Nevin Manimala

Eye Vis (Lond). 2022 May 7;9(1):18. doi: 10.1186/s40662-022-00289-z.

ABSTRACT

BACKGROUND: The ability to view the posterior segment in keratoprosthesis (Kpro) implanted patients is limited. The purpose of this retrospective, observational study was to investigate the use of ultra-wide field (UWF) scanning laser ophthalmoscopy imaging and its utility for serial evaluation of the retina and optic nerve in patients with either a Boston type I or II Kpro.

METHODS: A retrospective chart review was performed for patients with a Boston type I or II Kpro seen at The Ohio State University Wexner Medical Center. Images were graded for quality by two masked observers on a defined four-point scale (“Poor”, “Fair”, “Good”, or “Very good”) and assessed for visible posterior segment anatomy. Interobserver agreement was described using the Kappa statistic coefficient (κ) with 95% confidence intervals.

RESULTS: A total of 19 eyes from 17 patients were included in this study. Eighteen eyes had a type I Kpro, while one eye had a type II Kpro. UWF imaging from 41 patient visits were reviewed by two observers. Interobserver agreement between the two graders was fair for image quality (κ = 0.36), moderate for visibility of the macula with discernible details (κ = 0.59), moderate for visibility of the anterior retina with discernable details (κ = 0.60), and perfect agreement for visibility of the optic nerve with discernible details (κ = 1.0). In 6 eyes, UWF imaging was performed longitudinally (range 3-9 individual visits), allowing for long-term follow-up (range 3-46 months) of posterior segment clinical pathology.

CONCLUSIONS: UWF imaging provides adequate and reliable visualization of the posterior segment in Kpro implanted patients. This imaging modality allowed for noninvasive longitudinal monitoring of retinal and optic nerve disease in this selected patient population.

PMID:35526055 | DOI:10.1186/s40662-022-00289-z

Orphanet J Rare Dis. 2022 May 7;17(1):186. doi: 10.1186/s13023-022-02342-5.

ABSTRACT

BACKGROUND: Design and analysis of clinical trials for rare and ultra-rare disease pose unique challenges to the practitioners. Meeting conventional power requirements is infeasible for diseases where sample sizes are inherently very small. Moreover, rare disease populations are generally heterogeneous and widely dispersed, which complicates study enrollment and design. Leveraging all available information in rare and ultra-rare disease trials can improve both drug development and informed decision-making processes.

MAIN TEXT: Bayesian statistics provides a formal framework for combining all relevant information at all stages of the clinical trial, including trial design, execution, and analysis. This manuscript provides an overview of different Bayesian methods applicable to clinical trials in rare disease. We present real or hypothetical case studies that address the key needs of rare disease drug development highlighting several specific Bayesian examples of clinical trials. Advantages and hurdles of these approaches are discussed in detail. In addition, we emphasize the practical and regulatory aspects in the context of real-life applications.

CONCLUSION: The use of innovative trial designs such as master protocols and complex adaptive designs in conjunction with a Bayesian approach may help to reduce sample size, select the correct treatment and population, and accurately and reliably assess the treatment effect in the rare disease setting.

PMID:35526036 | DOI:10.1186/s13023-022-02342-5

BMC Musculoskelet Disord. 2022 May 7;23(1):430. doi: 10.1186/s12891-022-05370-1.

ABSTRACT

BACKGROUND: Fibrillin-1 (FBN1) is an extracellular matrix glycoprotein essential to the structural component of microfibrils and FBN1 gene polymorphisms can be associated with adolescent idiopathic scoliosis (AIS) susceptibility. This study aimed to evaluate the potential role of the FBN1 rs12916536 polymorphism in AIS development or severity and the variation in Cobb angle in relation to patient’s characteristics.

METHODS: DNA from 563 subjects (185 AIS patients and 378 controls) were genotyped using a validated TaqMan allelic discrimination assay. A multivariate logistic regression model evaluated the association between polymorphism and AIS, using the adjusted odds ratios (OR) with their respective 95% confidence intervals (95% CI). A linear regression analysis evaluated the variation in Cobb angle according to the patient’s age and body mass index (BMI).

RESULTS: Among the AIS group there was a predominance of females (12:1), low or normal BMI (90%), 58% had a Cobb angle greater than 45° and 74% were skeletally mature. Age was a risk factor (4-fold) for curve progression higher than BMI (P < 0.001). The allelic frequency of the rs12916536 G > A polymorphism was 40% in controls and 31% in AIS cases; and this difference was statistically significant (P = 0.004). FBN1 rs12916536 GA + AA genotypes were associated with a lower risk of AIS susceptibility (OR = 0.58 and 95% CI = 0.35-0.98), after adjustment for age, sex and BMI. However, no significant differences were detected in polymorphism distribution with the severity of the disease (Cobb < 45° or ≥ 45°).

CONCLUSION: Age was a risk factor for progression of the scoliotic curve and FBN1 rs12916536 polymorphism a protective factor for AIS susceptibility.

PMID:35526034 | DOI:10.1186/s12891-022-05370-1

Arthritis Res Ther. 2022 May 7;24(1):102. doi: 10.1186/s13075-022-02788-9.

ABSTRACT

OBJECTIVE: Aortic valve involvement is not uncommon in patients with Takayasu arteritis (TAK) and leading to poor prognosis. The aim of our study was to explore the risk factors of aortic valve involvement and to evaluate the prognosis in TAK patients with aortic valve involvement.

METHOD: In this retrospective study, 172 TAK patients were divided into groups with or without aortic valve involvement to identify the risk factors. Patients who underwent aortic valve surgical treatment were followed up to assess cumulative incidence of postoperative adverse events.

RESULTS: A total of 92 TAK patients (53.49%) had aortic valvular lesion. The infiltration of inflammatory cells was found in surgical specimens of aortic valve. Numano type IIb, elevated high-sensitivity C-reactive protein (hs-CRP) level, and dilation of ascending aorta and aortic root were statistically associated with aortic valvular lesion in TAK patients (OR [95%CI] 6.853 [1.685-27.875], p=0.007; 4.896 [1.646-14.561], p=0.004; 4.509 [1.517-13.403], p=0.007; 9.340 [2.188-39.875], p=0.003). The 1-, 5-, and 7-year cumulative incidence of postoperative adverse events were 14.7%, 14.7%, and 31.8%, respectively. Surgical methods (p=0.024, hazard ratio (HR) 0.082) and postoperatively anti-inflammatory therapy (p=0.036, HR 0.144) were identified as potential predictors of postoperative adverse events.

CONCLUSIONS: Regularly echocardiogram screening is suggested in patients with Numano type IIb and aggressive treatment should be performed early in TAK patients. As for TAK patients with aortic valve surgery, aortic root replacement seems to be the preferred option and regular anti-inflammatory therapy may reduce the occurrence of adverse events of them.

PMID:35526024 | DOI:10.1186/s13075-022-02788-9

BMC Psychol. 2022 May 7;10(1):119. doi: 10.1186/s40359-022-00818-4.

ABSTRACT

BACKGROUND: Racial, ethnic, religious, and cultural diversity in Australia is rapidly increasing. Although Indigenous Australians account for only approximately 3.5% of the country’s population, over 50% of Australians were born overseas or have at least one migrant parent. Migration accounts for over 60% of Australia’s population growth, with migration from Asia, Sub-Saharan African and the Americas increasing by 500% in the last decade. Little is known about Australian mental health care practitioners’ attitudes toward this diversity and their level of cultural competence.

AIM: Given the relationship between practitioner cultural competence and the mental health outcomes of non-White clients, this study aimed to identify factors that influence non-White and White practitioners’ cultural competence.

METHODS: An online questionnaire was completed by 139 Australian mental health practitioners. The measures included: the Balanced Inventory of Desirable Responding (BIDR); the Multicultural Counselling Inventory (MCI); and the Color-blind Racial Attitudes Scale (CoBRAS). Descriptive statistics were used to summarise participants’ demographic characteristics. One-way ANOVA and Kruskal-Wallis tests were conducted to identify between-group differences (non-White compared to White practitioners) in cultural competence and racial and ethnic blindness. Correlation analyses were conducted to determine the association between participants’ gender or age and cultural competence. Hierarchical multiple regression analysis was conducted to predict cultural competence.

RESULTS: The study demonstrates that non-White mental health practitioners are more culturally aware and have better multicultural counselling relationships with non-White people than their White counterparts. Higher MCI total scores (measuring cultural competence) were associated with older age, greater attendance of cultural competence-related trainings and increased awareness of general and pervasive racial and/or ethnic discrimination. Practitioners with higher MCI total scores were also likely to think more highly of themselves (e.g., have higher self-deceptive positive enhancement scores on the BIDR) than those with lower MCI total scores.

CONCLUSION: The findings highlight that the current one-size-fits-all and skills-development approach to cultural competence training ignores the significant role that practitioner diversity and differences play. The recommendations from this study can inform clinical educators and supervisors about the importance of continuing professional development relevant to practitioners’ age, racial/ethnic background and practitioner engagement with prior cultural competence training.

PMID:35526020 | DOI:10.1186/s40359-022-00818-4

J Cardiothorac Surg. 2022 May 7;17(1):107. doi: 10.1186/s13019-022-01853-3.

ABSTRACT

OBJECTIVE: Segmentectomy has been reported as an alternative to lobectomy for small-sized NSCLC without detriment to survival. The long-term benefits of segmentectomy over lobectomy on pulmonary function have not been firmly established. This meta-analysis aims to compare postoperative changes in pulmonary function in NSCLC patients undergoing segmentectomy or lobectomy.

METHODS: Medline, Embase, Web of Science and Scopus were searched through March 2021. Statistical comparisons were made when appropriate.

RESULTS: Fourteen studies (2412 participants) out of 324 citations were included in this study. All selected studies were high quality, as indicated by the Newcastle-Ottawa scale for assessing the risk of bias. Clinical outcomes were compared between segmentectomy and lobectomy. ΔFEV1 [10 studies, P < 0.01, WMD = 0.40 (0.29, 0.51)], ΔFVC [4 studies, P < 0.01, WMD = 0.16 (0.07, 0.24)], ΔFVC% [4 studies, P < 0.01, WMD = 4.05 (2.32, 5.79)], ΔFEV1/FVC [2 studies, P < 0.01, WMD = 1.99 (0.90, 3.08)], and ΔDLCO [3 studies, P < 0.01, WMD = 1.30 (0.69, 1.90)] were significantly lower in the segmentectomy group than in the lobectomy group. Subgroup analysis showed that in stage IA patients, the ΔFEV1% [3 studies, P < 0.01, WMD = 0.26 (0.07, 0.46)] was significantly lower in the segmentectomy group. The ΔDLCO% and ΔMVV% were incomparable.

CONCLUSION: Segmentectomy preserves more lung function than lobectomy. There were significantly smaller decreases in FEV1, FVC, FVC%, FEV1/FVC and DLCO in the segmentectomy group than in the lobectomy group.

PMID:35526006 | DOI:10.1186/s13019-022-01853-3

Trials. 2022 May 7;23(1):372. doi: 10.1186/s13063-022-06312-x.

ABSTRACT

BACKGROUND: Platform trial designs are used increasingly in cancer clinical research and are considered an efficient model for evaluating multiple compounds within a single disease or disease subtype. However, these trial designs can be challenging to operationalise. The use of platform trials in oncology clinical research has increased considerably in recent years as advances in molecular biology enable molecularly defined stratification of patient populations and targeted therapy evaluation. Whereas multiple separate trials may be deemed infeasible, platform designs allow efficient, parallel evaluation of multiple targeted therapies in relatively small biologically defined patient sub-populations with the promise of increased molecular screening efficiency and reduced time for drug evaluation. Whilst the theoretical efficiencies are widely reported, the operational challenges associated with these designs (complexity, cost, regulatory, resource) are not always well understood. MAIN: In this commentary, we describe our practical experience of the implementation and delivery of the UK plasmaMATCH trial, a platform trial in advanced breast cancer, comprising an integrated screening component and multiple parallel downstream mutation-directed therapeutic cohorts. plasmaMATCH reported its primary results within 3 years of opening to recruitment. We reflect on the operational challenges encountered and share lessons learnt to inform the successful conduct of future trials. Key to the success of the plasmaMATCH trial was well co-ordinated stakeholder engagement by an experienced clinical trials unit with expert methodology and trial management expertise, a federated model of clinical leadership, a well-written protocol integrating screening and treatment components and including justification for the chosen structure and intentions for future adaptions, and an integrated funding model with streamlined contractual arrangements across multiple partners. Findings based on our practical experience include the importance of early engagement with the regulators and consideration of a flexible resource infrastructure to allow adequate resource allocation to support concurrent trial activities as adaptions are implemented in parallel to the continued management of patient safety and data quality of the ongoing trial cohorts.

CONCLUSION: Platform trial designs allow the efficient reporting of multiple treatment cohorts. Operational challenges can be overcome through multidisciplinary engagement, streamlined contracting processes, rationalised protocol and database design and appropriate resourcing.

PMID:35526005 | DOI:10.1186/s13063-022-06312-x

J Orthop Surg Res. 2022 May 7;17(1):256. doi: 10.1186/s13018-022-03145-x.

ABSTRACT

BACKGROUND: Few studies focus on the trauma-specific functional outcomes after surgical revascularization and risk factors contributing to poor outcomes in patients with acute blunt popliteal artery injury (PAI). The objective of this study was to investigate the long-term trauma-specific functional outcomes in patients with acute blunt PAI and identify the associated risk factors.

METHODS: There were 36 patients with acute blunt PAI who require surgical revascularization at a national trauma center of West China Hospital of Sichuan University between March 2010 and April 2019. After propensity matching, each patient was matched to one patient who did not have a concomitant vascular injury in control cohort. Functional outcomes were assessed with trauma-specific functional scores, physical examination of range of motion, nerve functional status and knee stability. A logistics regression model was established to determine the independent risk factors.

RESULTS: The 5-year (range 2-10 years) follow-up showed that 22 patients (22/36, 61.1%) had functional deficit due to limited activity or chronic neurological symptoms. Patients in vascular cohort had significantly decreased FIM score and AHFS score compared with matched patients without vascular involvement (P = 0.003 and P < 0.001), whereas there was no statistically significant difference in KSS (P = 0.136). Spearman correlation analysis of functional scores in vascular cohort showed that the FIM score was positively correlated with AHFS score (r = 0.926, P < 0.001), but not correlated with the KSS (r = – 0.007, P = 0.967). Additionally, there was significant difference in the range of motion of ankle between two groups (P < 0.001 and P = 0.034). Logistic regression analysis further demonstrated nerve injuries and compartment syndrome were risk factors for poor ankle function after surgery (OR 22.580, P = 0.036 and OR 12.674, P = 0.041).

CONCLUSION: Most patients who sustained blunt PAI had significant functional deficit associated with limited activity and chronic neurological symptoms of ankle and foot, and poor functional outcomes were related to nerve injury and compartment syndrome. Therefore, early and effective decompression for compartment syndrome remains the only potentially modifiable risk factor for improving functional outcomes following PAI.

PMID:35525991 | DOI:10.1186/s13018-022-03145-x

BMC Bioinformatics. 2022 May 7;23(1):168. doi: 10.1186/s12859-022-04700-3.

ABSTRACT

BACKGROUND: Dimension reduction and variable selection play a critical role in the analysis of contemporary high-dimensional data. The semi-parametric multi-index model often serves as a reasonable model for analysis of such high-dimensional data. The sliced inverse regression (SIR) method, which can be formulated as a generalized eigenvalue decomposition problem, offers a model-free estimation approach for the indices in the semi-parametric multi-index model. Obtaining sparse estimates of the eigenvectors that constitute the basis matrix that is used to construct the indices is desirable to facilitate variable selection, which in turn facilitates interpretability and model parsimony.

RESULTS: To this end, we propose a group-Dantzig selector type formulation that induces row-sparsity to the sliced inverse regression dimension reduction vectors. Extensive simulation studies are carried out to assess the performance of the proposed method, and compare it with other state of the art methods in the literature.

CONCLUSION: The proposed method is shown to yield competitive estimation, prediction, and variable selection performance. Three real data applications, including a metabolomics depression study, are presented to demonstrate the method’s effectiveness in practice.

PMID:35525975 | DOI:10.1186/s12859-022-04700-3

Cost Eff Resour Alloc. 2022 May 7;20(1):21. doi: 10.1186/s12962-022-00344-5.

ABSTRACT

BACKGROUND: Harmonic ACE +7 Shears with Advanced Hemostasis is an upgraded ultrasonic device, an ultrasonic surgical and electrosurgical system (USES). The study aimed to evaluate the economic and clinical effectiveness of the USES compared with the conventional ultrasonic scalpel (CUS) in gastrectomy.

METHODS: We conducted a single-center, retrospective cohort study using the electronic medical records in China. We collected intraoperative and postoperative data from gastric cancer patients who underwent the endoscope-assisted distal gastrectomy from 2018 to June 30, 2019. Procedure-related costs were estimated. We used linear regression by controlling a set of covariates to assess the effect of USES on outcomes.

RESULT: Out of 87 eligible patients, the USES group (40 patients) and CUS group (47 patients) were comparable in terms of age, medical history and stages of cancer. Compared with the CUS, the USES saved 4.27 hemoclips per person (95% CI 0.57-7.97, p < 0.05) and 34.18 ml intraoperative blood per person (95% CI 8.74-59.62 ml, p < 0.05), respectively. Postoperative length of stay (LOS) was shorter in the USES group (7.90 ± 1.95 vs. 9.26 ± 2.81 days) but the difference was not statistically significant (p = 0.05).

CONCLUSIONS: The USES group was associated with fewer hemoclips use and intraoperative blood loss in patients undergoing laparoscopic gastrectomy at comparable costs.

PMID:35525958 | DOI:10.1186/s12962-022-00344-5