Tag: statistics

BMJ Paediatr Open. 2024 Mar 18;8(1):e002240. doi: 10.1136/bmjpo-2023-002240.

ABSTRACT

OBJECTIVES: This study aimed to explore the effects of short birth spacing (SBS), which is defined as a period of less than 33 months between two successive births, on multiple concurrent forms of child malnutrition (MCFCM) and at least one form of child malnutrition (ALOFCM) using propensity score matching (PSM).

METHODS: This study used data extracted from the 2017-18 Bangladesh Demographic and Health Survey. PSM with four different distance functions, including logistic regression, classification and regression tree, single hidden layer neural network and random forest, were performed to evaluate the effects of SBS on MCFCM and ALOFCM. We also explored how the effects were modified in different subsamples, including women’s empowerment, education and economic status (women’s 3E index)-constructed based on women’s decision-making autonomy, education level, and wealth index, and age at marriage, and place of residence.

RESULTS: The prevalence of SBS was 22.16% among the 4652 complete cases. The matched samples of size 2062 generated by PSM showed higher odds of MCFCM (adjusted OR (AOR)=1.25, 95% CI=1.02 to 1.56, p=0.038) and ALOFCM (AOR=1.20, 95% CI=1.01 to 1.42, p=0.045) for the SBS children compared with their counterparts. In the subsample of women with 3E index≥50% coverage, the SBS children showed higher odds of MCFCM (AOR: 1.43, 95% CI=1.03 to 2.00, p=0.041] and ALOFCM (AOR: 1.33, 95% CI=1.02 to 1.74, p=0.036). Higher odds of MCFCM (AOR=1.27, 95% CI=1.02 to 1.58, p=0.036) and ALOFCM (AOR=1.23, 95% CI=1.02 to 1.51, p=0.032) for SBS children than normal children were also evident for the subsample of mothers married at age≤18 years.

CONCLUSION: SBS was significantly associated with child malnutrition, and the effect was modified by factors such as women’s autonomy and age at marriage.

PMID:38499349 | DOI:10.1136/bmjpo-2023-002240

Thorax. 2024 Mar 18:thorax-2023-220436. doi: 10.1136/thorax-2023-220436. Online ahead of print.

ABSTRACT

RATIONALE: Life course trajectories of lung function development and decline influence the risk for lung disease but are poorly documented.

OBJECTIVE: To document lung function trajectories from childhood to mid-adult life.

METHODS: We modelled forced expiratory volume in 1 s (FEV₁), forced vital capacity (FVC) and FEV₁/FVC at ages 9, 11, 13, 15, 18, 21, 26, 32, 38 and 45 years from a population-based cohort using latent profile analysis to identify distinct subgroups of participants with similar lung function trajectories. Regression analyses were used to assess associations between the trajectories, early life factors and postbronchodilator airflow obstruction at age 45.

RESULTS: Among 865 participants with ≥6 measures of lung function, we identified 10 distinct FEV₁ trajectories. Most were approximately parallel except for a childhood airway hyper-responsiveness-related persistently low trajectory (3% of study population); two accelerated-decline trajectories, one of which (8%) was associated with smoking and higher adult body mass index (BMI) and a catch-up trajectory (8%). Findings for FEV₁/FVC trajectories were similar. Nine trajectories were identified for FVC: most were also approximately parallel except for a higher BMI-related accelerated-decline trajectory. The three FEV₁ trajectories leading to the lowest FEV₁ values comprised 19% of the cohort but contributed 55% of airflow obstruction at age 45.

CONCLUSIONS: Lung function trajectories to mid-adult life are largely established before adolescence, with a few exceptions: a childhood airway hyper-responsiveness-related persistently low trajectory, which starts low and gets worse with age, and accelerated adult decline trajectories associated with smoking and obesity. Adverse trajectories are associated with a high risk of airflow obstruction in mid-adult life.

PMID:38499347 | DOI:10.1136/thorax-2023-220436

BMJ Open Ophthalmol. 2024 Mar 18;9(1):e001608. doi: 10.1136/bmjophth-2023-001608.

ABSTRACT

OBJECTIVE: To compare multimodal structural and functional diagnostic methods in patients with systemic lupus erythematosus (SLE) treated with hydroxychloroquine, to identify the best complementary approach for detecting subclinical retinal toxicity.

METHODS: A cross-sectional, unicentric study was conducted on patients with SLE treated with hydroxychloroquine. Each patient underwent a comprehensive ophthalmic evaluation, comprising structural tests (spectral-domain optical coherence tomography (SD-OCT), en face OCT, en face OCT angiography (OCTA), fundus autofluorescence (FAF)) and functional tests (automated perimetry for visual field (VF) testing, multifocal electroretinography (mfERG)). A diagnosis of macular toxicity required the presence of abnormalities in at least one structural and functional test. The Kappa Concordance Index was used to assess the concordance among the different tests in detecting potential macular toxicity-associated alterations.

RESULTS: Sixty-six patients with SLE (132 eyes) were consecutively enrolled. Four (6.1%) patients developed subclinical hydroxychloroquine-induced retinal toxicity without visual acuity impairment. The proportion of abnormal results was 24% for both en face OCT and en face OCTA. Regarding functional analysis, VF was less specific than mfERG in detecting subclinical retinal toxicity (VF specificity 47.5%). En face OCT and en face OCTA structural findings showed better concordance, with a kappa index >0.8, and both identified the same cases of toxicity as FAF.

CONCLUSION: Although structural OCT and VF are frequently used to screen for hydroxychloroquine-induced retinal toxicity, our findings suggest that a combination of mfERG, en face OCT and en face OCTA could improve the diagnostic accuracy for subclinical retinal damage. This study emphasises the importance of a multimodal imaging strategy to promptly detect signs of hydroxychloroquine-induced retinal toxicity.

PMID:38499344 | DOI:10.1136/bmjophth-2023-001608

Stat Methods Med Res. 2024 Mar 18:9622802241236952. doi: 10.1177/09622802241236952. Online ahead of print.

ABSTRACT

Existing methods that use propensity scores for heterogeneous treatment effect estimation on non-experimental data do not readily extend to the case of more than two treatment options. In this work, we develop a new propensity score-based method for heterogeneous treatment effect estimation when there are three or more treatment options, and prove that it generates unbiased estimates. We demonstrate our method on a real patient registry of patients in Singapore with diabetic dyslipidemia. On this dataset, our method generates heterogeneous treatment recommendations for patients among three options: Statins, fibrates, and non-pharmacological treatment to control patients’ lipid ratios (total cholesterol divided by high-density lipoprotein level). In our numerical study, our proposed method generated more stable estimates compared to a benchmark method based on a multi-dimensional propensity score.

PMID:38499338 | DOI:10.1177/09622802241236952

J Med Genet. 2024 Mar 18:jmg-2023-109750. doi: 10.1136/jmg-2023-109750. Online ahead of print.

ABSTRACT

BACKGROUND: As gene-specific therapy for inherited retinal dystrophy (IRD) advances, unified variant interpretation across institutes is becoming increasingly important. This study aims to update the genetic findings of 86 retinitis pigmentosa (RP)-related genes in a large number of Japanese patients with RP by applying the standardised variant interpretation guidelines for Japanese patients with IRD (J-IRD-VI guidelines) built upon the American College of Medical Genetics and Genomics and the Association for Molecular Pathology rules, and assess the contribution of these genes in RP-allied diseases.

METHODS: We assessed 2325 probands with RP (n=2155, including n=1204 sequenced previously with the same sequencing panel) and allied diseases (n=170, newly analysed), including Usher syndrome, Leber congenital amaurosis and cone-rod dystrophy (CRD). Target sequencing using a panel of 86 genes was performed. The variants were interpreted according to the J-IRD-VI guidelines.

RESULTS: A total of 3564 variants were detected, of which 524 variants were interpreted as pathogenic or likely pathogenic. Among these 524 variants, 280 (53.4%) had been either undetected or interpreted as variants of unknown significance or benign variants in our earlier study of 1204 patients with RP. This led to a genetic diagnostic rate in 38.6% of patients with RP, with EYS accounting for 46.7% of the genetically solved patients, showing a 9% increase in diagnostic rate from our earlier study. The genetic diagnostic rate for patients with CRD was 28.2%, with RP-related genes significantly contributing over other allied diseases.

CONCLUSION: A large-scale genetic analysis using the J-IRD-VI guidelines highlighted the population-specific genetic findings for Japanese patients with IRD; these findings serve as a foundation for the clinical application of gene-specific therapies.

PMID:38499336 | DOI:10.1136/jmg-2023-109750

Arch Dis Child. 2024 Mar 18:archdischild-2024-326899. doi: 10.1136/archdischild-2024-326899. Online ahead of print.

NO ABSTRACT

PMID:38499322 | DOI:10.1136/archdischild-2024-326899

Brain Stimul. 2024 Mar 16:S1935-861X(24)00046-9. doi: 10.1016/j.brs.2024.03.010. Online ahead of print.

ABSTRACT

BACKGROUND: Vagus nerve stimulation (VNS) at low frequencies (≤30 Hz) has been an established treatment for drug-resistant epilepsy (DRE) for over 25 years.

OBJECTIVE: To examine the initial safety and efficacy performance of an investigational, high-frequency (≥250 Hz) VNS paradigm herein called “Microburst VNS” (μVNS). μVNS consists of short, high-frequency bursts of electrical pulses believed to preferentially modulate certain brain regions.

METHODS: Thirty-three (33) participants were enrolled into an exploratory feasibility study, 21 with focal-onset seizures and 12 with generalized-onset seizures. Participants were titrated to a personalized target dose of μVNS using an investigational fMRI protocol. Participants were then followed for up to 12 months, with visits every 3 months, and monitored for side-effects at all time points. This study was registered as NCT03446664 on February 27th, 2018.

RESULTS: The device was well-tolerated. Reported adverse events were consistent with typical low frequency VNS outcomes and tended to diminish in severity over time, including dysphonia, cough, dyspnea, and implant site pain. After 12 months of μVNS, the mean seizure frequency reduction for all seizures was 61.3% (median reduction: 70.4%; 90% CI of median: 48.9%-83.3%). The 12-month responder rate (≥50% reduction) was 63.3% (90% CI: 46.7%-77.9%) and the super-responder rate (≥80% reduction) was 40% (90% CI: 25.0%-56.6%). Participants with focal-onset seizures appeared to benefit similarly to participants with generalized-onset seizures (mean reduction in seizures at 12 months: 62.6% focal [n = 19], versus 59.0% generalized [n = 11]).

CONCLUSION: Overall, μVNS appears to be safe and potentially a promising therapeutic alternative to traditional VNS. It merits further investigation in randomized controlled trials which will help determine the impact of investigational variables and which patients are most suitable for this novel therapy.

PMID:38499287 | DOI:10.1016/j.brs.2024.03.010

Parasitol Int. 2024 Mar 16:102888. doi: 10.1016/j.parint.2024.102888. Online ahead of print.

ABSTRACT

Blastocystis sp. and Dientamoeba fragilis are intestinal protists, which are common worldwide, but the pathogenic role of these organisms in gastrointestinal diseases is still controversial. This study aimed to investigate the frequency of Blastocystis sp. and D. fragilis in stool samples from adult patients with celiac disease (CD) by using conventional and molecular methods. A total of 75 patients with CD and 75 healthy individuals were included in this study. Fresh stool specimens collected from each individual were analyzed by conventional and molecular methods. The overall prevalence of Blastocystis sp. and D. fragilis was 41.3% (31/75) and 24% (18/75) in patients with CD, and 46.7% (35/75) and 13.3% (10/75) in healthy controls, respectively. There was no statistically significant difference in the prevalence of Blastocystis sp. and D. fragilis between CD patients and healthy individuals. Blastocystis sp. subtypes were identified in 20 CD and 16 control patients and the overall subtype distribution was observed as ST1 13.9%, ST2 30.6%, and ST3 55.6%. The prevalence of Blastocystis sp. and D. fragilis in adults with CD is similar to the prevalence of protozoa in healthy adults. In this study, the most prevalent Blastocystis subtype was ST3 and the most frequent allele was a34 in both CD patients and healthy individuals. No significant difference was found between the two groups in terms of the detection rates of Blastocystis sp. and D. fragilis, and it is thought that both protists may be colonisers of the intestinal microbiome.

PMID:38499283 | DOI:10.1016/j.parint.2024.102888

J Dent. 2024 Mar 16:104935. doi: 10.1016/j.jdent.2024.104935. Online ahead of print.

ABSTRACT

OBJECTIVES: The recently introduced Implant Disease Risk Assessment (IDRA) identifies a restoration margin-alveolar bone crest (RM-AC) distance of less than 1.5 mm as a key risk factor for peri-implant disease among eight major risk factors. This study evaluated the impact of the RM-AC distance on marginal bone loss (MBL) through radiographic analysis.

METHODS: This retrospective cross-sectional study included 77 partially edentulous patients (39 females and 38 males, aged 22 to 76 years) with 202 platform-switched conical connection implants, cement-retained, implant-supported fixed restorations, and bone-level implants placed between 2016 and 2021. Dental implants were followed for least 6 to 36 months at follow up functional loading. Study participants were categorized into Group A (RM-AC distance ≤ 1.5 mm, n=69) and Group B (RM-AC distance > 1.5 mm, n=133). Twelve patients in Group B and five patients in Group A had no history of periodontal disease. The MBL was measured radiographically from the most coronal point of the implant shoulder to the alveolar bone, and the RM-AC distance was measured from the restoration margin to the alveolar crest. Multinomial logistic regression analysis was used for statistical evaluation.

RESULTS: The incidence of MBL in Group A was statistically significant and 3.42 times higher than that in Group B. The rate of MBL in periodontitis Stage 4 was found to be 26.31 times higher than that in periodontitis Stage 2. The incidence of MBL was 6.097 and 5.02 times higher with increasing implant diameter and length, respectively.

CONCLUSION: This study conclusively demonstrates that RM-AC distance ≤ 1.5 significantly increases the risk of MBL, particularly in patients with a history of periodontal disease.

CLINICAL SIGNIFICANCE: This study highlights the critical role of maintaining an RM-AC distance greater than 1.5 mm in the prevention of MBL, particularly in patients with a history of periodontal disease. Since implant diameter and length have a significant impact on the risk of MBL, it emphasizes that implant demographics should also be carefully evaluated.

PMID:38499282 | DOI:10.1016/j.jdent.2024.104935

Radiother Oncol. 2024 Mar 16:110232. doi: 10.1016/j.radonc.2024.110232. Online ahead of print.

ABSTRACT

BACKGROUND AND PURPOSE: Stereotactic radiotherapy (SRT) is an effective treatment for head & neck (H&N) paragangliomas. Nevertheless, the timeline for achieving a tumor-volume-reduction (TVR) remains unclear.

MATERIALS AND METHODS: Sixty-three cases with H&N paragangliomas received definitive SRT and were evaluated retrospectively. Statistical Package for the Social Sciences (SPSS) v23.0 (IBM, Armonk, NY, USA) was used for statistics.

RESULTS: Sixty-eight lesions were irradiated, with glomus jugulotympanicum being the most common location (44 %). Median tumor diameter and volume were 3 cm (range, 1-7.6 cm) and 15.4 cm³ (range,1-185 cm³), respectively. Median dose was 25 Gy (range, 12-37.5 Gy) in 5 fractions (range, 1-5 fractions). Median follow-up was 40 months (range, 3-184 months). Treatment response, evaluated at a median 4.6 months post-SRT (range: 3-11 months), revealed TVR in 26 cases (41 %). During follow-up, 13 additional cases showed TVR, resulting in an overall TVR rate of 62 %. The median duration for attaining TVR was 9 months (range, 3-36 months) after SRT, and TVR occurred ≥ 12 months in 42 % of cases. Patients without prior surgery (p = 0.03) and with a longer follow-up (p = 0.04) demonstrated a higher rate of TVR. The likelihood of TVR tends to increase as the SRT dose increases (p = 0.06). Overall local control (LC) rate was 100 %. No ≥ grade 3 acute or late toxicities were observed.

CONCLUSION: While SRT demonstrates an excellent LC rate for H&N paragangliomas, it’s important to note that the response to treatment may require time. TVR may last beyond the initial year of treatment in a substantial proportion of patients.

PMID:38499272 | DOI:10.1016/j.radonc.2024.110232