Total Point Score Research Articles

Efficacy and Safety of Branched Chain Amino Acids on Retinitis Pigmentosa: A Randomized, Double-Blind, Placebo-Controlled Clinical Trial.

The aim of this study was to investigate the efficacy and safety of orally administered branched-chain amino acids (BCAAs) on disease progression in patients with retinitis pigmentosa (RP). A double-blind, randomized, placebo-controlled study was conducted at the Kyoto University Hospital. Seventy patients with RP aged 20years or above were randomly assigned to the TK-98 (a combination of BCAAs in granule form) or placebo group. One packet (4.15g) of the study drug was administered orally thrice daily for 78weeks. There was no significant difference in the rate of change in the total point score, the primary endpoint, between the TK-98 (-52.4 ± 10.3dB/year) and placebo (-42.9 ± 13.8dB/year) groups. Ellipsoid zone length decreased by -76.5 ± 8.9 and -95.5 ± 12.2 µm/year in the TK-98 and placebo groups, respectively; although this difference was not significant, the TK-98 group showed slower degeneration. No serious adverse events were associated with the oral administration of TK-98 in patients with RP. This study did not yield conclusive evidence supporting BCAA combination granules' effectiveness in slowing visual field progression in patients with RP. An insignificant trend toward a slower reduction in ellipsoid zone length was found in morphological tests. Further studies are required to fully understand the potential benefits of BCAA supplementation in RP. Our study demonstrates the safety of administering BCAAs to patients with RP. Accordingly, larger, more homogeneous clinical studies with longer durations may suggest their potential as therapeutic agents.

Assessment of Basic Thoracic Ultrasound Skills in Immersive Virtual Reality: Gathering Validity Evidence

Background: Operator skills are essential for thoracic ultrasound (TUS) to ensure diagnostic accuracy. Immersive virtual reality (IVR) has shown potential within medical education but never to assess TUS skills.This study aimed to develop an IVR test for assessing TUS skills, gathering validity evidence, and establishing a pass/fail score.Methods: An expert panel developed a test based on the TUS protocol by the European Respiratory Society (ERS) including a tutorial and two clinical cases (pleural effusion and interstitial syndrome) using an IVR platform [VitaSim, Denmark]. Four anterior, four lateral, and six posterior zones were available for examination and decision of diagnosis. Each correct examination equalled one point. The contrasting groups’ method was used to set a pass/fail score.Results: Data was collected at the during the ERS Congress 2022. We included 13 novices (N, experience: 0 TUS), 22 intermediates (I, 1-50 TUS), and 11 experienced (E, >50 TUS). Cronbach's alpha was 0.86. The total mean point scores in case 1 (C1) were N: 5.0±2.7; I: 7.3±2.4; E: 8.7±1.3 and in case 2 (C2): N: 4.5±1.8; I: 6.7±2.3; E: 8.5±2.1. Significant differences were found between the N and I for C1 (p=0.007) and C2 (p=0.02), I and E for C1 (p= 0.04) and C2 (p=0.019), and N and E for C1 (p<0.001) and C2 (p<0.001). The pass/fail score was seven points in each case.Conclusion: We established an IVR test that can distinguish between operators with different TUS skills. This enables a standardized, objective, and evidence-based approach to assess TUS skills.

Exploration of a novel prognostic model based on nomogram in non-small cell lung cancer patients with distant organ metastasis: implications for immunotherapy.

Evidence for the effects of immunotherapy in non-small cell lung cancer (NSCLC) patients with distant organ metastasis is insufficient, and the predictive efficacy of established markers in tissue and blood is elusive. Our study aimed to determine the prognostic factors and develop a survival prognosis model for these patients. A total of 100 advanced NSCLC patients with distant organ metastases, who received single or combination immune checkpoint inhibitors (ICIs) in Xijing Hospital between June 2018 and June 2021, were enrolled for retrospective analysis. The major clinicopathological parameters were collected, and associated survival outcomes were followed up by telephone or inpatient follow-up for nearly 3 years to assess prognoses. The survival prognosis model was established based on univariate and multivariate Cox regression analyses to determine the candidate prognostic factors. From the start of immunotherapy to the last follow-up, 77 patients progressed and 42 patients died, with a median follow-up of 18 months [95% confidence interval (CI): 15-19.9]. The median progression-free survival (PFS) and overall survival (OS) were 8 months (95% CI: 5.6-10.4) and 21 months (95% CI: 8.9-33.1), respectively. Multivariate Cox proportional hazards analysis showed Eastern Cooperative Oncology Group performance status (ECOG PS), body mass index (BMI), age-adjusted Charlson comorbidity index (ACCI), lactate dehydrogenase (LDH), and absolute neutrophil count (ANC) were correlated significantly with OS. Based on these five predictive factors, a nomogram and corresponding dynamic web page were constructed with a concordance index (C-index) of 0.81 and a 95% CI of 0.778-0.842. Additionally, the calibration plot and time-receiver operating characteristic (ROC) curve validated the precision of the model at 6-, 12-, and 18-month area under the curves (AUCs) reached 0.934, 0.829, and 0.846, respectively. According to the critical point of the model, patients were further divided into a high-risk total point score (TPS) >258, middle-risk (204< TPS ≤258), and low-risk group (TPS ≤204), and significant OS differences were observed among the three subgroups (median OS: 4.8 vs. 13.0 vs. 32.9 months). A feasible and practical model based on clinical characteristics has been developed to predict the prognosis of NSCLC patients with distant organ metastasis undergoing immunotherapy.

Evaluating Students’ Performance Relative to Their Time Investment in an Asynchronous Online Class

COVID-19 lockdowns led to increased adoption of online teaching with courses that have an asynchronous format. It is recognized that some students could be online without dedicating all oftheir time to the class. This study aims to investigate whether there exists any association between the hours students invest in online classes with the point scores they achieve. Data on semester participation hours and total point scores were gathered in CNST 440-540, an asynchronous onlineclass at Eastern Michigan University from the winter semester of 2016 to the winter semester of 2021. Statistical modeling revealed a modest but significant relationship between student onlinetime and actual point scores.

Development of a Clinical Risk Score Prediction Tool for 5-, 9-, and 13-Year Risk of Dementia

Although researchers have devoted substantial efforts, money, and time to studying the causes of dementia and the means to prevent it, no effective treatment exists yet. Identifying preclinical risk factors of dementia could help prevent or delay its progression. To develop a point risk score prediction model of dementia. This study used a large UK population-based prospective cohort study conducted between March 13, 2006, and October 1, 2010. Data analysis was performed from June 7 to September 15, 2021. Individual analyses of time end points were concluded at the first dementia diagnosis during the follow-up period. The data were split into training and testing data sets to separately establish and validate a prediction model. Outcomes of interest included 5-, 9-, and 13-year dementia risk. Least absolute shrinkage and selection operator and multivariate Cox proportional hazards regression models were used to identify available and practical dementia predictors. A point risk score model was developed for the individual prediction of 5-, 9-, and 13-year dementia risk. A total of 502 505 participants were selected; the population after exclusions for missing data and dementia diagnosis at baseline was 444 695 (205 187 men; mean [SD] age, 56.74 [8.18] years; 239 508 women; mean [SD] age, 56.20 [8.01] years). Dementia occurrence during the 13 years of follow-up was 0.7% for men and 0.5% for women. The C statistic of the final multivariate Cox proportional hazards regression model was 0.86 for men and 0.85 for women in the training data set, and 0.85 for men and 0.87 for women in the testing data set. Men and women shared some modifiable risk and protective factors, but they also presented independent risk factors that accounted for 31.7% of men developing dementia and 53.35% of women developing dementia according to the weighted population-attributable fraction. The total point score of the risk score model ranged from -18 to 30 in men and -17 to 30 in women. The risk score model yielded nearly 100% prediction accuracy of 13-year dementia risk both in men and women. In this diagnostic study, a practical risk score tool was developed for individual prediction of dementia risk, which may help individuals identify their potential risk profile and provide guidance on precise and timely actions to promote dementia delay or prevention.

Prevalence of premature coronary artery disease (CAD) according to clinical familial hypercholesterolaemia (FH) diagnosis in Lithuania

Background and Aims : We aimed to determine the prevalence of CAD according to clinical diagnosis of Familial Hypercholesterolemia(FH) in Lithuania.Methods: Prospective observational cohort study enrolled patients with clinically diagnosed FH treated in Vilnius University Hospital Santaros Klinikos during the period of 2016-2021. According to Dutch Lipid Clinic Network (DLCN) diagnostic criteria for FH, definite FH was diagnosed when a total point score was>8, probable–6-8, possible-3-5,unlikely<3 points. Premature CAD was defined as occuring in men <55 years and women <60 years. Obstructive Atherosclerotic CAD was defined as the presence of stenosis ≥50% in at least one coronary vessel in Coronary Computed Tomography Angiography(CCTA) or coronary angiography, as well as performed percutaneous coronary intervention(PCI) or coronary arteries bypass grafting(CABG). Data of 119 study patients were included in the analysis. The prevalence of premature CAD was compared in different groups according to FH diagnosis. Statistical analysis was performed using R(v. 4.0.4) program package.Results: Of 119 examined patients 52,1%(n=62) were women and47,9%(n=57) were men.In the study population, premature CAD was diagnosed for 26%(n=31) patients. 21,8%(n=26) of study patients had definite FH diagnosis, 37,8%(n=45) had probable FH, 28,6%(n=34) - possible FH and11,8% (n=14) had unlikely FH diagnosis. Premature CAD was diagnosed to 38,5%(n=10) patients with definite FH, 33,3%(n=15) with probable FH, 14,7%(n=5) with possible FH and 7,1%(n=1) among people with unlikely FH.Conclusions: Our findings indicate that among individuals with premature CAD there is an opportunity to detect an index case for initiation of cascade FH screening, especially in definite and probable FH cases. Background and Aims : We aimed to determine the prevalence of CAD according to clinical diagnosis of Familial Hypercholesterolemia(FH) in Lithuania. Methods: Prospective observational cohort study enrolled patients with clinically diagnosed FH treated in Vilnius University Hospital Santaros Klinikos during the period of 2016-2021. According to Dutch Lipid Clinic Network (DLCN) diagnostic criteria for FH, definite FH was diagnosed when a total point score was>8, probable–6-8, possible-3-5,unlikely<3 points. Premature CAD was defined as occuring in men <55 years and women <60 years. Obstructive Atherosclerotic CAD was defined as the presence of stenosis ≥50% in at least one coronary vessel in Coronary Computed Tomography Angiography(CCTA) or coronary angiography, as well as performed percutaneous coronary intervention(PCI) or coronary arteries bypass grafting(CABG). Data of 119 study patients were included in the analysis. The prevalence of premature CAD was compared in different groups according to FH diagnosis. Statistical analysis was performed using R(v. 4.0.4) program package. Results: Of 119 examined patients 52,1%(n=62) were women and47,9%(n=57) were men.In the study population, premature CAD was diagnosed for 26%(n=31) patients. 21,8%(n=26) of study patients had definite FH diagnosis, 37,8%(n=45) had probable FH, 28,6%(n=34) - possible FH and11,8% (n=14) had unlikely FH diagnosis. Premature CAD was diagnosed to 38,5%(n=10) patients with definite FH, 33,3%(n=15) with probable FH, 14,7%(n=5) with possible FH and 7,1%(n=1) among people with unlikely FH. Conclusions: Our findings indicate that among individuals with premature CAD there is an opportunity to detect an index case for initiation of cascade FH screening, especially in definite and probable FH cases.

P1692: PREDICTIVE MODEL FOR VEIN THROMBOEMBOLIC EVENTS DEVELOPMENT IN PATIENTS WITH ACUTE MYELOID LEUKEMIA

Background: Patients with acute myeloid leukemia (AML) are at increased risk of vein thromboembolic events (VTE). However, thromboprophylaxis is largely underused, mainly due to increased risk of bleeding and limited number of studies analizing risk factors for VTE development. Aims: To assess potential risk factors for the VTE development and develop a simple predictive model, based on patients and disease-related parameters. Methods: We conducted a retrospective cohort study of adult patients with newly diagnosed AML who were treated in our institution between January 2009 and December 2021. Patients with AML-M3 were excluded. Primary end point of our study was imaging-confirmed VTE including simptomatic central venous catheter -related thrombosis (CVC). Participants were followed from the AML diagnosis to death or 6 months after the last cycle of therapy. We analyzed demographic factors (age, gender), Eastern Cooperative Oncology Group performance status (ECOG PS), body mass index (BMI), smoking status, comorbidities, Hematopoietic Cell Transplantation-specific Comorbidity Index (HCT-CI), baseline laboratory findings (complete bood counts, fibrinogen, PT, PTT, D dimer, LDH) leukemia related paremeters (cytogenetics, molecular genetics (FLT3, NPM), flow cytometry), Khorana score (KRS) as potential risk factors for the VTE development. Univariate and multivariate logistic regression model was used as the method for analyzing binary outcome (VTE development) and potential predictors. Based on our final model we cretaed a nomogram to enable calculalation of probability of VTE development. Results: The study population included 608 eligible AML patients, mean age 55 (range: 18-81) years, male/female 341/267 (56.1%/43.9%). During the follow-up period 81/608 (13.3%) patients developed VTE: CVL 61/608 (10.0%), deep vein thrombosis 16/608 (2.63%), pulmonary embolism 4/608 (0.01%). Before therapy, during induction, consolidation, transplantation and relapse VTE were developed in 5/81 (6.2%), 36/81 (44.4%), 30/81 (37%), 4/81 (4.9%), 6/81 (7.4%) patients respectively. Among all tested parameters five which are independent predictictors were included in predictive model: male sex (p=0.020, OR 1,92 95% CI: 1.11-3.31), prior history of TE (p=0.001, OR 3.83, 95% CI: 1.72-8.55), PT (p=0.107, OR 1.01, 95% CI: 0.99-1.03), ECOG PS (p=0.038, OR 0.72, 95% CI: 0.53-0.98), HCT-CI (p=0.032, OR 0.79, 95% CI: 0.64-0.98). All other patient and disease related paremeters, including Khorana score were not predictive. Our nomogram provides a visual depiction of the relative contribution of each prognostic factor to the total point score and, thus, the weight of factors regarding probability for VTE development (Picture 1). Image:Summary/Conclusion: Our study point out that: 1. A KRS score has no predictive value in AML; 2. Male patients and patients with history of prior thrombosis have 1.92 and 3.83 higher odds of developed VTE; 3. Increasing of ECOG and HCT-CI for one point decrease probability of develope thrombosis for 28% and 21%; 4. Thromboprophylaxis should be considered in patients with prior history of TE, especially with male sex, good PS and low HCT-CI. Our nomogram may help clinicians in clinical decision making. However, validation of our predictive model through prospective study with a larger number of patients are required.

Progressive Lacunar Strokes: A Predictive Score

Background and PurposeProgressive lacunar syndromes (PLS) occur in up to 20-30% of patients with lacunar strokes, increasing the risk of long term dependency. Our aim is to develop a predictive score to identify patients at high risk of presenting PLS. MethodsWe derived a risk score for PLS in a cohort of consecutive patients (n=187) presenting with one of the five classic lacunar syndromes (LS) and absence of vascular occlusion, perfusion deficit or symptomatic stenosis. A risk score was developed using the coefficients from the logistic regression model, and receiver operating characteristic (ROC) analysis was conducted to assess the prognostic value of the risk score. Sensitivity, specificity and accuracy were estimated for each total point score. ResultsOut of 187 patients included in our sample, 52 (27.8%) presented PLS. Previous history of diabetes mellitus (1 point), diastolic blood pressure at admission (2 points), clinical deficits consistent with a pure motor syndrome (1 point) and asymptomatic intracranial atheromatosis or stenosis in non-symptomatic territory (1 point) were independent predictors for PLS. The estimated area under the ROC curve for this model was 0.77 (95% CI,0.68 - 0.84). ConclusionThis score could be a useful tool in routine clinical practice to predict the occurrence of PLS, allowing the identification of those patients with LS who are at high risk of long term dependency due to early neurological worsening, and who would benefit the most from an intensive treatment.

Prediction of in-hospital mortality by using get with the guideline-heart failure (GWTG-HF) risk scores in patients hospitalized with heart failure

Abstract Funding Acknowledgements Type of funding sources: Public grant(s) – National budget only. Main funding source(s): University Grants Commission Bangladesh Background Prognostic stratification using readily available clinical data can assist clinical decision making in heart failure. Get with the Guideline- Heart Failure (GWTG-HF) score approved by American Heart Association (AHA) can be used to inform hospitals about the expected in-hospital mortality of their patients. Purpose This study aims to predict in-hospital mortality of patients with heart failure using GWTG-HF score in order to give evidence based therapy to high risk patients. Methods We have included patients who were hospitalized with a diagnosis of heart failure from February 2020 to 15 January 2021. A total of 2038 patients were admitted during this period of which 144 patients were diagnosed with heart failure (7%). The GWTG-HF risk score uses 7 clinical factors collected at admission. These are older age, low systolic blood pressure, elevated heart rate, low serum sodium, elevated BUN, presence of COPD, and ethnicity. The probability of in-hospital mortality is estimated for an individual patient by summing points assigned to the value of each predictor for a total point score within a range of 0-100. These scores were calculated for 144 heart failure patients involved in this study. Results Death occurred in 29 heart failure patients (20.13%). Mean age was 59 ±13 years. Male/female ratio was 3.65. All patients were ethnically Bangladeshi Asian. 60% of patients had left ventricular ejection fraction (LVEF) &amp;lt;40%. Overall LVEF was median (IQR) 36% (30-45). Those who died were more likely to have a prior heart failure diagnosis and an LVEF &amp;lt;40%. The observed mortality was 0% for the GWTG-HF score 0-33, 14.14% for 34-50, 42% for 51-57, 40% for 58-61 and 50% with a score of 62-65. The observed mortality of our patients were much higher than expected GWTG-HF score (20% vs 2.8%). The probable reasons for increased mortality in our study could be 1) The number of patients having LVEF &amp;lt;40% is much higher (60%), 2) surgically uncorrected/absence of intervention for underlying valvular disease/advance heart failure/ischaemic cardiomyopathy, 3) different treatment regimens and non-compliance of patients regarding long-term medication. Additionally, those who died had higher serum creatinine and BUN and lower serum sodium and haemoglobin levels at admission. Further multi-centre study involving large population is needed to clarify higher death rate in heart failure patients in our population. Conclusion The GWTG-HF risk score can identify patients at high risk of in-hospital death to aid clinical decision making. The risk score could be used to inform hospitals about the expected mortality rates of patients with heart failure admitted to their hospital. Although GWTG-HF risk score is validated in different populations, the mortality rate in this study is greater than expected mortality due to aforementioned reasons. Abstract Figure. GWTG-HF Risk Score (Reference 1) Abstract Figure. Predicted and Observed Mortality Rates

Students’ Cognitive Test Anxiety and Secondary School Examination Performance: Moderating Effect of Gender and School Sector

Despite a bulk of research in international context investigating the relationship of cognitive test anxiety with students’ academic performance, little work has yet been done in this area on samples of students in Pakistan. This correlational design study was, therefore, conducted to examine the relationship between secondary school students’ cognitive test anxiety and their examination performance in educational context of Pakistan and to ascertain how the direction and/or strength of this relationship may be moderated by students’ gender and their school sector. Data were collected on students’ cognitive test anxiety, total obtained marks in SSC examination and obtained marks in English along with students’ gender and school sector from 1712 mixed ability students enrolled in year 11 in 91 public and private institutions. Both the descriptive and inferential statistical techniques (i.e., Mean, SD and Independent samples t-test) along with Pearson’s R correlations and hierarchical multiple regression were performed to analyze the data. Results depicted that female sample students reported higher level of cognitive test anxiety in comparison to their male counterparts. Results also suggested a small but significant negative effect of cognitive test anxiety on students’ examination performance and that student cognitive test anxiety component accounted for 10% of variance in their examination performance. Results further revealed that the magnitude of relationship between students’ cognitive test anxiety and examination total point scores do not change much for the moderating variables and these variables accounted only for 3% of variance. The magnitude of moderating effect, however, found to be somewhat stronger in the school sector in comparison to students’ gender.

Development and Validation of a Nomogram for Predicting Prognosis to Immune Checkpoint Inhibitors Plus Chemotherapy in Patients With Non-Small Cell Lung Cancer.

BackgroundImmune checkpoint inhibitors (ICIs) plus chemotherapy improved the prognosis of patients with non-small cell lung cancer (NSCLC); however, reliable prognostic biomarkers are lacking. We explored factors associated with prognosis and developed a predictive model.MethodsWe retrospectively analyzed 130 consecutive stage IIIA–IVB NSCLC patients treated with ICIs combined with chemotherapy. Cox univariate and multivariate proportional hazards regression analyses were used to identify prognostic factors associated with progression-free survival (PFS). A nomogram was developed based on key factors in the training cohort (n = 86) and evaluated in the validation cohort (n = 44). According to the nomogram-based total point scores, we divided patients into low- and high-risk groups.ResultsIn the training cohort, bone metastases (p = 0.017) and an increased derived neutrophil-to-lymphocyte ratio (p = 0.018) were significantly associated with poor PFS, while smoking (p = 0.007) and programmed death-ligand 1 (PD-L1) ≥50% (p = 0.001) were associated with improved PFS. A nomogram based on these factors was developed to predict PFS at 3, 6, and 12 months. The C-index of the nomogram to predict PFS was 0.725 (95% CI: 0.711–0.739) in the training cohort and 0.688 (95% CI: 0.665–0.711) in the validation cohort. The area under the curve (AUC) exhibited an acceptable discriminative ability, and calibration curves demonstrated a consistency between the actual results and predictions. In the training cohort, the median PFS (mPFS) was 12.3 and 5.7 months in the low- and high-risk groups, respectively (p < 0.001). In the validation cohort, the mPFS was 12.6 and 6.2 months in the low- and high-risk groups, respectively (p = 0.021).ConclusionsA predictive nomogram was developed to help clinicians assess prognosis early for advanced NSCLC patients who received ICI plus chemotherapy.

THE EFFECT OF A GREEK TRADITIONAL DANCE PROGRAM ON THE MOTOR COMPETENCE OF CHILDREN WITH AUTISM SPECTRUM DISORDER

It is usual for individuals with autism spectrum disorder (ASD) to demonstrate deficits in motor behavior. Participation in movement programs has been suggested to elicit improvements in their motor competence (MC) level. The aim of the present study was to examine the effect of a Greek traditional dance (GTD) program on the MC of 6-14-year-old children with ASD. The study design included eight participants (two girls, six boys), who were randomly allocated into two equal-sized groups, group A (its members participated only in the regular Physical Education lessons provided by their school) and group B (its members additionally followed an 8-week GTD program of two 40-min sessions per week). Children’s MC was assessed prior and immediately after the completion of the GTD program by the Bruininks- Oseretsky Test of Motor Proficiency (BOT-2 SF). Due to study’s small sample size and participants’ heterogenous ASD characteristics, the results will be interpreted as outcomes of multiple single case studies. Overall, it was observed that the MC of all participants was considerably poor; however, members of group B made greater improvements in both the total BOT-2 point score and most item raw scores compared to those of group A, with balance and body coordination skills presenting the greater improvements. Even though the results of this study stress the positive effect of GTD on the MC of children with ASD, future studies employing wider sample sizes and/or implementing longer programs are required to further confirm its merit. &#x0D; &#x0D; &lt;p&gt; &lt;/p&gt;&lt;p&gt;&lt;strong&gt; Article visualizations:&lt;/strong&gt;&lt;/p&gt;&lt;p&gt;&lt;img src="/-counters-/edu_01/0874/a.php" alt="Hit counter" /&gt;&lt;/p&gt;

Detection Sensitivity of Retinitis Pigmentosa Progression Using Static Perimetry and Optical Coherence Tomography

PurposeTo compare the detection sensitivities of the progression of retinitis pigmentosa (RP) by automated perimetry to obtain the mean deviation (MD) and total point score and by optical coherence tomography (OCT) to determine the residual ellipsoid zone (EZ) length and thickness of retinal layers.MethodsTwenty-two eyes of 22 patients with RP who underwent annual automated perimetry (Humphrey Field Analyzer 10-2) and OCT examinations during the same period more than four times were included. Disease progression was evaluated using linear regression analysis with the least-squares method. The disease progression speed and interinspection fluctuations for the different examinations were compared using standardized values. The progression detection ability factor, defined as the average of the least squares divided by the square of annual change, was used to compare the sensitivities of the examinations for detecting the progression of RP.ResultsEZ length showed a high correlation with MD (R = 0.87; P = 1.12E-07) at baseline. Disease progression was detected more frequently using EZ length (12/22 eyes) than using MD (3/22 eyes; P = 0.004) or central retinal thickness (1/11 eyes; P = 0.012). Linear regression using standardized values showed that the EZ length had the fastest annual change, with the smallest least absolute values. EZ length was more sensitive for detecting RP progression than MD, total point score, visual acuity, or central retinal thickness.ConclusionsEZ measurement was sensitive for detecting RP progression, and the results of this study indicate that EZ length is appropriate for end points in clinical trials.Translational RelevanceThe study provides a basis for conducting future clinical trials.

The Gaucher earlier diagnosis consensus point-scoring system (GED-C PSS): Evaluation of a prototype in Finnish Gaucher disease patients and feasibility of screening retrospective electronic health record data for the recognition of potential undiagnosed patients in Finland

BackgroundGaucher disease (GD) is a rare inherited multiorgan disorder, yet a diagnosis can be significantly delayed due to a broad spectrum of symptoms and lack of disease awareness. Recently, the prototype of a GD point-scoring system (PSS) was established by the Gaucher Earlier Diagnosis Consensus (GED-C) initiative, and more recently, validated in Gaucher patients in UK. In our study, the original GED-C PSS was tested in Finnish GD patients. Furthermore, the feasibility of point scoring large electronic health record (EHR) data set by data mining to identify potential undiagnosed GD cases was evaluated. MethodsThis biobank study was conducted in collaboration with two Finnish biobanks. Five previously diagnosed Finnish GD patients and ~ 170,000 adult biobank subjects were included in the study. The original PSS was locally adjusted due to data availability issues and applied to the Finnish EHR data representing special health care recordings. ResultsAll GD patients had high levels of the biomarker lyso-Gb1 and deleterious GBA mutations. One patient was a compound heterozygote with a novel variant, potentially pathogenic mutation. Finnish EHR data allowed the retrospective assessment of 27–30 of the 32 original GED-C signs/co-variables. Total point scores of GD patients were high but variable, 6–18.5 points per patient (based on the available data on 28–29 signs/co-variables per patient). All GD patients had been recorded with anaemia while only three patients had a record of splenomegaly. 0.72% of biobank subjects were assigned at least 6 points but none of these potential “GD suspects” had a point score as high as 18.5. Splenomegaly had been recorded for 0.25% of biobank subjects and was associated with variable point score distribution and co-occurring ICD-10 diagnoses. DiscussionThis study provides an indicative GED-C PSS score range for confirmed GD patients, also representing potential mild cases, and demonstrates the feasibility of scoring Finnish EHR data by data mining in order to screen for undiagnosed GD patients. Further prioritisation of the “GD suspects” with more developed algorithms and data-mining approaches is needed. FundingThis study was funded by Shire (now part of Takeda).

Video-based, student tutor- versus faculty staff-led ultrasound course for medical students \u2013 a prospective randomized study

BackgroundUltrasound education is propagated already during medical school due to its diagnostic importance. Courses are usually supervised by experienced faculty staff (FS) with patient bedside examinations or students among each other but often overbooked due to limited FS availability. To overcome this barrier, use of teaching videos may be advantageous. Likewise, peer teaching concepts solely with trained student tutors have shown to be feasible and effective. The aim was to evaluate 1) objective learning outcomes of a combined video-based, student-tutor (ViST) as compared to a FS-led course without media support, 2) acceptance and subjective learning success of the videos.MethodsTwo ultrasound teaching videos for basic and advanced abdominal ultrasound (AU) and transthoracic echocardiography (TTE) were produced and six students trained as tutors. Fourth-year medical students (N = 96) were randomized to either the ViST- or FS course (6 students per tutor). Learning objectives were defined equally for both courses. Acquired practical basic and advanced ultrasound skills were tested in an objective structured clinical examination (OSCE) using modified validated scoring sheets with a maximum total score of 40 points. Acceptance and subjective learning success of both videos were evaluated by questionnaires based on Kirkpatrick’s evaluation model with scale-rated closed and open questions.Results79 of 96 medical students completed the OSCE and 77 could be finally analyzed. There was no significant difference in the mean total point score of 31.3 in the ViST (N = 42) and 32.7 in the FS course (N = 35, P = 0.31) or in any of the examined basic or advanced ultrasound skill subtasks. Of the 42 ViST participants, 29 completed the AU and 27 the TTE video questionnaire. Acceptance and subjective learning success of both videos was rated positively in 14–52% and 48–88% of the rated responses to each category, respectively. Attendance of either the student or faculty tutor was deemed necessary in addition to the videos.ConclusionsA ViST versus FS teaching concept was able to effectively teach undergraduate students in AU and TTE, albeit acceptance of the teaching videos alone was limited. However, the ViST concept has the potential to increase course availability and FS resource allocation.

Prognostic nomogram on clinicopathologic features and serum indicators for advanced non-small cell lung cancer patients treated with anti-PD-1 inhibitors.

BackgroundImmune checkpoint inhibitors (ICIs) have appeared as a promising therapy regimen for non-small cell lung cancer (NSCLC), but with an unsatisfying therapeutic response and inefficiency of a single predictive biomarker in patients’ selection.MethodsCentral data of clinicopathologic features, peripheral blood indicators, and treatment records were collected in advanced NSCLC patients accepting PD-1 inhibitors in Changhai Hospital from July 2016 to September 2019. The OS probability nomogram was developed according to Akaike Information Criterion (stepAIC) selected factors. The predictive accuracy of the nomogram was assessed by discrimination and calibration. C-index and decision curve analysis were used to compare with the previously reported model (Botticelli Model). Computers resampling 500 times (Bootstrap 500 times) were performed to validate the model internally. According to the nomogram-based total point scores (TPS), we divided patients into different risk groups.ResultsA total of 110 patients were enrolled in this study. Six predictors, including liver metastasis, Eastern Cooperative Oncology Group Performance Status (ECOG PS), second- or third-line immunotherapy, baseline levels of CRP, cytokeratin 19 fragment (CYFRA21-1), were selected to set up the nomogram. The C-index of the current nomogram was 0.81 (95% CI: 0.72–0.80), keeping the same accuracy as the earlier one. Calibration plots showed slight underestimation in patients with predictive mortality <44% at 12 months and overestimation in patients with predictive mortality >44%. Decision curve analysis showed that the current nomogram was with a higher net benefit rate than the earlier model. According to the cut-off points of TPS, patients were divided into three subgroups: low risk (TPS ≤118), intermediate-risk (118< TPS ≤189), and high risk (TPS >189). A significant OS difference was observed among subgroups. Median OS was 6.6, 4.5, 1.3 months, respectively.ConclusionsWe proposed a novel nomogram model on easily available and inexpensive clinicopathologic features, peripheral blood indicators which is beneficial in individual risk assessment for advanced NSCLC patients before receiving PD-1 inhibitors, and assisting clinicians in accurately determining therapeutic decisions.

Use of the Bruininks-Oseretsky test of motor proficiency (BOT-2) to assess efficacy of velmanase alfa as enzyme therapy for alpha-mannosidosis

ObjectivesAlpha-mannosidosis is a rare autosomal recessive lysosomal storage disorder resulting from deficient lysosomal alpha-mannosidase activity. Clinical manifestations include progressive balance disorders, immune deficiency, skeletal abnormalities and cognitive deficits beginning in early childhood. Enzyme replacement therapy with recombinant human alpha-mannosidase (velmanase alfa) is scheduled for clinical development in the US beginning in 2020 and has been approved in the EU for treatment of non-neurological manifestations in cases of mild to moderate disease. This study assessed effects of velmanase alfa on fine and gross motor proficiency in children and adults. MethodsIntegrated Bruininks-Oseretsky (BOT-2) test of Motor Proficiency data from velmanase alfa clinical trials was stratified by age for 14 adults and 19 children treated for up to 4 years. ResultsPatients showed global developmental delays at baseline. For the combined adult and pediatric group there was a statistically significant increase (improvement) in BOT-2 total point score of 13% (p = .035, 95% CI 1.0, 25.0) from baseline to last observation. When stratified by pediatric versus adult patients, there was improvement in BOT-2 total point score in patients <18 years (mean percent increase from baseline to last observation 23%) compared to adults (mean decrease of −0.7%). Subtest analysis of individual BOT-2 items captured some improvement following velmanase alfa treatment in pediatric patients. ConclusionsThere was limited ability to assess the BOT-2 change responses in adults. Pediatric patients showed stability or improvement in scaled scores relative to healthy peers, indicating continued skill acquisition, which may increase independence and contribute to improved patient quality of life.

Visual Field Changes Over 5 Years in Patients Treated With Panretinal Photocoagulation or Ranibizumab for Proliferative Diabetic Retinopathy

Preservation of peripheral visual field (VF) is considered an advantage for anti-vascular endothelial growth factor agents compared with panretinal photocoagulation (PRP) for treatment of proliferative diabetic retinopathy. Long-term data on VF are important when considering either treatment approach. To further evaluate changes in VF throughout 5 years among eyes enrolled in the Protocol S clinical trial, conducted by the DRCR Retina Network. Post hoc analyses of an ancillary study within a multicenter (55 US sites) randomized clinical trial. Individuals with eyes with proliferative diabetic retinopathy enrolled in Protocol S were included. Data were collected from February 2012 to February 2018. Analysis began in June 2018. Panretinal photocoagulation or intravitreous injections of 0.5-mg ranibizumab. Diabetic macular edema, whenever present, was treated with ranibizumab in both groups. Panretinal photocoagulation could be administered to eyes in the ranibizumab group when failure or futility criteria were met. Mean change in total point score on VF testing with the Humphrey Field Analyzer 30-2 and 60-4 test patterns. Of 394 eyes enrolled in Protocol S, 234 (59.4%) were targeted for this ancillary study. Of these, 167 (71.4%) had VF meeting acceptable quality criteria at baseline (median [interquartile range] age, 50 [43-58] years; 90 men [53.9%]). At 5 years, 79 (33.8%) had results available. The mean (SD) change in total point score in the PRP and ranibizumab groups was -305 (521) dB and -36 (486) dB at 1 year, respectively, increasing to -527 (635) dB and -330 (645) dB at 5 years, respectively (P = .04). After censoring VF results after PRP treatments in the ranibizumab group, the 5-year mean change in total point score was -201 (442) dB. In a longitudinal regression analysis of change in total point score including both treatment groups, laser treatment was associated with a mean point decrease of 208 (95% CI, 112-304) dB for the initial PRP session, 77 (95% CI, 21-132) dB for additional PRP sessions, and 325 (95% CI, 211-439) dB for endolaser. No association was found between change in point score and the number of ranibizumab injections during the previous year (-9 per injection [95% CI, -22 to 3]). The limited data available from Protocol S suggest that there are factors besides PRP associated with VF loss in eyes treated for proliferative diabetic retinopathy. Further clinical research is warranted to clarify the finding. ClinicalTrials.gov identifier: NCT01489189.

Зміна органолептичних показників сиркової пасти з лляною олією за різних умов зберігання

The development of new types of recipes for curd products, which have ingredients rich in essential fatty acids, which increase their biological value, are promising for introduction into production. The results of the investigated changes of organoleptic indicators and values of titrated acid in the rupture of curd spread with different content of the linseed oil stored under the temperature of +2 ± 0.5 and + 6 ± 1 ° C were presented at the article. The organoleptic qualities of the curd spread samples were determined according to SSU (State Standards of Ukraine) 4503: 2005 Curd products. General Technical Conditions and our improved 10 point scale. The organoleptic qualities of cottage cheese were evaluated according to SSU 4554: 2006 Cottage cheese. General Technical Conditions. In this case, the curd spread was considered acceptable for production by organoleptic qualities with total score of not less than 9 points. The titrated acidity of the samples of curd spread with flax oil and cottage cheese was determined by the titrimetric method. It was found that the storage of experimental samples of curd spread at a temperature of + 2 ± 0,5 ° C for 7 days did not significantly affect the overall score. Thus, in the test samples containing 8% and 10% linseed oil, the total score was reduced by 0.1 points, compared with freshly made samples. At the same time, in the 12% linseed oil sample, the score was reduced by 0.4 points. It was found that after 14 days of storage of curd paste in samples containing 8% and 10% f linseed oil, the total point score decreased to 9.0 ± 0.2 points, and in the sample with 12% oil to 7.1 ± 0.2 points. However, the samples of curd spread containing 8% and 10% linseed oil were still considered acceptable for production, as the total score was not lower than 9 points. It is established that during storage of curd spread with f linseed oil at + 6 ± 1 ° C, a gradual increase in titrated acidity is observed, which adversely affects the organoleptic characteristics of the product. Storage of curd spread samples for longer than 7 days under these temperature conditions is not desirable due to a decrease in the overall score. Key words: cheese paste, linseed oil, sour milk cheese, organoleptic evaluation, titrated acidity, storage temperature, expiration date.

Dyspareunia in Female Fibromyalgia patients

Objective: To evaluate tender points number, pressure pain threshold and incidence of fibromyalgia in women with dyspareuniaPatients and Methods: Married women dyspareunia were compared to 35 healthy controls. Females were asked if they encountered sexual intercourse throughout the preceding month and dyspareunia was rated from zero to 3 by usingMarinoff Dyspareunia Scale (MDS). Dolorimeter (pressure algometer) was employed to assess pressure pain threshold. Diagnosis of Fibromyalgia was based on the 1990 American College of Rheumatology (ACR) criteria. Depression status of was assessed using the Beck Depression Inventory (BDI).Results: No significant statistical difference was found among both patients and control groups in relation to age, body mass index (BMI). Myalgic score and total tender point score was significantly lower in the dyspareunia group. Tender points score among dyspareunia patients was significantly higher. Themean score of Beck Depression Inventory (BDI) was 18.1±6.7 among dyspareunia group diagnosed with fibromyalgia compared to 11.3±4.1 in the dyspareunia group without fibromyalgiaConclusion: fibromyalgia is a contributing factor to dyspareunia