Assessment Of Clinical Outcomes Research Articles

Meaningful within-patient change for clinical outcome assessments: model-based approach versus cumulative distribution functions

ABSTRACT Objectives The FDA recommends the use of anchor-based methods and empirical cumulative distribution function (eCDF) curves to establish a meaningful within-patient change (MWPC) for a clinical outcome assessment (COA). In practice, the estimates obtained from model-based methods and eCDF curves may not closely align, although an anchor is used with both. To help interpret their results, we investigated and compared these approaches. Methods Both repeated measures model (RMM) and eCDF approaches were used to estimate an MWPC on a target COA. We used both real-life (ClinicalTrials.gov: NCT02697773) and simulated data sets that included 688 patients with up to six visits per patient, target COA (range 0 to 10), and an anchor measure on patient global assessment of osteoarthritis from 1 (very good) to 5 (very poor). Ninety-five percent confidence intervals for the MWPC were calculated by the bootstrap method. Results The distribution of the COA score changes affected the degree of concordance between RMM and eCDF estimates. The COA score changes from simulated normally distributed data led to greater concordance between the two approaches than did COA score changes from the actual clinical data. The confidence intervals of MWPC estimate based on eCDF methods were much wider than that by RMM methods, and the point estimate of eCDF methods varied noticeably across visits. Conclusions Our data explored the differences of model-based methods over eCDF approaches, finding that the former integrates more information across a diverse range of COA and anchor scores and provides more precise estimates for the MWPC.

Evaluation of Outcomes and Complications of Sphenoid Wing Meningiomas in Tertiary Care Hospital of KPK

Objective: The objective of this study is to evaluate the outcomes and complications associated with pterional craniotomy and extended pterional craniotomy for the resection of Sphenoid Wing Meningiomas. Material and Methods: A prospective study was conducted on 34 patients at the Neurosurgery department, Prime Teaching Hospital, Pakistan, and Ali Institute of Neurosciences. We examined 34 recently diagnosed cases of sphenoid wing meningiomas, selected through a total enumerative sampling method. Diagnosis of these meningiomas was confirmed by comprehensive neurological assessment and imaging studies. Various surgical techniques, such as Pterional and Extended Pterional approaches, were employed in the procedures. Assessment of clinical outcomes and complications was conducted during follow-up evaluations. A total of 34 patients diagnosed with sphenoid wing meningiomas were covered as the study’s target population. All patients met certain criteria to be included in the study. Results: 25 (73.6%) of the total number of patients were females and 9 (26.4%) were males. The mean age at diagnosis was 47 ± 5 years. Presenting symptoms included headache, seizures, visual loss, motor deficit, and cognitive decline. Surgical techniques used in tumor resection were pterional craniotomy and extended pterional craniotomy. Post-operative evaluation after follow-up studies showed that the symptoms reversed, including a reduction in headache, vision restoration, seizure control, and motor and cognitive function improvement. Conclusion: Pterional approach and extended Pterional approaches related to SWM removal are satisfactory rates of an acceptable and safe surgical treatment with satisfactory results concerning the total resection rate and related clinical improvement.

Tenecteplase versus Alteplase before thrombectomy: A comprehensive evaluation of clinical and angiographic impact: Insights from the ETIS registry

IntroductionData on prior use of Tenecteplase versus Alteplase in acute stroke management by mechanical thrombectomy are controversial. Our primary objective was to make a comprehensive comparative assessment of clinical and angiographic efficacy and safety outcomes in a large prospective observational study. MethodsWe included stroke patients who were eligible for intravenous thrombolysis and endovascular thrombectomy between 2019 and 2021, from an ongoing registry in twenty comprehensive stroke centers in France. We divided patients into two groups based on the thrombolytic agent used (Alteplase vs Tenecteplase). We then compared their treatment times, and their angiographic (TICI scale), clinical (mRS at three months and sICH) and safety outcomes after controlling for potential confounders using propensity score methods. ResultsWe evaluated 1131 patients having undergone thrombectomy for the final analysis, 250 received Tenecteplase and 881 Alteplase. Both groups were of the same median age (75 vs 74 respectively), and had the same baseline NIHSS score (16) and ASPECTS (8). There was no significant difference for First Pass Effect (OR 0.93, 95 % CI 0.76–1.14, p = 0.75), time required for reperfusion (OR 0.03, 95 % CI 0.09–0.16, p = 0.49), or for final reperfusion status. Clinically, functional independence at 90 days was similar in both groups (OR 0.82, 95 % CI 0.61–1.10, p = 0.18) with the same risk of sICH (OR 1.36, 95 % CI 0.77–2.41, p = 0.28). However, Tenecteplase patients had shorter imaging-to-groin puncture times (99 vs 142 min, p < 0.05). ConclusionsTenecteplase showed no better clinical or angiographic impact on thrombectomy compared to Alteplase. Nevertheless, it appeared associated with a shorter thrombolysis-to-groin puncture time.

Development and validation of a brain fog scale for coeliac disease.

Brain fog is a subjective cognitive impairment commonly reported in coeliac disease. A standardised tool to define and assess it is an important unmet need. To develop a patient-informed tool to assess brain fog in coeliac disease to support clinical care, research and drug development. A pilot online study defined patient descriptors of brain fog. A second study evaluated the factor structure and performance of the scale across two-time points ('Now' and in the 'Past week'). One month later, participants were invited to repeat the study with two online cognitive processing tests, the Stroop task and the trail making test. Among adults with treated coeliac disease, 37 (91.9% F) participated in the pilot study and 510 (88.8% F) in the second study of whom 99 repeated the study 1 month later with 51 completing cognitive testing. The most common brain fog descriptors were 'difficulty focusing', 'difficulty thinking' and 'difficulty finding the right words and communicating'. The 12-item scale reflects 'cognitive impairment' and 'somatic and affective experience' and demonstrates strong psychometric properties. It tracked with patients report of brain fog being present or absent across two-time points. It did not significantly correlate with the cognitive tests. The brain fog assessment and severity scale is the first patient-informed clinical outcomes assessment tool measuring brain fog in coeliac disease. It is brief and validated for two time-based formats. Further research coupling it with biomarker discovery is needed to confirm its validity as a predictor of cognitive performance.

MRI-based vertebral bone quality score in cervical ossification of the posterior longitudinal ligament: a comparison with cervical spondylotic myelopathy using propensity score matching

BACKGROUNDBone mineral density plays a key role in the assessment of operative instrumentation complications and clinical outcomes. The MRI-based vertebral bone quality (VBQ) score has been introduced as a novel marker of bone quality. However, few studies have investigated the relationship between VBQ score and patients associated with cervical ossification of the posterior longitudinal ligament (OPLL). PURPOSEThe aims of the study were (1) to reveal bone mineral density between cervical OPLL and cervical spondylotic myelopathy (CSM) group by VBQ score, (2) to compare the VBQ score of cervical OPLL between male and female group, (3) to explore the relationship between segmental VBQ scores associated with OPLL. STUDY DESIGNRetrospective cohort study. PATIENT SAMPLEConsecutive series of 425 patients at a single academic institution. OUTCOME MEASURESMRI based measurements of C2–C7 VBQ scores. METHODSPreoperative noncontrast T1-weighted MRIs of the cervical spine was used to measure the VBQ score. The VBQ score was defined as the mean value of the signal intensity of the vertebrae divided by that of the cerebrospinal fluid (CSF) space at the cisterna magna. Patients with cervical OPLL and CSM were matched based on age, sex, body mass index (BMI), comorbidity, medication history, diet habit, smoking, alcohol consumption via propensity score matching (PSM). Normality of each VBQ score was tested by the Shapiro-Wilk test. Wilcoxon's rank-sum test was used to compare matched cohorts. Kruskal-Wallis test was performed to compare the VBQ scores between segments. Multivariate logistic regression analysis was used to evaluate factors associated with the development of cervical OPLL. RESULTSA total of 425 patients were assessed. For final analysis, 135 paired patients were compared between the cervical OPLL and CSM groups, and 22 paired patients were compared between male and female group associated with cervical OPLL. There were no statistically significant differences in age, sex, BMI, comorbidity, medication history, diet habit, smoking, alcohol between the matched cohorts. OPLL group was associated with lower VBQ score compared with CSM group at C3, while there were no differences in VBQ score for the other levels between the two groups. There were no differences between male and female group associated with OPLL in C2–C7 VBQ scores. VBQ scores of cervical OPLL are variable between segments, with significantly lower scores at C6, C7 compared with C1–C5. Multivariate logistic regression analysis showed that BMI was correlated with the development of OPLL (regression coefficient, 0.162; 95% confidence interval, 0.010–0.037). Additional risk factors included hypertension, calcium supple history and smoking. CONCLUSIONSThis study demonstrates that cervical OPLL is associated with lower VBQ score at C3, with no differences for the other levels when compared with CSM derived from measurements on MRI. No differences were found between male and female group associated with OPLL in C2–C7 VBQ scores. Cervical OPLL were found to have smaller VBQ score at C6, C7 compared with C1–C5. Our findings provide new insight for bone density assessment in cervical OPLL patient.

Sharing Experiences in Selecting Clinical Outcome and Approving Validated Questionnaires : Insights from an Elderly Registry Study

Objectives: Underpinned by the context of a Korean traditional medicine cohort study on healthy aging, this research primarily aims to guide the selection of Clinical Outcome Assessments (COAs) for elderly healthy aging patient registry research, offering insights into the selection process; and secondly, to streamline the resource-intensive process of obtaining permissions for validated COAs, benefiting future traditional Korean medicine clinical researchers.Methods : In this study, we identified outcomes through a review of previous studies, followed by a process involving expert consultations to select the final outcomes. Subsequently, for the selected outcomes that were Clinical Outcome Assessments (COAs) developed tools, we searched in commercial databases to confirm the availability of Korean versions and the necessity of obtaining permissions. Finally, we obtained permissions for their utilization and, when needed, acquired the original instrument questionnaire through payment.Results: Through a literature review of existing observational studies, a total of 57 outcomes were selected, with 19 of them identified as COA instruments. Upon verifying usage permissions for these 19 instruments, it was found that 17 required author-specific permissions, and among these, 2 needed a purchase as they were commercially available.Conclusion: This study provides a detailed overview of outcome selection and permission acquisition for elderly patient registry research. It underscores the importance of Clinical Outcome Assessment (COA) tools and the rigorous approval process, aiming to enhance research reliability. Continuous verification of COA information is essential, and future research should explore Core Outcome Set (COS) development through consensus-building approaches like Delphi studies.

Endovenous glue ablation for chronic venous insufficiency: A comprehensive 5-year assessment of clinical and hemodynamic outcomes.

Chronic venous insufficiency is mainly caused by reflux, obstruction, or both. Endovenous glue ablation has become one of the widely used methods for treating reflux in recent years. Duplex ultrasonography is the most commonly used method for diagnosing and evaluating treatment. However, there is important information that plethysmographic venous hemodynamics provides, which Duplex USG cannot provide. This retrospective study aimed to evaluate the 5-year clinical, anatomical, and hemodynamic results of endovenous glue ablation in the treatment of chronic venous insufficience, accompanied by the data from the plethysmographic study. Between January 2018 and August 2018, 133 patients with symptomatic CEAP 2-6 varicose veins with reflux of the great saphenous vein lasting longer than 0.5 seconds and a diameter of 5.5mm in the standing great saphenous vein underwent EVGA. CEAP, VCSS, CIVIQ 20, Doppler USG, GSV diameters and insufficiency times, and hemodynamically Venous Refilling Time and Venous Half-Value Time measurements were performed before the procedure. In the same way, measurements were made at the 1st, 3rd, 6th, 12th, 24th, and 60th months of the patients who were called and came to the postoperative follow-up. Procedural success was 100%, and complete occlusion was observed %93 after treatment, at the 60month. The improvement in VCSS (from 4.4 ± 1.3 to 1.7 ± 0.9), CIVIQ20 (from 8.5 ± 3.1 to 4.7 ± 2.0), VRT (from 20.3 ± 5.0 to 131.1 ± 4.0), and TH (from 2.8 ± 0.3 to 2.4 ± 0.2) was significant (p < .001 was for all). Endovenous glue ablation is a preferred method for the treatment of great saphenous vein insufficiency due to its ease of use and the comfort it provides to patients, as well as its effectiveness and safety. In particular, it can be considered an effective method for improving venous hemodynamics and relieving associated symptoms.

Assessment of clinical, functional and radiological outcomes in young patients with grade 3 and 4 osteoarthritis of the knee joint undergoing knee joint distraction with and without arthroscopic debridement and chondroplasty-a prospective, comparative and randomized controlled study

Background: Treatment of severe osteoarthritis (OA) in relatively young patients is challenging. Although successful, Total knee arthroplasty (TKA) has a limited lifespan, with the risk of revision surgery, especially in active young patients. Our study aims to assess the comparative clinical, functional and radiological outcomes of knee joint distraction (KJD) with and without arthroscopic debridement and chondroplasty in OA of knee in the young, in a randomized controlled trial. Methods: Our study was a prospective randomized trial with equal allocation. A total of 160 patients needing intervention for knee arthritis, in the age group of &lt;55 years were included as per the inclusion criteria, and were randomized into 2 groups. Group A included 80 patients who underwent KJD alone. Group B included 80 patients who underwent KJD with arthroscopic debridement and chondroplasty. All patients were followed up to 24 months post intervention. Clinical (Visual analogue scale-VAS), functional (Western Ontario and McMaster universities OA index-WOMAC) and radiological (Joint space width-JSW) outcomes were then compared and assessed. Results: Both the groups showed statistically significant improvement of clinical, functional and radiological outcome scores compared to baseline levels. The mean improvement in KJD+SCOPY group was statistically superior to that of KJD group with regard to clinical and functional outcome scores; however, the radiological improvement though being statistically significant in both groups compared to baseline levels, but one group was not superior to that of other. Conclusions: KJD in patients with OA of knee (Kellegren Lawrence grade 3-4) aged &lt;55 years results in improvement of clinical, functional and radiological parameters at 2 year follow up. Addition of arthroscopic debridement and chondroplasty to KJD makes it superior to KJD alone in terms of improvement in clinical, functional and radiological outcomes. We recommend arthroscopic debridement and chondroplasty coupled with KJD for compliant patients of less than 55 years with grade 3-4 OA of the knee.

Assessment of clinical, functional and radiological outcomes in patients with fracture of distal end radius managed with close reduction and internal fixation with multiple percutaneous K wires: a prospective, single centre study

Background: Distal end radius fractures are one of the commonest fractures, occurring in all the age groups. Different treatment modalities have been described in the literature ranging from conservative to operative including close/open reduction to external fixation. Our prospective study aimed at evaluating the outcomes and complications in these fractures managed by close means with multiple percutaneous K wires. Methods: All the patients with distal end radius fracture were treated with close reduction and internal fixation with multiple percutaneous K wires. They were then evaluated clinically with visual analogue scale (VAS) score, functionally with Mayo wrist score and radiologically with Sarmiento’s modification of Lindstrom’s criteria on the follow ups. Results: The study included a total of 152 patients (88 females and 64 males) with mean age being 49.5±10.4 years, with fall on outstretched hand being the most common mode of injury, and dorsal displacement (73.7%) being the most common X-ray finding. Maximum patients in the study had A2.2 fracture (80 patients) followed by C1.2 fracture type (36 patients). There was clinically and statistically significant improvement in the mean VAS scores on follow ups. Excellent functional and radiological outcomes were seen in 124 patients and 108 patients respectively. Conclusions: Close reduction and internal fixation with multiple percutaneous K wires is a cost effective and easy method to treat all distal end radius fractures, provided good anatomical reduction is achieved before the wires are put.

Assessment of clinical outcome after sudden alteration of home parenteral nutrition program from customised to commercially premixed admixtures. Experience with short bowel syndrome treatment

Background & AimsDisruptions in the home parenteral nutrition (HPN) process may lead to failure to achieve the intended treatment purposes. This study aimed to evaluate the mid-term clinical outcome in a group of patients with short bowel syndrome (SBS) after a sudden change in the type of home parenteral nutrition programs from customized to commercially premixed admixtures. MethodsThe study conducted in 2020 identified 51 patients with SBS: 27 (53%) women and 24 (47%) men. The SBS were classified as SBS with end jejunostomy 23 (45%) patients, with jejuno-colon anastomosis 23 (45%) patients, with jejuno-ileo anastomosis and total colon in continuity 5 (10%) patients. The following therapeutic program-related data were analysed: changes in nutritional status, body mass index (BMI), Controlling Nutritional Status (CONUT) score, and biochemical assessment. For statistical analysis, the Wilcoxon rank-sum and signed-rank paired tests with continuity corrections were used to compare the results. A p-value of <0.001 was considered statistically significant. ResultsThere was no statistically significant difference between the analysed groups in total energy, amino acid concentrations, and intravenous volume supplementation. BMI and CONUT assessments of nutritional status and selected biochemical parameters were stable during the study period. ConclusionsThe study demonstrated that a sudden change in the HPN therapy program from parenteral admixtures, tailored to meet individual patients’ needs, to commercially premixed admixtures had no significant impact on the mid-term clinical condition of patients with SBS.

Acromioclavicular joint dislocation Rockwood type III and V show no difference in functional outcome and 91% recovered well without the need for surgery.

Acromioclavicular (AC) joint dislocations are common injuries, but the indication for and timing of surgery is debated. The objective of the study was to evaluate the results after acute AC joint dislocations Rockwood type III and V treated nonsurgically with the option of delayed surgical intervention. This is a prospective cohort study with clinical, radiological and patient-reported outcome assessment at baseline, 6 weeks, 3 months, 6 months and 1 year after acute AC joint dislocation. Patients aged 18-60 with acute AC joint dislocation and a baseline panorama (Zanca) radiograph with an increase in the coracoclavicular distance of >25% compared to the uninjured side were eligible for inclusion. All patients were treated nonsurgically with 3 months of home-based training and with the option of delayed surgical intervention. The primary outcome was the Western Ontario Shoulder Instability Index (WOSI). Secondary outcomes were surgery yes/no and the Shoulder Pain and Disability Index (SPADI). Ninety-five patients were included. Fifty-seven patients were Rockwood type III and 38 patients were type V. There were no statistically significant differences in WOSI and SPADI between patients with type III and V injuries at any time point. Nine patients (9.5%) were referred for surgery; seven type III and two type V (ns). Ninety-one percent of patients with acute AC joint dislocation Rockwood type III and V recovered without surgery and there were no differences in outcome scores between type III and V at any time point. Level IV.

Association of Blood Metabolomics Biomarkers with Brain Metabolites and Patient-Reported Outcomes as a New Approach in Individualized Diagnosis of Schizophrenia.

Given its polygenic nature, there is a need for a personalized approach to schizophrenia. The aim of the study was to select laboratory biomarkers from blood, brain imaging, and clinical assessment, with an emphasis on patients' self-report questionnaires. Metabolomics studies of serum samples from 51 patients and 45 healthy volunteers, based on the liquid chromatography-electrospray ionization-mass spectrometry (LC-ESI-MS/MS), led to the identification of 3 biochemical indicators (cortisol, glutamate, lactate) of schizophrenia. These metabolites were sequentially correlated with laboratory tests results, imaging results, and clinical assessment outcomes, including patient self-report outcomes. The hierarchical cluster analysis on the principal components (HCPC) was performed to identify the most homogeneous clinical groups. Significant correlations were noted between blood lactates and 11 clinical and 10 neuroimaging parameters. The increase in lactate and cortisol were significantly associated with a decrease in immunological parameters, especially with the level of reactive lymphocytes. The strongest correlations with the level of blood lactate and cortisol were demonstrated by brain glutamate, N-acetylaspartate and the concentrations of glutamate and glutamine, creatine and phosphocreatine in the prefrontal cortex. Metabolomics studies and the search for associations with brain parameters and self-reported outcomes may provide new diagnostic evidence to specific schizophrenia phenotypes.

Considering Functional Outcomes as Efficacy Endpoints in Pediatric Low-Grade Glioma Clinical Trials: An FDA Educational Symposium.

In October 2022, the FDA Oncology Center of Excellence hosted an educational symposium entitled, "Considering Functional Outcomes as Efficacy Endpoints in Pediatric Low-Grade Glioma (pLGG) Clinical Trials." The symposium brought together patient advocates, regulators from the FDA and the European Medicines Agency (EMA), and an international group of academic thought leaders in the field of pediatric neuro-oncology to discuss the potential role of functional outcomes, including visual acuity, motor function, and neurocognitive performance, as endpoints in clinical trials enrolling patients with pLGG. The panel discussed challenges and opportunities regarding the selection, implementation, and evaluation of clinical outcome assessments in these functional domains and outlined key considerations for their inclusion in future clinical trial design and role in new drug development.

Cerebral Mechanism of Tuina on the Descending Pain Inhibitory System in Knee Osteoarthritis: Protocol for a Randomized Controlled Trial.

Knee osteoarthritis (KOA) is reputedly the most common musculoskeletal disease of the lower limbs and the main cause of pain and disability among older individuals. Pain is the most significant and widespread symptom of KOA. The descending pain inhibitory system has a cardinal role in normal pain consciousness, and its malfunction may be one of the pathophysiological mechanisms in KOA. Crucially, the rostral ventromedial medulla (RVM) and periaqueductal gray (PAG), as important components of the descending pain inhibitory system, directly modulate the activity of the spinal neurons involved in pain transmission. Tuina, a manual therapy, is effective and safe for reducing clinical symptoms of KOA; however, the mechanism that influences pain through the descending pain inhibitory system in KOA is unclear. This study aims to investigate the modulatory implications of Tuina on the RVM and PAG, which have critical roles in the descending pain inhibitory system in patients with KOA. This randomized controlled parallel trial will be conducted at the Tuina Clinic of the Third Affiliated Hospital of Henan University of Chinese Medicine (Zhengzhou, China). Patients with KOA will be randomly assigned (1:1) to 6 weeks of health education or Tuina. All patients in both groups will accept a resting-state functional magnetic resonance scan at the beginning and end of the experiment, and the resting-state functional connectivity and the voxel-based morphometry analysis will be performed to detect the RVM and PAG function and structure changes. The clinical outcome assessments will be (1) the pressure pain thresholds, (2) the Numerical Rating Scale, (3) the Hamilton Depression Scale (HAMD), and (4) the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC). Considering that this trial is a study of resting-state functional magnetic resonance imaging technology, resting-state functional connectivity and voxel-based morphometry are the primary outcomes, and clinical outcome assessments are secondary outcomes. Adverse events will be documented and assessed throughout. All main analyses will be carried out on the basis of the intention-to-treat principle. The outcome evaluators and data statisticians will be masked to the treatment group assignment to reduce the risk of bias. This trial was approved by the ethics committee of the Third Affiliated Hospital of Henan University of Chinese Medicine. Enrollment began in December 2023, and the results of this trial are expected to be submitted for publication in May 2025. This trial will identify a possible relationship between function and structure changes of RVM and PAG and the improvement of clinical variables, elucidating the effect of Tuina on the descending pain inhibitory system of patients with KOA. This trial will provide much-needed knowledge for Tuina for patients with KOA. Chinese Clinical Trial Registry ChiCTR2300070289; https://www.chictr.org.cn/showproj.html?proj=182570. PRR1-10.2196/52820.

Drug-related side effects and adverse reactions in the treatment of migraine: a bibliometric and visual analysis.

Migraine imposes a substantial global burden, impacting patients and society. Pharmacotherapy, as a primary treatment, entails specific adverse reactions. Emphasizing these reactions is pivotal for improving treatment strategies and enhancing patients' well-being. Thus, we conducted a comprehensive bibliometric and visual analysis of relevant literature. We conducted a comprehensive search on the Science Citation Index Expanded within the Web of Science, restricting the literature for analysis based on criteria such as document type, publication date, and language. Subsequently, we utilized various analytical tools, including VOSviewer, Scimago Graphica, the R package 'bibliometrix', CiteSpace, and Excel programs, for a meticulous examination and systematic organization of data concerning journals, authors, countries/regions, institutions, keywords, and references. By August 31, 2023, the literature was distributed across 379 journals worldwide, authored by 4,235 individuals from 1726 institutions. It featured 2,363 keywords and 38,412 references. 'HEADACHE' led in publication count, with 'SILBERSTEIN S' as the most prolific author. The United States ranked highest in publication volume, with 'UNIV COPENHAGEN' leading among institutions. Our research findings indicate that researchers in the field continue to maintain a focus on the calcitonin gene-related peptide (CGRP) system and explore diverse mechanisms for drug development through the application of novel biotechnological approaches. Furthermore, it is imperative to enhance the assessment of clinical trial outcomes, consistently monitor the efficacy and safety of prominent drugs such as Erenumab and Fremanezumab. There is a need for further evaluation of acute and preventive treatments tailored to different populations and varying types of migraine.

UK Medical Cannabis Registry: Assessment of clinical outcomes in patients with insomnia.

The primary aim of this study was to assess changes in sleep-specific health-related quality of life (HRQoL) for those prescribed cannabis-based medicinal products (CBMPs) for insomnia. A case series of UK patients with insomnia was analyzed. Primary outcomes were changes in the Single-Item Sleep-Quality Scale (SQS), Generalized Anxiety Disorder-7 (GAD-7), and EQ-5D-5L at up to 6 months from baseline. Statistical significance was identified as a p value < .050. 61 patients were included in the analysis. There was an improvement in the SQS from baseline at 1, 3, and 6 months (p < .001). There were also improvements in the EQ-5D-5L Index value and GAD-7 at 1, 3, and 6 months (p < .050). There were 28 (45.9%) adverse events recorded by 8 patients (13.1%). There were no life-threatening/disabling adverse events. Patients with insomnia experienced an improvement in sleep quality following the initiation of CBMPs in this medium-term analysis. Fewer than 15% of participants reported one or more adverse events. However, due to the limitations of the study design, further investigation is required before definitive conclusions can be drawn on the efficacy of CBMPs in treating insomnia.

Unveiling Assessment Gaps in Parkinson's Disease Psychosis: A Scoping Review.

Parkinson's disease psychosis (PDP) is a multidimensional construct that is challenging to measure. Accurate assessment of PDP requires comprehensive and reliable clinical outcome assessment (COA) measures. To identify PDP measurement gaps in available COAs currently used in clinical and research settings. We conducted a scoping review using Preferred Reporting Items for Systematic Review and Meta-Analysis Extension for Scoping Reviews (PRISMA-ScR) guidelines. We implemented a three-step search strategy in international databases with keywords related to Parkinson's disease (PD), psychosis, and COA. We analyzed studies using COA to assess PDP, classifying their items according to domains and subdomains. From 5673 identified studies, we included 628 containing 432 PDP core items from 32 COAs. Among the 32 COAs, 19 were PD-specific, containing 266 items, constructed as clinician-reported outcomes (ClinRO) (148 items), patient-reported outcomes (PRO) (112 items), and observer-reported outcomes (ObsRO) (six items). Across all PD-specific COAs, regardless of structure, 89.4% of the items from 27 COAs focused primarily on assessing PDP symptoms' severity, and only 9.7% of items probed the impact of PDP on a person's daily functioning. Symptom-based domains are currently prioritized for measuring the severity of PDP, with limited coverage of the functional impact of PDP on patients' lives. Whereas the International Parkinson and Movement Disorder Society has traditionally developed a "Unified" COA that culls items from prior COAs to form a new one, a new COA will largely need newly developed items if the functional impact of PDP is prioritized. © 2024 International Parkinson and Movement Disorder Society.

Changes in Digital Speech Measures in Asymptomatic Carriers of Pathogenic Variants Associated With Frontotemporal Degeneration.

Clinical trials developing therapeutics for frontotemporal degeneration (FTD) focus on pathogenic variant carriers at preclinical stages. Objective, quantitative clinical assessment tools are needed to track stability and delayed disease onset. Natural speech can serve as an accessible, cost-effective assessment tool. We aimed to identify early changes in the natural speech of FTD pathogenic variant carriers before they become symptomatic. In this cohort study, speech samples of picture descriptions were collected longitudinally from healthy participants in observational studies at the University of Pennsylvania and Columbia University between 2007 and 2020. Participants were asymptomatic but at risk for familial FTD. Status as "carrier" or "noncarrier" was based on screening for known pathogenic variants in the participant's family. Thirty previously validated digital speech measures derived from automatic speech processing pipelines were selected a priori based on previous studies in patients with FTD and compared between asymptomatic carriers and noncarriers cross-sectionally and longitudinally. A total of 105 participants, all asymptomatic, included 41 carriers: 12 men [30%], mean age 43 ± 13 years; education, 16 ± 2 years; MMSE 29 ± 1; and 64 noncarriers: 27 men [42%]; mean age, 48 ± 14 years; education, 15 ± 3 years; MMSE 29 ± 1. We identified 4 speech measures that differed between carriers and noncarriers at baseline: mean speech segment duration (mean difference -0.28 seconds, 95% CI -0.55 to -0.02, p = 0.04); word frequency (mean difference 0.07, 95% CI 0.008-0.14, p = 0.03); word ambiguity (mean difference 0.02, 95% CI 0.0008-0.05, p = 0.04); and interjection count per 100 words (mean difference 0.33, 95% CI 0.07-0.59, p = 0.01). Three speech measures deteriorated over time in carriers only: particle count per 100 words per month (β = -0.02, 95% CI -0.03 to -0.004, p = 0.009); total narrative production time in seconds per month (β = -0.24, 95% CI -0.37 to -0.12, p < 0.001); and total number of words per month (β = -0.48, 95% CI -0.78 to -0.19, p = 0.002) including in 3 carriers who later converted to symptomatic disease. Using automatic processing pipelines, we identified early changes in the natural speech of FTD pathogenic variant carriers in the presymptomatic stage. These findings highlight the potential utility of natural speech as a digital clinical outcome assessment tool in FTD, where objective and quantifiable measures for abnormal behavior and language are lacking.

The impacts of migraine on functioning: Results from two qualitative studies of people living with migraine.

To describe the impact of migraine on functioning based on comprehensive data collection, analysis, and reporting of patients' experiences. Qualitative research conducted to understand patients' perspectives on living with migraine has often focused on narrow topics or specific groups of patients or has been selectively reported. Qualitative interviews with 71 participants were conducted during two concept elicitation studies as part of the Migraine Clinical Outcome Assessment System (MiCOAS) project, an FDA grant-funded program designed to develop a core set of patient-centered outcome measures for migraine clinical trials. Participants self-reported being diagnosed with migraine by a healthcare professional and participated in semi-structured qualitative interviews about their experiences with the symptoms and impacts of migraine. Interview transcripts were coded to identify and define concepts, which were then grouped into broad domains based on conceptual similarities. A total of 66 concepts were identified: 12 for physical functioning, 16 for cognitive functioning, 10 for social role functioning, 19 for emotional and psychological functioning, and 9 related to migraine management. Participants described a complex and varied relationship between migraine attack symptoms and impacts on functioning. Impacts from migraine were further influenced by numerous contextual factors, such as people's individual social environments and the level of day-to-day demand for functioning they face. Findings showed that migraine impacted individual functioning in multiple ways and the nature of these impacts was dependent on social-contextual factors. The results are being used in the development of core measures designed to improve our understanding of the burden of migraine and the efficacy of migraine therapies. The results also offer new insights and raise new questions about migraine experience that can be used to guide future research.

Pregnant patients undergoing cholecystectomy: nationwide assessment of clinical characteristics and outcomes

Gallstone disease in pregnancy is one of the most common indications for nonobstetrical surgery during pregnancy. National-level data on contemporary surgical practice and outcomes are limited. This study aimed to assess the clinical characteristics and outcomes of patients undergoing cholecystectomy during pregnancy. This cross-sectional study examined the Healthcare Cost and Utilization Project's 2 nationwide databases in the United States: the National Inpatient Sample and the Nationwide Ambulatory Surgery Sample. The study population included 18,630 patients who had cholecystectomy during pregnancy from January 2016 to December 2020. The exposure was gestational age, grouped sequentially into the following 5 groups: first trimester (<14 weeks), early second trimester (14-20 weeks), late second trimester (21-27 weeks), early third trimester (28-36 weeks), and late third trimester (≥37 weeks). The main outcomes were clinical demographics, medical comorbidities, surgical information, and pregnancy characteristics and outcomes, assessed by gestational age. Cholecystectomy was most common in the early second trimester (32.1%), followed by the first trimester (25.2%), late second trimester (23.1%), early third trimester (12.4%), and late third trimester (7.2%). Patients in the first-trimester group were more likely to be aged ≥35 years, to smoke, and to have acute cholecystitis, severe hyperemesis gravidarum including metabolic disturbance, pregestational diabetes, multifetal gestation, and sepsis/shock (P<.001). Patients in the early-third-trimester group were more likely to be obese and have gestational diabetes, Charlson Comorbidity Index of ≥1, premature rupture of membranes, and intrauterine growth restriction, whereas those in the late-third-trimester group were more likely to have gallstone pancreatitis, biliary colic, chorioamnionitis, gestational hypertension, preeclampsia, and severe maternal morbidity including sepsis (P<.001). At the cohort level, a laparoscopic approach was used in most cholecystectomy procedures (97.5%), and bile duct injury was uncommon (<0.1%). Delivery during the admission occurred in 0.3%, 0%, 0.6%, 17.8%, and 60.6% in the 5 gestational age groups, respectively (P<.001). Among the cases that had delivery in the early- and late-third-trimester groups, the delivery event preceded cholecystectomy in 61.4% and 86.2%, respectively, whereas both delivery and cholecystectomy occurred on the same day in 34.3% and 13.8%, respectively. This nationwide analysis suggests that clinical and pregnancy characteristics and outcomes of patients undergoing cholecystectomy differ by pregnancy stage with a bimodal distribution. Although patients in the first and third trimesters have distinct medical conditions, more clinically significant pregnancy and maternal outcomes were found in both groups compared with patients in the second trimester.