UNASSIGNED: The heterogeneity in outcome measures of post stroke rehabilitation trials suggests the need for consensus approach in stroke recovery measurement. To reach this aim, it is important to understand the past and current use of outcome measures in randomized control trials (RCTs) of stroke rehabilitation.
UNASSIGNED: To systematically review RCTs of post stroke UE rehabilitation interventions to understand the use of UE outcome measures in research and their changes over time.
UNASSIGNED: CINAHL, Embase, PubMed, Scopus and Web of Science were searched from 1960 to 1 April 2021. Studies were eligible for inclusion if they (1) were RCTs or crossovers published in English (2) ≥50% of participants were affected by stroke, 3) included adults ≥ 18 years old, and (4) applied an intervention to the hemiparetic UE as the primary objective of the study.
UNASSIGNED: 1,276 RCTs met inclusion criteria, and 112 different outcome measures were identified. Outcome measures were classified according to the International Classification of Functioning, Disability and Health (ICF) framework. Outcome measures most frequently assessed body function and structure (n = 1,692), followed by activities (n = 1,572) and participation (n = 162). The most used outcome measures were the Fugl-Meyer Assessment (n = 619), the modified Ashworth Scale (n = 255), Action Research Arm Test (n = 211), Wolf Motor Function Test (n = 184), and Box and Block Test (n = 178).
UNASSIGNED: Understanding the breadth of outcome measures that have been used over time emphasizes the need for proposed standardization of outcome measures but also the need to adjust and expand consensus recommendations based on past and ongoing research trends.

The progression of vitiligo is unpredictable, emphasizing the need to identify periods of activity early for tailored treatment. Confetti-like depigmentation, hypochromic areas/borders and Koebner\'s phenomenon are clinical visible signs associated with disease activity in vitiligo. However, their true clinical significance requires further investigation using standardized scoring systems. In the present study, the Vitiligo Signs of Activity Score (VSAS) and the Vitiligo Disease Activity Score (VDAS) were applied to assess disease activity signs and disease progression over time, respectively. Individuals with at least one disease activity sign had a 76.9% likelihood of having active vitiligo. The simultaneous presence of multiple signs or their appearance across body locations increased the likelihood to 94% and 87.1%, respectively. Patients with no disease activity signs had a 60.3% likelihood of having stable disease. This research provides an important nuance about the disease activity signs in vitiligo, which may help guide disease management. The risk of active disease increases when at least two types of vitiligo activity signs are present, or when they are present on different body locations. However, the absence of vitiligo activity signs does not rule out active vitiligo.

Only few years after the first report on diagnosing acute pulmonary embolism (PE) with pulmonary angiography, studies began to investigate the effectiveness and safety of thrombolytic therapy for achieving early reperfusion. In 1992, Guy Meyer demonstrated the fast improvement of pulmonary haemodynamics after alteplase administration; this drug has remained the mainstay of thrombolysis for PE over almost 35 years. In the meantime, algorithms for PE risk stratification continued to evolve. The landmark Pulmonary Embolism International Thrombolysis (PEITHO) trial, led by Guy Meyer, demonstrated the clinical efficacy of thrombolysis for intermediate-risk PE, albeit at a relatively high risk of major, particularly intracranial bleeding. Today, systemic thrombolysis plays an only minor role in the real-world treatment of acute PE in the United States and Europe, but major trials are underway to test safer reperfusion regimens. Of those, the PEITHO-3 study, conceived by Guy Meyer and other European and North American experts, is an ongoing randomised, placebo-controlled, double-blind, multinational academic trial. The primary objective is to assess the efficacy of reduced-dose intravenous thrombolytic therapy against the background of heparin anticoagulation in patients with intermediate-high-risk PE. In parallel, trials with similar design are testing the efficacy and safety of catheter-directed local thrombolysis or mechanical thrombectomy. Increasingly, focus is being placed on long-term functional and patient-reported outcomes, including quality of life indicators, as well as on the utilization of health care resources. The pioneering work of Guy Meyer will thus continue to have a major impact on the management of PE for years to come.

UNASSIGNED: Stroke-induced upper limb disabilities can be characterized by both motor impairments and activity limitations, commonly assessed using Fugl-Meyer Motor Assessment for Upper Extremity (FMMA-UE) and Action Research Arm Test (ARAT), respectively. The relationship between the two assessments during recovery is largely unstudied. Expectedly they diverge over time when recovery of impairment (restitution) plateaus, but compensation-driven improvements still occur. The objective of this study is to evaluate the alignment between FMMA-UE and ARAT in defining upper limb functional recovery categories by ARAT scores. We aimed to establish cut-off scores for both measures from the acute/early subacute, subacute and chronic stages of stroke recovery.
UNASSIGNED: Secondary analysis of four prospective cohort studies (acute/early subacute: n = 133, subacute: n = 113, chronic: n = 92) stages post-stroke. Receiver operating characteristic curves calculated the area under the curve (AUC) to establish optimal FMMA-UE cut-offs based on predefined ARAT thresholds distinguishing five activity levels from no activity to full activity. Weighted kappa was used to determine agreement between the two assessments. We used minimally clinically important difference (MCID) and minimal detectable change (MDC95) for comparison.
UNASSIGNED: FMMA-UE and ARAT scores showed no relevant divergence across all recovery stages. Results indicated similar cut-off scores in all recovery stages with variability below MCID and MDC95 levels. Cut-off scores demonstrated robust AUC values from 0.77 to 0.86 at every recovery stage. Only in highly functional patients at the chronic stage, we found a reduced specificity of 0.55. At all other times sensitivity ranged between 0.68 and 0.99 and specificity between 0.71 and 0.99. Weighted kappa at the acute/early subacute, subacute and chronic stages was 0.76, 0.83, and 0.81, respectively.
UNASSIGNED: Our research shows a strong alignment between FMMA-UE and ARAT cut-off scores throughout stroke recovery, except among the subgroup of highly recovered patients at the chronic stage. Discrepancies in specificity potentially stem from fine motor deficits affecting dexterity outcomes that are not captured by FMMA-UE. Additionally, the high congruence of both measures suggests they are not suited to distinguish between restitution and compensation. Calling for more comprehensive assessment methods to better understand upper limb functionality in rehabilitation.

Myotonic dystrophy type 1 (DM1) is a heterogeneous neuromuscular disorder characterized by progressive muscle weakness and myotonia. This study investigates the progression of muscular strength and function over a four-year period. Patients with DM1 were examined at baseline and four years later. The following metrics were assessed over time: muscle strength (Medical Research Council-sumscore), hand-grip strength (Martin-Vigorimeter), hand-grip relaxation time (myotonia), and limitations in activities of daily living and (DM1ActivC questionnaire). A total of 648 patients entered the registry. Recruitment and follow-up is ongoing. In our manuscript, we focus on, 187 patients who were followed for 4 years. A significant decline in MRC sum score was observed, with distal muscles showing more deterioration. Hand-grip strength decreased significantly, with notable differences between sex and phenotype classified by disease onset. Surprisingly, an improvement of myotonia was observed. Follow-up analysis revealed a significant interaction between myotonia and grip-strength over time. Thus, the improvement in myotonia is likely explained by decreased in grip strength. Finally, there was a significant reduction in DM1ActivC score, indicating decreased activity and social participation. This study demonstrated variability in disease progression depending on sex, phenotype and disease status. This research demonstrates a nuanced pattern of disease progression, highlighting the need to combine different outcome measures to fully understand the complexity of DM1.

UNASSIGNED: To investigate the feasibility, discriminative and convergent validity, and reliability of a lower limb sensor-based proprioception measure in children with upper motor neuron (UMN) lesions.
UNASSIGNED: We assessed three proprioception modalities (joint movement, joint position, and dynamic position sense) of the lower limbs in 49 children with UMN lesions and 50 typically developing (TD) peers (5-19 years). Forty-three children with UMN lesion had a congenital and six an acquired brain lesion and 82% were able to walk without a walking aid. We evaluated the feasibility, compared the test results between children with UMN lesions and TD peers, and calculated Spearman correlations (rs) between the modalities. We quantified relative reliability with Intra-Class Correlation Coefficients (ICC) and absolute reliability with Smallest Detectable Changes (SDC).
UNASSIGNED: Most children with UMN lesions (>88%) found the tests easy to perform. The children with UMN lesions had significantly (p < 0.001) lower proprioceptive function than the TD children. The correlation between the three proprioceptive modalities was moderate to high (0.50 ≤ rs ≤ 0.79). The relative reliability for test-retest and the inter-rater reliability was moderate to high (ICCs = 0.65-0.97), and SDC was between 2° and 15°.
UNASSIGNED: The three tests are feasible, and discriminative and convergent validity and reliability were confirmed. Further studies should investigate the influence on motor function and performance in children with UMN lesions.

OBJECTIVE: Behavioral variant frontotemporal dementia (bvFTD) and primary psychiatric disorders (PPD), such as mood, psychotic, and autism spectrum disorders, share similar clinical characteristics of behavior and social cognition. Better understanding of clinical progression in bvFTD and PPD is essential for adequate disease monitoring and trial design.
METHODS: In this longitudinal study (N = 89), patients with bvFTD and PPD with at least one follow-up assessment were included from the Social Brain Project of the Alzheimer Center Amsterdam. Behavioral change and social cognitive decline were assessed via informant-rated questionnaires (Cambridge Behavioral Inventory-Revised, Frontal Behavioral Inventory [FBI], Stereotypy Rating Inventory, Frontotemporal Dementia Rating Scale, Revised Self-Monitoring Scale [RSMS]-caregiver) and patient assessment (Ekman 60-Faces Test, RSMS-patient, Emotional Contagion Scale). Clinical trajectories (median = 1.4 years, interquartile range = 1.0-2.2) were examined using linear mixed models. In a subsample, associations with baseline serum neurofilament light (sNfL) were examined.
RESULTS: At baseline, behavioral and social cognitive symptoms were similar between diagnosis groups, except for poorer emotion recognition in bvFTD. Over time, behavioral symptoms worsened in bvFTD, whereas most measures remained stable and the FBI improved in PPD. Regarding social cognition, emotion recognition and caregiver-reported socioemotional sensitivity worsened in bvFTD and remained stable in PPD. Patient-reported social cognitive measures did not change over time. Higher sNfL was associated with faster behavioral change.
CONCLUSIONS: Trajectories of behavior and social cognition differentiate bvFTD from PPD, provided that social cognition is not patient-reported. Therefore, we stress the need to optimize longitudinal social cognitive assessment in bvFTD. sNfL may be a useful prognostic marker of behavioral progression in neuropsychiatric populations.

BACKGROUND: Assessment of functional status is an integral part of older adult healthcare and research. Therefore, it is essential that tools to capture function are contextually appropriate. Many tools designed to evaluate extended Activities of Daily Living (eADLs) were developed decades ago.
OBJECTIVE: Our aim was to explore which eADL tasks are commonly performed by older adults and map these tasks to popular eADL scales, seeking stakeholder perspectives to inform recommendations on scale content.
METHODS: Online survey with quantitative questions and free text followed-by semi-structured interviews.
METHODS: Older adults (≥60-years), carers and health, social care and voluntary/community professionals.
METHODS: We extracted eADL tasks from existing scales to develop a survey on frequency of performance, with space for additional comment. The survey was disseminated via Join Dementia Research and other clinical and professional networks. Online semi-structured interviews were undertaken with thematic analysis of verbatim transcripts.
RESULTS: From 2244 online survey responses, 87% came from older people and two-thirds of respondents were female. Some eADL tasks were never performed by many participants. Tasks not part of existing tools included online banking and mobile phone use. From fifteen interviews, a recurring theme was that technological and societal changes have altered daily tasks.
CONCLUSIONS: Commonly used eADL scales contain obsolete tasks and omit tasks older people consider part of their everyday lives. There is a need to work collaboratively to update eADL tools to reflect the priorities and activities of older people to ensure these remain valid measures for use in practice and research. To complement this abstract, a video abstract is available online. A more detailed video-based summary of the content is also available as supplemental material.

UNASSIGNED: The Pediatric Rheumatology Care and Outcomes Improvement Network (PR-COIN) is a North American learning health network focused on improving outcomes of children with juvenile idiopathic arthritis (JIA). JIA is a chronic autoimmune disease that can lead to morbidity related to persistent joint and ocular inflammation. PR-COIN has a shared patient registry that tracks twenty quality measures including ten outcome measures of which six are related to disease activity. The network\'s global aim, set in 2021, was to increase the percent of patients with oligoarticular or polyarticular JIA that had an inactive or low disease activity state from 76% to 80% by the end of 2023.
UNASSIGNED: Twenty-three hospitals participate in PR-COIN, with over 7,200 active patients with JIA. The disease activity outcome measures include active joint count, physician global assessment of disease activity, and measures related to validated composite disease activity scoring systems including inactive or low disease activity by the 10-joint clinical Juvenile Arthritis Disease Activity Score (cJADAS10), inactive or low disease activity by cJADAS10 at 6 months post-diagnosis, mean cJADAS10 score, and the American College of Rheumatology (ACR) provisional criteria for clinical inactive disease. Data is collated to measure network performance, which is displayed on run and control charts. Network-wide interventions have included pre-visit planning, shared decision making, self-management support, population health management, and utilizing a Treat to Target approach to care.
UNASSIGNED: Five outcome measures related to disease activity have demonstrated significant improvement over time. The percent of patients with inactive or low disease activity by cJADAS10 surpassed our goal with current network performance at 81%. Clinical inactive disease by ACR provisional criteria improved from 46% to 60%. The mean cJADAS10 score decreased from 4.3 to 2.6, and the mean active joint count declined from 1.5 to 0.7. Mean physician global assessment of disease activity significantly improved from 1 to 0.6.
UNASSIGNED: PR-COIN has shown significant improvement in disease activity metrics for patients with JIA. The network will continue to work on both site-specific and collaborative efforts to improve outcomes for children with JIA with attention to health equity, severity adjustment, and data quality.

BACKGROUND: The palliative care (PC) needs of patients with head and neck cancer (HNC) are complex, due to high and unique symptom burdens. Uniform outcome measures are critical to assessing the impact of PC interventions in HNC.
METHODS: A scoping review of outcome measures used in patients with HNC receiving PC was performed using PubMed, Embase, and Web of Science from 1980 to 2022.
RESULTS: Of 20 eligible studies, 19 unique instruments were identified which assessed 22 physical, 5 mental, 4 social, 7 related quality of life, and 9 advanced care planning outcomes. Instruments were underutilized, with a larger number of outcomes measurable for instruments used than were reported. The average instrument assessed three domains whereas the average study only reported outcomes from two domains.
CONCLUSIONS: Comparison across studies is limited due to heterogeneity in outcome measures. Future work is needed to develop core PC outcome measures for use in HNC care.