Dataset Information

The Clinical Efficacy of Imiglucerase versus Eliglustat in Patients with Gaucher's Disease Type 1: A Systematic Review.

ABSTRACT: Gaucher's disease (GD) is one of the most common lysosomal diseases in humans. It results from ?-glucosidase deficiency and leads to necrosis, especially in macrophages with the accumulation of glucosylceramidase in cells. Most of the deleterious effects of the disease are seen in the liver, spleen, and bone marrow. The aim of this study was to compare the efficacy of Imiglucerase with Eliglustat in treating patients with GD. PubMed/Medline, Cochrane Library, Scopus, Web of Science, Embase, and Google Scholar were searched from inception to August, 2018. Predefined inclusion criteria for included studies were based on search methodology and are as follows: All randomized, quasi-randomized controlled, and cohort studies about patients with GD Type 1 that Imiglucerase was compared with Eliglustat were included. Two authors independently choose the papers based on the inclusion criteria. From 2979 recognized studies, three studies including two randomized clinical trials and one cohort study were recognized to meet the inclusion criteria. The primary outcomes were hemoglobin level, platelets count, liver, and spleen size, and the secondary outcomes were the immunological side effects of the medicines and bone complications. The results showed that there is no meaningful difference between the two medicines in terms of increasing blood hemoglobin, platelets count, and reducing the liver and spleen size. The findings of this review showed that both medicines are effective in the treatment of GD Type 1 and there is no statistically significant difference between their efficacies.

SUBMITTER: Nabizadeh A

PROVIDER: S-EPMC6298139 | biostudies-other | 2018 Oct-Dec

REPOSITORIES: biostudies-other

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The Clinical Efficacy of Imiglucerase versus Eliglustat in Patients with Gaucher's Disease Type 1: A Systematic Review.

Nabizadeh Azita A Amani Bahman B Kadivar Maliheh M Toroski Mahdi M Asl Akbar Abdollahi AA Bayazidi Yahya Y Mojahedian Mahdi M Davari Majid M

Journal of research in pharmacy practice 20181001 4

Gaucher's disease (GD) is one of the most common lysosomal diseases in humans. It results from β-glucosidase deficiency and leads to necrosis, especially in macrophages with the accumulation of glucosylceramidase in cells. Most of the deleterious effects of the disease are seen in the liver, spleen, and bone marrow. The aim of this study was to compare the efficacy of Imiglucerase with Eliglustat in treating patients with GD. PubMed/Medline, Cochrane Library, Scopus, Web of Science, Embase, and ...[more]

PMID: 30622983

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Project description:BackgroundHis-Purkinje system pacing (HPSP), including his-bundle pacing (HBP) and left bundle branch area pacing (LBBaP), imitates the natural conduction of the heart as an alternative to biventricular pacing (BVP) in cardiac resynchronization therapy (CRT). However, the feasibility and efficacy of HPSP were currently only evidenced by studies with a limited sample size, so this study aimed to provide a comprehensive assessment through a systematic review and meta-analysis.MethodsIn order to compare the clinical outcomes associated with HPSP and BVP in patients for CRT, PubMed, EMBASE, Cochrane Library and Web of Science database were searched from inception to April 10, 2023. Clinical outcomes of interest including QRS duration (QRSd), left ventricular (LV) function and New York Heart Association (NYHA) functional classification, pacing threshold, echocardiographic and clinical response, hospitalization rate of HF and all-cause mortality were also extracted and summarized for meta-analysis.ResultsA total of 13 studies (ten observational studies and three randomized studies) involving 1,121 patients were finally included. The patients were followed up for 6-27 months. Compared with BVP, CRT patients treated by HPSP presented shorter QRSd [mean difference (MD): -26.23 ms, 95% confidence interval (CI): -34.54 to -17.92, P < 0.001, I2 = 91%], greater LV functional improvement with increased left ventricular ejection fraction (LVEF) (MD: 6.01, 95% CI: 4.81 to 7.22, P < 0.001, I2 = 0%), decreased left ventricular end-diastolic dimension (LVEDD) (MD: -2.91, 95% CI: -4.86 to -0.95, P = 0.004, I2 = 35%), and more improved NYHA functional classification (MD: -0.45, 95% CI: -0.67 to -0.23, P < 0.001, I2 = 70%). In addition, HPSP was more likely to have higher echocardiographic [odds ratio (OR): 2.76, 95% CI: 1.74 to 4.39, P < 0.001, I2 = 0%], clinical (OR: 2.10, 95% CI: 1.16 to 3.80, P = 0.01, I2 = 0%) and super clinical (OR: 3.17, 95% CI: 2.09 to 4.79, P < 0.001, I2 = 0%) responses than BVP, and a lower hospitalization rate of HF (OR: 0.34, 95% CI: 0.22 to 0.51, P < 0.001, I2 = 0%), while presented no difference (OR: 0.68, 95% CI: 0.44 to 1.06, P = 0.09, I2 = 0%) in all-cause mortality compared with BVP. With threshold change taking into account, BVP was less stable than LBBaP (MD: -0.12 V, 95% CI: -0.22 to -0.03, P = 0.01, I2 = 57%), but had no difference with HBP (MD: 0.11 V, 95% CI: -0.09 to 0.31, P = 0.28, I2 = 0%).ConclusionThe present findings suggested that HPSP was associated with greater improvement of cardiac function in patients with indication for CRT and was a potential alternative to BVP to achieve physiological pacing through native his-purkinje system.

Project description:OBJECTIVE:To evaluate the clinical efficacy and effectiveness of using 3D printing to develop medical devices across all medical fields. DESIGN:Systematic review compliant with Preferred Reporting Items for Systematic Reviews and Meta-Analyses. DATA SOURCES:PubMed, Web of Science, OVID, IEEE Xplore and Google Scholar. METHODS:A double-blinded review method was used to select all abstracts up to January 2017 that reported on clinical trials of a three-dimensional (3D)-printed medical device. The studies were ranked according to their level of evidence, divided into medical fields based on the International Classification of Diseases chapter divisions and categorised into whether they were used for preoperative planning, aiding surgery or therapy. The Downs and Black Quality Index critical appraisal tool was used to assess the quality of reporting, external validity, risk of bias, risk of confounding and power of each study. RESULTS:Of the 3084 abstracts screened, 350 studies met the inclusion criteria. Oral and maxillofacial surgery contained 58.3% of studies, and 23.7% covered the musculoskeletal system. Only 21 studies were randomised controlled trials (RCTs), and all fitted within these two fields. The majority of RCTs were 3D-printed anatomical models for preoperative planning and guides for aiding surgery. The main benefits of these devices were decreased surgical operation times and increased surgical accuracy. CONCLUSIONS:All medical fields that assessed 3D-printed devices concluded that they were clinically effective. The fields that most rigorously assessed 3D-printed devices were oral and maxillofacial surgery and the musculoskeletal system, both of which concluded that the 3D-printed devices outperformed their conventional comparators. However, the efficacy and effectiveness of 3D-printed devices remain undetermined for the majority of medical fields. 3D-printed devices can play an important role in healthcare, but more rigorous and long-term assessments are needed to determine if 3D-printed devices are clinically relevant before they become part of standard clinical practice.

Project description:Acute myeloid leukemia (AML) is a rapidly progressive hematological malignancy that is difficult to cure. The prognosis is poor and treatment options are limited in case of relapse. A comprehensive assessment of current disease burden and the clinical efficacy of non-intensive therapies in this population are lacking. We conducted two systematic literature reviews (SLRs). The first SLR (disease burden) included observational studies reporting the incidence and economic and humanistic burden of relapsed/refractory (RR) AML. The second SLR (clinical efficacy) included clinical trials (phase II or later) reporting remission rates (complete remission [CR] or CR with incomplete hematologic recovery [CRi]) and median overall survival (mOS) in patients with RR AML or patients with de novo AML who are ineligible for intensive chemotherapy. For both SLRs, MEDLINE®/Embase® were searched from January 1, 2008 to January 31, 2020. Clinical trial registries were also searched for the clinical efficacy SLR. After screening, two independent reviewers determined the eligibility for inclusion in the SLRs based on full-text articles. The disease burden SLR identified 130 observational studies. The median cumulative incidence of relapse was 29.4% after stem cell transplant and 46.8% after induction chemotherapy. Total per-patient-per-month costs were $28,148-$29,322; costs and health care resource use were typically higher for RR versus non-RR patients. Patients with RR AML had worse health-related quality of life (HRQoL) scores than patients with de novo AML across multiple instruments, and lower health utility values versus other AML health states (i.e. newly diagnosed, remission, consolidation, and maintenance therapy). The clinical efficacy SLR identified 50 trials (66 total trial arms). CR/CRi rates and mOS have remained relatively stable and low over the last 2 decades. Across all arms, the median rate of CR/CRi was 18.3% and mOS was 6.2 months. In conclusion, a substantial proportion of patients with AML will develop RR AML, which is associated with significant humanistic and economic burden. Existing treatments offer limited efficacy, highlighting the need for more effective non-intensive treatment options.

Project description:Vancomycin and teicoplanin are the glycopeptides currently in use for the treatment of infections caused by invasive beta-lactam-resistant gram-positive organisms. We conducted a systematic review and meta-analysis of randomized controlled trials that have compared vancomycin and teicoplanin administered systemically for the treatment of suspected or proven infections. A comprehensive search of trials without year, language, or publication status restrictions was performed. The primary outcome was all-cause mortality. Two reviewers independently extracted the data. Risk ratios (RRs) with 95% confidence intervals (CIs) were pooled by using the fixed-effect model (RRs of >1 favor vancomycin). Twenty-four trials were included. All-cause mortality was similar overall (RR, 0.95; 95% CI, 0.74 to 1.21), and there was no significant heterogeneity. In trials that used adequate allocation concealment, the results favored teicoplanin (RR, 0.82; 95% CI, 0.63 to 1.06), while in trials with unknown methods or inadequate concealment, the results favored vancomycin (RR, 3.61; 95% CI, 1.27 to 10.30). The latter trials might have recruited more severely ill patients. No other variable affected the RRs for mortality, including the assessment of glycopeptides administered empirically or for proven infections, neutropenia, the participant's age, and drug dosing. There were no significant differences between teicoplanin and vancomycin with regard to clinical failure (RR, 0.92; 95% CI, 0.81 to 1.05), microbiological failure (RR, 1.24; 95% CI, 0.93 to 1.65), and other efficacy outcomes. Lower RRs (in favor of teicoplanin) for clinical failure were observed with a lower risk of bias and when treatment was initiated for infections caused by gram-positive organisms rather than empirically. Total adverse events (RR, 0.61; 95% CI, 0.50 to 0.74), nephrotoxicity (RR, 0.44; 95% CI, 0.32 to 0.61), and red man syndrome were significantly less frequent with teicoplanin. Teicoplanin is not inferior to vancomycin with regard to efficacy and is associated with a lower adverse event rate than vancomycin.

Project description:ObjectiveTo determine the effects and complications of arthroscopic surgery compared with conservative management strategies in patients with degenerative knee disease.DesignSystematic review.Main outcome measuresPain, function, adverse events.Data sourcesMEDLINE, EMBASE, the Cochrane Central Register of Controlled Trials (CENTRAL), Google Scholar and Open Grey up to August 2016.Eligibility criteriaFor effects, randomised clinical trials (RCTs) comparing arthroscopic surgery with a conservative management strategy (including sham surgery) in patients with degenerative knee disease. For complications, RCTs and observational studies.Review methodsTwo reviewers independently extracted data and assessed risk of bias for patient-important outcomes. A parallel guideline committee (BMJ Rapid Recommendations) provided input on the design and interpretation of the systematic review, including selection of patient-important outcomes. We used the GRADE approach to rate the certainty (quality) of the evidence.ResultsWe included 13 RCTs and 12 observational studies. With respect to pain, the review identified high-certainty evidence that knee arthroscopy results in a very small reduction in pain up to 3?months (mean difference =5.4 on a 100-point scale, 95% CI 2.0 to 8.8) and very small or no pain reduction up to 2?years (mean difference =3.1, 95% CI -0.2 to 6.4) when compared with conservative management. With respect to function, the review identified moderate-certainty evidence that knee arthroscopy results in a very small improvement in the short term (mean difference =4.9 on a 100-point scale, 95% CI 1.5 to 8.4) and very small or no improved function up to 2?years (mean difference =3.2, 95% CI -0.5 to 6.8). Alternative presentations of magnitude of effect, and associated sensitivity analyses, were consistent with the findings of the primary analysis. Low-quality evidence suggested a very low probability of serious complications after knee arthroscopy.ConclusionsOver the long term, patients who undergo knee arthroscopy versus those who receive conservative management strategies do not have important benefits in pain or function.Trial registration numberPROSPERO CRD42016046242.

Project description:BackgroundArtificial intelligence (AI) has been extensively used in a range of medical fields to promote therapeutic development. The development of diverse AI techniques has also contributed to early detections, disease diagnoses, and referral management. However, concerns about the value of advanced AI in disease diagnosis have been raised by health care professionals, medical service providers, and health policy decision makers.ObjectiveThis review aimed to systematically examine the literature, in particular, focusing on the performance comparison between advanced AI and human clinicians to provide an up-to-date summary regarding the extent of the application of AI to disease diagnoses. By doing so, this review discussed the relationship between the current advanced AI development and clinicians with respect to disease diagnosis and thus therapeutic development in the long run.MethodsWe systematically searched articles published between January 2000 and March 2019 following the Preferred Reporting Items for Systematic reviews and Meta-Analysis in the following databases: Scopus, PubMed, CINAHL, Web of Science, and the Cochrane Library. According to the preset inclusion and exclusion criteria, only articles comparing the medical performance between advanced AI and human experts were considered.ResultsA total of 9 articles were identified. A convolutional neural network was the commonly applied advanced AI technology. Owing to the variation in medical fields, there is a distinction between individual studies in terms of classification, labeling, training process, dataset size, and algorithm validation of AI. Performance indices reported in articles included diagnostic accuracy, weighted errors, false-positive rate, sensitivity, specificity, and the area under the receiver operating characteristic curve. The results showed that the performance of AI was at par with that of clinicians and exceeded that of clinicians with less experience.ConclusionsCurrent AI development has a diagnostic performance that is comparable with medical experts, especially in image recognition-related fields. Further studies can be extended to other types of medical imaging such as magnetic resonance imaging and other medical practices unrelated to images. With the continued development of AI-assisted technologies, the clinical implications underpinned by clinicians' experience and guided by patient-centered health care principle should be constantly considered in future AI-related and other technology-based medical research.

Dataset Information

The Clinical Efficacy of Imiglucerase versus Eliglustat in Patients with Gaucher's Disease Type 1: A Systematic Review.

Publications

The Clinical Efficacy of Imiglucerase versus Eliglustat in Patients with Gaucher's Disease Type 1: A Systematic Review.

Similar Datasets

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