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肢端肥大症的治疗方法:系统评价和网络荟萃分析。

Medical Treatments for Acromegaly: A Systematic Review and Network Meta-Analysis.

机构信息

Graduate Program in Pharmaceutical Sciences, Universidade Federal do Paraná, Curitiba, Paraná, Brazil.

Department of Social Pharmacy, Faculty of Pharmacy, Research Institute for Medicines (iMed.ULisboa), Universidade de Lisboa, Lisbon, Portugal.

出版信息

Value Health. 2018 Jul;21(7):874-880. doi: 10.1016/j.jval.2017.12.014. Epub 2018 Feb 8.

DOI:10.1016/j.jval.2017.12.014
PMID:30005760
Abstract

BACKGROUND

Acromegaly results from the hypersecretion of growth hormone. Because of the low incidence rates of this disease worldwide, few clinical trials evaluating drug treatments have been conducted.

OBJECTIVES

To conduct the first network meta-analysis simultaneously comparing all available drugs used in acromegaly treatment so as to provide more robust evidence in this field.

METHODS

A systematic review was performed according to Preferred Reporting Items for Systematic Reviews and Meta-Analyses and Cochrane Collaboration recommendations (PROSPERO database under the registration number CRD42017059880). The electronic searches were conducted in PubMed (MEDLINE), Scopus, and Web of Science databases. Randomized controlled trials comparing any drug for the treatment of acromegaly head-to-head or versus placebo were included. Outcomes concerning the efficacy and safety of treatments were evaluated. The statistical analyses were performed using Aggregate Data Drug Information System version 1.16.8 (drugis.org, Groningen, The Netherlands).

RESULTS

The initial search retrieved 2059 articles. Of these, 10 randomized controlled trials were included in a qualitative analysis and 7 in a quantitative analysis. The network meta-analysis for the efficacy outcome (number of patients achieving insulinlike growth factor 1 control) showed that pegvisomant and lanreotide autogel were statistically superior to placebo (odds ratio [95% credible interval] 0.06 [0.00-0.55] and 0.09 [0.01-0.88]). No further differences were found. The probability rank indicated that pegvisomant and pasireotide have the highest probabilities (33% and 34%, respectively) of being the best therapeutic options. No major side effects were noted.

CONCLUSIONS

Pegvisomant is still a good option for acromegaly treatment, but pasireotide seems to be a promising alternative. Nevertheless, other important key factors such as drug costs and effectiveness (real-world results) should be taken into account when selecting acromegaly treatment.

摘要

背景

肢端肥大症是由生长激素过度分泌引起的。由于这种疾病在全球的发病率较低,因此很少有评估药物治疗的临床试验。

目的

进行首次网络荟萃分析,同时比较肢端肥大症治疗中所有可用的药物,以便为该领域提供更有力的证据。

方法

根据系统评价和荟萃分析首选报告项目以及 Cochrane 协作组织的建议(PROSPERO 数据库,注册号 CRD42017059880)进行系统评价。电子检索在 PubMed(MEDLINE)、Scopus 和 Web of Science 数据库中进行。纳入比较任何药物治疗肢端肥大症的头对头或与安慰剂对照的随机对照试验。评估治疗效果和安全性的结局。使用 Aggregate Data Drug Information System 版本 1.16.8(drugis.org,荷兰格罗宁根)进行统计分析。

结果

最初的搜索检索到 2059 篇文章。其中,10 项随机对照试验纳入定性分析,7 项纳入定量分析。疗效结局(达到胰岛素样生长因子 1 控制的患者人数)的网络荟萃分析显示,培维索孟和兰瑞肽自动凝胶在统计学上优于安慰剂(比值比[95%可信区间]0.06[0.00-0.55]和 0.09[0.01-0.88])。没有发现进一步的差异。概率排名表明,培维索孟和帕瑞肽具有最高的可能性(分别为 33%和 34%)成为最佳治疗选择。没有发现主要的副作用。

结论

培维索孟仍然是肢端肥大症治疗的一个较好选择,但帕瑞肽似乎是一个有前途的替代选择。然而,在选择肢端肥大症治疗时,还应考虑药物成本和有效性(实际结果)等其他重要关键因素。

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Cureus. 2024 Dec 11;16(12):e75553. doi: 10.7759/cureus.75553. eCollection 2024 Dec.
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