Core Set Of Measures Research Articles

Renal injury, biomarkers, and myositis, an understudied aspect of disease: prospective study in the MyoCite cohort.

The mechanisms leading to chronic kidney disease (CKD) in patients with idiopathic inflammatory myopathies (IIMs) are poorly understood. We assessed the prevalence of subclinical renal injury in patients with IIMs, through elevation in biomarker levels of tubular injury and fibrosis (NGAL, KIM1, Activin A, CD163, and Cys-c), and assessed differences between subtypes of IIMs, and the effect of disease activity and duration. Clinical data, core set measures, sera and urine were prospectively collected from all patients enrolled in the MyoCite cohort from 2017 to 2021. Twenty healthy subjects (HC) and 16 patients with acute kidney injury (AKI) were included as controls. Baseline and follow up data for IIMs were included. Enzyme-linked immunosorbent assay (ELISA) was used to measure urine NGAL (Human Lipocalin-2/NGAL Duoset ELISA, Cat no: DY1757), KIM1 (Human TIM-1/KIM 1/HAVCR Duoset ELISA, Cat.no: DY1750B), Activin A (Human Activin A Duoset ELISA, Cat no: DY338), CD163 (Human CD163 Duoset ELISA,Cat no: DY1607-05), and Cys-c (Human Cystatin C Duoset ELISA, Cat. no.: DY1196) levels, while eGFR (unit mL/min/1.73 m2) was calculated by the Cockcroft-Gault formula and CKD-EPI formula. Analysis of 201 visits of 110 adult patients with IIMs indicated higher normalized biomarker levels compared to HCs, and comparable to patients with AKI, with the exception of NGAL, which was higher in the AKI group. Notably 72 (49%) patients with IIMs had eGFR<90; the levels of the 5 biomarkers were comparable between active and inactive IIMs, and different subtypes of IIMs. Similarly, a poor correlation between urine biomarker levels and core set measures of activity and damage was found. Changes in biomarker levels on follow-up did not correlate with eGFR changes. This exploratory analysis of urinary biomarkers identified low eGFR and elevated biomarkers of CKD in nearly half of the patients with IIMs, comparable to patients with AKI and higher than HCs, indicative of potential renal damage in IIMs that may have a lead to complications in other systems.

Therapeutic Equivalence of Biosimilar and Reference Biologic Drugs in Rheumatoid Arthritis

Biosimilar drugs are potentially lower-cost versions of biologics that may improve access to therapy. However, there is a lack of adequate systematic reviews demonstrating equivalence between these drugs for the treatment of rheumatoid arthritis (RA). To assess the efficacy, safety, and immunogenicity associated with biosimilars of adalimumab, etanercept, and infliximab compared with their reference biologics in patients with RA. MEDLINE via PubMed, Embase, Cochrane Central Register of Controlled Trials, and LILACS databases were searched from inception to September 2021. Head-to-head randomized clinical trials (RCTs) of biosimilars of adalimumab, etanercept, and infliximab and their biologic reference drugs for RA were assessed. Two authors independently abstracted all data. Meta-analysis was conducted with bayesian random effects using relative risks (RRs) for binary outcomes and standardized mean differences (SMDs) for continuous outcomes, with 95% credible intervals (CrIs) and trial sequential analysis. Specific domains were assessed for the risk of bias in equivalence and noninferiority trials. This study was conducted according to the Preferred Reporting Items for Systematic Reviews and Meta-analyses (PRISMA) guideline. Equivalence was tested using prespecified margins for the American College of Rheumatology criteria, with at least 20% improvement in the core set measures (ACR20) (ie, RR, 0.94 to 1.06), and for the Health Assessment Questionnaire-Disability Index (HAQ-DI) (ie, SMD, -0.22 to 0.22). Secondary outcomes included 14 items measuring safety and immunogenicity. A total of 25 head-to-head trials provided data on 10 642 randomized patients with moderate to severe RA. Biosimilars met equivalence with reference biologics in terms of ACR20 response (24 RCTs with 10 259 patients; RR, 1.01; 95% CrI, 0.98 to 1.04; τ2 = 0.000) and change of HAQ-DI scores (14 RCTs with 5579 patients; SMD, -0.04; 95% CrI, -0.11 to 0.02; τ2 = 0.002) considering prespecified margins of equivalence. Trial sequential analysis found evidence for equivalence for ACR20 since 2017 and HAQ-DI since 2016. Overall, biosimilars were associated with similar safety and immunogenicity profiles compared with reference biologics. In this systematic review and meta-analysis, biosimilars of adalimumab, infliximab, and etanercept were associated with clinically equivalent treatment effects compared with their reference biologics for the treatment of RA.

Performance of the 2016 ACR-EULAR myositis response criteria in juvenile dermatomyositis therapeutic trials and consensus profiles.

The 2016 ACR-EULAR Response Criteria for JDM was developed as a composite measure with differential weights of six core set measures (CSMs) to calculate a Total Improvement Score (TIS). We assessed the contribution of each CSM, representation of muscle-related and patient-reported CSMs towards improvement, and frequency of CSM worsening across myositis response criteria (MRC) categories in validation of MRC. Data from JDM patients in the Rituximab in Myositis trial (n = 48), PRINTO JDM trial (n = 139), and consensus patient profiles (n = 273) were included. Observed vs expected CSM contributions were compared using Sign test. Characteristics of MRC categories were compared by Wilcoxon tests with Bonferroni adjustment. Spearman correlation of changes in TIS and individual CSMs were examined. Agreement between physician-assessed change and MRC categories was evaluated by weighted Cohen's kappa. Of 457 JDM patients with IMACS CSMs and 380 with PRINTO CSMs, 9-13% had minimal, 19-23% had moderate and 41-50% had major improvement. The number of improved and absolute percentage change of CSMs increased by MRC improvement level. Patients with minimal improvement by MRC had a median of 0-1 CSM worsened, and those with moderate/major improvement had a median of zero worsening CSMs. Of patients improved by MRC, 94-95% had improvement in muscle strength and 93-95% had improvement in ≥1 patient-reported CSM. IMACS and PRINTO CSMs performed similarly. Physician-rated change and MRC improvement categories had moderate-to-substantial agreement (Kappa 0.5-0.7). The ACR-EULAR MRC perform consistently across multiple studies, supporting its further use as an efficacy end point in JDM trials.

Performance of the 2016 ACR-EULAR Myositis Response Criteria in adult dermatomyositis/polymyositis therapeutic trials and consensus profiles.

The ACR-EULAR Myositis Response Criteria (MRC) were developed as a composite measure using absolute percentage change in six core set measures (CSMs). We aimed to further validate the MRC by assessing the contribution of each CSM, frequency of strength vs extramuscular activity improvement, representation of patient-reported outcome measures (PROM), and frequency of CSM worsening. Data from adult dermatomyositis/polymyositis patients in the rituximab (n = 147), etanercept (n = 14), and abatacept (n = 19) trials, and consensus patient profiles (n = 232) were evaluated. The Total Improvement Score (TIS), number of improving vs worsening CSMs, frequency of improvement with and without muscle-related CSMs, and contribution of PROM were evaluated by MRC category. Regression analysis was performed to assess contribution of each CSM to the MRC. Of 412 adults with dermatomyositis/polymyositis, there were 37%, 24%, 25%, and 14% with no, minimal, moderate, and major MRC improvement, respectively. The number of improving CSMs and absolute percentage change in all CSMs increased by improvement category. In minimal-moderate improvement, only physician-reported disease activity contributed significantly more than expected by MRC. Of patients with at least minimal improvement, 95% had improvement in muscle-related measures and a majority (84%) had improvement in PROM. Patients with minimal improvement had worsening in a median of 1 CSM, and most patients with moderate-major improvement had no worsening CSMs. Physician assessment of change generally agreed with MRC improvement categories. The ACR-EULAR MRC performs consistently across multiple studies, further supporting its use as an efficacy end point in future myositis therapeutic trials.

Accelerating Medicines Partnership® Schizophrenia (AMP® SCZ): developing tools to enable early intervention in the psychosis highrisk state.

Accelerating Medicines Partnership® Schizophrenia (AMP® SCZ): developing tools to enable early intervention in the psychosis highrisk state.

Traumatic Brain Injury Research: A Closer Examination of Outcome Measure Use

<h3>Objective(s)</h3> To conduct a systematic review of outcome measures currently used in the traumatic brain injury (TBI) scientific literature and to evaluate how they fit within the International Classification of Functioning, Disability and Health (ICF) Framework. <h3>Data Sources</h3> Scopus, PubMed, CINAHL, EMBASE and PsycINFO. <h3>Study Selection</h3> Articles published up to and including June 30, 2021 meeting the following criteria were included: (1) human subjects with a mean of ≥18 years old, (2) ≥50% of the sample had moderate to severe TBI, (3) the study design was a randomized controlled trial (RCT), and (4) written in English. Protocols and post-hoc analyses of RCTs were not included. <h3>Data Extraction</h3> Author(s), country, year, and all behavioural outcome measures used within a study were extracted; non-behavioural parameters (e.g., neurophysiological measurements) were not included. <h3>Data Synthesis</h3> A total of 674 RCTs met inclusion. Over 600 outcome measures were identified, with the majority appearing in less than 5 articles. The most commonly used measures included: the Glasgow Coma Scale, the Glasgow Outcome Scale, Injury Severity Score and the Trail Making Test. ICF domains of body function, activities and participation, environmental factors and personal factors were all well covered by the literature; the greatest number of outcome measures fell under ICF Mental Functions. <h3>Conclusions</h3> Given the large number of outcome measures identified, continued efforts should be made in identifying a core set of outcomes to be used in TBI trials. A core set of measures would assist researchers in selecting strong instruments for their trials and allow for consistency and comparisons across the literature. <h3>Author(s) Disclosures</h3> None.

Randomized Trial of Tocilizumab in the Treatment of Refractory Adult Polymyositis and Dermatomyositis.

To assess the efficacy and tolerability of tocilizumab in a multicenter, randomized, double-blind, placebo-controlled trial in refractory adult patients with dermatomyositis (DM) and polymyositis (PM). Thirty-six subjects with probable or definite DM/PM were enrolled in a 6-month phase 2B clinical trial and randomized 1:1 to receive tocilizumab (8 mg/kg intravenously) or placebo every 4 weeks for 24 weeks. Eligible subjects had either a DM rash, a myositis-associated autoantibody or an adjudicated PM diagnosis. Active disease was defined by at least three of six abnormal core set measures (CSMs), including a manual muscle testing (MMT)-8 score of less than 136/150. If the MMT-8 score was greater than 136, then a cutaneous score of 3 or more (10 cm visual analogue scale) was required along with three additional abnormal CSMs indicating disease activity. The primary endpoint compared the Total Improvement Score (TIS) between both arms from week 4 to 24. Secondary outcomes included time to meeting minimal TIS improvement, changes in CSMs, time to worsening, steroid-sparing effect, proportion of subjects meeting more stringent improvement criteria, and safety outcomes. There was no significant difference (P=0.86) in the TIS over 24 weeks between tocilizumab and placebo arms. The secondary endpoints of time to improvement (minimal, moderate, or major), time to worsening, CSM changes, safety outcomes, and steroid-sparing effect were also not significantly different between arms. Tocilizumab was safe and well tolerated but did not meet the primary or secondary efficacy outcomes in refractory DM and PM in this 24-week phase 2B study.

Low-dose rituximab is efficacious in refractory idiopathic inflammatory myopathies.

Rituximab (RTX) use early in the course of refractory idiopathic inflammatory myopathy (IIM) is not well studied. This study sought to determine the short-term efficacy of RTX in a registry-based cohort of refractory IIM. Registry-based observational data about IIM patients receiving RTX between 2018 and 2021 were included. Total improvement score was calculated from the core set measures as per International Myositis Assessment and Clinical Studies group (IMACS) at baseline, 6 months and 12 months of follow-up. Forty-two patients (F:M, 29:13), with a mean (s.d.) age of 39.5 (11.5) years were studied. Majority of patients received RTX for refractory myositis, after a median (interquartile range) duration of 8 (4,18) months. Twenty-eight received RTX at a dosage of 1 g × two doses, while 14 received 500 mg × two doses with an interval of 15 days. At 6 months and 12 months post-RTX, the improvement was recorded in manual muscle testing (MMT-8) scores, physician global assessment (PGA), patient global assessment (PtGA) and median steroid dosage as compared with the baseline (P < 0.01 for all). A mean (s.d.) improvement of 44.5 (16) and 48.7 (19.2) in total improvement score was recorded at 6 and 12 months, respectively. The change in MMT-8, PGA and PtGA scores from baseline between the two dosage regimens of RTX were comparable at 6 and 12 months. Severe lower respiratory tract infections requiring hospitalization occurred in three patients of the cohort. RTX improved IMACS core set measures and had steroid sparing efficacy at 6 and 12 months in patients with IIM in this registry-based study. Rituximab as an induction regimen of two doses of 500 mg can be as efficacious as 1 g at 6 months and 12 months of follow-up.

Current status of clinical outcome measures in inclusion body myositis: a systematised review.

Sporadic inclusion body myositis (IBM) is a debilitating idiopathic inflammatory myopathy (IIM) which affects hand function, ambulation, and swallowing. There is no approved pharmacological therapy for IBM, and there is a lack of suitable outcome measure to assess the effect of an intervention. The IBM scientific interest group under IMACS reviewed the previously used outcome measures in IBM clinical studies to lay the path for developing a core set of outcome measures in IBM. In this systematised review, we have extracted all outcome measures reported in IBM clinical studies to determine what measures were being used and to assess the need for optimising outcome measures in IBM. We found 13 observational studies, 17 open-label clinical trials, and 15 randomised control trials (RCTs) in IBM. Six-minute walk distance, IBM-functional rating scale (IBM-FRS), quantitative muscle testing, manual muscle testing, maximal voluntary isometric contraction testing, and thigh muscle volume measured by MRI were used as primary outcome measures. Twelve different outcome measures of motor function were used in IBM clinical trials. IBM-FRS was the most used measure of functionality. Swallowing function was reported as a secondary outcome measure in only 3 RCTs. There are inconsistencies in using outcome measures in clinical studies in IBM. The core set measures developed by the IMACS group for other IIMs are not directly applicable to IBM. As a result, there is an unmet need for an IBM-specific core set of measures to facilitate the evaluation of new potential therapeutics for IBM.

A core outcome set for clinical studies of adhesive small bowel obstruction.

Adhesive small bowel obstruction (ASBO) is a common surgical emergency condition. Research in the field is plentiful; however, inconsistency in outcome reporting makes comparisons challenging. The aim of this study was to define a core outcome set (COS) for studies of ASBO. The long list of outcomes was identified through systematic review, and focus groups across different geographical regions. A modified Delphi consensus exercise of three rounds was undertaken with stakeholder groups (patients and clinicians). Items were rated on a 9-point Likert scale. Items exceeding 70% rating at 7-9 were passed to the consensus meeting. New item proposals were invited in round 1. Individualised feedback on prior voting compared to other participants was provided. An international consensus meeting was convened to ratify the final COS. In round 1, 56 items were rated by 118 respondents. A total of 18 items reached consensus, and respondents proposed an additional 10 items. Round 2 was completed by 90 respondents, and nine items achieved consensus. In round 3, 80 surveys were completed; one item achieved consensus, and five borderline items were identified. The final COS included 26 outcomes, mapped to the following domains: Interventions, need for stoma, septic complications, return of gut function, patient reported outcomes, and recurrence of obstruction, as well as mortality, failure to rescue, and time to resolution. This COS should be used in future studies in the treatment of adhesive SBO. Further studies to define a core measurement set are needed to identify the optimum tools to measure each outcome.

Advancing Policy and Practice in Medicaid Home and Community-Based Services Quality.

This policy brief highlights recent developments and future directions in the Medicaid Home and Community Based Services (HCBS) quality policy and practice within the US. Background is provided about the structure of Medicaid HCBS within the US, the changing landscape of payment and service delivery, and implications for HCBS quality measurement and use. An overview of a HCBS quality framework is provided that was developed with stakeholder input. Frequently used survey tools, existing quality measures, and measure development are discussed. Actionable recommendations are made, including establishment of stakeholder input mechanisms, enhanced federal guidance on a core set of measures, improved data collection and stratification to address equity, multiple mechanisms to assess quality, and increased federal investment in HCBS quality infrastructure.

Development of a core measurement set for research in degenerative cervical myelopathy: a study protocol (AO Spine RECODE-DCM CMS)

IntroductionProgress in degenerative cervical myelopathy (DCM) is hindered by inconsistent measurement and reporting. This impedes data aggregation and outcome comparison across studies. This limitation can be reversed by developing a...

P114 A service evaluation of the Myositis clinic at London North West University Healthcare Trust

Abstract Background/Aims The idiopathic inflammatory myopathies (IIM) are a heterogeneous group of rare diseases, primarily characterised by inflammation of skeletal muscle which leads to muscle weakness, but often involving several other organ systems. The myositis service at London North West University Healthcare Trust (LNWUH) was created in 2017 with the aim of standardising care and improving clinical outcomes for patients. The International Myositis Assessment and Clinical Studies Group (IMACS) established disease activity core set measures to validate clinical response to treatment in studies, but the optimal treatment and real world data on clinical outcomes for patients with IIM is not well described. We undertook a service evaluation to understand the cohort, assess the effectiveness of the service, and in order to inform future service improvements for patients with IIM. Methods We performed a retrospective analysis of the IIM cohort at LNWUH as of January 2021, which consisted of 40 patients. Data were gathered from electronically stored notes and results. Results 65% of our cohort were female, 35% were male. The age range was 18-85. The most common subgroup was dermatomyositis (22.5%). The presenting symptoms included muscle weakness (82.5%), dysphagia (32.5%) and interstitial lung disease (ILD) (27.5%). The most common primary antibody was HMG-CoA reductase, followed by anti-Jo1. More than one antibody was present in 47.5% of patients. three patients had no detectable antibody. 1 in 5 patients had a normal creatine kinase (CK) at baseline. 89% had a raised troponin at baseline, with only two of these having confirmed cardiac involvement on imaging (echocardiogram or cardiac MRI). 30% of the cohort are currently taking mycophenolate mofetil (MMF), 27.5% are taking methotrexate, and 20% are taking hydroxychloroquine. 30% of patients have received rituximab, 20% intravenous immunoglobulin (IVIg), and 7.5% cyclophosphamide. Normalisation of CK was seen in 80% of patients, compared with 20% for pulmonary function tests (PFTs). 63% of patients were in clinical remission one year after diagnosis. Three patients died, two from MDA5-positive rapidly progressive interstitial lung disease. Conclusion In our cohort of patients with IIM, most patients had a significant improvement in their CK and muscle strength with treatment, but there remain challenges. We have established a baseline demographic and clinical outcome data for our cohort that will be used to make service improvements in line with the BSR guideline in development “Adult patients with idiopathic inflammatory myopathy”, and to assess future performance of the service. Disclosure I. Welding: None. A. Isaacs: None.

Stakeholder Engagement: Bridging Research and Policy to Improve Measurement and Dental Care for Children in Medicaid.

Stakeholder Engagement: Bridging Research and Policy to Improve Measurement and Dental Care for Children in Medicaid.

Characterization of stroke-related upper limb motor impairments across various upper limb activities by use of kinematic core set measures

BackgroundUpper limb kinematic assessments provide quantifiable information on qualitative movement behavior and limitations after stroke. A comprehensive characterization of spatiotemporal kinematics of stroke subjects during upper limb daily living activities is lacking. Herein, kinematic expressions were investigated with respect to different movement types and impairment levels for the entire task as well as for motion subphases.MethodChronic stroke subjects with upper limb movement impairments and healthy subjects performed a set of daily living activities including gesture and grasp movements. Kinematic measures of trunk displacement, shoulder flexion/extension, shoulder abduction/adduction, elbow flexion/extension, forearm pronation/supination, wrist flexion/extension, movement time, hand peak velocity, number of velocity peaks (NVP), and spectral arc length (SPARC) were extracted for the whole movement as well as the subphases of reaching distally and proximally. The effects of the factors gesture versus grasp movements, and the impairment level on the kinematics of the whole task were tested. Similarities considering the metrics expressions and relations were investigated for the subphases of reaching proximally and distally between tasks and subgroups.ResultsData of 26 stroke and 5 healthy subjects were included. Gesture and grasp movements were differently expressed across subjects. Gestures were performed with larger shoulder motions besides higher peak velocity. Grasp movements were expressed by larger trunk, forearm, and wrist motions. Trunk displacement, movement time, and NVP increased and shoulder flexion/extension decreased significantly with increased impairment level. Across tasks, phases of reaching distally were comparable in terms of trunk displacement, shoulder motions and peak velocity, while reaching proximally showed comparable expressions in trunk motions. Consistent metric relations during reaching distally were found between shoulder flexion/extension, elbow flexion/extension, peak velocity, and between movement time, NVP, and SPARC. Reaching proximally revealed reproducible correlations between forearm pronation/supination and wrist flexion/extension, movement time and NVP.ConclusionSpatiotemporal differences between gestures versus grasp movements and between different impairment levels were confirmed. The consistencies of metric expressions during movement subphases across tasks can be useful for linking kinematic assessment standards and daily living measures in future research and performing task and study comparisons.Trial registration: ClinicalTrials.gov Identifier NCT03135093. Registered 26 April 2017, https://clinicaltrials.gov/ct2/show/NCT03135093.

Clinical outcome measures and their evidence base in degenerative cervical myelopathy: a systematic review to inform a core measurement set (AO Spine RECODE-DCM)

ObjectivesTo evaluate the measurement properties of outcome measures currently used in the assessment of degenerative cervical myelopathy (DCM) for clinical research.DesignSystematic reviewData sourcesMEDLINE and EMBASE were searched through 4 August...

Toward a Model for Measuring Social and Structural Determinants of Alzheimer’s Disease and Related Dementias

Social and structural determinants of health (SSDoH) are conditions in the environments in which individuals are born, live, learn, work, play, worship, and age that affect health, functioning, and quality-of-life outcomes across the life course. Growing evidence suggests that SSDoH can help to explain heterogeneity in cognitive, functional, and interventional outcomes in Alzheimer’s disease and related disorders research and clinical practice. The National Institute on Aging (NIA) has prioritized collecting SSDoH data in order to elucidate disease mechanisms and aid discovery of a disease modifying treatments. However, a major nexus of ADRD research, the national network of Alzheimer’s Disease Research Centers (ADRCs), does not routinely collect SSDoH data. We describe a model for feasibly gathering and analyzing SSDoH data across Alzheimer’s Disease Research Centers (ADRCs). We lay out theoretical underpinnings of key constructs and their measure, empirical evidence for their importance, and their potential for elucidating disease and prevention mechanisms. Toward a goal of translation, we describe a general approach to measuring SSDoH along with core set of measures. We also describe empirical support and rationales for assessing SSDoH in standing geographically and culturally diverse research cohorts, and guiding considerations in selecting modules to serve unique communities. We specifically address SSDoH in Black, Hispanic/Latin, and refugee populations with an eye toward conveying how geographic proximity, socioeconomic status, ethnoracial factors, and sex\\gender\\sexual orientation affect populations in ways directly relevant to Alzheimer’s disease (AD) and Alzheimer’s disease related dementias (ADRD).

Establishing a Framework for Gathering Structural and Social Determinants of Health in Alzheimer's Disease Research Centers.

Structural and social determinants of health (SSDoH) are environmental conditions in which individuals are born, live, learn, work, play, worship, and age that affect health, functioning, and quality-of-life outcomes across the life course. Growing evidence suggests that SSDoH can help to explain heterogeneity in outcomes in Alzheimer's disease and Alzheimer's disease and related dementias (AD/ADRD) research and clinical practice. The National Institute on Aging has prioritized collecting SSDoH data to elucidate disease mechanisms and aid discovery of disease-modifying treatments. However, a major nexus of AD/ADRD research, the national network of Alzheimer's Disease Research Centers (ADRCs), collects few SSDoH data. We describe a framework for feasibly gathering and modeling SSDoH data across ADRCs. We lay out key constructs, their measures, and empirical evidence for their importance in elucidating disease and prevention mechanisms. Toward a goal of translation, the framework proposes a modular structure with a core set of measures and options for adjunctive modules. We describe considerations for measuring SSDoH in existing geographically and culturally diverse research cohorts. We also outline a rationale for universal implementation of a set of SSDoH measures and juxtapose the approach with alternatives aimed at collecting SSDoH data.

Assessment of disability in idiopathic inflammatory myopathy: a call for linearity.

ObjectivesTo evaluate the clinimetric properties of the Academic Medical Centre Disability Score (ALDS) in patients with idiopathic inflammatory myopathy (IIM).MethodsWe used prospectively collected data of IIM patients who completed a phase-2 study with first-line IVIG monotherapy. The ALDS is a patient-reported questionnaire which contains 25 items relevant for disability in myositis. ALDS and all core set measures (CSMs) for myositis [including HAQ-Disability Index (HAQ-DI)] were evaluated at baseline and 9 weeks follow-up. In addition, the 2016 ACR/EULAR myositis response criteria outcome called Total Improvement Score (TIS) was evaluated at 9 weeks. We examined floor/ceiling effects, reliability and construct validity of the ALDS. To examine known-group validity, ALDS change scores over time were compared with TIS and physician impression of clinical response.ResultsNineteen patients with IIM [median age 59 years, 12 (63%) female] were enrolled. At baseline, ALDS showed a median score of 65.4 (IQR 58.2–73.5), good Cronbach’s alpha (α = 0.84) and a small ceiling effect (11%). Construct validity was confirmed by moderate to strong correlations between ALDS and HAQ-DI [rs = −0.57 (baseline); −0.86 (follow-up)]. ALDS change score correlated with TIS (rs = 0.70), discriminated between responders and non-responders (TIS ≥ 40; P = 0.001), between groups based on physician impression of clinical response (P = 0.03), and detected deterioration.ConclusionThe ALDS showed promising clinimetric properties and detected relevant changes in disability in patients with myositis. These results warrant further investigations.

Validation of two simple patient-centered outcome measures for virtual monitoring of patients with idiopathic inflammatory myositis.

BackgroundTelemedicine has changed the landscape of patient care with wider use of patient-centered outcome measures (PCOMs). We evaluated two novel task–based PCOMs namely ten times arm lift (AL) test and two-minute walk distance (2MWD) in idiopathic myositis (IIM).MethodsThis was a cross-sectional observational study with the enrolment of adult IIM (ACR/EULAR criteria) patients with active/inactive disease. Active disease was defined as any two of increase in immunosuppression within 3 months, elevated muscle enzymes, physician VAS ≥ 2, worsened cutaneous disease, or fall in MMT8 < 76. Standard myositis core set measures (CSMs) were evaluated and test–retest validity [Cronbach’s alfa (CA)], construct validity (Pearson’s correlation), and discriminant validity (between active/inactive IIM) were assessed. The results were further validated in a separate tele-rheumatology cohort.ResultsAmong 22 IIM patients (68%-female) of age 30.5(19–62) years, AL and 2MWD showed excellent test–retest reliability (CA-0.987, 0.99). AL exhibited moderate-strong correlation with all CSMs except CK levels and MDI. In contrast, 2MWD values were highly variable without CSM correlation. A higher AL time discriminated active and inactive myositis (16.6 vs 11 s, p = 0.006) with an AUC of 0.882 (p = 0.006). AL > 12.8 s had 94% negative predictive value (NPV) for active muscle disease. In the validation cohort (47 patient visits among 26 patients), AL significantly differentiated between active vs. inactive disease with an NPV of 95%.ConclusionsAL test exhibits pilot evidence of construct and discriminant validity in patients with IIM requiring further evaluation. 2MWD was not a good test for outcome evaluation of IIM patients. Key Points• Novel task–based patient-centered outcome measures were evaluated for remote monitoring of muscle strength in IIM.• Ten times arm lift (AL) test showed strong test–retest reliability as well as provide pilot evidence of construct and discriminant validity in patients with IIM unlike 2-min walk distance.• This provides preliminary evidence to further evaluate the role of AL as patient-centered outcome measure in patients with IIM for virtual clinical trials.Supplementary InformationThe online version contains supplementary material available at 10.1007/s10067-021-05990-3.