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雄激素类药物在遗传性血管性水肿治疗中的应用:并不像想象中那么便宜。

Anabolic androgen use in the management of hereditary angioedema: Not so cheap after all.

机构信息

Department of Allergy, Kaiser Permanente Medical Center, San Diego, California.

Division of Rheumatology, Allergy, and Immunology, Department of Medicine, University of California at San Diego, La Jolla, California.

出版信息

Ann Allergy Asthma Immunol. 2017 Apr;118(4):456-460.e1. doi: 10.1016/j.anai.2017.01.015. Epub 2017 Mar 7.

DOI:10.1016/j.anai.2017.01.015
PMID:28283277
Abstract

BACKGROUND

Hereditary angioedema due to C1 inhibitor deficiency (HAE) is a rare, life-threatening disease that imposes a significant burden on affected patients. 17α-alkylated androgens (anabolic androgens) decrease attack frequency and severity but carry the risk of potentially serious dose-related adverse effects. Despite the emergence of targeted therapies for HAE, continued anabolic androgen use has been driven in part by their low cost.

OBJECTIVE

To examine the hidden cost of anabolic androgen use related to the risk of developing non-HAE comorbidities.

METHODS

Patients with HAE were identified in the Southern California Kaiser Permanente database using clinical and laboratory findings compatible with HAE. These patients were stratified into anabolic androgen exposed and nonexposed groups. Matched controls were selected from the Kaiser database who did not have HAE or anabolic androgen exposure. Using multivariate analysis, we determined the number of non-HAE comorbidities linked to anabolic androgen use. We next determined the association between dosing and increasing exposure to anabolic androgens and the likelihood of having various comorbidities.

RESULTS

Patients with HAE exposed to anabolic androgens had a 28% increase (P = .04) in non-HAE comorbidities when compared with their matched (nonexposed) controls. With each gram per month increase in exposure, a 12% increase in non-HAE comorbidities is observed (P < .01). The most commonly occurring non-HAE comorbidities were psychiatric, muscle cramps, obesity, and hyperlipidemia.

CONCLUSION

Our data suggest that long-term anabolic androgen use enhances the risk of developing comorbid health conditions, thus amplifying the cost of care. Our report provides additional support for the preferred use of newer, targeted therapies for the management of HAE.

摘要

背景

C1 抑制剂缺乏引起的遗传性血管性水肿(HAE)是一种罕见的、危及生命的疾病,给患者带来了巨大的负担。17α- 烷基化雄激素(同化雄激素)可降低发作频率和严重程度,但存在潜在严重剂量相关不良反应的风险。尽管针对 HAE 的靶向治疗已经出现,但同化雄激素的持续使用部分是由于其成本低。

目的

研究与非 HAE 合并症风险相关的同化雄激素使用的隐性成本。

方法

使用与 HAE 相符的临床和实验室发现,在南加州 Kaiser Permanente 数据库中确定 HAE 患者。将这些患者分为同化雄激素暴露和非暴露组。从 Kaiser 数据库中选择未患有 HAE 或同化雄激素暴露的匹配对照者。通过多变量分析,我们确定了与同化雄激素使用相关的非 HAE 合并症的数量。我们接下来确定了剂量和暴露于同化雄激素增加与各种合并症发生的可能性之间的关系。

结果

与匹配的(未暴露)对照者相比,暴露于同化雄激素的 HAE 患者非 HAE 合并症增加了 28%(P =.04)。暴露量每月增加 1 克,非 HAE 合并症增加 12%(P <.01)。最常见的非 HAE 合并症是精神疾病、肌肉痉挛、肥胖和血脂异常。

结论

我们的数据表明,长期使用同化雄激素会增加发生合并健康状况的风险,从而增加医疗费用。我们的报告为使用新型靶向治疗来管理 HAE 提供了更多支持。

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