Discordance in perception of disease activity between adolescent patients with lupus and their providers may influence disease outcomes. We found that patients endorsed higher perceptions of disease activity than providers. Discordance was present at all levels of disease activity, particularly in patients with high activity, nephritis, and/or taking corticosteroids or mycophenolate mofetil.

BACKGROUND: SMA is a hereditary neuromuscular disease that causes progressive muscle weakness and atrophy. Several studies have shown that the burden of SMA is very high at many levels. Functional assessment tools currently used do not completely address the impact of the disease in patients\' life. The objective of this qualitative study was to identify aspects of SMA that are relevant to patients and to design items useful for assessment purposes.
RESULTS: Five focus group sessions were run during an annual SMA families meeting in Madrid, Spain. Focus groups were composed by parents of SMA type I children, sitter children type II-III, parents of sitter children type II-III, adult patients, and parents of walker children. Two trained facilitators conducted the focus groups using a semi-structured guideline to cover previously agreed topics based on the input of a Scientific and Patient Advisory Committee. The guideline was adapted for the different groups. According to what was communicated by participants, SMA entails a high burden of disease for both patients and their parents. Burden was perceived in physical, psychological, and social areas. Patient\'s physical domain was the most relevant for participants, especially for parents of non-ambulant children, followed by limitations of motor scales to capture all changes, parents psychological burden, treatment expectations and patient\'s psychological burden. Ten domains were the main areas identified as impacted by the disease: mobility and independence, fatigue and fatigability, infections and hospital consultations, scoliosis and contractures, vulnerability, pain, feeding, time spent in care, breathing, and sleep and rest.
CONCLUSIONS: This study confirms the necessity of evaluating other aspects of the disease that are not assessed in the functional motor scale. Measures of other aspects of the disease, such as pain, fatigue, feeding, should be also considered. A patient-reported outcomes instrument measuring such aspects in a valid and reliable way would be very useful. This study generated a list of new items relevant to be systematically measured in the assessment of the impact of SMA on the patients\' everyday life.

UNASSIGNED: Person-centered goals capture individual priorities in personal contexts. Goal Attainment Scaling (GAS) has been used in drug trials involving people living with dementia (PLWD) but GAS has been characterized as difficult to incorporate into trials and clinical practice. We used GAS in a trial of New Interventions for Independence in Dementia Study (NIDUS)-family, a manualized care and support intervention, as the primary outcome and to tailor the interventions to goals set. We aimed to assess the feasibility and content of baseline goal-setting.
UNASSIGNED: We developed training for nonclinical facilitators to set individualized GAS goals remotely with PLWD and family carer dyads, or carers alone, in the intervention trial, during the COVID-19 pandemic. A qualitative content analysis of the goals set explored participants\' priorities and unmet needs, to consider how existing GAS goal domains might be extended in a psychosocial intervention trial context.
UNASSIGNED: Eleven facilitators were successfully trained to set and score GAS goals. A total of 313/328 (95%) participants were able to collaboratively set three to five goals with the facilitators. Of these, 302 randomized participating dyads set 1043 (mean 3.5, range 3 to 5) goals. We deductively coded 719 (69%) goals into five existing GAS domains (mood, behavior, self-care, cognition, and instrumental activities of daily living); 324 (31%) goals were inductively coded into four new domains: carer break, carer mood, carer behavior, and carer sleep. The most frequently set goals pertained to social support. There was little variation in types of goals set based on the context of who set them or level of pandemic restrictions in place.
UNASSIGNED: It is feasible for people without clinical training to set GAS holistic goals for PLWD and family carers in the community. GAS has potential to facilitate personalization of care and support interventions, such as NIDUS-family, and facilitate the roll out of more personalized care.
UNASSIGNED: Goal Attainment Scaling (GAS) can capture meaningful priorities of people with dementia and their family carers.A psychosocial intervention RCT used GAS as the primary outcome measure and goals were set collaboratively by non-clinically trained facilitators.The findings underscore the feasibility of using GAS as an outcome measure with this population.The content analysis findings unveiled the diversity in experiences and priorities of the study participants.GAS has the potential to support the implementation of more person-centred approaches to dementia care.

BACKGROUND: Patient-reported outcomes measures (PROMs) are increasingly being collected within cancer clinical trials, yet limited literature on the feasibility and acceptability of doing so.
METHODS: We collected parent-proxy and adolescent (≥12 years old) PROMs through a longitudinal, psychosocial sub-study (\'PRISM-Impact\') embedded in a precision medicine trial for children with poor prognosis cancer (\'PRISM\'). We report on feasibility (response, participation, and attrition rates; follow-up and responding to elevated distress) and acceptability (parents\' perceived benefit/burden of participation; and impact on decision to participate in PRISM) of PRISM-Impact.
RESULTS: Over the reporting period, 462 families were eligible for PRISM-Impact. Family and adolescent response rates were 53% and 45%, respectively. Parents whose child had relapsed were more likely to participate in PRISM-Impact than parents whose child had not (p < 0.001). Parent and adolescent attrition rates were 30% and 56% respectively. We conducted 478 calls for intake and to follow-up on missing questionnaires, and 122 calls to respond to elevated distress. Parents reported wanting to participate in PRISM-Impact for altruistic reasons and because they valued psychosocial research. Parents reported little-to-no burden and some benefit from participating in PRISM-Impact, with little change in ratings overtime. Most parents felt that participating in PRISM-Impact did not impact their desire to participate in PRISM (72%), with some feeling more eager to participate (19%).
CONCLUSIONS: PRISM-Impact response rates were comparable to other psycho-oncology studies, despite the poor prognosis population. Integration of PROMs within a paediatric oncology trial is acceptable to parents, and may provide a more comprehensive assessment of the impact of trial participation.

BACKGROUND: With treatment trials on the horizon, this study aimed to identify candidate digital-motor gait outcomes for autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS), capturable by wearable sensors with multicenter validity, and ideally also ecological validity during free walking outside laboratory settings.
METHODS: Cross-sectional multicenter study (four centers), with gait assessments in 36 subjects (18 ARSACS patients; 18 controls) using three body-worn sensors (Opal, APDM) in laboratory settings and free walking in public spaces. Sensor gait measures were analyzed for discriminative validity from controls, and for convergent (ie, clinical and patient relevance) validity by correlations with SPRSmobility (primary outcome) and Scale for the Assessment and Rating of Ataxia (SARA), Spastic Paraplegia Rating Scale (SPRS), and activities of daily living subscore of the Friedreich Ataxia Rating Scale (FARS-ADL) (exploratory outcomes).
RESULTS: Of 30 hypothesis-based digital gait measures, 14 measures discriminated ARSACS patients from controls with large effect sizes (|Cliff\'s δ| > 0.8) in laboratory settings, with strongest discrimination by measures of spatiotemporal variability Lateral Step Deviation (δ = 0.98), SPcmp (δ = 0.94), and Swing CV (δ = 0.93). Large correlations with the SPRSmobility were observed for Swing CV (Spearman\'s ρ = 0.84), Speed (ρ = -0.63), and Harmonic Ratio V (ρ = -0.62). During supervised free walking in a public space, 11/30 gait measures discriminated ARSACS from controls with large effect sizes. Large correlations with SPRSmobility were here observed for Swing CV (ρ = 0.78) and Speed (ρ = -0.69), without reductions in effect sizes compared with laboratory settings.
CONCLUSIONS: We identified a promising set of digital-motor candidate gait outcomes for ARSACS, applicable in multicenter settings, correlating with patient-relevant health aspects, and with high validity also outside laboratory settings, thus simulating real-life walking with higher ecological validity. © 2024 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

UNASSIGNED: Effective rehabilitation of upper limb musculoskeletal disorders requires multimodal assessment to guide clinicians\' decision-making. Furthermore, a comprehensive assessment must include reliable tests. Nevertheless, the interrelationship among various upper limb tests remains unclear. This study aimed to evaluate the reliability of easily applicable upper extremity assessments, including absolute values and asymmetries of muscle mechanical properties, pressure pain threshold, active range of motion, maximal isometric strength, and manual dexterity. A secondary aim was to explore correlations between different assessment procedures to determine their interrelationship.
UNASSIGNED: Thirty healthy subjects participated in two experimental sessions with 1 week between sessions. Measurements involved using a digital myotonometer, algometer, inclinometer, dynamometer, and the Nine-Hole Peg test. Intraclass correlation coefficients, standard error of the mean, and minimum detectable change were calculated as reliability indicators. Pearson\'s correlation was used to assess the interrelationship between tests.
UNASSIGNED: For the absolute values of the dominant and nondominant sides, reliability was \'good\' to \'excellent\' for muscle mechanical properties, pressure pain thresholds, active range of motion, maximal isometric strength, and manual dexterity. Similarly, the reliability for asymmetries ranged from \'moderate\' to \'excellent\' across the same parameters. Faster performance in the second session was consistently found for the Nine-Hole Peg test. No systematic inter-session errors were identified for the values of the asymmetries. No significant correlations were found between tests, indicating test independence.
UNASSIGNED: These findings indicate that the sensorimotor battery of tests is reliable, while monitoring asymmetry changes may offer a more conservative approach to effectively tracking recovery of upper extremity injuries.

UNASSIGNED: Persistent symptoms post-mild traumatic brain injury (mTBI) includes autonomic dysregulation (AD). The composite autonomic symptoms score, (COMPASS-31), was developed to quantify AD symptom severity in the last year, which limits clinical utility. The primary aim was to determine validity of a modified-COMPASS-31 measuring symptoms in the last month compared to the original, secondarily to compare both original and modified versions to the Neurobehavioral Symptom Inventory (NSI), and tertiarily to detect change post-treatment of the modified-COMPASS-31 compared to NSI and headache intensity (HI).
UNASSIGNED: Thirty-three military personnel with persistent headache post-mTBI.
UNASSIGNED: Total and domain scores for COMPASS-31 (original vs. modified) NSI and HI at baseline. Change in modified-COMPASS-31. NSI, and HI.
UNASSIGNED: Baseline COMPASS-31 versions were comparable and highly correlated (r = 0.72, p < 0.001), they were moderately correlated at best to the NSI (r < 0.6), which may suggest differences in measurement metrics. The mean change in modified-COMPASS-31 scores (15.4/100, effect size 0.8) was mild to moderately correlated to the change in HI (r = 0.39) score, but not to NSI (r = 0.28).
UNASSIGNED: The modified-COMPASS-31 appears to be valid, can measure change of AD symptom severity, and is recommended as an outcome measure.

OBJECTIVE: Constitutional symptoms (fatigue, lymphadenopathy, and weight loss) are not included in the SLE disease activity index-2000 (SLEDAI-2K). In this pilot study, we assessed the concurrent and construct validity of a revised SLEDAI-2K (SLED-R) that included these symptoms with the original SLEDAI-2K (SLED-O), using the physician global assessment of disease activity (PGA) as the reference.
METHODS: Our revised SLED-R substituted the SLED-O\'s fever descriptor with a constitutional descriptor that included fever, fatigue, lymphadenopathy, and/or weight loss. SLED-O, SLED-R, PGA and patient global assessment (PtGA) scores were collected prospectively. Bland-Altman correlations for repeated measures were calculated and Meng\'s z-test was used to compare correlations between dependent and overlapping correlation coefficients. Associations between constitutional symptoms and disease activity measures were analyzed using Mann-Whitney U, Kruskal-Wallis, Chi-square tests and repeated measures correlations.
RESULTS: 1123 SLED-O, SLED-R, PGA, and 1066 PtGA were collected in 239 subjects. The new descriptor was scored in 45 subjects (18.8%) and 92 instances (8.1%), while the original descriptor, fever, was scored in only 4 subjects (1.7%) and 5 instances (0.4%). Mean SLED-O, PGA and PtGA scores were higher when the constitutional descriptor was scored versus not (p < .001). The correlation between SLED-R and PGA was marginally higher than between SLED-O and PGA (p < .001). Fatigue contributed most to this increase (p = .001) and associated with both higher PGA and PtGA scores (p < .001). Mean SLED-O and PGA scores were higher when ≥1 constitutional symptom(s) were scored versus not (p < .002). Correlations between PGA and PtGA when the new descriptor was scored versus not were similar (p = .860). The frequency of concordance between PGA and PtGA was lower when the new descriptor was scored (55%) versus not (72.5%), with PGA > PtGA when the new descriptor was scored (p < .001).
CONCLUSIONS: The addition of constitutional symptoms to SLEDAI-2K, particularly fatigue, resulted in a marginal increase in its correlation with PGA, and new constitutional symptoms associated with higher SLED-O and PGA scores. As fatigue is subjective and difficult to attribute to SLE, its validity and inter-rater reliability in scoring remains uncertain. The clinical utility of SLED-R remains unclear, and further studies of its validity and reliability are needed.

UNASSIGNED: Although most surgeons treating patients with lumbar spinal stenosis (LSS) believe that surgical treatment is superior to conservative measures, systematics reviews have concluded that no solid evidence support this.
UNASSIGNED: To compare change at 1-year of walking ability, health-related quality of life, leg and back pain in patients with symptomatic LSS referred to a spine surgery clinic who opted for surgery and those who did not.
UNASSIGNED: The study included 149 operated and 149 non-operated patients seen by spine surgeons and diagnosed with LSS. The non-operated patients were propensity-matched to a cohort retrieved from the Danish national spine registry. Matching was done on demographics and baseline outcome measures. The outcomes was walking improvement measured by item 4 of the Oswestry Disability Index, EQ-5D-3L, global assessment (GA) of back/leg pain, back and leg pain on the Visual Analogue Scale and the Short Form 36 transition item 2.
UNASSIGNED: Less than half of the non-operated reached MCID on EQ-5D-3L, VAS pain legs or VAS pain back where 2/3 of the operated did. The largest difference was VAS back pain where 27.5% of the non-operated reached an MCID of 12 points compared to 71.8% in the operated group.
UNASSIGNED: Surgical treated patients improved better than non-operated on all outcome measures. However, further research is required to compare the effectiveness of surgical decompression with non-operative care for LSS patients.

BACKGROUND: Managing postoperative pain while minimizing opioid-related adverse drug events (ORADEs) remains a significant challenge. The OPI•AID Zone Tool is proposed as a novel clinical decision support tool that - both graphically and in a scoring-system - represents the relationship between pain management and the occurrence of ORADEs, aiming to enhance patient outcomes in postoperative care. The OPI•AID Zone Tool places pain score on the x-axis and an ORADE score on the y-axis, and stratifies patients into five zones to reflect the composite impact of pain severity and ORADEs on the quality of postoperative patient care. The study will have two key aims: (1) to explore whether the OPI•AID Zone Tool can function as a composite outcome measure for postoperative pain and ORADEs, and (2) to evaluate the use of the OPI•AID Zone Tool in visual presentations and for evaluation of patients\' postoperative pain management quality.
METHODS: This prospective observational cohort study will include 200 adults undergoing various surgical procedures in general anesthesia with a subsequent stay in the post-anesthesia care unit (PACU) at Bispebjerg Hospital, Denmark. Substudy 1 primary outcome: To assess whether a zone score in the OPI•AID Zone Tool is associated with patient-perceived health (EQ VAS), quality of recovery (QoR-PACU), and time to discharge readiness in PACU, and if the zone score has a stronger association than pain and ORADE score in themselves. Substudy 2 primary outcome: To assess how the use of intraoperative non-opioid analgesics impact where patients are placed in the OPI•AID Zone Tool\'s XY scatterplot right after surgery. To assess if patients who receive more comprehensive non-opioid analgesic basic regimens, generally fall into lower zones.
CONCLUSIONS: The OPI•AID Zone Tool could potentially be a valuable clinical decision-making tool for optimizing postoperative care by simultaneously addressing pain management and the risk of ORADEs. By computing a composite measure of these two critical outcomes, the tool could guide more nuanced and patient-centered analgesic regimens, potentially improving patient satisfaction and operational efficiency in postoperative settings. The tool\'s applicability will be explored in this observational pilot and followed up in a planned series of studies (opiaid.dk).