Category: Statistics

Egypt Heart J. 2023 Dec 2;75(1):99. doi: 10.1186/s43044-023-00427-5.

ABSTRACT

BACKGROUND: Hypertrophic cardiomyopathy (HCM) is a hereditary myocardial disorder, often due to sarcomere gene mutations, characterized by the left ventricular hypertrophy. Current treatments offer symptomatic relief but lack specificity. Mavacamten, an allosteric inhibitor, has shown significant improvements in HCM patients in trials, reducing the requirement for invasive treatments. This meta-analysis assesses Mavacamten’s efficacy and safety as a targeted HCM intervention.

METHODS: This study examined four randomized controlled trials comparing Mavacamten to placebo in HCM patients. Each trial had a unique primary endpoint, and secondary outcomes included improvements in NYHA-FC, eligibility for septal reduction therapy (SRT) or undergoing it, adverse events (serious and treatment-related), atrial fibrillation, and non-sustained ventricular tachycardia. Statistical analysis involved calculating risk ratios (RRs) and assessing heterogeneity.

RESULTS: The four included studies showed minimal risk of bias and involved 503 patients with HCM (273 Mavacamten and 230 placebo). Mavacamten significantly increased the primary endpoint (RR 2.15, 95% CI 1.20-3.86, P = 0.01) and ≥ 1 NYHA-FC class (RR 2.21, 95% CI 1.48-3.3, P = 0.0001). Mavacamten group had lower rates of SRT compared to those receiving placebo (RR, 0.30, 95% CI 0.22-0.40; P < 0.00001). No significant differences existed in rates adverse events between the Mavacamten and placebo groups.

CONCLUSIONS: Our study suggests that Mavacamten may have therapeutic benefits for HCM patients, as indicated by its positive impact on certain endpoints. Further research with larger samples, longer follow-up, and comprehensive analysis is needed to understand Mavacamten’s safety and efficacy in HCM patients.

PMID:38041770 | DOI:10.1186/s43044-023-00427-5

Biochem Genet. 2023 Dec 2. doi: 10.1007/s10528-023-10580-w. Online ahead of print.

ABSTRACT

Data on the role of CYP2D6 and CYP3A4/5 polymorphisms in relation to risperidone (RIS) pharmacokinetics (PK) in children are relatively limited and inconsistent. This is partially attributable to the limited coverage of CYP2D6 and CYP3A4/5 metabolizer phenotypes, particularly those of poor and ultrarapid metabolizers (PMs and UMs), which has led to calls for studies of populations with a non-European background that may carry variants that are less frequent in Europeans. Children ≤ 18 years old with at least 8 weeks of a RIS-based regimen were recruited from three autism centers in Riyadh, Saudi Arabia. The primary outcomes measured were plasma concentrations of RIS and 9-hydroxyrisperidone (9-OH-RIS) and their dose-adjusted (C/D) ratios as a function of phenotypes and activity score (AS). For accurate DNA genotyping, targeted pharmacogenomic testing with the Axiom PharmacoFocus Array was performed via examination of a broad collection of probesets targeting CYP2D6 and CYP3A4/5 variants. The frequency of genotypes/phenotypes and the impact of their allele translation and phenoconversion-predicted enzyme activity were examined. The final cohort included 83 individuals. The most common CYP2D6 phenotype in our population was normal metabolizers (NMs, 66.3%). Inconsistent with some previous studies, the three phenotypes of intermediate metabolizers (IMs), NMs, and UMs were significantly different in terms of RIS concentration, the RIS/9-OH-RIS ratio, the RIS C/D ratio and the 9-OH-RIS C/D ratio. According to AS analyses, there were statistically significant differences in the RIS concentration (P = 0.013), RIS/9-OH-RIS ratio (P < 0.001) and RIS C/D ratio (P = 0.030) when patients were categorized into AS ≤ 1 vs. AS > 1. None of the CYP3A4/5 star allele translated phenotypes revealed a significant influence on any of the RIS PK parameters. Notably, neither CYP2D6 nor CYP3A4/5 phenotyping demonstrated a significant impact on the total active moiety, suggesting that other gene variants could modulate RIS PK. The study confirmed the previously reported partial impact of the CYP2D6 gene on RIS PK. However, future studies using contemporary genotyping techniques targeting a wide range of variants in other candidate genes must be conducted to further examine their interactive effects on RIS PK and the clinical response.

PMID:38041757 | DOI:10.1007/s10528-023-10580-w

Ir J Med Sci. 2023 Dec 2. doi: 10.1007/s11845-023-03583-4. Online ahead of print.

ABSTRACT

OBJECTIVE: The current study assessed the knowledge of the medical students regarding the nature of radiation, associated risks, and protective measures.

METHOD: A cross-sectional study was conducted among international students enrolled in different medical colleges/universities in Xian, PR China. A self-developed and self-reported questionnaire was used for the current study. The descriptive statistics was carried out to summarize the finding of the study. Chi-square and Fisher exact tests were conducted to assess the association of the demographics with knowledge level.

RESULTS: A total of 796 students responded among 980. Most of the participants fell in the age range of 20-25 years (42.6%), being male (67.1%) and having bachelor’s education (56.7%). Participants with previous experience was 58.7%. The age group of 20-25 years (p < 0.001), being male (p < 0.001), having bachelor education (p < 0.001), and previous experience (p = 0.009) was significantly associated with moderate to good knowledge. The overall knowledge about the nature of radiation (96%) and associated risk (82%) was good, but regarding protection, 59% of the participants showed good knowledge.

CONCLUSION: Most of the participants showed satisfactory result. However, the knowledge regarding protection measures was poor in almost half of the population.

PMID:38041750 | DOI:10.1007/s11845-023-03583-4

Pediatr Nephrol. 2023 Dec 2. doi: 10.1007/s00467-023-06224-1. Online ahead of print.

ABSTRACT

BACKGROUND: Split kidney function (SKF) is critical for treatment decision in pediatric patients with hydronephrosis and is commonly measured using renal scintigraphy (RS). Non-contrast-enhanced magnetic resonance urography (NCE-MRU) is increasingly used in clinical practice. This study aimed to investigate the feasibility of using NCE-MRU as an alternative to estimate SKF in pediatric patients with hydronephrosis, compared to RS.

METHODS: Seventy-five pediatric patients with hydronephrosis were included in this retrospective study. All patients underwent NCE-MRU and RS within 2 weeks. Kidney parenchyma volume (KPV) and texture analysis parameters were obtained from T2-weighted (T2WI) in NCE-MRU. The calculated split KPV (SKPV) percent and texture analysis parameters percent of left kidney were compared with the RS-determined SKF.

RESULTS: SKPV showed a significant positive correlation with SKF (r = 0.88, p < 0.001), while inhomogeneity was negatively correlated with SKF (r = – 0.68, p < 0.001). The uncorrected and corrected prediction models of SKF were established using simple and multiple linear regression. Bland-Altman plots demonstrated good agreement of both predictive models. The residual sum of squares of the corrected prediction model was lower than that of the uncorrected model (0.283 vs. 0.314) but not statistically significant (p = 0.662). Subgroup analysis based on different MR machines showed correlation coefficients of 0.85, 0.95, and 0.94 between SKF and SKPV for three different scanners, respectively (p < 0.05 for all).

CONCLUSIONS: NCE-MRU can be used as an alternative method for estimating SKF in pediatric patients with hydronephrosis when comparing with RS. Specifically, SKPV proves to be a simple and universally applicable indicator for predicting SKF. A higher resolution version of the Graphical abstract is available asSupplementary information.

PMID:38041747 | DOI:10.1007/s00467-023-06224-1

Neurosurg Rev. 2023 Dec 2;46(1):323. doi: 10.1007/s10143-023-02210-1.

ABSTRACT

The endoscopic transventricular transchoroidal approach facilitates entry into the posterior part of the third ventricle, allowing a visualization field from the foramen of Monro to the pineal region through this anatomical corridor. Combined surgery to treat the target lesion and possible endoscopic third ventriculostomy (ETV) can be performed through a single burr hole. A detailed description of this surgical technique is given, and a series of cases from our center is presented. This retrospective study included patients with lesions in the pineal region or posterior zone of the third ventricle who underwent surgery between 2004 and 2022 in our center for tumor biopsy or endoscopic cyst fenestration. In nine cases, the transchoroidal approach was performed. Demographic and clinical variables were collected: sex, age at diagnosis, clinical presentation, characteristics of the lesion, pathological diagnosis, characteristics of the procedure, complications, subsequent treatments, evolution, follow-up time, and degree of success of the endoscopic procedure. The mean and range of the quantitative variables and frequency of the qualitative variables were analyzed, together with the statistical significance (p < 0.05). Surgical planning was carried out by performing a preoperative MRI, calculating the ideal entry point and trajectory for each case. The preoperative planning of the surgical technique is described in detail. Of our sample, 55.6% were women, with a mean age of 35 years (7-78). The most common clinical presentation was intracranial hypertension (55.6%), with or without a focus. Eight patients presented hydrocephalus at diagnosis. The most frequent procedure was endoscopic biopsy with ETV (66.7%). The pathological diagnosis varied widely. Procedure-related complications included one case of self-limited bleeding of the choroidal fissure at its opening and one intraventricular hemorrhage due to tumor bleeding in the postoperative period. Non-procedure-related complications comprised two ETV failures and one case of systemic infection, while late complications included one case of disease progression and one case of radionecrosis. Four patients died, one due to poor neurological evolution after post-surgical tumor bleeding and three due to causes unrelated to the procedure. The rest of the patients had a favorable evolution and were asymptomatic or stable. The transchoroidal approach through a single burr hole is a feasible and safe option for access to the posterior part of the third ventricle. Proper planning of each case is necessary to avoid complications.

PMID:38041741 | DOI:10.1007/s10143-023-02210-1

Eur J Clin Microbiol Infect Dis. 2023 Dec 2. doi: 10.1007/s10096-023-04720-8. Online ahead of print.

ABSTRACT

PURPOSE: To evaluate the performance of core genome multilocus sequence typing (cgMLST) for genotyping Mycobacterium tuberculosis (M.tuberculosis) Strains in regions where the lineage 2 strains predominate.

METHODS: We compared clustering by whole-genome SNP typing with cgMLST clustering in the analysis of WGS data of 6240 strains from five regions of China. Using both the receiver operating characteristic (ROC) curve and epidemiological investigation to determine the optimal threshold for defining genomic clustering by cgMLST. The performance of cgMLST was evaluated by quantifying the sensitivity, specificity and concordance of clustering between two methods. Logistic regression was used to gauge the impact of strain genetic diversity and lineage on cgMLST clustering.

RESULTS: The optimal threshold for cgMLST to define genomic clustering was determined to be ≤ 10 allelic differences between strains. The overall sensitivity and specificity of cgMLST averaged 99.6% and 96.3%, respectively; the concordance of clustering between two methods averaged 97.1%. Concordance was significantly correlated with strain genetic diversity and was 3.99 times (95% CI, 2.94-5.42) higher in regions with high genetic diversity (π > 1.55 × 10^-4) compared to regions with low genetic diversity. The difference missed statistical significance, while concordance for lineage 2 strains (96.8%) was less than that for lineage 4 strains (98.3%). CONCLUSION : cgMLST showed a discriminatory power comparable to whole-genome SNP typing and could be used to genotype clinical M.tuberculosis strains in different regions of China. The discriminative power of cgMLST was significantly correlated with strain genetic diversity and was slightly lower with strains from regions with low genetic diversity.

PMID:38041721 | DOI:10.1007/s10096-023-04720-8

Ophthalmol Ther. 2023 Dec 2. doi: 10.1007/s40123-023-00823-9. Online ahead of print.

ABSTRACT

INTRODUCTION: Goniosynechialysis (Phaco-GSL) is a logical therapeutic approach for patients with primary angle-closure glaucoma (PACG) and cataract. The aim of this study was to compare the long-term effectiveness and safety of Phaco-GSL and trabeculectomy (TRB) in the management of PACG with coexisting cataract.

METHODS: A review was conducted on 96 Chinese patients (96 eyes) with PACG and cataract from Peking Union Medical College Hospital (PUMCH). Among them, 56 patients underwent Phaco-GSL, while 40 underwent TRB. Intraocular pressure (IOP), best corrected visual acuity (BCVA), use of supplemental antiglaucoma medical therapy, surgery success rates, and complications for both procedures were assessed.

RESULTS: The average follow-up period was 50.3 ± 18.7 months in the Phaco-GSL group and 61.2 ± 15.1 months in the TRB group. At the final follow-up, IOP decreased from 27.0 ± 11.1 mmHg to 13.5 ± 2.1 mmHg in the Phaco-GSL group and in the TRB group IOP decreased from 27.1 ± 7.7 mmHg to 16.5 ± 5.5 mmHg. The long-term postoperative IOP in the Phaco-GSL group was significantly lower than that in the TRB group. There was a statistically significant reduction in medication usage in both groups, with the TRB group having a higher number of postoperative medications at the final follow-up. The incidence of postoperative complications was significantly higher in the TRB group compared to the Phaco-GSL group.

CONCLUSIONS: Phaco-GSL is the recommended surgical approach for Chinese patients with primary angle-closure glaucoma (PACG) and cataract. Compared to traditional TRB, eyes undergoing Phaco-GSL show a reduced requirement for antiglaucomatous medications, improved management of IOP, decreased risk of complications, and higher long-term cumulative probability of treatment success for patients with PACG.

PMID:38041720 | DOI:10.1007/s40123-023-00823-9

Trop Anim Health Prod. 2023 Dec 2;55(6):427. doi: 10.1007/s11250-023-03854-3.

ABSTRACT

Our objective was to use measures of intake and productive performance to adjust prediction models for the carcass traits of non-castrated Nellore cattle finished in a feedlot. Individual data from 168 non-castrated male Nellore steers finished in feedlot between the years 2016-2021 were used. Descriptive statistical analyzes and Pearson correlation coefficients were performed. The outliers were tested by evaluating the studentized residuals in relation to the values predicted by the equations. Residues that were outside the range of -2.5 to 2.5 were removed. The goodness of fit of the developed equations was evaluated by the coefficients of determination (R²) and root mean square error (RMSE). Models for carcass yield, subcutaneous fat thickness, ribeye area, and shear force were adjusted. Means of 53.5% carcass yield, 4.8 mm subcutaneous fat thickness, 73 cm² loin eye area, and 8.1 kg shear force were observed. The observed average intakes were 9.9 kg/day of dry matter, 3.3 kg/day of neutral detergent fiber content, 1.5 kg/day of crude protein, and 7.1 kg/day of total digestible nutrients. The average confinement time was 113 days, the average total weight gain was 152.2 kg and the average daily gain was 1.35 kg/day. Intake measures significantly correlated with shear force and subcutaneous fat thickness and ribeye area. Carcass yield was significantly correlated with total weight gain, feedlot time, and hot carcass weight. Measures of nutrient intake, performance, and confinement time can be used as predictors of carcass yield, ribeye area, fat thickness, and shear force of non-castrated Nellore cattle finished in a feedlot. The prediction equations for ribeye area, carcass yield, subcutaneous fat thickness, and shear force showed sufficient precision and accuracy for non-castrated Nellore cattle finished in confinement systems under tropical conditions. All equations can be used with caution to estimate carcass traits of cattle finished in a feedlot using measures of intake and productive performance.

PMID:38041713 | DOI:10.1007/s11250-023-03854-3

Curr Pain Headache Rep. 2023 Dec 2. doi: 10.1007/s11916-023-01189-0. Online ahead of print.

ABSTRACT

PURPOSE OF REVIEW: This systematic review aims to inform the current state of evidence about the efficacy and effectiveness of medical cannabis use for the treatment of LBP, specifically on pain levels and overall opioid use for LBP. Searches were conducted in MEDLINE (PubMed), Embase, and CINAHL. The search was limited to the past 10 years (2011-2021). Study inclusion was determined by the critical appraisal process using the Joanna Briggs Institute framework. Only English language articles were included. Participant demographics included all adult individuals with LBP who were prescribed medical cannabis for LBP and may be concurrently using opioids for their LBP. Study quality and the risk of bias were both evaluated. A narrative synthesis approach was used.

RECENT FINDINGS: A total of twelve studies were included in the synthesis: one randomized controlled trial (RCT), six observational studies (one prospective, four retrospective, and one cross-over), and five case studies. All study results, except for the RCT, indicated a decrease in LBP levels or opioid use over time after medical cannabis use. The RCT reported no statistically significant difference in LBP between cannabis and placebo groups. Low back pain (LBP) affects 568 million people worldwide. In the United States, LBP treatment represents more than half of regular opioid users. With the opioid epidemic, alternative methods, particularly medical cannabis, is now increasingly sought by practicing physicians and patients. Due to its infancy, there is minimal high-quality evidence to support medical cannabis use as a first line treatment for LBP.

PMID:38041708 | DOI:10.1007/s11916-023-01189-0

Br J Ophthalmol. 2023 Nov 27:bjo-2023-323480. doi: 10.1136/bjo-2023-323480. Online ahead of print.

ABSTRACT

BACKGROUND: Orbital decompression is commonly performed to correct proptosis in patients with Graves’ orbitopathy (GO). In literature, the incidence of new-onset constant diplopia after orbital decompression varies. Few studies have evaluated changes in duction and cyclodeviation after orbital decompression. We evaluated the changes in duction, cyclodeviation, eye position and degree of diplopia after orbital decompression.

METHODS: We retrospectively analysed data from patients who underwent orbital decompression at our hospital between January 2016 and July 2020. Data regarding the type of decompression, eye position, duction, cyclodeviation and level of diplopia according to the Gorman score were recorded.

RESULTS: Data from 281 eyes/orbits of 156 patients were analysed. Proptosis decreased from 23.8±2.5 to 20.9±2.5 mm. Horizontal and vertical duction range decreased (horizontal d=0.40 and vertical 0.30; p=0.000) after surgery; however, the change was not clinically significant (≤5°). Horizontal deviation changed towards esodeviation (d=-0.45; p=0.000), whereas vertical deviation remained stable (d=0.15; p=0.161). Preoperative cyclotorsion in the primary and downgaze positions changed towards incyclodeviation (primary: d=0.30, p=0.021; downgaze d=0.30, p=0.039). Diplopia improved in 22% (18 patients), whereas new-onset constant diplopia developed in 11% (12 patients). Elevation has an excellent predictive value of causing new-onset constant diplopia when measured preoperatively as <19°, sensitivity 88.0%, specificity 41.7% (area under the curve 0.812 (95% CI 0.660 to 0.963); p=0.000).

CONCLUSION: After orbital decompression, incidence of new-onset constant diplopia was 11% and 22% of the patients had partial or complete improvement of their diplopia. The changes in duction, cyclodeviation and horizontal deviation showed a statistical but not clinical difference. Patients with GO and a preoperatively restricted elevation of <19° are at risk of developing new-onset constant diplopia. In addition, preoperative incyclodeviation may worsen after decompression surgery.

PMID:38041682 | DOI:10.1136/bjo-2023-323480