Category: Statistics

Semin Thromb Hemost. 2022 Jan 12. doi: 10.1055/s-0041-1742092. Online ahead of print.

NO ABSTRACT

PMID:35021248 | DOI:10.1055/s-0041-1742092

Eur J Orthod. 2022 Jan 11:cjab080. doi: 10.1093/ejo/cjab080. Online ahead of print.

ABSTRACT

BACKGROUND: There has been an increased interest in conducting healthcare economic evaluations. Also, orthodontic treatments have gathered focus from an economic point of view, however orthodontic research seldom examines both clinical and economic outcomes.

OBJECTIVE: To evaluate and compare the costs of three retention methods: a bonded retainer to the maxillary four incisors, a bonded retainer to the maxillary four incisors and canines, and a removable vacuum-formed retainer (VFR) in the maxilla. The null hypothesis was that there was no difference in costs for the three types of retention methods.

TRIAL DESIGN: Three-arm, parallel group, single-centre, randomized controlled trial.

MATERIALS AND METHODS: Ninety adolescent patients, 54 girls and 36 boys, treated with fixed or removable retainers in the maxilla, were recruited to the study. The patients were randomized in blocks of 30, by an independent person, to one of three groups: bonded multistranded PentaOne (Masel Orthodontics) retainer 13-23, bonded multistranded PentaOne (Masel Orthodontics) retainer 12-22, and removable VFR. A cost analysis was made regarding chair time costs based on the costs per hour for the specialist in orthodontics, and material costs plus any eventual costs for repairs of the appliance. Changes in Little’s irregularity index and in single contact point discrepancies (CPDs) were measured on digitalized three-dimensional study casts. Data were evaluated on an intention-to-treat basis. The analysis was performed at 2 years of retention.

RESULTS: No statistically significant difference in costs between the maxillary fixed retainers and the VFRs was found, however, the material and emergency costs were significantly higher for the VFR compared with the bonded retainers. All three retention methods showed equally effective retention capacity, and no statistically significant differences in irregularity or CPDs of the maxillary anterior teeth in the three groups was detected.

LIMITATIONS: It was a single-centre trial, and hence less generalizable. Costs depended on local factors, and consequently, cannot be directly transferred to other settings.

CONCLUSIONS: All three retention methods can be recommended when considering costs and retention capacity.

TRIAL REGISTRATION: NCT04616755.

PMID:35021204 | DOI:10.1093/ejo/cjab080

Eur J Cardiothorac Surg. 2022 Jan 11:ezab575. doi: 10.1093/ejcts/ezab575. Online ahead of print.

ABSTRACT

OBJECTIVES: Type 2 diabetes mellitus (DM) is a frequent comorbidity among patients suffering from advanced heart failure necessitating a left ventricular assist device (LVAD) implant. The goal of this study was to evaluate the impact of type 2 DM on early and long-term outcomes of patients following an LVAD implant.

METHODS: We performed an observational cohort study in a large tertiary care centre in Israel. All data of patients who underwent a continuous flow LVAD implant between 2006 and 2020 were extracted from our departmental database. Patients were divided into 2 groups: group I (patients without diabetes) and group II (patients with diabetes). We compared short-term (30-day and 3-month) mortality, intermediate-term (1- and 3-year) mortality and long-term (5 year) mortality between the 2 groups.

RESULTS: The study population included 154 patients. Group I (patients without diabetes) comprised 88 patients and group II (patients with diabetes) comprised 66 patients. The mean follow-up duration was 38.2 ± 30.3 months. Short- and intermediate-term mortality (30 days, 1 year and 3 years) was higher in the group with DM compared with the group without DM but did not reach any statistically significant difference: 16.1% vs 9.8% (P = 0.312), 24.2% vs 17.3% (P = 0.399) and 30.6% vs 21.9% (P = 0.127) respectively. Long-term 5-year mortality was significantly higher in the group with DM compared to the group without: 38.7% vs 24.4% (P = 0.038). Furthermore, predictors of long-term mortality included diabetes (hazard ratio 2.09, confidence interval 1.34-2.84, P = 0.004), as demonstrated by regression analysis.

CONCLUSIONS: Patients with diabetes and those without diabetes have similar 30-day and short- and intermediate-term mortality rates. The mortality risk of diabetic patients begins to increase 3 years after an LVAD implant. Diabetes is an independent predictor of long-term, 5-year mortality after an LVAD implant.

CLINICAL TRIAL REGISTRATION: Ethical Committee of Sheba Medical Centre, Israel, on 2 December 2014, Protocol 4257.

PMID:35021207 | DOI:10.1093/ejcts/ezab575

Brief Bioinform. 2022 Jan 11:bbab567. doi: 10.1093/bib/bbab567. Online ahead of print.

ABSTRACT

The cell type identification is among the most important tasks in single-cell RNA-sequencing (scRNA-seq) analysis. Many in silico methods have been developed and can be roughly categorized as either supervised or unsupervised. In this study, we investigated the performances of 8 supervised and 10 unsupervised cell type identification methods using 14 public scRNA-seq datasets of different tissues, sequencing protocols and species. We investigated the impacts of a number of factors, including total amount of cells, number of cell types, sequencing depth, batch effects, reference bias, cell population imbalance, unknown/novel cell type, and computational efficiency and scalability. Instead of merely comparing individual methods, we focused on factors’ impacts on the general category of supervised and unsupervised methods. We found that in most scenarios, the supervised methods outperformed the unsupervised methods, except for the identification of unknown cell types. This is particularly true when the supervised methods use a reference dataset with high informational sufficiency, low complexity and high similarity to the query dataset. However, such outperformance could be undermined by some undesired dataset properties investigated in this study, which lead to uninformative and biased reference datasets. In these scenarios, unsupervised methods could be comparable to supervised methods. Our study not only explained the cell typing methods’ behaviors under different experimental settings but also provided a general guideline for the choice of method according to the scientific goal and dataset properties. Finally, our evaluation workflow is implemented as a modularized R pipeline that allows future evaluation of new methods. Availability: All the source codes are available at https://github.com/xsun28/scRNAIdent.

PMID:35021202 | DOI:10.1093/bib/bbab567

ORL J Otorhinolaryngol Relat Spec. 2022 Jan 12:1-11. doi: 10.1159/000520672. Online ahead of print.

ABSTRACT

INTRODUCTION: Despite multiple prognostic indicators described for oral cavity squamous cell carcinoma (OCSCC), its management still continues to be a matter of debate. Machine learning is a subset of artificial intelligence that enables computers to learn from historical data, gather insights, and make predictions about new data using the model learned. Therefore, it can be a potential tool in the field of head and neck cancer.

METHODS: We conducted a systematic review.

RESULTS: A total of 81 manuscripts were revised, and 46 studies met the inclusion criteria. Of these, 38 were excluded for the following reasons: use of a classical statistical method (N = 16), nonspecific for OCSCC (N = 15), and not being related to OCSCC survival (N = 7). In total, 8 studies were included in the final analysis.

CONCLUSIONS: ML has the potential to significantly advance research in the field of OCSCC. Advantages are related to the use and training of ML models because of their capability to continue training continuously when more data become available. Future ML research will allow us to improve and democratize the application of algorithms to improve the prediction of cancer prognosis and its management worldwide.

PMID:35021182 | DOI:10.1159/000520672

Brief Bioinform. 2022 Jan 11:bbab572. doi: 10.1093/bib/bbab572. Online ahead of print.

ABSTRACT

With the increasing volume of human sequencing data available, analysis incorporating external controls becomes a popular and cost-effective approach to boost statistical power in disease association studies. To prevent spurious association due to population stratification, it is important to match the ancestry backgrounds of cases and controls. However, rare variant association tests based on a standard logistic regression model are conservative when all ancestry-matched strata have the same case-control ratio and might become anti-conservative when case-control ratio varies across strata. Under the conditional logistic regression (CLR) model, we propose a weighted burden test (CLR-Burden), a variance component test (CLR-SKAT) and a hybrid test (CLR-MiST). We show that the CLR model coupled with ancestry matching is a general approach to control for population stratification, regardless of the spatial distribution of disease risks. Through extensive simulation studies, we demonstrate that the CLR-based tests robustly control type 1 errors under different matching schemes and are more powerful than the standard Burden, SKAT and MiST tests. Furthermore, because CLR-based tests allow for different case-control ratios across strata, a full-matching scheme can be employed to efficiently utilize all available cases and controls to accelerate the discovery of disease associated genes.

PMID:35021184 | DOI:10.1093/bib/bbab572

Lifestyle Genom. 2022 Jan 12:1-10. doi: 10.1159/000519916. Online ahead of print.

ABSTRACT

INTRODUCTION: The oral cavity is home to a diverse and distinct microbiome. While the role of oral bacteria in cariogenic and other dental diseases is irrefutable, their beneficial effects in the form of probiotics (PB) has been less studied, especially pertaining to oral diseases in children. This study compares the efficacy of a PB mouthrinse with 0.12% chlorhexidine (CHX) and 0.05% sodium fluoride (NaF) mouthrinse on the colony counts of mutans streptococci (MS) in children.

METHODS: A triple-blind crossover randomized trial between interventional groups was planned. Fifty-one children between 8 to 12 years of age were divided into three groups (I, II, and III) and were exposed to all three mouthrinses (A, B, and C) by randomized allocation for a period of two weeks with an inter-phase washout period of four weeks. Pre- and post-interventional MS counts (CFU/mL) were assessed, and the mean change was analysed using the t test (intragroup) and ANOVA (intergroup and crossover).

RESULTS: The mean changes in the colony counts obtained with the use of PB, CHX, and NaF mouthrinses were -1.0223 (-1.2201 to -0.8246), -0.9564 (-1.1503 to -0.7626), and -0.9511 (-1.1554 to -0.7467), respectively, which were statistically significant (p < 0.0001). However, the intergroup comparison for the mean change in colony counts revealed no statistically significant differences (p > 0.05).

CONCLUSION: The study concluded that the PB mouthrinse was equally efficacious as compared to CHX and NaF mouthrinses against MS in 8- to 12-year-old children. However, further studies are recommended to strengthen the evidence.

PMID:35021171 | DOI:10.1159/000519916

Transplant Cell Ther. 2022 Jan 9:S2666-6367(22)00002-1. doi: 10.1016/j.jtct.2022.01.002. Online ahead of print.

ABSTRACT

Haploidentical related donor (HRD) is a common alternative donor strategy used when matched sibling or unrelated donors are not available for hematopoietic stem cell transplantation (HSCT). However, there have been no studies comparing HRD HSCT with post-transplant cyclophosphamide (PTCy) and matched unrelated donor (MUD) HSCT with antithymocyte globulin, using similar busulfan-based myeloablative conditioning regimen in pediatric acute leukemia. Here, we compared the outcomes in children and adolescents with high-risk acute leukemia after HRD HSCT with PTCy (n=35) and MUD HSCT (n=45) after targeted busulfan-based myeloablative conditioning using intensive pharmacokinetic monitoring. The median follow-up times of the HRD and MUD groups were 3.7 and 4.6 years, respectively. No engraftment failure was observed in both groups. The cumulative incidence of acute graft-versus-host disease (GVHD) grades II-IV (34.3% versus 48.9%, p=0.142), grades III-IV (2.9% vs. 8.9%, p=0.272), moderate to severe chronic GVHD (11.4% vs. 18.3%, p=0.417), relapse (25.6% vs. 28.0%, p=0.832), and non-relapse mortality (0% vs. 2.2%, p=0.420) were not significantly different between the two groups. The 3-year severe chronic GVHD-free/relapse-free (GRFS), leukemia-free (LFS) and overall survival (OS) rates in the HRD and MUD groups were 62.9% (95% confidence intervals [CI], 45.8%, 80.0%) versus 49.8% (95% CI, 34.9%, 64.7%; p=0.318), 74.4% (95% CI, 58.7%, 90.1%) versus 67.5% (95% CI, 53.4%, 81.6%; p=0.585), and 88.6% (95% CI, 78.0%, 99.2%) versus 83.7% (95% CI, 72.5%, 94.9%; p=0.968), respectively. In a subgroup analysis of acute lymphoblastic leukemia patients (HRD, n=17; MUD, n=26), the 3-year GRFS, LFS, and OS rates of the HRD and MUD groups were 49.4% (95% CI, 24.3%, 74.5%) versus 39.5% (95% CI, 19.7%, 59.3%; p=0.601), 61.8% (95% CI, 37.5%, 86.1%) versus 63.6% (95% CI, 44.4%, 82.8%; p=0.872), and 82.4% (95% CI, 64.4%, 100%) versus 84.2% (95% CI, 70.1%, 98.3%; p=0.445), respectively. In acute myeloid leukemia patients (HRD, n=16; MUD, n=16), the 3-year GRFS, LFS, and OS rates of the HRD and MUD groups were 80.8% (95% CI, 61.2%, 100%) versus 61.9% (95% CI, 37.8%, 86.0%; p=0.326), 87.1% (95% CI, 70.2%, 100%) versus 73.9% (95% CI, 51.8%, 96.0%; p=0.478), and 93.8% (95% CI, 81.8%, 100%) versus 85.6% (95% CI, 67.0%, 100%; p=0.628), respectively. Although the difference was not statistically significant and the number of patients was small, the promising outcomes of HRD HSCT in AML patients were encouraging. Our results demonstrated that HRD HSCT with PTCy using a targeted busulfan-based myeloablative conditioning shows outcomes similar to those of MUD HSCT with antithymocyte globulin. HRD HSCT with PTCy could be a feasible option for pediatric high-risk acute leukemia patients who lack an HLA-matched related or unrelated donor.

PMID:35021131 | DOI:10.1016/j.jtct.2022.01.002

Folia Phoniatr Logop. 2022 Jan 12. doi: 10.1159/000521906. Online ahead of print.

ABSTRACT

INTRODUCTION: Assessment of intelligibility in dysarthria tends to rely on oral reading of sentences or words. However, self-generated utterances are closer to a clients’ natural speech. This study investigated how transcription of utterances elicited by picture description can be used in the assessment of intelligibility in speakers with Parkinson’s disease.

METHODS: Speech samples from eleven speakers with Parkinson’s disease and six neurologically healthy persons were audio-recorded. Forty-two naive listeners completed transcriptions of self-generated sentences from a picture description task and orally read sentences from the Swedish Test of Intelligibility, as well as scaled ratings of narrative speech samples.

RESULTS: Intelligibility was higher in orally read than self-generated sentences and higher for content words than for the whole sentence in self-generated sentences for most of the speakers, although these within-group differences were not statistically significant at group level. Adding contextual leads for the listeners increased intelligibility in self-generated utterances significantly, but with individual variation. Although correlations between the intelligibility measures were at least moderate or strong, there was a considerable inter- and intra-speaker variability in intelligibility scores between tasks for the speakers with Parkinson’s disease, indicating individual variation of factors that impact intelligibility. Intelligibility scores from neurologically healthy speakers were generally high across tasks with no significant differences between the conditions.

DISCUSSION/CONCLUSION: Within-speaker variability support literature recommendations to use multiple methods and tasks when assessing intelligibility. The inclusion of transcription of self-generated utterances elicited by picture description to the intelligibility assessment has the potential to provide additional information to assessment methods based on oral reading of pre-scripted sentences, and to inform the planning of interventions.

PMID:35021169 | DOI:10.1159/000521906

Curr Probl Cardiol. 2022 Jan 9:101111. doi: 10.1016/j.cpcardiol.2022.101111. Online ahead of print.

ABSTRACT

BACKGROUND: Obstructive sleep apnea (OSA) is increasingly recognized to be a risk factor for cardiovascular disease. This pilot study assessed the association of OSA and invasive coronary microvascular function in patients with ischemia with no obstructive coronary artery disease (INOCA).

METHODS: Forty-two patients with angina, were prospectively screened at a single tertiary centre covering the northern metropolitan area of South Australia, from February 2018 to December 2020 (ACTRN12618000149268). Forty patients were invited into to this observational study after coronary angiography demonstrated INOCA and functional coronary vasomotor disorder (n = 40). Twenty one participants subsequently underwent a sleep study for OSA diagnosis while 9 participants had prior formal diagnosis of OSA (ACTRN12618000227291).

RESULTS: Of the 30 participants with OSA data, 87% (n=26) had a diagnosis of OSA. Accordingly, 11 with mild severity, 7 with moderate severity and 8 with severe OSA. No OSA was observed in 4 participants. Participants with OSA were older [61.4±8.7 vs 49.9±9.7, p=0.002] with similar clinical characteristics for the pattern and severity of angina and other co-morbidities. 73.3% (n=22) had abnormal functional disorders of the epicardial coronary arteries and/or coronary microcirculation. On multivariate analysis, OSA was the only statistically significant association with functional coronary microvascular disorders [OR 53.95, 1.41-2065.01, p=0.032].

CONCLUSION: This study provided an observation of a significant correlation between INOCA with abnormal coronary vasomotor function and OSA in an Australian cohort. This correlation supports a possible pathophysiological interplay between these two conditions that needs to be further evaluated. The benefit of treatment of OSA in this subset remains unknown.

PMID:35021111 | DOI:10.1016/j.cpcardiol.2022.101111