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Contemporary Clinical Trials Jun 2024Despite a growing body of literature in the area of recruitment modeling for multicenter studies, in practice, statistical models to predict enrollments are rarely used...
Despite a growing body of literature in the area of recruitment modeling for multicenter studies, in practice, statistical models to predict enrollments are rarely used and when they are, they often rely on unrealistic assumptions. The time-dependent Poisson-Gamma model (tPG) is a recently developed flexible methodology which allows analysts to predict recruitments in an ongoing multicenter trial, and its performance has been validated on data from a cohort study. In this article, we illustrate and further validate the tPG model on recruitment data from randomized controlled trials. Additionally, in the appendix, we provide a practical and easy to follow guide to its implementation via the tPG R package. To validate the model, we show the predictive performance of the proposed methodology in forecasting the recruitment process of two HIV vaccine trials conducted by the HIV Vaccine Trials Network in multiple Sub-Saharan countries.
PubMed: 38908745
DOI: 10.1016/j.cct.2024.107607 -
Clinical Orthopaedics and Related... May 2024In patients with a degenerative tear of the medial meniscus, recent meta-analyses and systematic reviews have shown no treatment benefit of arthroscopic partial...
Arthroscopic Partial Meniscectomy for a Degenerative Meniscus Tear Is Not Cost Effective Compared With Placebo Surgery: An Economic Evaluation Based on the FIDELITY Trial Data.
BACKGROUND
In patients with a degenerative tear of the medial meniscus, recent meta-analyses and systematic reviews have shown no treatment benefit of arthroscopic partial meniscectomy (APM) over conservative treatment or placebo surgery. Yet, advocates of APM still argue that APM is cost effective. Giving advocates of APM their due, we note that there is evidence from the treatment of other musculoskeletal complaints to suggest that a treatment may prove cost effective even in the absence of improvements in efficacy outcomes, as it may lead to other benefits, such as diminished productivity loss and reduced costs, and so the question of cost effectiveness needs to be answered for APM.
QUESTIONS/PURPOSES
(1) Does APM result in lower postoperative costs compared with placebo surgery? (2) Is APM cost-effective compared with placebo surgery?
METHODS
One hundred forty-six adults aged 35 to 65 years with knee symptoms consistent with a degenerative medial meniscus tear and no knee osteoarthritis according to the American College of Rheumatology clinical criteria were randomized to APM (n = 70) or placebo surgery (n = 76). In the APM and placebo surgery groups, mean age was 52 ± 7 years and 52 ± 7 years, and 60% (42 of 70) and 62% (47 of 76) of participants were men, respectively. There were no between-group differences in baseline characteristics. In both groups, a standard diagnostic arthroscopy was first performed. Thereafter, in the APM group, the torn meniscus was trimmed to solid meniscus tissue, whereas in the placebo surgery group, APM was carefully mimicked but no resection of meniscal tissue was performed; as such, surgical costs were the same in both arms and were not included in the analyses. All patients received identical postoperative care including a graduated home-based exercise program. At the 2-year follow-up, two patients were lost to follow-up, both in the placebo surgery group. Cost effectiveness over the 2-year trial period was computed as incremental net monetary benefit (INMB) for improvements in quality-adjusted life years (QALY), using both the societal (primary) and healthcare system (secondary) perspectives. To be able to consider APM cost effective, the CEA analysis should yield a positive INMB value. Nonparametric bootstrapping was used to assess uncertainty. Several one-way sensitivity analyses were also performed.
RESULTS
APM did not deliver lower postoperative costs, nor did it convincingly improve quality of life scores when compared with placebo surgery. From a societal perspective, APM was associated with € 971 (95% CI -2013 to 4017) higher costs and 0.015 (95% CI -0.011 to 0.041) improved QALYs over 2-year follow-up compared with placebo surgery. Both differences were statistically inconclusive (a wide 95% CI that crossed the line of no difference). Using the conventional willingness to pay (WTP) threshold of € 35,000 per QALY, APM resulted in a negative INMB of € -460 (95% CI -3757 to 2698). In our analysis, APM would result in a positive INMB only when the WTP threshold rises to about € 65,000 per QALY. The wide 95% CIs suggests uncertain cost effectiveness irrespective of chosen WTP threshold.
CONCLUSION
The results of this study lend further support to clinical practice guidelines recommending against the use of APM in patients with a degenerative meniscus tear. Given the robustness of existing evidence demonstrating no benefit or cost effectiveness of APM over nonsurgical treatment or placebo surgery, future research is unlikely to alter this conclusion.Level of Evidence Level III, economic analysis.
PubMed: 38905520
DOI: 10.1097/CORR.0000000000003094 -
The Journal of International Medical... Jun 2024Immunoglobulin G4-related disease (IgG4-RD) is a fibroinflammatory condition characterized by chronic activation of the immune system and a tendency to form tumorous...
Immunoglobulin G4-related disease (IgG4-RD) is a fibroinflammatory condition characterized by chronic activation of the immune system and a tendency to form tumorous lesions. IgG4-RD is frequently characterized by the presence of tumor-like masses affecting multiple organs and is easily mistaken for a malignant neoplasm. However, IgG4-RD affecting the appendix is extremely rare, with only seven cases reported previously. We report the case of a woman in her early 60s who presented with insidious abdominal pain and radiological findings mimicking appendiceal neoplasms. After diagnosing appendiceal neoplasms, surgery was performed. The patient had a serum IgG4 concentration of <1.35 g/L, which did not satisfy one of the three revised comprehensive diagnostic criteria for IgG4-RD. A pathological examination was conducted, and the patient was diagnosed with appendiceal IgG4-RD. To the best of our knowledge, there have been no previously reported cases of IgG4-RD affecting the appendix in patients with low serum IgG4 concentrations. This report may prove beneficial for the future understanding of IgG4-RD and for the revision of diagnostic and treatment strategies.
Topics: Humans; Female; Appendiceal Neoplasms; Immunoglobulin G4-Related Disease; Diagnosis, Differential; Middle Aged; Immunoglobulin G; Tomography, X-Ray Computed; Appendix
PubMed: 38902205
DOI: 10.1177/03000605241260540 -
The Lancet. Diabetes & Endocrinology Jul 2024
Topics: Humans; Diabetes Mellitus, Type 1; Prospective Studies; Feeding and Eating Disorders; Female; Male; Adult; Proof of Concept Study; Mental Health; Cohort Studies
PubMed: 38901894
DOI: 10.1016/S2213-8587(24)00123-2 -
Journal of Obstetrics and Gynaecology... Jun 2024To formulate strategies for clinical assessments for endometrial thickening on ultrasound in a postmenopausal woman without bleeding.
OBJECTIVE
To formulate strategies for clinical assessments for endometrial thickening on ultrasound in a postmenopausal woman without bleeding.
TARGET POPULATION
Postmenopausal women of any age.
OUTCOMES
To reduce unnecessary invasive interventions and investigations in women with asymptomatic endometrial thickening while selectively investigating women at risk for endometrial cancer.
BENEFITS, HARMS, AND COSTS
It is anticipated that the adoption of these recommendations would save postmenopausal women unnecessary anxiety, pain, and risk of procedural complications. It is also expected to decrease the cost to the health care system by eliminating unnecessary interventions.
EVIDENCE
English language articles from Medline, Cochrane, and PubMed databases for relevant peer-reviewed articles dating from 1995 to 2022 (e.g., asymptomatic endometrial thickness, endometrial cancer, postmenopausal bleeding, transvaginal ultrasound, endometrial biopsy, cervical stenosis, hormone therapies and the endometrium, tamoxifen, tibolone, aromatase inhibitors). Results were restricted to systematic reviews and meta-analyses, randomized controlled trials/controlled clinical trials, and observational studies.
VALIDATION METHODS
The authors rated the quality of evidence and strength of recommendations using the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach. See Appendix A (Tables A1 for definitions and A2 for interpretations of strong and conditional [weak] recommendations).
INTENDED AUDIENCE
Physicians, including gynaecologists, obstetricians, family physicians, radiologists, pathologists, and internists; nurse practitioners and nurses; medical trainees, including medical students, residents, and fellows; and other providers of health care of the postmenopausal population.
SOCIAL MEDIA ABSTRACT
Postmenopausal women often have a thickening of the lining of the uterus found during ultrasound. Without bleeding, an endometrium <11 mm is rarely a serious problem but should be evaluated by a health care provider.
PubMed: 38901794
DOI: 10.1016/j.jogc.2024.102591 -
Educational and Psychological... Apr 2024Regression factor score predictors have the maximum factor score determinacy, that is, the maximum correlation with the corresponding factor, but they do not have the...
Regression factor score predictors have the maximum factor score determinacy, that is, the maximum correlation with the corresponding factor, but they do not have the same inter-correlations as the factors. As it might be useful to compute factor score predictors that have the same inter-correlations as the factors, correlation-preserving factor score predictors have been proposed. However, correlation-preserving factor score predictors have smaller correlations with the corresponding factors (factor score determinacy) than regression factor score predictors. Thus, higher factor score determinacy goes along with bias of the inter-correlations and unbiased inter-correlations go along with lower factor score determinacy. The aim of the present study was therefore to investigate the size and conditions of the trade-off between factor score determinacy and bias of inter-correlations by means of algebraic considerations and a simulation study. It turns out that under several conditions very small gains of factor score determinacy of the regression factor score predictor go along with a large bias of inter-correlations. Instead of using the regression factor score predictor by default, it is proposed to check whether substantial bias of inter-correlations can be avoided without substantial loss of factor score determinacy using a correlation-preserving factor score predictor. A syntax that allows to compute correlation-preserving factor score predictors from regression factor score predictors, and to compare factor score determinacy and inter-correlations of the factor score predictors is given in the Appendix.
PubMed: 38898877
DOI: 10.1177/00131644231171137 -
Otology & Neurotology : Official... Jun 2024To determine the cost-effectiveness or cost-utility of cochlear implants (CI) in adults with severe to profound bilateral hearing loss.
OBJECTIVE
To determine the cost-effectiveness or cost-utility of cochlear implants (CI) in adults with severe to profound bilateral hearing loss.
DATABASES REVIEWED
PubMed (Medline), The Cochrane Library, Embase via Elsevier, EBSCOhost CINAHL, and Scopus.
METHODS
The study included adult participants with severe to profound bilateral neurosensory hearing loss. The analysis encompassed quality of life improvements, costs, cost-effectiveness, cost-utility, cost-benefit, quality-adjusted life year (QALY), incremental cost-effectiveness ratio (ICER), and incremental cost-utility ratio (ICUR). Systematic reviews, meta-analyses, case series, and prospective or retrospective cohort studies published in English between 2010 and 2023 were retrieved. Exclusion criteria included incomplete studies, abstracts, clinical cases, editorials, letters, studies involving pediatric populations, single-side deafness, methodology research, noneconomic aspects of CI, mixed child and adult data, and studies published before 2010. The risk of bias was assessed following the criteria outlined in Appendix I of the economic evaluation's quality assessment as per the NICE Guideline Development Method.
RESULTS
Ten articles met the criteria and were included in the qualitative synthesis. One study conducted a prospective cost-utility analyses, one carried out a cost-benefit analyses, one was a randomized controlled clinical trial focusing on cost-utility, and another was a clinical trial addressing cost-effectiveness. Six studies employed Markov models, and one study utilized uniquely the Monte Carlo method. None quantified the economic impact of improved hearing on cognitive function.
CONCLUSIONS
The heterogeneity of sources impacted data quality. Unilateral and sequential bilateral CI appeared to be cost-effective when compared with bilateral hearing aids or nontechnological support. When properly indicated, simultaneous bilateral CIs are cost-effective compared to no interventions and to unilateral cochlear implantation through differential discounting or variations from the base cases, especially with a life expectancy of 5-10 years or longer.
PubMed: 38896785
DOI: 10.1097/MAO.0000000000004225 -
Cureus May 2024Intrauterine appendicular perforation leading to meconium peritonitis is exceptionally rare, with few reported cases in the literature. This case underscores the...
Intrauterine appendicular perforation leading to meconium peritonitis is exceptionally rare, with few reported cases in the literature. This case underscores the diagnostic challenges and high mortality associated with neonatal appendicular perforation. Neonatal appendicitis and subsequent perforation are uncommon due to the funnel shape of the fetal appendix, which reduces susceptibility to luminal obstruction. While advances in neonatal care and diagnostic modalities have improved outcomes, challenges persist in timely diagnosis and management. We present the case of a preterm infant, one of dichorionic-diamniotic (DCDA) twins delivered via cesarean section, who developed gross abdominal distension and respiratory distress shortly after birth. Diagnostic abdominocentesis revealed meconium-stained fluid, prompting further investigation with imaging and subsequent exploratory laparotomy. Extensive adhesions and cecal perforation were observed, necessitating a cecostomy. Despite interventions, the infant's condition deteriorated, leading to a fatal outcome. Intrauterine appendicular perforation leading to meconium peritonitis is a rare and difficult-to-diagnose condition. Antenatal suspicion and early surgical intervention are crucial for improving outcomes. Factors contributing to neonatal appendicular perforation include ischemia, obstruction, and infective etiologies. Neonatal appendicular perforation is a rare but life-threatening condition requiring a high index of suspicion for prompt diagnosis and management. Advances in diagnostic tools and antenatal monitoring have contributed to improved outcomes, highlighting the importance of considering this diagnosis in cases of unexplained neonatal abdominal distension.
PubMed: 38894799
DOI: 10.7759/cureus.60576 -
Cancers May 2024Neuroendocrine carcinomas (NECs) of the tubular gastrointestinal tract (GI-NECs) are rare and associated with worse clinical outcomes. This population-based study aims...
Neuroendocrine carcinomas (NECs) of the tubular gastrointestinal tract (GI-NECs) are rare and associated with worse clinical outcomes. This population-based study aims to highlight key demographics, clinicopathological factors, and survival outcomes in the US population. Data from 10,387 patients with GI-NECs were extracted from the Surveillance, Epidemiology, and End Result (SEER) database from 2000 to 2020. Most patients were >40 years old at the time of presentation with a median age of 63 years old, with almost equal ethnic distribution per US population data. The most common primary tumor site was the small intestine (33.6%). The metastatic spread was localized in 34.8%, regional in 27.8%, and distant in 37.3% of cases, and the liver was the most common site of metastasis (19.9%) in known cases of metastases. Most NEC patients underwent surgery, presenting the highest 5-year overall survival of 73.2% with a 95% confidence interval (CI) (95% CI 72.0-74.4%), while chemotherapy alone had the lowest 5-year survival of 8.0% (95% CI 6.4-10.0%). Compared to men, women had a superior 5-year survival rate of 59.0% (95% CI 57.6-60.5%). On multivariate analysis, age > 65 (HR 2.49, 95% CI 2.36-2.54%, ≤ 0.001), distant metastasis (HR 2.57, 95% CI 2.52-2.62%, ≤ 0.001), tumor size > 4 mm (HR 1.98, 95%, CI 1.70-2.31%, ≤ 0.001), esophageal (HR 1.49, 95% CI 0.86-2.58%, ≤ 0.001), transverse colon (HR 1.95, 95% CI 1.15-3.33%, ≤ 0.01), descending colon (HR 2.12, 95% CI 1.12, 3.97%, = 0.02) anorectal sites, and liver or lung metastases were associated with worse survival. Surgical intervention and tumors located in the small intestine or appendix showed a better prognosis. GI-NECs are a group of rare malignancies associated with a poor prognosis. Therefore, epidemiological studies analyzing national databases may be the best alternative to have a more comprehensive understanding of this condition, assess the impact of current practices, and generate prognosis tools.
PubMed: 38893117
DOI: 10.3390/cancers16111998 -
Journal of Clinical Medicine May 2024The role of infestation in the context of appendicitis is largely overlooked, but is considered an unexpected and significant appendicectomy finding. The aim of this...
The role of infestation in the context of appendicitis is largely overlooked, but is considered an unexpected and significant appendicectomy finding. The aim of this study was to investigate the frequency of findings in appendectomies and to evaluate the clinical and histopathologic features of patients with -associated acute appendicitis and those with appendiceal infestation. The medical records of all children who underwent an appendectomy in two large pediatric centers in Croatia between 1 January 2009 and 1 January 2024 were retrospectively reviewed. Of 6359 appendectomies, 61 (0.96%) children were diagnosed with on histopathology and included in further analysis. The groups were compared with regard to demographic characteristics, laboratory values, clinical features and histopathological findings. : The incidence of enterobiasis fluctuated slightly in the individual study years, but was constant overall. The median age of all patients was 11 years (IQR 8.5, 13), with females predominating (60.7%). Acute appendicitis was observed in 34% of the appendiceal species. The patients with infestation, without appendicitis, were younger (9 years (IQR 8, 13) vs. 12 years (IQR 10, 15); = 0.020), had longer duration of symptoms (36 h (IQR, 12, 48) vs. 24 h (IQR, 12, 36); = 0.034), lower body temperature (37 °C (IQR 36.8, 37.4) vs. 37.6 °C (IQR, 37, 38.6) = 0.012), lower Appendicitis Inflammation Response (AIR) score (3 (IQR 2, 5) vs. 7 (IQR 5, 9.5) < 0.001), lower incidence of rebound tenderness (57.1% vs. 20%; = 0.003) and less frequent vomiting (12.5% vs. 47.6%; = 0.004) compared to the patients with -associated acute appendicitis. Acute inflammatory markers in the laboratory showed significantly higher values in the group of patients with acute appendicitis: C-reactive protein ( = 0.009), White blood cells ( = 0.001) and neutrophils ( < 0.001). Eosinophilia was not found in any of the groups, although eosinophil counts were significantly higher in children who had infestation than in those with -related appendicitis (2.5% (IQR 0.9, 4.3) vs. 1.8% (IQR 0.7, 2.1); = 0.040). : Pediatric surgeons should consider infestation as a differential diagnosis when removing a vermiform appendix. Younger age, longer duration of symptoms, lower body temperature, lower AIR score, lower diameter of the appendix and normal laboratory inflammatory markers could predict infection in children presenting with right iliac fossa pain and avoid unnecessary appendectomy.
PubMed: 38892909
DOI: 10.3390/jcm13113198