported outcome measures (PROMs, CROMs) are used in rehabilitation to evaluate and track the patient's health status and recovery. However, controversy still exists regarding their relevance and validity when assessing a change in health status. METHODS: We retrospectively analyzed the changes in a CROM (Fingertip-To-Floor Test - FTF) and PROMs (ODI, HAQ-DI, NPRS, EQ5D) and the associations between these outcomes in 395 patients with lower back pain (57.2 ± 11.8 years, 49.1% female). We introduced a new way to measure and classify outcome performance using a distribution-based approach (t2D). Outcome measures were assessed at baseline and after 21 days of inpatient rehabilitation. RESULTS: Overall, the rehabilitation (Cohens d = 0.94) resulted in a large effect size outcome. Medium effect sizes were observed for FTF (d = 0.70) and PROMs (d > 0.50). Best performance rating was observed for pain (NPRS). We found that 13.9% of patients exhibited a deterioration in the PROMs, but only 2.3%, in the FTF. The correlation between the PROMs and FTF were low to moderate, with the highest identified for HAQ-DI (rho = 0.30-0.36); no significant correlations could be shown for changes. High consistency levels were observed among the performance scores (t2D) in 68.9% of the patients. CONCLUSIONS: Different and complementary assessment modalities of PROMs and CROMs can be used as valuable tools in the clinical setting. Results from both types of measurements and individual performance assessments in patients provide a valid basis for the meaningful interpretation of the patients' health outcomes. TRIAL REGISTRATION: This clinical study was entered retrospectively on August 14, 2020 into the German Clinical Trials Register (DRKS, registration number: DRKS00022854).

New complementary perspectives for inpatient physical function assessment: matched clinician-report and patient-report short form measures from the PROMIS adult physical function item bank.

PURPOSE: To develop two item content-matched, precise, score-level targeted inpatient physical function (PF) short form (SF) measures: one clinician-reported, one patient-reported. Items were derived from PROMIS PF bank content; scores are reported on the PROMIS PF T-score metric. METHODS: The PROMIS PF item bank was reviewed for content measuring lower-level PF status (T-scores 10-50) with high item set score-level reliability (≥ 0.90). Selected patient-reported (PR) items were also edited to function as clinician-reported (CR) items. Items were reviewed by clinicians and field tested; responses were assessed for meeting PROMIS measure development standards. New CR and PR items were calibrated using patient responses to the original PROMIS PF items as anchoring data. SFs were constructed, based on content and precision. RESULTS: Nine PROMIS PF items were candidates for CR and PR inpatient PF assessment; three new items were written to extend content coverage. An inpatient sample (N = 515; 55.1% female; mean age = 66.2 years) completed 12 PR items and was assessed by physical therapists (using 12 CR items). Analyses indicated item sets met expected measure development standards. Twelve new CR and three new PR items were linked to the PROMIS PF metric (raw score r = 0.73 and 0.90, respectively). A 5-item CR SF measure was constructed; score-level reliabilities were ≥ 0.90 for T-scores 13-45. A 5-item PR SF measure was assembled, mirroring CR SF content. CONCLUSIONS: Two item content-matched SFs have been developed for clinician and patient reporting and are an effective, efficient means of assessing inpatient PF and offer complementary perspectives.

Differences among the observers in the assessments of Japanese orthopedic association hip scores between surgeons and physical therapists and the correlations to patients' reported outcomes after total hip arthroplasty.

BACKGROUND: We aimed to assess the utility of a clinician-reported outcome (the Japanese Orthopedic Association [JOA] hip score) as evaluated by clinicians and physiotherapists. This assessment was made by comparing these scores to those of the JOA hip disease evaluation questionnaire (JHEQ), which is a measurement of patient-reported outcomes after total hip arthroplasty. METHODS: In this retrospective case-control study, 52 hips that underwent primary total hip arthroplasty were included in the analyses. The mean age of the participants was 66.8 years (sex, seven male and 45 female participants). The JOA hip score included four categories: pain, range of motion, ability to walk, and active daily living. The JHEQ included three categories: pain, movement, and mental health. These scores were evaluated preoperatively and postoperatively by clinicians or physiotherapists. Pearson's correlation coefficients were utilized to analyze the association of the JOA hip scores to those of the JHEQ. RESULTS: The JOA hip scores were determined by clinicians and physiotherapists (scores of 46.8 and 57.3, respectively) preoperatively and at 24 months (scores of 94.4 and 91.7, respectively) postoperatively. The JHEQ points were 28.8 and 66.2 preoperatively and at 24 months postoperatively, respectively. The correlation coefficients between the JOA hip and JHEQ scores were .66 and .69 preoperatively and .57 and .76 at 24 months postoperatively, as evaluated by clinicians and physiotherapists, respectively. CONCLUSIONS: Although the JHEQ scores were positively correlated to the JOA hip scores by clinicians and physiotherapists preoperatively and postoperatively, this study implies that clinicians may interpret the results in a way that might have been beneficial to them. To comprehend a patients' health status, we should inclusively understand the varying range of information among different evaluators.

Outcomes reporting in systematic reviews on non-surgical root canal treatment: A scoping review for the development of a core outcome set.

BACKGROUND: Studies related to non-surgical root canal treatment are amongst the most frequently performed clinical studies in endodontics. However, heterogeneity in reporting outcomes and lack of standardization is a significant challenge to evidence synthesis and guideline development. OBJECTIVES: The aims of the present scoping review were to (a) identify outcomes reported in systematic reviews evaluating non-surgical root canal treatment; (b) identify how and when the reported outcomes were measured; (c) assess possible selective reporting bias in the included studies. The information obtained in this study should inform the development of a core outcome set (COS) for non-surgical root canal treatment. METHODOLOGY: Structured literature searches were performed to identify systematic reviews on non-surgical root canal treatments published in English between January 1990 and December 2020. Two reviewers undertook study selection and data extraction. Outcomes were categorized according to a healthcare taxonomy into five core areas (survival, clinical/physiological changes, life impact, resource use, and adverse events). The outcome measurement tools and length of follow-up were recorded. RESULTS: Seventy-five systematic reviews were included, of which 40 included meta-analyses. Most reviews reported on physiological and clinical outcomes, primarily pain and/or radiographic assessment of periapical status, and a variety of measurement tools and scales were used. Few reviews focused on tooth survival, life impact, resources, and adverse events. The heterogeneity amongst the reviews was large on all parameters. Less than 40% of the reviews assessed the risk of selective reporting. DISCUSSION: Overall aims of the included reviews were highly heterogenic; thus, outcomes and how they were measured also varied considerably. Patient-centred outcomes and the use of resources were rarely reported on. CONCLUSIONS: Most studies reported on physiological and clinical outcomes, in particular pain and/or radiographic healing. Measurement tools, scales, thresholds, and follow-up periods varied greatly within each outcome, making comparison across studies complicated. Less than 40% of the reviews assessed risk of selective reporting; thus, selective bias could not be ruled out. The presented information on reported outcomes, measurement tools and scales, and length of follow-up may guide the planning of future research and inform the development of a COS for non-surgical root canal treatment.

Clinical update on psychiatric outcome measurement: what is the purpose, what is known and what should be done about it?

OBJECTIVE: To provide a clinical update on the purposes, evidence-base and recommendations for both clinician and patient-rated outcome measures in psychiatric practice. CONCLUSIONS: Private and public sector funders have implemented outcome measurement systems in Australian mental healthcare, in order to improve cost-effectiveness. It is important to consider the ultimate aims of outcome measurement from various perspectives in evaluating the evidence-base, as there are a number of measurement dimensions to address. For individual clinicians, the purpose may be to guide treatment-planning, as well as to assess treatment and clinician efficacy. For patients, the purpose is to assess outcomes in terms of their goals for recovery, as well as to evaluate their satisfaction with the care provided, and their healthcare providers. The other orthogonal dimensions of measurement comprise, the proximal to illness measures of symptomatic severity, and the distal measures of disability, which apply to both clinician and patient outcomes. In turn, these measures may be used by healthcare funders in public or private sectors as proxy measures of the cost-effectiveness of psychiatric care provided. Clinical registries linked to service-mapping would provide better data for patients, providers and funders to assess the availability and effectiveness of psychiatric care in Australia.

Global multi-site, prospective analysis of cataract surgery outcomes following ICHOM standards: the European CAT-Community.

PURPOSE: To evaluate in a large sample of patients from 10 different European centers the results of cataract surgery, characterizing the relationship between patient-reported outcomes (PROMs) and clinician-reported outcome measures (CROMs). METHODS: Prospective non-interventional multicenter observational descriptive study analyzing the clinical outcomes of a total of 3799 cases undergoing cataract surgery (mean age: 72.7 years). In all cases, the cataract surgery standard developed by the International Consortium for Health Outcomes Measurements (ICHOM) was used to register the clinical data. Three-month postoperative visual acuity and refraction data were considered CROMs, whereas Rasch-calibrated item 2 (RCCQ2) and total Catquest-9SF score (CQ) were considered PROMs. RESULTS: Postoperative corrected distance visual acuity (CDVA) was 0.3 logMAR or better in 88.7% (2505/2823) of eyes. Mean differences between preoperative and postoperative RCCQ2 and CQ scores were -3.09 and -2.39, respectively. Visual function improvement with surgery was reported by 91.5% (2163/2364) of patients. Statistically significant, although weak, correlations of postoperative CDVA with postoperative refraction, PROMs, and complications were found (0.133 ≤ r ≤0.289, p < 0.001). A predictive model (R2: 0.254) of postoperative CDVA considering 10 variables was obtained, including preoperative CDVA, different ocular comorbidities, age, gender and intraoperative complications. Likewise, another predictive model (R2: 0.148) of postoperative CQ considering a total of 14 variables was obtained, including additionally preoperative CQ, target refraction and previous surgeries. CONCLUSIONS: Cataract surgery provides an improved functional vision in most of patients although this improvement can be limited by ocular comorbidities and complications. The relationship between PROMs and CROMs is multifactorial and complex.

Comparison between patient-reported and clinician-reported outcomes: Validation of the Japanese version of the Integrated Palliative care Outcome Scale for staff.

OBJECTIVES: The goal of palliative and supportive care is to improve patients' quality of life (QoL). Patient-reported outcome measures (PROMs) are the gold standard for the assessment of QoL and symptoms; however, when self-reporting is complicated, PROMs are often substituted with proxy-reported outcome measures, such as clinician-reported outcome measures. The objective of this study was to assess the validity and reliability of the Japanese version of the Integrated Palliative care Outcome Scale (IPOS) for staff (IPOS-Staff). METHODS: This multicenter, cross-sectional observational study was conducted concurrently with the validation of the IPOS for patients (IPOS-Patient). Japanese adult patients with cancer and their staff were recruited. We assessed the characteristics of the patients and staff members, missing values, prevalence, and total IPOS scores. For the analysis of criterion validity, intra-rater, and inter-rater reliability, we calculated intraclass correlations (ICCs). RESULTS: One hundred and forty-three patients completed the IPOS-Patient, and 79 medical staff members completed the IPOS-Staff. The most common missing values from IPOS-Staff were Family Anxiety (3.5%) and Sharing Feelings (3.5%). Over half of the patients scored themselves moderate or worse for Poor Mobility, Anxiety, and Family Anxiety, while staff members scored patients moderate or worse for Weakness, Anxiety, and Family Anxiety. For criterion validity (patient-staff agreement) as well as intra-rater and inter-rater reliability, ICCs ranged from 0.114 (Sharing Feelings) to 0.826 (Nausea), 0.720 (Anxiety) to 0.933 (Nausea), and -0.038 (Practical Problems) to 0.830 (Nausea), respectively. SIGNIFICANCE OF RESULTS: The IPOS-Staff is easy to respond to; it has fair validity and reliability for physical items but poor for psycho-social items. By defining the context and objectives of its use and interpretation, the IPOS-Staff can be a useful tool for measuring outcomes in adult patients with cancer who cannot complete self-evaluations.

Outcome Measures in Gender-Confirming Chest Surgery: A Systematic Scoping Review.

BACKGROUND: The aim of this scoping review was to provide an overview of outcome measures in gender-confirming chest surgery. METHODS: A comprehensive literature search was performed in PubMed, EMBASE, CINAHL, PsycINFO, Scopus and the Cochrane Library to find studies evaluating gender-confirming chest surgery in a non-cis gender population. The systematic scoping review followed the PRISMA extension for scoping reviews. Data were charted for outcome measures including complications, reoperations, revision surgery, aesthetic outcome and patient-reported outcome measures. RESULTS: Our search yielded 849 records, which were screened on title, abstract and full text. Of these, 47 were included in the review. Feminising gender-confirming chest surgery was evaluated in 11 studies, and masculinising gender-confirming chest surgery was evaluated in 39 studies. Clinician-reported outcome categories were used in 40 studies and included complications, reoperation, revision surgery and aesthetic outcome. Categories of patient-reported outcomes were used in 29 studies and included aesthetic outcome, functional outcome and mental health parameters. The summary of outcome domains and classifications showed that there are large variations in outcome evaluation between studies. Although several studies reported on similar outcome categories, there was a high level of heterogeneity of domains and classifications of outcomes. CONCLUSIONS: Evaluation of outcomes in gender-confirming chest surgery showed large variations in reporting, and further streamlining of reporting is therefore required to be able to compare surgical outcomes between studies. LEVEL OF EVIDENCE III: This journal requires that authors assign a level of evidence to each article. For a full description of these Evidence-Based Medicine ratings, please refer to the Table of Contents or the online Instructions to Authors www.springer.com/00266.

Validation of the Investigator Global Assessment of Chronic Hand Eczema (IGA-CHE): a new clinician reported outcome measure of CHE severity.

The Investigator Global Assessment of Chronic Hand Eczema (IGA-CHE) is a novel Clinician-Reported Outcome measure that allows investigators to assess cross-sectional CHE global disease severity using clinical characteristics of erythema, scaling, lichenification/hyperkeratosis, vesiculation, oedema, and fissures as guidelines for overall severity assessment. This study aimed to evaluate the psychometric properties of the IGA-CHE for use as an outcome measure in CHE clinical trials and clinical practice. Psychometric analyses were performed using data from a sample of 280 patients with moderate to severe CHE from a phase 3 trial of delgocitinib cream, pooled across treatment groups. Test-retest reliability results were moderate to strong with kappa coefficients ranging from 0.63 to 0.76. Correlations with measures assessing related concepts were moderate or strong (range 0.65-0.72) and exceeded a priori hypotheses, providing evidence of convergent validity. Known-groups validity was supported by statistically significant differences between severity groups (< 0.001). Within-group effect sizes were consistently larger for improved groups compared to stable groups, providing evidence of ability to detect change. Anchor-based analyses generated within-subject meaningful change estimates ranging from - 0.8 to - 2.3. A correlation weighted average suggested a single value of - 1.7 in change from baseline. These findings provide evidence the IGA-CHE scale has strong reliability, construct validity, and ability to detect change, supporting its use as an endpoint in CHE clinical trials and clinical practice. Based on the evidence, 2-level changes in IGA-CHE score are considered a conservative meaningful change threshold; however, findings also indicate 1-level change in IGA-CHE scores reflects a clinically meaningful improvement for patients.Clinical trial registration: NCT04871711.

French translation and validation of the cross-cultural adaptation of the MSTS functional assessment questionnaire completed after tumor surgery.

INTRODUCTION: Malignant tumors of the extremities are uncommon. Their surgical treatment, whether conservative or not, may cause sequelae. Functional assessment of patients is essential for research purposes, but also follow-ups and adaptation of treatments. The Musculoskeletal Tumor Society Rating Scale (MSTS) is a disease-specific functional scoring system often used in English literature. Currently, no studies have been published on a valid French translation of the MSTS, whether for the lower or upper extremities. We, therefore, conducted a prospective study to answer the following questions: (1) Are the proposed adapted French versions of the MSTS valid? (2) Do the psychometric properties of the two versions obtained make their use relevant? MATERIALS AND METHODS: The translation and cross-cultural adaptation were carried out following the recommendations of Beaton and Guillemin to obtain two versions: one for patients who had undergone upper extremity surgery (MSTS-UE) and one for those who had undergone lower extremity surgery (MSTS-LE). A prospective multicenter cohort study was then carried out to analyze the psychometric properties of these two versions. RESULTS: A total of 250 patients from 3 referral centers were enrolled in this study. A confirmatory factor analysis (CFA) demonstrated that the two French versions of the MSTS (MSTS-LE and MSTS-UE) were a good fit with a root mean square error of approximation (RMSEA)<0.08 and a comparative fit index (CFI)>0.90. The psychometric properties of the two versions were validated with internal consistency (Cronbach alpha>0.7), convergent validity of each item with its score (> 0.4), and sufficient criterion validity (Pearson correlation coefficient>0.4). The discriminant validity analysis showed that there was a significant correlation between each version and the performance status (PS) (p<0.05). CONCLUSION: This study produced a French version of the MSTS scoring system and validated the psychometric properties of the two versions obtained (MSTS-UE and MSTS-LE). Therefore, the French MSTS scoring system is a valid measurement that can be used in international studies. LEVEL OF EVIDENCE: I.

Efficacy of Baricitinib in Patients with Various Degrees of Alopecia Areata Severity: Post-Hoc Analysis from BRAVE AA1 and BRAVE AA2.

BACKGROUND: Baricitinib, an oral selective JAK1/JAK2 inhibitor, is approved for the treatment of adults with severe alopecia areata (AA). OBJECTIVE: To evaluate differences in response up to week 52 among subgroups based on the baseline severity of AA assessed with the Severity of Alopecia Tool (SALT) score. METHODS: Data were pooled from BRAVE-AA1 and BRAVE-AA2, two randomized, placebo-controlled, phase 3 trials, which enrolled adults with a SALT score ≥ 50. Patients were subdivided by the degree of AA severity at baseline. RESULTS: Among the 855 patients treated with baricitinib 2 mg and 4 mg, improvements in scalp hair growth continued through to week 52. A superior response was observed in patients with a SALT score of 50-94 versus a score of 95-100. Patients on baricitinib 4 mg had a faster and higher response rate compared to baricitinib 2 mg. CONCLUSION: Across all degrees of severity for baricitinib 2 mg and 4 mg doses, the proportion of patients responding was yet to plateau up to week 52. Response to treatment was longer for patients with a baseline SALT score 95-100. Further studies are needed to analyze other parameters that may impact observed response rates.

An Assessment of Current Clinician-Reported and Patient-Reported Outcome Measures for Alopecia Areata: A Scoping Review.

Although progress has been made in developing outcome measures for AA, the use of these measures remains unstandardized. A scoping review was conducted to identify the clinician-reported outcome measures (ClinROMs) and patient-reported outcome measures (PROMs) used in assessing and treating AA, the results of which revealed heterogeneity in AA outcome measures. Of 23 research studies ultimately included, only 2 ClinROMs were used by >15% of studies; likewise, of 110 clinical trials evaluated, numerous outcome instruments were used, but only one ClinROM was used by >5% of trials (Severity of Alopecia Tool). These results suggest the need for consensus and standardization in both research and trial settings.

Unlocking the Power of Benchmarking: Real-World-Time Data Analysis for Enhanced Sarcoma Patient Outcomes.

Benchmarking is crucial for healthcare providers to enhance quality and efficiency, notably for complex conditions like sarcomas. Multidisciplinary teams/sarcoma boards (MDT/SBs) are vital in sarcoma management, but differences in their processes can affect patient outcomes and treatment costs, despite adherence to international guidelines. To address this issue, this study aimed to compare two MDT/SBs and establish an interoperable digital platform, Sarconnector®, for real-time-world data assessment and automated analysis. The study included 983 patients, 46.0% of whom female, with a median age of 58 years, and 4.5% of patients presented with metastasis at diagnosis. Differences were observed in the number of first-time presentations, follow-up presentations, primary sarcomas, biopsies and chemotherapy indications between the two MDT/SB. The results highlight the importance of benchmarking and utilizing a harmonized data approach, such as the RWT approach provided by the Sarconnector®, to standardize and evaluate quality and cost metrics. By identifying areas of improvement and making data-driven decisions on the meta-level, healthcare providers can optimize resources and improve patient outcomes. In conclusion, benchmarking with the RWT harmonized data approach provided by the Sarconnector® can help healthcare providers improve the overall effectiveness of the healthcare system and achieve better outcomes for their patients in terms of both outcomes and costs.