Clinician-reported Outcome Measures Research Articles

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.

Effectiveness of the KC@H programme compared with clinic-based rehabilitation in patients recovering from ACL reconstruction: a study protocol for a single-centre, two-arm, single-blinded, randomised controlled superiority trial

Patients who cannot fully comply with conventional clinic-based rehabilitation (CR) sessions after ACL reconstruction (ACLR) may find additional internet-based sessions beneficial. These remote sessions include therapeutic exercises that can be...

Validity of the modified Rankin Scale in patients with aneurysmal subarachnoid hemorrhage: a randomized study

PurposeThe modified Rankin Scale (mRS), a clinician-reported outcome measure of global disability, has never been validated in patients with aneurysmal subarachnoid hemorrhage (aSAH). The aims of this study are to assess: (1) convergent validity of the mRS; (2) responsiveness of the mRS; and (3) the distribution of mRS scores across patient-reported outcome measures (PROMs).MethodsThis is a prospective randomized multicenter study. The mRS was scored by a physician for all patients, and subsequently by structured interview for half of the patients and by self-assessment for the other half. All patients completed EuroQoL 5D-5L, RAND-36, Stroke Specific Quality of Life scale (SS-QoL) and Global Perceived Effect (GPE) questionnaires. Convergent validity and responsiveness were assessed by testing hypotheses.ResultsIn total, 149 patients with aSAH were included for analysis. The correlation of the mRS with EQ-5D-5L was r = − 0.546, while with RAND-36 physical and mental component scores the correlation was r = − 0.439and r = − 0.574 respectively, and with SS-QoL it was r = − 0.671. Three out of four hypotheses for convergent validity were met. The mRS assessed through structured interviews was more highly correlated with the mental component score than with the physical component score of RAND-36. Improvement in terms of GPE was indicated by 83% of patients; the mean change score of these patients on the mRS was − 0.08 (SD 0.915). None of the hypotheses for responsiveness were met.ConclusionThe results show that the mRS generally correlates with other instruments, as expected, but it lacks responsiveness. A structured interview of the mRS is best for detecting disabling neuropsychological complaints.RegistrationURL: https://trialsearch.who.int; Unique identifier: NL7859, Date of first administration: 08-07-2019

CO92 Clinician-Reported Outcome Measure for Palonosetron Versus Standard of Care in Chemotherapy-Induced Nausea and Vomiting in Egypt

CO92 Clinician-Reported Outcome Measure for Palonosetron Versus Standard of Care in Chemotherapy-Induced Nausea and Vomiting in Egypt

Occupational Therapy Assessments of Cortical or Cerebral Visual Impairment: A Systematic Review.

Cortical or cerebral visual impairment (CVI), a leading cause of visual dysfunction in children across the globe, can result in delayed development of fine and gross motor skills, functional mobility, and socialization, which affects all areas of occupation. No assessments are recommended for occupational therapy practitioners working with children with CVI. To appraise the measurement properties and clinical utility of CVI assessments appropriate for use by occupational therapy practitioners. MEDLINE via PubMed, Scopus, and Web of Science. We followed the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) guidelines for systematic reviews of patient-reported outcome measures for relevant studies. A total of 590 records yielded 15 studies of the measurement properties of 12 assessment tools, 8 patient-reported outcome measures and 4 clinician-reported or performance-based outcome measures. We report on the quality of measurement properties of 10 of the 12 outcome measures. None had high-quality evidence of content validity, structural validity, or test-retest reliability. Most had indeterminate internal consistency as a result of a lack of confirmatory factor analysis. Three instruments demonstrated high quality and sufficient construct validity. On the basis of the COSMIN guidelines, all 12 outcome measures have the potential to be recommended but require further study before definitive recommendations can be made. What This Article Adds: This is the first systematic review of assessments of CVI feasible for use by occupational therapy practitioners. High-quality studies of measurement properties are lacking. Further examination of the measurement properties of outcome measures is required.

Orofacial Pain Clinician Reported Outcome Measure: Development of a Scoring System Related to Patient Symptoms

Orofacial Pain Clinician Reported Outcome Measure: Development of a Scoring System Related to Patient Symptoms

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

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. 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. 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. 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.

Botulinum Toxin for the Management of Pelvic Floor Tension Myalgia and Persistent Pelvic Pain: A Systematic Review and Meta-analysis.

To systematically review the literature on use of botulinum toxin to treat pelvic floor tension myalgia and persistent pelvic pain. The ClinicalTrials.gov, PubMed, EMBASE, and Scopus databases were searched from inception to November 2022 by two independent assessors (B.L.K. and F.G.L.). Identified studies were screened by title and abstract and included after full-text review. Data extraction was subsequently performed and recorded in Microsoft Excel. This study was performed in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-analyses guidelines after registration in PROSPERO (CRD42022289132). All randomized studies, prospective studies with more than five participants, and retrospective studies with more than 10 participants published in English or French and assessing the use of botulinum toxin for the treatment of pelvic floor tension myalgia and persistent pelvic pain in women were included. Meta-analyses were performed on randomized data. Of 4,722 articles identified, 24 satisfied inclusion criteria. A meta-analysis of five randomized controlled trials totaling 329 participants demonstrated no differences in patient- and clinician-reported outcome measures, including pain, dyspareunia, sexual function, and vaginal manometry. Mean duration of follow-up was 6 months. A qualitative analysis of 14 prospective and four retrospective studies including 804 participants is supportive of botulinum toxin; however, the quality of data is low, and there is marked heterogeneity between studies. Meta-analyses of randomized data do not support the use of botulinum toxin to treat pelvic floor tension myalgia and persistent pelvic pain. Failure of these data to confirm the findings of nonrandomized prospective studies that suggest a treatment benefit may be attributable to the absence of placebo control and confounding outcomes obtained from an active comparator group. Further randomized controlled trials with true placebo are strongly recommended. PROSPERO, CRD42022289132.

Clinician-reported outcome measures for the assessment of vitiligo: A scoping review.

Clinician-reported outcome measures (ClinROMs) are essential for assessment of vitiligo in clinical trials and daily practice. Several instruments have been developed and tested to measure, for example, vitiligo extent, repigmentation and activity. The goal of this review was to identify all introductory publications of ClinROMs for vitiligo that include at least some aspects of validation and to describe the instruments' characteristics, intention for use and practical strengths and limitations. A search strategy was conducted in PubMed, Embase and Cochrane Library (CENTRAL) from inception to July 2022. Based on the literature search (n = 2860), 10 articles were identified, describing 14 different ClinROMs. Six ClinRoms measured disease extent and/or repigmentation, seven evaluated disease activity and one was a composite score. The Vitiligo Area Scoring Index (VASI), and Vitiligo Extent Score (VES and VESplus) measure overall disease extent and/or repigmentation. The VASI relies on hand units (1% body surface area), whereas the VES and VESplus use a picture-based scoring technique. The Vitiligo Extent Score for a Target Area (VESTA) measures repigmentation percentage for target lesions. One global assessment score for extent has been validated. Vitiligo disease activity scores included a static measure of clinical activity signs (Vitiligo Signs of Activity Score [VSAS]) and two measures assessing dynamic evolution (Vitiligo Disease Activity Score [VDAS] and Vitiligo Disease Improvement Score [VDIS]). The Vitiligo European Task Force assessment tool (VETFa) is a composite score. Depending on the practical strengths and limitations as well as the research question and setting (clinical trials vs. daily practice), the choice of an appropriate ClinROM may differ. Fourteen ClinROMs in vitiligo were identified to measure vitiligo extent, repigmentation, and activity. Further research evaluating the validity, reliability, and responsiveness of each instrument and worldwide consensus on which instrument to use for a specific outcome (domain) is greatly needed.

P64 Efficacy of baricitinib in patients with different degrees of severe alopecia areata: 52-week results from BRAVE-AA1 and BRAVE-AA2

Abstract Baricitinib, an oral selective Janus kinase (JAK)1/JAK2 inhibitor, demonstrated efficacy in adults with severe alopecia areata (AA) over 36 weeks in two phase III, randomized, double-blind, placebo-controlled trials, BRAVE-AA1 (NCT03570749) and BRAVE-AA2 (NCT03899259). Using integrated data from the long-term extension periods of both trials, we report treatment response to baricitinib by the AA severity subgroup based on the Severity of Alopecia Tool (SALT) score over 52 weeks. Patients randomized to baricitinib at baseline (n = 855) retained their treatment allocation (2 mg/4 mg) through to week 52 in both trials and were included in week 52 analyses; placebo nonresponders were rescued at week 36. Severity of AA at baseline was defined as severe (SALT score 50–94) or very severe (SALT score 95–100). Key inclusion criteria were SALT score ≥ 50, current AA episode lasting &amp;gt; 6 months to &amp;lt; 8 years without spontaneous improvement within 6 months prior to screening, and no previous inadequate response to oral JAK inhibitors. Efficacy endpoints included the proportion of patients achieving a SALT score ≤ 20 and the proportion of patients achieving Clinician-Reported Outcome (ClinRO) Measure for Eyebrow/Eyelash Hair Loss™ 0,1 (no, minimal gaps) with ≥ 2-point improvement from baseline among patients with baseline scores 2,3 (significant gaps, no notable eyebrows/eyelashes). Analyses were presented descriptively for each subgroup up to week 52. Nonresponder imputation was used for missing data. Data collected after permanent study drug discontinuation or at remote visits due to the COVID-19 pandemic were excluded. In total, 1200 patients were enrolled (BRAVE-AA1, n = 654; BRAVE-AA2, n = 546). At baseline, the overall mean (SD) age was 37.5 (12.9) years; 60.7% were female and 46.8% and 53.2% of patients had severe and very severe AA, respectively. At week 52, a SALT score ≤ 20 was achieved by 36.1% and 51.2% of patients on baricitinib 2 mg and baricitinib 4 mg, respectively, with severe AA and 12.4% and 27.7% of patients on baricitinib 2 mg and baricitinib 4 mg, respectively, with very severe AA. Eyebrow/Eyelash Hair Loss™ scores 0,1 with ≥ 2-point improvement from baseline at week 52 were achieved by 19.4% and 26.0% (baricitinib 2 mg) and 37.5% and 38.5% (baricitinib 4 mg) of patients with severe AA and 24.3% and 25.3% (baricitinib 2 mg) and 47.6% and 48.1% (baricitinib 4 mg) of patients with very severe AA. Following 52 weeks of treatment with both baricitinib doses, clinical responses of SALT score ≤ 20 were observed both in patients with severe and very severe AA. Proportions of patients achieving full coverage or minimal gaps in eyebrows and eyelashes were similar at week 52, regardless of scalp hair-loss severity. Funding sources: Eli Lilly and Company.

The International Standard Set of Outcome Measures for the Assessment of Hearing in People with Osteogenesis Imperfecta.

The aim is to recommend a minimum standard set of clinician-reported outcome measures (CROMs) and patient-reported outcome measures (PROMs) on hearing for people with osteogenesis imperfecta (OI). This project is part of the larger "Key4OI" project initiated by the "Care4BrittleBones foundation" of which the goal is to improve quality of life of people with OI. Key4OI provides a standard set of outcome measures and covers a large set of domains affecting the well-being of people with OI. An international team of experts in OI, comprising specialists in audiological science, medical specialists, and an expert patient representative, used a modified Delphi consensus process to select CROMs and PROMs to evaluate hearing problems in people with OI. In addition, focus groups of people with OI identified key consequences of their hearing loss. These criteria were matched to categories of preselected questionnaires to select a PROM that matched their specific hearing-related concerns best. Consensus on PROMs for adults and CROMs for adults and children was reached. The focus of the CROMs was on specific audiological outcome measures and standardized follow-up. This project resulted in a clear consensus statement for standardization of hearing-related PROMs and CROMs and follow-up management of patients with OI. This standardization of outcome measurements will facilitate comparability of research and easier international cooperation in OI and hearing loss. Furthermore, it can improve standard of care in people with OI and hearing loss by incorporating the recommendations into care pathways.

Developing an Edema Clinician-Reported Outcome Measure for Nephrotic Syndrome

Introduction: Edema is a common manifestation of proteinuric kidney diseases, but there is no consensus approach for reliably evaluating edema. The objective of this study was to develop an edema clinician-reported outcome measure for use in patients with nephrotic syndrome. Methods: A literature review was conducted to assess existing clinician-rated measures of edema. Clinical experts were recruited from internal medicine, nephrology, and pediatric nephrology practices to participate in concept elicitation using semi-structured interviews and cognitive debriefing. Qualitative analysis methods were used to collate expert input and inform measurement development. In addition, training and assessment modules were developed using an iterative process that also utilized expert input and cognitive debriefing to ensure interrater reliability. Results: While several clinician-rated measures of edema have been proposed, our literature review did not identify any studies to support the reliability or validity of these measures. Fourteen clinician experts participated in the concept elicitation interviews, and twelve participated in cognitive debriefing. A clinician-reported outcome measure for edema was developed. The measure assesses edema severity in multiple individual body parts. An online training module and assessment tool were generated and refined using additional clinician input and investigative team expertise. Conclusion: The Edema ClinRO (V1) measure is developed specifically to measure edema in nephrotic syndrome. The tool assesses edema across multiple body parts, and it includes a training module to ensure standardized administration across raters. Future examination of this measure is ongoing to establish its reliability and validity.

Implementation of the vitiligo area scoring index in clinical studies of patients with vitiligo: a scoping review.

The vitiligo area scoring index (VASI) is a validated, reliable clinician-reported outcome measure widely used to assess the extent of skin depigmentation seen in patients with vitiligo and to measure patient responses to therapies for vitiligo in clinical trials. However, its implementation in studies is inconsistent and makes comparing results across different studies difficult. The aim of this scoping review is to summarize interventional clinical studies that applied the VASI to measure vitiligo and identify variability in VASI implementation. A systematic search of Ovid Medline, Embase, Web of Science, Cochrane, and ClinicalTrials.gov was performed. Interventional studies published between January 1946 and October 2020 that used the VASI as an outcome measure for assessing vitiligo response were reviewed for methodological approach. Great heterogeneity was found within the 55 included interventional studies that used VASI as an outcome measure. A total of 9 VASI subtypes were described by the authors within 10 intervention categories. VASI determined study eligibility in one study. Body surface area was most frequently established using inconsistent methods. We found unclear or ambiguously scaled assessments of depigmentation. Most VASI outcomes were reported as mean absolute difference, percentage VASI improvement, and percentage of patients who achieved the VASI endpoint. The VASI score was over 100 in one study. Our scoping review revealed many VASI methodology variations in interventional clinical studies of vitiligo. While VASI is a standard method to measure vitiligo changes, substantial heterogeneity in methodology limits reliable comparison and interpretation of findings from different clinical trials. Our findings may be used as a foundation to standardize the VASI outcome measure methodology, allowing for improved clinician training and rigorous data analysis across vitiligo research groups worldwide.

Evaluation of the LFA-REAL clinician-reported outcome (ClinRO) and patient-reported outcome (PRO): prespecified analysis of the phase III ustekinumab trial in patients with SLE

ObjectiveThe Lupus Foundation of America Rapid Evaluation of Activity in Lupus (LFA-REAL) system is a novel and simple SLE disease activity instrument, consisting of a tandem clinician-reported (ClinRO) and patient-reported...

The Association between Acute and Late Genitourinary and Gastrointestinal Toxicities: An Analysis of the PACE B Study

Several studies have demonstrated the association between acute and late radiotherapy toxicity in prostate cancer using older radiotherapy techniques. However, whether this association is present with newer techniques such as stereotactic body radiotherapy (SBRT), remains unclear. We use univariable and multivariable logistic regression to analyse the association between grade 2 or worse acute gastrointestinal (GI) and genitourinary (GU) toxicities with equivalent late toxicities in patients treated with SBRT and conventional or moderately fractionated radiotherapy (CRT) within the PACE-B study. 842 patients were included in this analysis. Common Terminology Criteria for Adverse Events (CTCAE) was the primary clinician reported outcome measure used in this analysis. In univariable analysis, experiencing a grade 2+ acute GU toxicity was significantly associated with developing a grade 2+ late GU toxicity after SBRT (OR 4.63, 95% CI (2.96-7.25), p < 0.0001) and CRT (OR 2.83, 95% CI (1.69-4.71), p < 0.0001). This association remained significant in multivariable analysis. In univariable analysis, experiencing a grade 2+ acute GI toxicity was also associated with developing a grade 2+ late GI toxicity after SBRT (OR 3.67, 95% CI (1.91-7.03), p < 0.0001) and CRT (OR 4.4, 95% CI (2.04-9.47), p < 0.0001). This association also remained significant in multivariable analysis. Grade 2+ baseline GU symptoms were also associated with grade 2+ late urinary toxicity in both univariable and multivariable analysis. Overall, acute toxicity is an important predictor variable for late GU/GI toxicity after localised prostate radiotherapy using SBRT and CRT. Future work should test whether optimising symptoms pre-treatment and early intervention in those with significant acute toxicities could mitigate the development late of toxicity.

What outcomes have been reported on patients following open lower limb fracture, and how have they been measured?

Open lower limb fracture is a life-changing injury affecting 11.5 per 100,000 adults each year, and causes significant morbidity and resource demand on trauma infrastructures. This study aims to identify what, and how, outcomes have been reported for people following open lower limb fracture over ten years. Systematic literature searches identified all clinical studies reporting outcomes for adults following open lower limb fracture between January 2009 and July 2019. All outcomes and outcome measurement instruments were extracted verbatim. An iterative process was used to group outcome terms under standardized outcome headings categorized using an outcome taxonomy. A total of 532 eligible studies were identified, reporting 1,803 outcomes with 786 unique outcome terms, which collapsed to 82 standardized outcome headings. Overall 479 individual outcome measurement instruments were identified, including 298 outcome definitions, 27 patient- and 18 clinician-reported outcome measures, and six physical performance measures. The most-reported outcome was 'bone union/healing' reported in over 50% of included studies, while health-related quality of life was only measured in 6% of included studies. Outcomes reported for people recovering from open lower limb fracture are heterogeneous, liable to outcome reporting bias, and vary widely in the definitions and the measurement tools used to collect them. Outcomes likely to be important to patients, such as quality of life and measures of physical functioning, have been neglected. This systematic review identifies the need to unify outcome measures reported on patients recovering from open lower limb fracture; this may be addressed by creating a core outcome set.

358 A poor correlation between clinician-reported outcomes and patient-reported outcomes is observed in non-White patients with atopic dermatitis

Abstract Atopic dermatitis (AD) manifests differently across diverse patient populations, potentially leading to challenges in its assessment and management.1 Additionally, pigment masking may obscure erythema and other AD signs leading to misclassification of AD severity in patients with darker phototypes.2,3 We hypothesized that clinician-reported outcome measures may not perform as well in non-White vs. White patients. To test this, we examined whether clinician-reported outcome measures have weaker correlations with established patient-reported outcome measures across different races or ethnicities. A prospective, dermatology practice-based study was performed in children and adults with AD as defined by the Hanifin-Rajka diagnostic criteria. Patients were enrolled sequentially between January 2014 and September 2019. The study was approved by the institutional review board of Northwestern University. Informed consent was obtained electronically. Electronic surveys were completed by patients/caregivers, including self-identified race and Hispanic ethnicity, Numerical Rating Scale (NRS) for average-itch in the past 7 days, and Patient-Oriented Eczema Measure (POEM). Investigator assessments of AD severity were performed by a dermatologist (J.I.S.) and included the Eczema Area and Severity Index (EASI), and the objective component of SCORAD (oSCORAD). Spearman correlations were performed for POEM and NRS-itch vs. oSCORAD and EASI. Correlation coefficients were interpreted as≥0.70 or ≤ −0.70 = very strong, 0.50 to 0.69 or −0.69 to −0.50 = strong, 0.30 to 0.49 or −0.49 to −0.30 = moderate and 0.10 to 0.29 or −0.29 to −0.10 = weak.4 Overall, 1987 patients were included in the study (age &amp;lt;18 years: 101 [5.08%], ≥ 18 years: 1886 [94.92%]), including 198 (9.96%) Black, 360 (18.12%) Asian, 8 (0.40%) multiracial/other, 1313 (66.08%) White race and 108 (5.44%) Hispanic ethnicity. In White patients, POEM and NRS average-itch had strong correlations with oSCORAD and EASI. Whereas in Black patients, POEM had only moderate correlations with oSCORAD and EASI NRS average-itch had weak-moderate correlations with oSCORAD and EASI. Asian/Pacific Islander patients also had numerically weaker correlations of POEM with oSCORAD and EASI compared to White patients, but strong or very strong correlations of NRS average-itch with oSCORAD and EASI. Patients with Hispanic ethnicity also showed weaker correlations for POEM with oSCORAD and EASI compared to Whites, as well as weaker correlations of NRS average-itch with EASI. The modest correlation observed between PROMs and clinician-reported outcome measures in general highlights the importance of measuring both signs and symptoms to fully describe the severity of AD. This is particularly important in non-White patients who had notably weaker correlations between PROMs and clinician-reported outcome measures. The poor correlation between patient-reported outcome measures and clinician-reported outcome measures may be multifactorial, including pigment masking limiting assessment of erythema and other AD signs in patients with darker phototypes,3 more severe pruritus and morphologic variants occurring in Blacks and Asians/Pacific Islanders that may not adequately be represented in oSCORAD and EASI. AD severity is often underestimated in patients with darker phototypes.3 Future efforts are needed to optimize clinician assessments of AD severity in diverse patient populations.

Toward the Definition of Patient-Reported Outcome Measurements in Hereditary Spastic Paraplegia.

Hereditary spastic paraplegias (HSPs) are a heterogeneous group of rare neurodegenerative diseases, characterized by a progressive spastic paraparesis. Currently, there is a HSP-specific clinician-reported outcome measure (CROM) called Spastic Paraplegia Rating Scale (SPRS). There are, however, no specific patient-reported outcome measures (PROMs) for HSP. In the present cohort study, we prospectively follow up a well-examined Austrian HSP cohort using validated rating scales and compared PROM with disease-specific and non-disease-specific CROM. Patients were recruited and followed up at the Center for Rare Movement Disorders, Innsbruck, Austria. CROM included the SPRS, Scale for the Assessment and Rating of Ataxia (SARA), Barthel Index (BI), and Mini-Mental State Examination (MMSE). PROM included the EQ-5D questionnaire and the Patient Health Questionnaire 9 (PHQ-9). Standardized response means (SRMs) were calculated for all scales at follow-up (FU) after 1 year. A total of 55 patients (36 males) with HSP were included in the study. FU was performed for 30 patients (21 males). Apart from females reporting more problems in the EQ-5D domain of anxiety and depression (p = 0.008), other clinician-reported outcomes (CROs) or patient-reported outcomes (PROs) did not differ significantly across sex. SPRS showed significant correlations with SARA (p < 0.001), mainly driven by the gait item, as well as the BI. Although SPRS did not correlate with EQ-5D visual analogue scale and PHQ-9 scores, several EQ-5D domains correlated significantly with SPRS. At FU, SPRS showed the highest responsiveness (SRM 1.11), followed by SARA (SRM 0.47). Neither MMSE nor PRO significantly increased at FU. In this study, we present an Austrian cohort of patients with HSP and a prospective study evaluating correlations of CRO and PRO as well as their progression. Demographics from our cohort are comparable with several other European cohort studies. Our data highlight the capabilities of the SPRS to show clinical progression and warrant consideration of ataxia rating scales such as SARA in HSP cohorts. We also show that the generic PROMs are not suitable to detect change in HSP, and thus, we propose to create a disease-specific PROM fully depicting the effect of HSP on the patients' lives.

The U-shaped association between urbanization and post-stroke disability: A nationwide longitudinal study in China

BackgroundIn the 21st century, China experienced a rapid increase in urbanization, which has enhanced medical service availability, but has also increased exposure to adverse environmental and socioeconomic factors. Urbanization-related factors can affect the prognosis of stroke patients, particularly the progression of neurological disabilities. However, the overall association between urbanization and post-stroke disability remains unknown. MethodA total of 21,678 stroke patients were selected from the China National Stroke Screening Survey (2013–2018); all included patients had at least one follow-up record. The modified Rankin Scale (mRS), a clinician-reported functional outcome measure for recovery from stroke, was utilized to assess post-disability at each visit. A larger mRS score means severer disability. The urbanization level before each visit was evaluated based on 5-year average satellite measurements of night-time light (NTL) or urban land cover (i.e., proportion of impervious surface). We used a fixed-effects model to estimate the association between urbanization level and risk of disability (i.e., mRS score). The non-linear relationship was modelled by penalized spline functions. ResultsAccording to linear models with multivariate adjustment, every 10 digital number (DN) increment in NTL was associated with a 0.050 (95% confidence interval [CI]: 0.026, 0.074) increase in mRS score, and every 10% increment in impervious surface was associated with a 0.052 (95% CI: 0.034, 0.070) increase in mRS score. The association was stronger in patients diagnosed with atrial fibrillation, dyslipidemia, hypertension, or diabetes compared with the corresponding reference group. Non-linear analysis showed a U-shaped relationship between urbanization and the mRS score, which indicates that high and low urbanization levels were both associated with an increased risk of post-stroke disability. ConclusionsOur study revealed a complex association between urbanization and post-stroke disability. The optimal level of urbanization to prevent neurological disabilities in stroke patients could be inferred from the U-shaped curve. The reasons for the complex association between urbanization and post-stroke disability should be further explored to establish causality.

Content Validation of the Movement Disorder-Childhood Rating Scale (MD-CRS) for Dyskinetic Cerebral Palsy

BackgroundDyskinetic cerebral palsy (DCP), a lifelong neurological disorder beginning in early childhood, manifests with hyperkinetic movements and dystonia. The Movement Disorder-Childhood Rating Scale (MD-CRS) is a clinician-reported outcome measure assessing the intensity of movement disorders and their effect on daily life in pediatric patients. Content validity of clinical outcome assessments is key to accurately capturing patient perspective. Evidence demonstrating content validity of the MD-CRS in patients with DCP is needed. This study captures input from patients with DCP and their caregivers regarding the content validity of the MD-CRS. MethodsThis qualitative, noninterventional, cross-sectional study included interviews with children/adolescents (aged six to 18 years) with DCP and caregivers of children with DCP. Participants were asked to describe body regions and daily functions affected by DCP. Caregivers also reviewed MD-CRS Part I to evaluate the relevance of the items and corresponding response options. Descriptions of DCP were coded and mapped to MD-CRS items and response options. Caregiver feedback on MD-CRS Part I was analyzed using inductive content analysis. ResultsEight patients and 12 caregivers were interviewed. Participants confirmed that the body regions and activities listed in the MD-CRS were affected by DCP and that involuntary movements interfered with all motor, oral/verbal, self-care, and video protocol activities. Caregivers endorsed the response options for 12 of 15 items in MD-CRS Part I and suggested clarifications for others. ConclusionsParticipants confirmed that affected body regions and activities listed in the MD-CRS were relevant to their experience with DCP, demonstrating the content validity of this tool in children/adolescents with DCP.