Meta-analysis Of Real-world Data Research Articles

Burosumab Efficacy and Safety in Patients with X-Linked Hypophosphatemia: Systematic Review and Meta-analysis of Real-World Data.

To assess the efficacy and safety of burosumab in children and adults with X-linked hypophosphatemia based on real-world evidence. MEDLINE (via PubMed) and Cochrane Library were searched until 18 October 2023 for single-arm (before-after) studies. Registries including Clinicaltrials.gov, EU Clinical Trials, WHO International Clinical Trials Registry Platform, and conference abstracts. The outcomes were a change in serum phosphorus concentrations and change in RSS, a change in serum ALP, bone-specific ALP, a change in the ratio of Tubular maximum reabsorption of Phosphate to Glomerular Filtrate rate, a change in serum 1,25(OH)2D and 25(OH)2D concentrations, change in height Z-score, McMaster Universities Osteoarthritis Index (WOMAC) and safety outcomes. An inverse variance random-effects meta-analysis was applied for data synthesis. Fifteen studies (289 participants) were included. Burosumab treatment improved serum phosphate concentrations [mean difference 0.88mg/dl, 95% confidence interval 0.70 to 1.07, I2 = 92%), Rickets Severity score (mean difference -1.86, 95% confidence interval -2.5 to -1.21, I2 = 71%), serum alkaline phosphate concentrations (mean difference -1.86, 95% confidence interval -2.5 to -1.21, I2 = 71%), serum 1,25(OH)2D concentrations (mean difference 18.91pg/ml, 95% confidence interval 6.39 to 31.43, I2 = 96%) and renal phosphate reabsorption (mean difference 1.22 mg/dl, 95% confidence interval 0.70 to 1.74, I2 93%). Burosumab treatment improved overall clinical and laboratory findings in patients with X-linked hypophosphatemia.

Health-related Quality of Life of Patients with Rosacea: A Systematic Review and Meta-analysis of Real-world Data.

Patients with rosacea commonly experience stigmatization, which induces stress and thereby exacerbates their symptoms. Given the strong effects of rosacea on health-related quality of life (HRQoL), addressing the physical and psychosocial aspects of rosacea is essential. To examine the effects of rosacea on HRQoL, we conducted a systematic review and meta-analysis involving real-world data. PubMed, EMBASE, and the Cochrane Library were searched, and randomized controlled trials (RCTs), cross-sectional studies, and case series evaluating the HRQoL of patients with rosacea were included. HRQoL assessment tools such as the Dermatology Life Quality Index (DLQI) and Rosacea-Specific Quality-of-Life Questionnaire (RosaQoL) were used. Data on 13,453 patients were retrieved from 52 eligible studies: 4 RCTs, 15 case series, and 33 cross-sectional studies. Compared with healthy controls, patients with rosacea had significantly lower DLQI scores (standardized mean difference [SMD] = -1.09, 95% confidence interval [CI] = -0.81 to -1.37). The DLQI scores after treatment were higher than those before treatment (SMD = -1.451, 95% CI = -1.091 to -1.810). The pooled estimates for the overall DLQI and RosaQoL scores were 8.61 and 3.06, respectively. In conclusion, patients with rosacea have lower HRQoL compared with healthy individuals, and treatment for rosacea improves their HRQoL.

Time for detailed clinical management of Paget's disease of the breast.

Time for detailed clinical management of Paget's disease of the breast.

Precise application of sentinel lymph node biopsy in patients with ductal carcinoma in situ: A systematic review and meta-analysis of real-world data

PurposeAlthough ductal carcinoma in situ (DCIS) seldom involves lymph nodes, some patients may upstage to invasive disease, thus requiring a second surgery for sentinel lymph node (SLN) biopsy (SLNB). However, the indications of SLNB remain inconclusive and clinical trials are rarely available. Our aim is to systematically review the real-world data to evaluate whether SLNB is precisely applied in patients with a high risk of upstaging from DCIS to invasive carcinoma. MethodsPubMed, EMBASE, and Cochrane library databases were searched. Prospective and retrospective cohort studies that evaluated the pathological outcomes of SLNB and the upstaging rate in women with DCIS were included. The primary outcomes were the upstaging and SLN-positive rates of patients initially diagnosed as having DCIS. We analyzed factors, namely biopsy methods, clinical presentations, histological patterns, and hormone receptor status, that potentially indicate nodal involvement risk. ResultsWe retrieved 43 prospective and 69 retrospective studies including 44,001 patients. The pooled estimates of upstaging and SLN-positive rates were 25.8% (95% confidence interval [CI]: 0.230–0.286) and 4.9% (95% CI: 0.042–0.055), respectively. In subgroup analysis, the upstaging rate was significantly higher in patients with estrogen receptor-negative status, palpable mass, tumor size >2 cm on imaging, and high-nuclear grade and those who received a preoperative diagnosis through core needle biopsy. ConclusionThe upstaging and SLN-positive rates of DCIS were 25.8% and 4.9%, respectively. By selecting patients with high risk DCIS, surgeons may increase the precision of and reduce the excess and incomplete treatment rates of SLNB.

Efficacy & safety of PCSK9-inhibitors: A systematic review and meta-analysis of real-world data

Efficacy & safety of PCSK9-inhibitors: A systematic review and meta-analysis of real-world data

ASO Visual Abstract: Outcomes of Sentinel Node Biopsy in Women with Breast Cancer Following Neoadjuvant Therapy—Systematic Review and Meta-Analysis of Real-World Data

ASO Visual Abstract: Outcomes of Sentinel Node Biopsy in Women with Breast Cancer Following Neoadjuvant Therapy—Systematic Review and Meta-Analysis of Real-World Data

Outcomes of Sentinel Node Biopsy for Women with Breast Cancer After Neoadjuvant Therapy: Systematic Review and Meta-Analysis of Real-World Data.

Evidence on the accuracy of sentinel lymph node biopsy (SLNB) after neoadjuvant therapy (NAT) for patients with breast cancer is inconclusive. This study reviewed the real-world data to determine the acceptability of SLNB after NAT. The study searched for articles in the PubMed, EMBASE, and Cochrane Library databases. The primary outcomes were the identification rate for sentinel lymph nodes (SLNs) and the false-negative rate (FNR) for SLNB. The study also evaluated the FNR in subgroups defined by tumor stage, nodal stage, hormone receptor status, human epidermal growth factor receptor-2 status, tumor response, mapping technique, and number of SLNs removed. The study retrieved 61 prospective and 18 retrospective studies with 10,680 initially cN± patients. The pooled estimate of the identification rate was 0.906 (95 % confidence interval [CI], 0.891-0.922), and the pooled FNR was 0.118 (95 % CI, 0.103-0.133). In subgroup analysis, the FNR was significantly higher for the patients with estrogen receptor (ER)-negative status and fewer than three SLNs removed. The FNR did not differ significantly between the patients with and those without complete tumor response. Among the patients with initial clinical negative axillary lymph nodes, the incidence of node metastasis was 26.8 % (275/1041) after NAT. Real-world evidence indicates that the FNR of SLNB after NAT in breast cancer is 11.8 %, exceeding only slightly the commonly adopted threshold of 10 %. The FNR is significantly higher for patients with ER-negative status and removal of fewer than three SLNs. Using a dual tracer and removing at least three SLNs may increase the accuracy of SLNB after NAT.

Adjuvant use of ribavirin with treatment of hepatitis C virus in kidney transplant recipients: A systematic review and meta-analysis of real-world data

Hepatitis C virus infection among kidney transplant recipients (KTRs) is an important public health concern worldwide. Evidence on available treatments given shows a greater variability for safety and efficacy, thus we performed this large analysis to present the pooled findings. PubMed/Medline, ScienceDirect, and Google Scholar were searched to identify observational studies of both interferon-based conventional treatment (CT) and recently approved direct-acting antivirals (DAAs), published before November 2020. Meta-analysis of effectiveness outcome (sustained virological response [SVR12]) and safety data (adverse events and discontinuation rate) was performed using a random-effect model. Subgroup analysis based on ± ribavirin with both treatments was performed to assess its relevance in clinical practice. Chi-square and I2 tests were used to assess heterogeneity between the studies. Twenty-five and 9 observational records reporting on treatment with DAAs (n = 943) and CT (n = 125), respectively, are analyzed. The overall pooled estimate of SVR12 rate was found as 95.3%, (range: 92.6%–97%) with DAAs in comparison to 46.4% (range: 33.7%–59.6%) with CT. The pooled prevalence of adverse drug reactions (ADRs) in CT was higher as 47.8% than in DAAs as 23.8%, consequently showing a high discontinuation rate of 31.7% in CT as compared to 6.5% with DAAs. Subgroup analyses of both DAAs and interferon-based treatment ± ribavirin showed decreased effectiveness and increased ADR rates on adding ribavirin. The effectiveness of DDAs was found significantly higher than CT with a better safety profile in KTRs. Ribavirin did not provide any additional benefits while given in combination with either of the treatments available.

Accuracy of point-of-care diagnostic tests for SARS-CoV-2 antibodies (IgM/IgG) is heterogeneous.

Riccò M, Ferraro P, Gualerzi G, et al. Point-of-care diagnostic tests for detecting SARS-CoV-2 antibodies: a systematic review and meta-analysis of real-world data. J Clin Med. 2020;9:1515. 32443459.

Point-of-Care Diagnostic Tests for Detecting SARS-CoV-2 Antibodies: A Systematic Review and Meta-Analysis of Real-World Data.

SARS-CoV-2 is responsible for a highly contagious infection, known as COVID-19. SARS-CoV-2 was discovered in late December 2019 and, since then, has become a global pandemic. Timely and accurate COVID-19 laboratory testing is an essential step in the management of the COVID-19 outbreak. To date, assays based on the reverse-transcription polymerase chain reaction (RT-PCR) in respiratory samples are the gold standard for COVID-19 diagnosis. Unfortunately, RT-PCR has several practical limitations. Consequently, alternative diagnostic methods are urgently required, both for alleviating the pressure on laboratories and healthcare facilities and for expanding testing capacity to enable large-scale screening and ensure a timely therapeutic intervention. To date, few studies have been conducted concerning the potential utilization of rapid testing for COVID-19, with some conflicting results. Therefore, the present systematic review and meta-analysis was undertaken to explore the feasibility of rapid diagnostic tests in the management of the COVID-19 outbreak. Based on ten studies, we computed a pooled sensitivity of 64.8% (95%CI 54.5–74.0), and specificity of 98.0% (95%CI 95.8–99.0), with high heterogeneity and risk of reporting bias. We can conclude that: (1) rapid diagnostic tests for COVID-19 are necessary, but should be adequately sensitive and specific; (2) few studies have been carried out to date; (3) the studies included are characterized by low numbers and low sample power, and (4) in light of these results, the use of available tests is currently questionable for clinical purposes and cannot substitute other more reliable molecular tests, such as assays based on RT-PCR.

ECOG performance status ≥2 as a prognostic factor in patients with advanced non small cell lung cancer treated with immune checkpoint inhibitors—A systematic review and meta-analysis of real world data

ObjectivesICIs have been approved and are routinely administered regardless of performance status (PS), despite randomized clinical trials of ICIs alone or combined with chemotherapy or target therapy enrolled patients with ECOG PS 0 or 1, while patients with ECOG PS 2 or more were excluded. Materials and methodsWe carried out a meta-analysis of available clinical studies exploring the prognostic impact of PS ≥ 2 on Overall Survival (OS), Progression Free Survival (PFS) or Overall Response Rate (ORR) in patients with non small cell lung cancer (NSCLC) treated with immunotherapy (any line). ResultsWe reviewed 19 studies, comprising 3600 NSCLC patients, 757 of whom with ECOG PS > 1 (average 21.0%, range 6.0–48.6%). In the overall population PS ≥ 2 resulted in worse OS, PFS and ORR (OS pooled hazard ratio of 2.72; 95% CI: 2.03–3.63; I2 72.70%, p < 0.001; PFS pooled hazard ratio of 2.39; 95% CI 1.81–3.15, p < 0.0001; I2 73.03%; ORR pooled odds ratio 0.25; 95% CI 0.11–0.56, p 0.001; I2 0.00%). ConclusionECOG PS ≥ 2 retains an important prognostic validity in patients treated with ICI similar, in terms of effect size, to that reported for chemotherapy in NSCLC. The high level of heterogeneity for OS and PFS analysis (but not for ORR), not completely explained by the different proportion of ECOG 3−4 patients (ranging from 0% to 50% of the PS ≥ 2 population), could be the result of both patient heterogeneity within the PS 2 population and the subjectivity of ECOG PS assessment. Whether poorer PS is also a predictor of lower immunotherapy efficacy remains still to be demonstrated.

Abstract P2-17-02: Locoregional therapy targeted at the primary tumour improves overall survival in patients presenting with de novo stage IV metastatic breast cancer: A systematic review and meta-analysis of real-world data with 201598 patients

Abstract Background: De novo stage IV metastatic breast cancer is a complex disease that is traditionally treated using systemic therapy. There is mounting evidence that locoregional therapy (LRT), defined as resection of the primary tumour and/or localised radiotherapy, could be associated with survival improvements. We aimed to conduct a meta-analysis to inform decision making. Methods: Using the PubMed, Cochrane and Ovid SP databases, a literature review and meta-analysis was undertaken to assess whether LRT of the primary tumour in metastatic breast cancer prolongs survival. Results: 48 studies met the criteria for analysing the efficacy of all locoregional treatments (radiotherapy and/or surgery) and 44 studies were suitable for the analysis of surgery-only treatment of the primary. Studies were analysed for the impact of LRT on survival. All LRT resulted in a significant 32.9% reduction in mortality with LRT (N=48; HR=0.671: 95% CI 0.624-0.721). Primary resection alone resulted in a significant 36.9% mortality (N=44; HR=0.631: 95% CI 0.591-0.674). Conclusions: This is the largest meta-analysis regarding this question to date. LRT seems to improve overall survival in stage IV disease at initial diagnosis and should be considered in selected patients after a multidisciplinary discussion. Citation Format: Salim Tayeh, Ritika Gera, Hiba El Hage Chehade, Umar Wazir, Abdul Kasem, Kefah Mokbel. Locoregional therapy targeted at the primary tumour improves overall survival in patients presenting with de novo stage IV metastatic breast cancer: A systematic review and meta-analysis of real-world data with 201598 patients [abstract]. In: Proceedings of the 2019 San Antonio Breast Cancer Symposium; 2019 Dec 10-14; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2020;80(4 Suppl):Abstract nr P2-17-02.

Abstract P4-13-09: Oncoplastic breast conserving surgery (OBCS): A meta-analysis of real-world data

Abstract Background: The role of oncoplastic breast conserving surgery (OBCS) is that of a middle ground between standard breast conserving surgery (SBCS) and mastectomy - it allows adequate resection margins of tumours unsuitable for traditional breast conserving surgery whilst allowing for a better cosmetic outcome and a reduced morbidity rate when compared to a traditional mastectomy. However, due to this being a relatively new type of procedure, there is limited evidence on its oncological safety, which is the ultimate goal of any breast cancer surgery. Methods: This study aims to compare oncological safety of OBCS with SBCS and mastectomy by examining the relative risk of cancer recurrence and re-operation rates through meta-analysis. Literature search of Pubmed and Web of Science databases was conducted looking for studies up until February 2019. Meta-Analysis was performed using R Statistical Software (www.r-project.org). Results: Initial finding of 645 studies was narrowed down to a pool of 19 studies including 18,163 patients. For the primary outcome measure of recurrence there was found to be no significant difference between the OBCS and SBCS or mastectomy (RR 0.861; 95% CI 0.640-1.160; p&amp;lt;0.296). The secondary outcome measure of re-operation was initially found to be significant in favour of OBCS (RR 0.64; 95% CI 0.46-0.89; p&amp;lt;0.01), however after adjustment for publication bias this was attenuated to an insignificant difference between the two study groups (RR 0.86; 95% CI 0.56-1.31; p&amp;gt;0.05). Conclusions: For both recurrence of cancer and re-operation rate, there was not found to be a significant difference between OBCS and more traditional techniques. This would suggest that OBCS is of comparable oncological safety to more established operations and a useful option in suitable patients. Citation Format: Sebastian Kosasih, Salim Tayeh, Kefah Mokbel, Abdul Kasem. Oncoplastic breast conserving surgery (OBCS): A meta-analysis of real-world data [abstract]. In: Proceedings of the 2019 San Antonio Breast Cancer Symposium; 2019 Dec 10-14; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2020;80(4 Suppl):Abstract nr P4-13-09.

Breast Conserving Surgery Is An Oncologically Safe Procedure, Does The Addition Of Autologous Fat Grafting Compromise Its Oncological Safety? A Meta-Analysis of Real-World Data

Breast Conserving Surgery Is An Oncologically Safe Procedure, Does The Addition Of Autologous Fat Grafting Compromise Its Oncological Safety? A Meta-Analysis of Real-World Data

Balloon-expandable transcatheter aortic valve implantation with or without pre-dilation \u2013 results of a meta-analysis of 3 multicenter registries

BackgroundTo evaluate the outcomes of transcatheter aortic valve implantation (TAVI) without balloon aortic valvuloplasty (BAV) in a real-world setting through a patient-level meta-analysis.MethodsThe meta-analysis included patients of three European multicenter, prospective, observational registry studies that compared outcomes after Edwards SAPIEN 3 or XT TAVI with (n = 339) or without (n = 355) BAV. Unadjusted and adjusted pooled odds ratios (with 95% confidence intervals) were calculated for procedural and 30-day outcomes.ResultsMedian procedural time was shorter in the non-BAV group than in the BAV group (73 versus 93 min, p = 0.001), as was median fluoroscopy time (7 versus 11 min, p = 0.001). Post-delivery balloon dilation (15.5% versus 22.4%, p = 0.02) and catecholamine use (9.0% vs. 17.9%; p = 0.016) was required less often in the non-BAV group than in the BAV group with the difference becoming insignificant after multiple adjustment. There was a reduced risk for periprocedural atrioventricular block during the intervention (1.4% versus 4.1%, p = 0.035) which was non-significant after adjustment. The rate of moderate/severe paravalvular regurgitation post-TAVI was 0.6% in the no-BAV group versus 2.7% in the BAV group. There were no between-group differences in the risk of death, stroke or other adverse clinical outcomes at day 30.ConclusionsThis patient-level meta-analysis of real-world data indicates that TAVI performed without BAV is advantageous as it has an adequate device success rate, reduced procedure time and no adverse effects on short-term clinical outcomes.

Vinflunine in the treatment of relapsed metastatic urothelial cancer: A systematic review and meta-analysis of real-world series.

Vinflunine (VFL) is approved in Europe as second-line treatment of metastatic urothelial cancer after failure of platinum-containing therapy. We performed a systematic review and meta-analysis of real-world data (RWD) to assess utilization, efficacy and safety of VFL. We performed a MEDLINE search for the period of 1/1/2000-31/8/2017. Full-length articles providing post-marketing RWD on VFL in patients failing previous chemotherapy were eligible. Interventional clinical trials were excluded. Ten studies with 797 patients were identified. According to pooled REs analysis, overall response rate was 19%, most frequent, all-grade toxicities were fatigue (41%), constipation (39%), nausea/vomiting (25%), and most prevalent Grade 3-4 toxicities were neutropenia (13%), anaemia (9%), fatigue (8%). Median OS was comparable to results reported in recent randomized studies. Our findings confirm the efficacy and safety of VFL in an unselected population and support the use of VFL in the changing treatment paradigm of relapsed mUC.

P3447Mortality and stroke prevention in atrial fibrillation; network meta-analysis of real world data

P3447Mortality and stroke prevention in atrial fibrillation; network meta-analysis of real world data

RAS mutation prevalence among patients with metastatic colorectal cancer: a meta-analysis of real-world data.

Aim:A confirmed wild-type RAS tumor status is commonly required for prescribing anti-EGFR treatment for metastatic colorectal cancer. This noninterventional, observational research project estimated RAS mutation prevalence from real-world sources.Materials & methods:Aggregate RAS mutation data were collected from 12 sources in three regions. Each source was analyzed separately; pooled prevalence estimates were then derived from meta-analyses.Results:The pooled RAS mutation prevalence from 4431 tumor samples tested for RAS mutation status was estimated to be 43.6% (95% CI: 38.8–48.5%); ranging from 33.7% (95% CI: 28.4–39.3%) to 54.1% (95% CI: 51.7–56.5%) between sources.Conclusion:The RAS mutation prevalence estimates varied among sources. The reasons for this are not clear and highlight the need for further research.

RAS mutation prevalence among patients with metastatic colorectal cancer: A meta-analysis of real-world data.

513 Background: Guidelines for prescribing anti-EGFR therapy for metastatic colorectal cancer (mCRC) require prior testing to confirm RAS (exons 2, 3, 4 of KRAS and NRAS) wild-type status in Europe and the USA. There is limited published evidence reporting the prevalence of RAS mutations in patients with mCRC in a real-world setting. The aim of this study was to use data from a range of real-world sources to obtain RAS mutation prevalence estimates for different geographic regions. Methods: Aggregated RAS mutation prevalence data were collected from 13 sources, including individual pathology centers (n = 7), mCRC registries (n = 3), and Amgen-sponsored studies (n = 3). Data sources included in this study originated in Europe (n = 10), the Middle East (n = 2), and South America (n = 1). A meta-analysis of all collected data was carried out to investigate the effect of heterogeneity amongst the data sources and obtain pooled RAS prevalence estimates for each, using a mixed regression model. Results: Aggregate data from 4322 patients with mCRC were included in the pooled meta-analysis. The overall RAS mutation prevalence across all data sources was estimated to be 43.5% (95% CI: 41.5–45.5%). RAS mutation prevalence varied between data sources, ranging from low estimates of 33.7% (95% CI: 28.4–39.3%) for a Middle Eastern pathology dataset, and 34.6% (95% CI: 27.7–42.1%) for a Middle Eastern mCRC registry, to the highest estimates of 53.6% (95% CI: 43.2–63.8%) and 54.1% (95% CI: 51.7–56.5%) in two European pathology centers, in the Czech Republic and Poland, respectively. Conclusions: This is one of the first studies to carry out a large pooled analysis of RAS mutation prevalence, based on real-world data. There was a high degree of heterogeneity between the sources included, possibly due to the types of data source, patient selection criteria, geographic location, or differences in laboratory testing methods. The estimated RAS mutation prevalence reported here is lower than that in a recent pooled analysis of past clinical trials, which reported an overall prevalence of 55.5% (95% CI: 53.9–57.9%) (Peeters M, et al. Eur J Cancer 2015;51:1704–13). Further investigations are required to explain the disparity in these findings.

Trastuzumab-induced cardiotoxicity in elderly women with HER-2-positive breast cancer: a meta-analysis of real-world data

Background: We conducted a meta-analysis to assess the overall risk of cardiac toxicity associated with trastuzumab treatment in elderly breast cancer patients. Methods: We searched databases from PubMed, EMBASE and Cochrane Central Registry of Controlled Trials to identify relevant studies. Statistical analyses were conducted to calculate the incidence rate, overall hazard ratio (HR) and 95% CIs using a fixed effects model. Results: A total of 116,342 and 360 elderly patients from five cohort studies and two randomized clinical trials (RCTs) were included for analysis. The pooled incidences of symptomatic congestive heart failure (CHF) and CHF/HF/CM were 6.4% (95% CI 4.1% – 9.4) and 16.4% (95% CI 16.19% – 16.61) in patients with median age of 67.5 years from two RCTs and in patients with median age of 67.5 (60 – 75), 71 (66 – 80+), 74.5 (65 – 89), 75 (66 – 81+) and 79.5 (60 – 99) from five cohort studies, respectively. Trastuzumab was significantly correlated with an increased risk of defined cardiac toxicities in five cohort studies (HR = 1.89, 95% CI 1.72 – 2.07, p < 0.00001) and two RCTs (HR = 3.00, 95% CI 1.71, 5.26, p < 0.00001). Sub-group analysis showed that the anthracycline-based chemotherapy increased the risk of CHF/HF and CM in patients among five cohort studies (HR = 2.16, 95% CI: 1.8 – 1.87, p < 0.00001). Conclusion: Trastuzumab is likely associated with an increased risk of cardiac toxicity in elderly patients with HER-2-positive breast cancer. Carefully monitoring cardiac function in elderly patients receiving trastuzumab, particularly with concurrent use of anthracycline, is warranted.