Category: Statistics

Stat Med. 2024 Jun 20. doi: 10.1002/sim.10157. Online ahead of print.

ABSTRACT

Meta-analysis is an essential tool to comprehensively synthesize and quantitatively evaluate results of multiple clinical studies in evidence-based medicine. In many meta-analyses, the characteristics of some studies might markedly differ from those of the others, and these outlying studies can generate biases and potentially yield misleading results. In this article, we provide effective robust statistical inference methods using generalized likelihoods based on the density power divergence. The robust inference methods are designed to adjust the influences of outliers through the use of modified estimating equations based on a robust criterion, even when multiple and serious influential outliers are present. We provide the robust estimators, statistical tests, and confidence intervals via the generalized likelihoods for the fixed-effect and random-effects models of meta-analysis. We also assess the contribution rates of individual studies to the robust overall estimators that indicate how the influences of outlying studies are adjusted. Through simulations and applications to two recently published systematic reviews, we demonstrate that the overall conclusions and interpretations of meta-analyses can be markedly changed if the robust inference methods are applied and that only the conventional inference methods might produce misleading evidence. These methods would be recommended to be used at least as a sensitivity analysis method in the practice of meta-analysis. We have also developed an R package, robustmeta, that implements the robust inference methods.

PMID:38899515 | DOI:10.1002/sim.10157

Mov Disord. 2024 Jun 20. doi: 10.1002/mds.29883. Online ahead of print.

ABSTRACT

BACKGROUND: Biallelic ZBTB11 variants have previously been associated with an ultrarare subtype of autosomal recessive intellectual developmental disorder (MRT69).

OBJECTIVE: The aim was to provide insights into the clinical and genetic characteristics of ZBTB11-related disorders (ZBTB11-RD), with a particular emphasis on progressive complex movement abnormalities.

METHODS: Thirteen new and 16 previously reported affected individuals, ranging in age from 2 to 50 years, with biallelic ZBTB11 variants underwent clinical and genetic characterization.

RESULTS: All patients exhibited a range of neurodevelopmental phenotypes with varying severity, encompassing ocular and neurological features. Eleven new patients presented with complex abnormal movements, including ataxia, dystonia, myoclonus, stereotypies, and tremor, and 7 new patients exhibited cataracts. Deep brain stimulation was successful in treating 1 patient with generalized progressive dystonia. Our analysis revealed 13 novel variants.

CONCLUSIONS: This study provides additional insights into the clinical features and spectrum of ZBTB11-RD, highlighting the progressive nature of movement abnormalities in the background of neurodevelopmental phenotype. © 2024 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

PMID:38899514 | DOI:10.1002/mds.29883

Nutr Rev. 2024 Jun 19:nuae073. doi: 10.1093/nutrit/nuae073. Online ahead of print.

ABSTRACT

CONTEXT: Diet quality indices (DQIs) assess the level of adherence to dietary recommendations and a specified dietary pattern in populations; however, there is limited evidence regarding the construct criteria and validation methodology of DQIs used in pregnant women.

OBJECTIVES: The objectives of this study were to (i) identify and describe characteristics of DQIs that have been validated for use in pregnant women in high-income countries, and (ii) evaluate criteria used to develop DQIs and validation methodologies employed.

DATA SOURCES: CINAHL, Embase, Medline, SCOPUS, and Web of Science were systematically searched for eligible articles published between 1980 and November 2022 that focused on DQIs validated for use in pregnant women from high-income countries.

DATA EXTRACTION: Characteristics, development criteria, and validation methodologies used in the included articles were extracted by one reviewer and checked by a second reviewer.

DATA ANALYSIS: A narrative synthesis and descriptive statistics were used to summarize the review findings. Reporting was guided by the Preferred Reporting Items for Systematic Reviews and Meta-analyses statement.

RESULTS: Six articles with 5 validated DQIs were identified. In total, 3777 participants were included – five articles had pregnant women aged 31-50 years and in their second trimester. Food frequency questionnaires were used as the dietary assessment method in all studies, and 3 DQIs were used to assess dietary intake at 1 time point, using 2 different dietary assessment methods. No indices fulfilled preferred features for the DQI development criteria developed by Burggraf et al (2018). Construct validity was assessed by all DQIs, followed by criterion validity (n = 4) and test-retest reliability (n = 2).

CONCLUSION: Limited high-quality validated DQIs for use in pregnant women in high-income countries were identified. Scoring for DQI components were not specific to nutrient requirements for pregnant women. Findings from this review may inform the development of DQIs that evaluate specific dietary requirements and specific food safety considerations applicable to pregnancy.

SYSTEMATIC REVIEW REGISTRATION: https://osf.io/u2hrq.

PMID:38899508 | DOI:10.1093/nutrit/nuae073

J Interprof Care. 2024 Jun 20:1-9. doi: 10.1080/13561820.2024.2367424. Online ahead of print.

ABSTRACT

Undertaking an authentic interprofessional simulation experience may be a useful and consistent strategy for healthcare professional students to build competencies required for a rural healthcare context. An observational comparative study design was adopted to evaluate a clinical simulation experience created to develop the interprofessional competencies of a sample of healthcare professional students at a regional university situated on multiple campuses in New South Wales (NSW), Australia. Over 200 students across three campuses of the university were involved in a simulation experience that included four interprofessional activities. Of these students, 189 (89%) agreed to participate in the study. The healthcare professional students who participated in the study were from second year occupational therapy, physiotherapy, and podiatry, and third year speech pathology programs. Retrospective pre and post self-assessed interprofessional collaborative competencies were compared for all students using the revised Interprofessional Collaborative Attainment Survey (ICCAS). Results demonstrated a statistically significant improvement in self-perceived scores using the validated revised ICCAS survey. The findings of this study suggest that carefully designed and authentic interprofessional simulation experiences can facilitate the development of competencies required for effective interprofessional practice, which are necessary for successful rural practice.

PMID:38899500 | DOI:10.1080/13561820.2024.2367424

Cancer Med. 2024 Jun;13(12):e7302. doi: 10.1002/cam4.7302.

ABSTRACT

INTRODUCTION: Severe immune-related adverse events (irAEs) due to immune checkpoint inhibitors (ICIs) can lead to admission to the intensive care unit (ICU). In this retrospective study, we determined the incidence, treatment patterns and survival outcomes of this patient population at a comprehensive cancer center.

METHODS: All patients admitted to the ICU due to irAEs from ICI treatment between January 2015 and July 2022 were included. Descriptive statistics were reported on patient characteristics and treatment patterns during hospital admission. Overall survival (OS) from the time of ICU discharge to death was estimated using the Kaplan-Meier method.

RESULTS: Over the study period, 5561 patients received at least one ICI administration, of which 32 patients (0.6%) were admitted to the ICU due to irAEs. Twenty patients were treated with anti-PD-1 plus anti-CTLA-4 treatment, whereas 12 patients were treated with ICI monotherapy. The type of irAEs were de novo diabetes-related ketoacidosis (n = 8), immune-related gastrointestinal toxicity (n = 8), myocarditis or myositis (n = 10), nephritis (n = 3), pneumonitis (n = 2), and myelitis (n = 1). The median duration of ICU admission was 3 days (interquartile range: 2-6 days). Three patients died during ICU admission. The median OS of the patients who were discharged from the ICU was 18 months (95% confidence interval, 5.0-NA).

CONCLUSION: The incidence of irAEs leading to ICU admission in patients treated with ICI was low in this study. ICU mortality due to irAEs was low and a subset of this patient population even had long-term survival.

PMID:38899457 | DOI:10.1002/cam4.7302

Scand J Rheumatol. 2024 Jun 20:1-8. doi: 10.1080/03009742.2024.2361542. Online ahead of print.

ABSTRACT

OBJECTIVE: To investigate changes in socioeconomic inequalities in patient-related outcomes and pain medication use, following participation in a digital self-management intervention for osteoarthritis (OA) in Sweden.

METHOD: Participants with hip/knee OA enrolled in the digital intervention were included. Self-reported outcomes collected were the numerical rating scale (NRS) pain, activity impairment, general health, Knee/Hip injury and Osteoarthritis Outcome Score (KOOS-12, HOOS-12) Pain, Function, and Quality of Life subscales, 5-level EuroQol 5 Dimensions (EQ-5D-5L), Patient Acceptable Symptom State (PASS) for function, walking difficulties, fear of movement, wish for surgery, pain medication use, physical function measured by the 30s chair-stand test, and level of physical activity. Educational attainment was used as a socioeconomic measure and the concentration index was used to assess the magnitude of inequalities at baseline and 3 month follow-up.

RESULTS: The study included 21,688 participants (mean ± sd age 64.1 ± 9.1 years, 74.4% females). All outcomes except for PASS demonstrated inequalities in favour of highly educated participants at both time-points, with highly educated participants reporting better outcomes. At 3 month follow-up, the magnitude of inequality widened for activity impairment, but narrowed for NRS pain, EQ-5D-5L, KOOS-12/HOOS-12 Pain and Function, physical function, and wish for surgery. There were no statistically conclusive changes in the magnitude of inequalities for the remaining outcomes.

CONCLUSIONS: There were inequalities in patient-related outcomes in favour of those with higher education among participants of a digital self-management intervention for OA, although the magnitude of these pre-existing inequalities generally narrowed after the 3 month intervention.

PMID:38899452 | DOI:10.1080/03009742.2024.2361542

Elife. 2024 Jun 20;13:RP93694. doi: 10.7554/eLife.93694.

ABSTRACT

Comprehensive biodiversity data is crucial for ecosystem protection. The Biome mobile app, launched in Japan, efficiently gathers species observations from the public using species identification algorithms and gamification elements. The app has amassed >6 million observations since 2019. Nonetheless, community-sourced data may exhibit spatial and taxonomic biases. Species distribution models (SDMs) estimate species distribution while accommodating such bias. Here, we investigated the quality of Biome data and its impact on SDM performance. Species identification accuracy exceeds 95% for birds, reptiles, mammals, and amphibians, but seed plants, molluscs, and fishes scored below 90%. Our SDMs for 132 terrestrial plants and animals across Japan revealed that incorporating Biome data into traditional survey data improved accuracy. For endangered species, traditional survey data required >2000 records for accurate models (Boyce index ≥ 0.9), while blending the two data sources reduced this to around 300. The uniform coverage of urban-natural gradients by Biome data, compared to traditional data biased towards natural areas, may explain this improvement. Combining multiple data sources better estimates species distributions, aiding in protected area designation and ecosystem service assessment. Establishing a platform for accumulating community-sourced distribution data will contribute to conserving and monitoring natural ecosystems.

PMID:38899444 | DOI:10.7554/eLife.93694

J Laparoendosc Adv Surg Tech A. 2024 Jun 20. doi: 10.1089/lap.2024.0144. Online ahead of print.

ABSTRACT

Introduction: Right colon cancer often requires surgical intervention, and complete mesocolic excision (CME) has emerged as a standard procedure. The study aims to evaluate and compare the safety and efficacy of robotic and laparoscopic CME for patients with right colon cancer and 5-year survival rates examined to determine the outcomes. Materials and Methods: Patients who underwent CME for right-sided colon cancer between 2014 and 2021 were included in this study. Group differences of age, body mass index, operation time, bleeding amount, total harvested lymph nodes, and postoperative stay were analyzed by the Mann-Whitney U test. Group differences of sex, American Society of Anesthesiology, and tumor, node, and metastasis stage were analyzed by the Chi-squared test. Disease-free and overall survival were assessed using Kaplan-Meier curves with the log-rank Mantel-Cox test. Results: From 109 patients, 74 of them were 1:1 propensity score matched and used for analysis. Total harvested lymph node (P ≤ .001) and estimated blood loss (P = .031) were found to be statistically significant between the groups. We found no statistically significant difference between the groups in terms of disease-free and overall survival (P = .27, .86, respectively), and the mortality rate was 9.17%, with no deaths directly attributed to the surgery. Conclusions: Study shows that minimally invasive surgery is a feasible option for CME in right colon cancers, with acceptable overall survival rates. Although the robotic approach has a higher lymph node yield, there was no significant difference in survival rates. Further randomized trials are needed to determine the clinical significance of both approaches.

PMID:38899434 | DOI:10.1089/lap.2024.0144

J Med Virol. 2024 Jun;96(6):e29757. doi: 10.1002/jmv.29757.

ABSTRACT

No effective treatments can ameliorate symptoms of long COVID patients. Our study assessed the safety and efficacy of human umbilical cord-derived mesenchymal stem cells (UC-MSCs) in the treatment of long COVID patients. Ten long COVID patients were enrolled and received intravenous infusions of UC-MSCs on Days 0, 7, and 14. Adverse events and clinical symptoms were recorded, and chest-high-resolution CT (HRCT) images and laboratory parameters were analyzed. During UC-MSCs treatment and follow-up, we did not observe serious adverse events, the symptoms of long COVID patients were significantly relieved in a short time, especially sleep difficulty, depression or anxiety, memory issues, and so forth, and the lung lesions were also repaired. The routine laboratory parameters did not exhibit any significant abnormalities following UC-MSCs transplantation (UMSCT). The proportion of regulatory T cells gradually increased, but it was not statistically significant until 12 months. The proportion of naive B cells was elevated, while memory B cells, class-switched B-cells, and nonswitched B-cells decreased at 1 month after infusion. Additionally, we observed a transient elevation in circulating interleukin (IL)-6 after UMSCT, while tumor necrosis factor (TNF)-α, IL-17A, and IL-10 showed no significant changes. The levels of circulating immunoglobulin (Ig) M increased significantly at month 2, while IgA increased significantly at month 6. Furthermore, the SARS-CoV-2 IgG levels remained consistently high in all patients at Month 6, and there was no significant decrease during the subsequent 12-month follow-up. UMSCT was safe and tolerable in long COVID patients. It showed potential in alleviating long COVID symptoms and improving interstitial lung lesions.

PMID:38899432 | DOI:10.1002/jmv.29757

Muscle Nerve. 2024 Jun 20. doi: 10.1002/mus.28184. Online ahead of print.

ABSTRACT

INTRODUCTION/AIMS: Efgartigimod, a neonatal Fc-receptor inhibitor, has recently been approved as treatment for myasthenia gravis (MG). In this retrospective cohort study, we aimed to systematically assess short- and long-term effectiveness of efgartigimod in patients with refractory MG.

METHODS: Sixteen patients with refractory autoimmune acetylcholine receptor MG were treated with efgartigimod. Data were collected from January 2021 to March 2023 on Myasthenia Gravis Activities of Daily Living (MG-ADL), Quantitative Myasthenia Gravis score (QMG), Myasthenia Gravis Composite score (MGC) and the 15-item revised version of the Myasthenia Gravis Quality of Life questionnaire (MG-QoL15r).

RESULTS: A favorable outcome was seen in 56% of patients at the last measurement. Out of 16 patients, 50% were an MG-ADL responder after the first treatment cycle. After 4 weeks, a clinically meaningful improvement compared to baseline was seen on the MG-ADL, QMG, and MGC. There was a statistically significant improvement on the MGQoL15r from baseline to week 4. The improvement was maintained until the last measurement for the MGC and the MGQoL15r. At the last visit, all patients had discontinued 4-weekly dosages, shifting to administration frequencies of 1, 2, or 3 weeks. Drug doses could be decreased for prednisolone (n = 7), azathioprine (n = 2), and intravenous immunoglobulin (n = 9). Frequency of plasma exchange was decreased in nine patients.

DISCUSSION: In patients with refractory MG, efgartigimod was effective for at least half of all patients. Patients required more frequent dosing compared to the ADAPT phase 3 trial. In 80% of the patients concurrent medication could be reduced or discontinued.

PMID:38899431 | DOI:10.1002/mus.28184