Tag: statistics

Neurol Sci. 2024 May 21. doi: 10.1007/s10072-024-07584-8. Online ahead of print.

ABSTRACT

BACKGROUND: Rett syndrome (RTT) is an uncommon inherited neurodevelopmental disorder that affects brain development, mostly in females. It results from mutation in MECP2 gene in the long arm (q) of the X chromosome.

OBJECTIVE: Trofinetide is a recently developed drug that has a neuroprotective effect on neurons, and it is our aim in this meta-analysis to evaluate its efficacy and safety in treating Rett syndrome patients.

METHODS: We searched 5 databases (PubMed, Scopus, Embase, Web of Science, and Cochrane Library databases) to identify randomized controlled trials (RCTs) comparing Trofinetide and placebo in patients with Rett syndrome until August 13, 2023.Our primary outcomes were the Clinical Global Impression-Improvement (CGI) and the Rett syndrome Behavior Questionnaire (RSBQ). We used Risk of Bias Assessment tool-2 (ROB2) to assess the methodological quality of the included randomized controlled trials.

RESULTS: Three RCTs with a total of 325 patients were included with a follow-up duration ranging from one month to three months. 186 patients received the intervention drug (Trofinetide) and 138 received the placebo. Trofinetide was found to reduce CGI and RSBQ significantly more than placebo (MD = -0.35, 95% CI [-0.52 to -0.18], P 0.0001), (MD = -3.40, 95% CI [-3.69 to -3.12], P 0.00001) respectively. Most adverse events did not show any statistical difference between Trofinetide and the placebo.

CONCLUSION: Trofinetide offers promise as a potential effective and safe therapeutic opportunity for a population without many available treatments, with improvements seen on both CGI and RSBQ assessments and no severe adverse effects reported.

PMID:38771525 | DOI:10.1007/s10072-024-07584-8

Int Urogynecol J. 2024 May 21. doi: 10.1007/s00192-024-05778-4. Online ahead of print.

ABSTRACT

INTRODUCTION AND HYPOTHESIS: Bladder pain syndrome (BPS) is poorly understood with both the aetiology and pathophysiology being unknown. Symptoms overlap with other disorders, such as overactive bladder (OAB) and chronic pelvic pain disorders such as endometriosis, making a consensus on how to diagnosis and manage patients challenging. The development of biomarkers for BPS may be the key to understanding more about its pathophysiology, as well as aiding diagnosis, subclassification, and discovering new drug targets for its management. As inflammation is widely understood to hold a central role in BPS, the evaluation of cytokines has gained interest. This article summarises the current literature and understanding of urinary, serum, and bladder tissue cytokines found elevated in patients with bladder pain syndrome.

METHODS: literature search using Pub Med with the keywords “bladder pain syndrome”, “painful bladder syndrome”, “bladder pain”, “Interstitial cystitis” AND “cytokines” or “inflammation”. This study was except from institutional approval.

RESULTS: Thirty-six cytokines have been identified as being statistically significantly elevated in either the serum, urine, or bladder tissue of patients with bladder pain syndrome in the 22 studies identified in this review of the literature. These cytokines include those from the interleukin group (n = 14), the CXC chemokine group (n = 5), and the C-C chemokine group (n = 7).

CONCLUSIONS: CXCL-1, CXCL-8, CXCL-9, CXCL-10, CXCL-11 from the CXC chemokine group, and CCL2, CCL4, CCL5, CCL7, and CCL11 from the C-C chemokine group have been found to be significantly elevated in patients with bladder pain in the literature. Many of these analytes also have supporting evidence for their roles in bladder pain from animal models and studies in other chronic inflammatory conditions. It is likely that a single cytokine will not serve as an adequate biomarker of disease in bladder pain syndrome for either diagnosis or disease severity. Instead, panels of inflammatory mediators may reveal more about the different pathways of inflammation leading to similar presentations of bladder pain in patients.

PMID:38771505 | DOI:10.1007/s00192-024-05778-4

Clin Exp Med. 2024 May 21;24(1):105. doi: 10.1007/s10238-024-01373-5.

ABSTRACT

Chimeric antigen receptor T cell (CAR-T) therapy is an effective treatment for B cell malignancies. A certain fraction of patients, however, experience post-CAR-T relapse, and due to the difficulty of precise relapse prediction, biomarkers that can predict the strength and duration of CAR-T efficacy are needed before CAR-T infusion. Therefore, we performed a single-center cohort study including 91 diffuse large B cell lymphoma (DLBCL) patients treated with CAR-T in order to identify such a new prognostic biomarker. After confirming that each of the already reported prognostic parameters (disease status at leukapheresis, primary refractoriness, number of treatment lines, CD3⁺ cell counts at leukapheresis) has only limited predictive performance, we established a new composite parameter by integrating these four variables, and found that it predicts progression-free survival (PFS) after CAR-T infusion with statistical significance. Moreover, after comprehensive correlation analyses of this new composite parameter with all individual laboratory variables, we determined that the standard deviation of red blood cell distribution width (RDW-SD) at leukapheresis shows significant correlation with the composite parameter and may be a prognostic biomarker (R² = 0.76, p = 0.02). Validation analysis indicated that a higher RDW-SD is significantly associated with poorer PFS after CAR-T cell therapy (HR, 3.46, P = 0.03). Thus, this study suggests that a single parameter, RDW-SD at leukapheresis, is a novel, useful biomarker that can be obtained early to predict therapeutic effects of CAR-T cell therapy. Post-CAR-T maintenance or re-induction therapies should be adopted for higher risk patients, who may relapse after CAR-T therapy.

PMID:38771501 | DOI:10.1007/s10238-024-01373-5

Arch Womens Ment Health. 2024 May 21. doi: 10.1007/s00737-024-01471-z. Online ahead of print.

ABSTRACT

PURPOSE: Adjuvant endocrine therapy has a vital role in reducing breast cancer mortality. The beliefs in adjuvant endocrine therapy is a very important factor in the medication adherence of breast cancer survivors. Therefore, it is necessary to develop a standardized scale for assessment of adjuvant endocrine therapy. The purpose of this study was to identify the attributes of adjuvant endocrine therapy beliefs, and to evaluate adjuvant endocrine therapy beliefs scale psychometric properties.

METHODS: A hybrid model was applied to identify the concept of adjuvant endocrine therapy beliefs and measurement question were developed by the scale development process. Statistical analysis using validity analysis and Rasch analysis based on item response theory were performed. A total of 228 breast cancer survivors in South Korea participated in the study.

RESULTS: The finally developed adjuvant endocrine therapy beliefs scale consisted of 22 items. The items extracted by 4 factors explained 59.72% of the total variance. The model fit showed an acceptable level. The adjuvant endocrine therapy beliefs scale was excellent in convergent and discriminant validity with reliability.

CONCLUSION: This scale is expected to be practical and useful in identifying adjuvant endocrine therapy beliefs and developing intervention strategies to promote adjuvant endocrine therapy adherence. In addition, continuous education and support should be accompanied so that breast cancer survivors can maintain positive beliefs in adjuvant endocrine therapy adherence.

PMID:38771495 | DOI:10.1007/s00737-024-01471-z

Epidemiol Prev. 2024 Mar-Apr;48(2):149-157. doi: 10.19191/EP24.2.A622.042.

ABSTRACT

BACKGROUND: the peer-review process, which is the foundation of modern scientific production, represents one of its essential elements. However, despite numerous benefits, it presents several critical issues.

OBJECTIVES: to collect the opinions of a group of researchers from the epidemiological scientific community on peer-review processes.

DESIGN: cross-sectional study using a questionnaire evaluation.

SETTING AND PARTICIPANTS: a 29-question survey was administered to 516 healthcare professionals through the SurveyMonkey platform. The questions focused on the individual characteristics of the respondents and their perceived satisfaction with some characteristics of the review process as well as their propensity of changing some aspects of it. In addition, three open-ended questions were included, allowing respondents to provide comments on the role that reviewers and the review process should play. Descriptive statistics were produced in terms of absolute frequencies and percentages for the information collected through the questionnaire. Secondly, a multiple logistic regression analysis was conducted to assess the willingness to change certain aspects of peer review, adjusting for covariates such as age, sex, being the author of at least one scientific work, being a reviewer of at least one scientific work, and belonging to a specific discipline. The results are expressed as odds ratios (ORs) and their 95% confidence intervals (95%CI). Text analysis and representation using word cloud were also used for an open-ended question.

MAIN OUTCOMES MEASURES: level of satisfaction regarding some characteristics of the peer-review process.

RESULTS: a total of 516 participants completed the questionnaire. Specifically, 87.2% (N. 450) of the participants were the authors of at least one scientific publication, 78.7% were first authors at least once (N. 406), and 71.5% acted as reviewers within the peer-review process (N. 369). The results obtained from the multiple logistic regression models did not highlight any significant differences in terms of propensity to change for age and sex categories, except for a lower propensity of the under 35 age group towards unmasking, defined as the presence of reviewers and editorial boards names on the publish article (OR <35 years vs 45-54 years: 0.51; 95%CI 0.29-0.89) and a higher propensity for post-formatting proposals, defined as the possibility of formatting the article following journal guidelines after the acceptance, among those under 45 (OR <35 years vs 45-54 years: 1.73; 95%CI 0.90-3.31; OR 35-44 years vs 45-54 years: 2.02; 95%CI 1.10-3.72). Finally, approximately 50% of respondents found it appropriate to receive credits for the revision work performed, while approximately 30% found it appropriate to receive a discount on publication fees for the same journal in which they acted as reviewers.

CONCLUSIONS: the peer-review process is considered essential, but imperfect, by the professionals who participated in the questionnaire, thus providing a clear picture of the value that peer-review adds rigorously to each scientific work and the need to continue constructive dialogue on this topic within the scientific community.

PMID:38770732 | DOI:10.19191/EP24.2.A622.042

Epidemiol Prev. 2024 Mar-Apr;48(2):118-129. doi: 10.19191/EP24.2.A720.039.

ABSTRACT

BACKGROUND: according to the International Agency for Cancer Research on Cancer, in 2022, breast cancer is the most common cancer in the Italian population, followed by colorectal cancer. Oncological screenings represent an effective secondary prevention strategy to counteract colorectal and breast cancers, significantly reducing mortality. In Lombardy Region (Northern Italy), screening programmes have been active since 2007, but adherence, especially in specific population subgroups, remains lower than expected.

OBJECTIVES: to analyse potential predictors of non-adherence to colorectal and breast cancer screening in the Lombardy Region during the pre-pandemic period of 2018-2019.

DESIGN: a retrospective cohort study aimed at investigating the role of sociodemographic variables, health status, and access to the healthcare system on non-adherence to colorectal and breast cancer screening. Statistical analyses were conducted separately by each Agency for Health Protection (ATS). The results of the models were synthesized across the Lombardy region through random-effects meta-analysis.

SETTING AND PARTICIPANTS: residents within the territory of each ATS in Lombardy as of 01.01.2018 and aged between 49 and 69 years at the beginning of the follow-up.

MAIN OUTCOMES MEASURES: adherence to colorectal and breast cancer screenings.

RESULTS: during the study period, across the Lombardy Region, 2,820,138 individuals were eligible to participate in colorectal cancer screening, and 1,357,344 women were eligible to participate in breast cancer screening, with an invitation coverage of 87% and 86%, respectively.For breast cancer screening, older age, cardiopathy, chronic obstructive pulmonary disease (COPD), inflammatory bowel diseases (IBD), autoimmune diseases, and presence of a rare disease are associated with a reduced risk of non-adherence. Conversely, foreign citizenship, oncological diagnosis, transplant, chronic kidney disease/dialysis, diabetes, heart failure, arterial or cerebral vasculopathy, and presence of a neurological diagnosis are associated with significant excess risks of non-participation. For colorectal cancer screening, factors favouring adherence include female gender, older age, cardiopathy, COPD, autoimmune diseases, and having access/utilization of primary care. Non-adherence is associated with foreign citizenship, transplant, chronic kidney disease/dialysis, diabetes, heart failure, arterial or cerebral vasculopathy, IBD, neurological diseases, residence in assisted living facilities, use of integrated home care, and presence of disability.

CONCLUSIONS: this is the first study conducted in the Lombardy Region which explores the theme of equity of access to organized screenings. This analysis highlights how sociodemographic determinants, chronic conditions, and access to the healthcare and social healthcare system constitute significant risk factors for non-adherence to screening programmes. Based on the results of this analysis, communication and/or organizational change interventions will be developed to counteract inequalities in access to effective prevention procedures.

PMID:38770729 | DOI:10.19191/EP24.2.A720.039

Acta Oncol. 2024 May 21;63:351-357. doi: 10.2340/1651-226X.2024.33241.

ABSTRACT

BACKGROUND: Electrochemotherapy (ECT) is a combined treatment method based on electroporation and simultaneous chemotherapy. In cases where radiotherapy has previously been used, surgery is often the only treatment option for vulvar cancer recurrence with potential resection of clitoris, vagina, urethra or anal sphincter. The unique advantage of ECT is its selectivity for cancer cells while sparing the surrounding healthy tissue. The aim of the study was to compare the ECT treatment of vulvar cancer recurrence for non-palliative purposes with surgical treatment.

MATERIALS AND METHODS: Eleven patients with single vulvar cancer recurrence were treated with ECT and followed up for 12 months. As a control group, 15 patients with single vulvar cancer recurrence were treated with wide local excision. The following data were collected, analyzed and compared: Age, body mass index, comorbidities, histological type, location and size of vulvar cancer recurrence, treatment history, details of procedures and hospital stay.

RESULTS: The probability curves for local tumor control did not differ between the ECT group and the surgical group (p = 0.694). The mean hospital stay and the mean duration of procedure were statistically significantly shorter in the ECT group (p < 0.001). There were no statistically significant differences between the ECT and surgical groups in terms of mean body mass index, associated diseases, previous treatments, presence of lichen sclerosus, p16 status, gradus, anatomical site of the tumor, and type of anesthesia.

CONCLUSION: In this case-control study, treatment of vulvar cancer recurrence with ECT for non-palliative purposes was comparable to surgical treatment in terms of effectiveness. The results need to be confirmed in larger randomized trials.

PMID:38770722 | DOI:10.2340/1651-226X.2024.33241

Knee Surg Sports Traumatol Arthrosc. 2024 May 21. doi: 10.1002/ksa.12244. Online ahead of print.

ABSTRACT

PURPOSE: Rotational ankle instability can be diagnosed in up to 18% of cases of chronic lateral ankle instability. It is characterised by an abnormal increase of talar rotation within the tibiofibular mortise, due to an injury in the most anterior component of the deltoid ligament secondary to a chronic deficiency of the lateral collateral ligament. The aim of this prospective observational study was to investigate the clinical outcomes following arthroscopic all-inside medial and lateral ligament reconstruction for rotational ankle instability.

METHODS: A prospective observational study of consecutive patients undergoing arthroscopic all-inside medial and lateral ligament reconstruction for rotational ankle instability with minimum 6-month follow-up. The primary outcome was a validated patient-reported outcome measure (PROM), the Manchester-Oxford Foot Questionnaire. Secondary outcomes included the EQ-5D, European Foot and Ankle Society score and complications.

RESULTS: Between 2020 and 2023, 12 patients underwent primary arthroscopic all-inside medial and lateral ligament reconstruction for rotational ankle instability with pre- and post-operative PROMs available for all 12 patients. The mean ± standard deviation age was 33.9 ± 7.2 years and the mean follow-up was 1.9 ± 1.2 (range: 0.5-3.8, interquartile range: 0.9-3.0) years. There was a significant improvement in all Manchester-Oxford Foot Questionnaire domain scores (p < 0.05): Index 53.1 ± 19.1 to 26.4 ± 27.6, Pain 46.7 ± 20.3 to 26.2 ± 26.8, Walking/Standing 58.7 ± 26.0 to 27.0 ± 30.0 and Social Interaction 51.2 ± 19.5 to 25.6 ± 30.1. There were improvements in EQ-5D-5L Index, VAS and VAS Pain; however, these were not statistically significant. There was one complication-a superficial peroneal nerve injury which resolved with a corticosteroid injection.

CONCLUSION: The arthroscopic all-inside medial and lateral ligament reconstruction technique is a reliable and safe method for treating rotational ankle instability, demonstrating significant improvement in PROMs at a mean 1.9-year follow-up.

LEVEL OF EVIDENCE: Level IV.

PMID:38770701 | DOI:10.1002/ksa.12244

J Obstet Gynaecol. 2024 Dec;44(1):2354575. doi: 10.1080/01443615.2024.2354575. Epub 2024 May 21.

ABSTRACT

BACKGROUND: Epidural analgesia has emerged as one of the best methods that can be used to reduce labour pain. This study was conducted to assess awareness, attitudes, and practices of pregnant women who visited maternity and antenatal healthcare clinics about epidural analgesia during normal vaginal birth.

METHODS: This multicentre study was conducted in a cross-sectional design among pregnant women using a pre-tested questionnaire. The study population in this study was pregnant women who visited maternity and antenatal healthcare clinics in Palestine.

RESULTS: In this study, a total of 389 pregnant women completed the questionnaire. Of the pregnant women, 381 (97.9%) were aware of the existence of epidural analgesia, 172 (44.2%) had already used epidural analgesia, and 57 (33.1%) experienced complications as a result of epidural analgesia. Of the pregnant women, 308 (79.2%) stated that epidural analgesia should be available during vaginal birth. Of the pregnant women, 243 (62.5%) stated that they would use epidural analgesia if offered for free or covered by insurance. Multivariate logistic regression showed that women who were younger than 32 years, who have used epidural analgesia, and those who stated that epidural analgesia should be available during vaginal birth were 2.78-fold (95% CI: 1.54-5.04), 4.96-fold (95% CI: 2.71-9.10), and 13.57-fold (95% CI: 6.54-28.16) more likely to express willingness to use epidural analgesia, respectively.

CONCLUSIONS: Pregnant women had high awareness of the existence, moderate knowledge, and positive attitudes towards epidural analgesia for normal vaginal birth. Future studies should focus on educating pregnant women about all approaches that can be used to reduce labour pain including their risks and benefits.

PMID:38770655 | DOI:10.1080/01443615.2024.2354575

Cancer Med. 2024 May;13(10):e7290. doi: 10.1002/cam4.7290.

ABSTRACT

BACKGROUND: This study aimed to establish the standardized procedure of trans-areola single site endoscopic parathyroidectomy (TASSEP), and to compare the performance of TASSEP with that of conventional open parathyroidectomy (COP).

METHODS: This study enrolled 40 patients with primary hyperparathyroidism (PHPT) who underwent TASSEP, and included 40 of 176 PHPT patients who underwent COP based on propensity score matching. The retrospective analysis was conducted based on prospectively collected data. Perioperative outcomes, including surgical profile, surgical burden and cosmetic results and follow-up were reported. The learning curve was described using a cumulative sum (CUSUM) analysis.

RESULTS: 40 TASSEPs were completed successfully without conversions or severe complications. There was no statistically significant difference in operation time between TASSEP and COP groups (80.83 ± 11.95 vs. 76.95 ± 7.30 min, p = 0.084). Experience of 17 cases was necessitated to reach the learning curve of TASSEP. Postoperative pain score and traumatic index (C-reactive protein and erythrocyte sedimentation rate) in TASSEP were apparently lower than those in COP group (p < 0.05). During the proliferation and stabilization phases, TASSEP was associated with significantly better incision recovery and cosmetic scores. Postoperative serum calcium and PTH levels throughout the follow-up period indicated satisfactory surgical qualities in both groups.

CONCLUSION: Based on precise preoperative localization and intraoperative planning facilitated by three-dimensional (3D) virtual modeling, TASSEP can be feasibly performed on selected patients with satisfactory success rates and low complication rates, providing preferable cosmetic results and alleviating the surgical burden to a certain extent.

PMID:38770646 | DOI:10.1002/cam4.7290