Topical Beta Blockers in Small Infantile Hemangioma: State of the Art?

Author(s):  
Stefan Bittmann

Infantile hemangiomas (IH) are benign forms of endothelial proliferation in the first 8 weeks of life. Leaute-Labreze et al. published the first spontaneous cases of IH treated with beta blockers in 2008 due to their cardiac disease in the New England Journal of Medicine. This was a revolutionary new understanding of treatment in infantile hemangioma (IH). Not only systemic but also topical beta blockers seem to be an effective treatment option for small infantile hemangioma. Efficacy and safety of topical beta blockers in the treatment of infantile hemangioma (IH) were proved in systematic reviews and meta-analysis.

2016 ◽  
Vol 10 (02) ◽  
pp. 108 ◽  
Author(s):  
Philippe Denis ◽  

Ultrasound ciliary plasty (UCP) is a novel, non-invasive procedure for the control of intraocular pressure (IOP) in patients with openangle glaucoma (OAG), and is particularly useful for refractory glaucoma after failed filtering surgery and patients with elevated risk of surgical failure due to high risk of conjunctival bleb scarring. A meta-analysis was performed of seven clinical trials, involving 251 patients, which evaluated the efficacy and safety of the procedure. The procedure was effective in reducing mean IOP across all indications and IOP reductions were similar in patients with refractory and non-refractory glaucoma. Safety and tolerability were good, with conjunctival hyperaemia being the most common side effect. Serious complications were rare. Procedures using the second-generation therapy probe were associated with superior reproducibility of IOP reduction compared with the first-generation probe. In summary, the procedure is a promising and effective treatment option for patients with refractory and non-refractory OAG.


BMC Cancer ◽  
2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Kabirraaj Toor ◽  
Mark R. Middleton ◽  
Keith Chan ◽  
Adenike Amadi ◽  
Andriy Moshyk ◽  
...  

Abstract Background Immune checkpoint inhibitors and targeted therapies are approved for adjuvant treatment of patients with resected melanoma; however, they have not been compared in randomized controlled trials (RCTs). We compared the efficacy and safety of adjuvant nivolumab with other approved treatments using available evidence from RCTs in a Bayesian network meta-analysis (NMA). Methods A systematic literature review was conducted through May 2019 to identify relevant RCTs evaluating approved adjuvant treatments. Outcomes of interest included recurrence-free survival (RFS)/disease-free survival (DFS), distant metastasis-free survival (DMFS), all-cause grade 3/4 adverse events (AEs), discontinuations, and discontinuations due to AEs. Time-to-event outcomes (RFS/DFS and DMFS) were analyzed both assuming that hazard ratios (HRs) are constant over time and that they vary. Results Of 26 identified RCTs, 19 were included in the NMA following a feasibility assessment. Based on HRs for RFS/DFS, the risk of recurrence with nivolumab was similar to that of pembrolizumab and lower than that of ipilimumab 3 mg/kg, ipilimumab 10 mg/kg, or interferon. Risk of recurrence with nivolumab was similar to that of dabrafenib plus trametinib at 12 months, however, was lower beyond 12 months (HR [95% credible interval] at 24 months, 0.46 [0.27–0.78]; at 36 months, 0.28 [0.14–0.59]). Based on HRs for DMFS, the risk of developing distant metastases was lower with nivolumab than with ipilimumab 10 mg/kg or interferon and was similar to dabrafenib plus trametinib. Conclusion Adjuvant therapy with nivolumab provides an effective treatment option with a promising risk–benefit profile.


2021 ◽  
Vol 70 (10) ◽  
Author(s):  
Jielin Qin ◽  
Han Yang ◽  
Zhiming Shan ◽  
Lingzhi Jiang ◽  
Qingxian Zhang

Antifungal drugs have already been established as an effective treatment option for Candida parapsilosis infections, but there is no universal consensus on the ideal target for clinical efficacy and safety of antifungal drugs for the treatment of C. parapsilosis infections. Few studies have directly compared the efficacies of antifungal drugs for the treatment of C. parapsilosis infections. We hypothesize that different antifungal drugs offer differing clinical efficacy and safety for the treatment of C. parapsilosis infections. We performed a comprehensive network meta-analysis on different strategies for C. parapsilosis infection treatment and compared the clinical efficacy and safety of antifungal drugs as interventions for C. parapsilosis infections. The Cochrane Database of Systematic Reviews, Medline, Embase, PubMed, Web of Science, China National Knowledge Infrastructure (CNKI), Technology of Chongqing VIP database, Wan Fang Data, and SinoMed databases were searched to identify appropriate randomized trials. Among the extracted C. parapsilosis cases, the survival and death rates with treatment of C. parapsilosis infection were compared among groups treated with different antifungal drugs. According to the evidence-network analysis, echinocandins were a better choice than other drugs for treating C. parapsilosis infections, and more importantly, caspofungin showed a more preferable effect for decreasing the risk of 30 day mortality. In conclusion, this study systematically evaluated the effectiveness and safety of antifungal drugs for the purpose of helping clinicians choose the most appropriate antifungal drugs. Future studies with larger samples are needed to evaluate the effects of patient factors on the clinical efficacy and safety of antifungal drugs for C. parapsilosis infections.


2021 ◽  
Vol 42 (Supplement_1) ◽  
Author(s):  
M Perrett ◽  
N Gohil ◽  
O Tica ◽  
K.V Bunting ◽  
D Kotecha

Abstract Background Intravenous (IV) beta-blockers are commonly used to manage patients with acute atrial fibrillation (AF) and atrial flutter (AFl). Important pharmacodynamic differences exist amongst beta-blockers, and the choice of beta-blocker or other therapy is often not evidence-based. Purpose Systematic review and meta-analysis of randomised controlled trials (RCTs) in the setting of acute AF/AFl to assess the efficacy and safety of IV beta-blockers against other pharmacological interventions (diltiazem, verapamil, digoxin, anti-arrhythmic drugs or placebo). Methods Prospectively registered (PROSPERO: CRD42020204772). MEDLINE, EMBASE and Cochrane Register were searched from inception to August 2020. The primary outcomes were reduction in heart rate and proportion of patients achieving study-defined target heart rate. Secondary outcomes included conversion to sinus rhythm and incidence of adverse events. Beta-blockers were divided according to beta-1 adrenoreceptor selectivity. Meta-analysis was performed to calculate risk ratios (RR) and standardised mean differences (SMD) using random-effects, and fixed-effects within beta-1 subgroups. Results From 5974 studies, 12 RCTs were included with variable risk of bias, encompassing 1152 participants with mean age 62 years, 38% women and baseline heart rate 137 beats/minute. With high heterogeneity (I2=87%; p<0.001), there was no difference in the reduction in heart rate between beta-blockers and comparators (SMD −0.65, 95% CI −1.63–0.21; p=0.19), and no difference in the proportion that achieved target heart rate (RR 0.85, 95% CI 0.36–1.97; p=0.70). Analysis by beta-1 selectivity demonstrated that conventional beta-1 blockers (metoprolol, esmolol) were inferior for target heart rate reduction (RR 0.33, 95% CI 0.17–0.64; p=0.001), whereas super-selective agents (landiolol) were superior (RR 1.98, 95% CI 1.54–2.54; p<0.001). There was no difference in the rate of conversion to sinus rhythm between beta-blockers and comparators (RR 1.15, 95% CI 0.92–1.44; p=0.21). Adverse events were similar in both groups overall, with no significant difference in hypotension (RR 1.74, 95% CI 0.85–3.58; p=0.13), bradycardia (RR 0.86, 95% CI 0.19–3.81; p=0.153) or events leading to drug discontinuation (RR 1.16, 95% CI 0.26–5.15; p=0.84). Assessment by beta-1 selectivity showed that hypotension and bradycardia were more frequent with non-selective beta-blockers (sotalol) than comparators (RR 4.57, 95% CI 1.92–10.85; p=0.001 and 5.97, 95% CI 1.34–26.57; p=0.019). Conclusion There is no difference between IV beta-blockers overall and other medications for the control of acute AF/AFl, although better efficacy was shown for beta-blockers with higher beta-1 selectivity. IV beta-blockers were as safe as comparator agents, with the exception of non-selective beta-blockers. FUNDunding Acknowledgement Type of funding sources: Private company. Main funding source(s): Independent systematic review commissioned by Amomed Pharma. Target heart rate and hypotension events


VASA ◽  
2019 ◽  
Vol 48 (2) ◽  
pp. 134-147 ◽  
Author(s):  
Mirko Hirschl ◽  
Michael Kundi

Abstract. Background: In randomized controlled trials (RCTs) direct acting oral anticoagulants (DOACs) showed a superior risk-benefit profile in comparison to vitamin K antagonists (VKAs) for patients with nonvalvular atrial fibrillation. Patients enrolled in such studies do not necessarily reflect the whole target population treated in real-world practice. Materials and methods: By a systematic literature search, 88 studies including 3,351,628 patients providing over 2.9 million patient-years of follow-up were identified. Hazard ratios and event-rates for the main efficacy and safety outcomes were extracted and the results for DOACs and VKAs combined by network meta-analysis. In addition, meta-regression was performed to identify factors responsible for heterogeneity across studies. Results: For stroke and systemic embolism as well as for major bleeding and intracranial bleeding real-world studies gave virtually the same result as RCTs with higher efficacy and lower major bleeding risk (for dabigatran and apixaban) and lower risk of intracranial bleeding (all DOACs) compared to VKAs. Results for gastrointestinal bleeding were consistently better for DOACs and hazard ratios of myocardial infarction were significantly lower in real-world for dabigatran and apixaban compared to RCTs. By a ranking analysis we found that apixaban is the safest anticoagulant drug, while rivaroxaban closely followed by dabigatran are the most efficacious. Risk of bias and heterogeneity was assessed and had little impact on the overall results. Analysis of effect modification could guide the clinical decision as no single DOAC was superior/inferior to the others under all conditions. Conclusions: DOACs were at least as efficacious as VKAs. In terms of safety endpoints, DOACs performed better under real-world conditions than in RCTs. The current real-world data showed that differences in efficacy and safety, despite generally low event rates, exist between DOACs. Knowledge about these differences in performance can contribute to a more personalized medicine.


2018 ◽  
Vol 09 (05) ◽  
pp. 242-243
Author(s):  
Dr. Susanne Krome

Zehntausende nichtproteinkodierende RNAs haben die Kenntnisse über die normale Physiologie sowie die Entstehung und Behandlung von Krankheiten auf den Kopf gestellt, schreibt Prof. Frank Slack, Harvard Medical School, Boston/USA, im New England Journal of Medicine über den überwiegenden Teil unseres Genoms. Diese RNA-Sub typen regulieren Wachstum, Entwicklung und Organfunktion. Ihre Gewebespezifität eröffnet neue, unerwartete Möglichkeiten in der Onkologie. Der größte Teil ihrer Funktionen ist allerdings noch nicht erforscht.


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