Current status and challenges of drug development for hormonal treatment of endometriosis: a systematic review of randomized control trials

Abstract

Objective

The aim of this systematic review is to summarize the data obtained from randomized controlled trials looking at new pharmacologic treatments for endometriosis published over the last decade with a focus on hormonal therapeutic options for endometriosis-associated pelvic pain (EAPP), excluding studies focusing on fertility.

Methods

We identified relevant original studies in the English language through a search of the MEDLINE, Scopus, and EMBASE (2012 to present) databases using the appropriate MeSH terms and applying the article type filter ‘randomized controlled trials’. A total of 219 records were found during the electronic search. After a detailed evaluation and review of the manuscripts, 11 primary articles met the inclusion criteria. A systematic review of the data was conducted.

Results

This review included several emerging drug therapies for EAPP. Randomized control trials showed promising results with several oral gonadotropin-releasing hormone antagonists (elagolix, relugolix, ASP1707, linzagolix). However, studies of other hormonal agents such as aromatase inhibitors and selective progesterone receptor modulators have not yielded significant or new advantages. Selective estrogen receptor modulators have not been represented in randomized control trials and have failed to demonstrate clinical efficacy.

Conclusion

Although numerous novel agents are being investigated for the treatment of endometriosis, there is still no significant progress in the development of curative rather than suppressive drugs. Therefore, further efforts are needed to develop an effective and hopefully curative treatment for this chronic, costly, and overwhelming disease.

摘要

目的

本系统评价的目的是总结过去十年发表的随机对照试验的数据, 以寻找子宫内膜异位症的新药物治疗, 重点是子宫内膜异位症相关盆腔疼痛（EAPP）的激素疗法的选择, 不包括以生育为重点的研究。

方法

相关的英语原始研究是通过检索MEDLINE, Scopus和EMBASE（2012年至今）数据库, 使用适当的医学主题术语并应用文章类型过滤器“随机对照试验”来确定的。在电子检索过程中共发现219条记录。通过对稿件的详细评估和审查, 11篇主要文章符合收录标准。对数据进行了系统审查。

结果

本综述包括几种治疗EAPP的新兴药物疗法。随机对照试验显示, 几种口服促性腺激素释放激素拮抗剂（恶拉戈利、瑞卢戈利、ASP1707、林扎戈利）具有良好的疗效。然而, 对其他激素药物如芳香化酶抑制剂和选择性孕激素受体调节剂的研究并没有取得显著或新的优势。选择性雌激素受体调节剂尚未出现在随机对照试验中, 也未能证明其临床疗效。

结论

尽管许多新型药物正在被研究用于治疗子宫内膜异位症, 但在开发治疗性药物而非抑制性药物方面仍没有重大进展。因此, 需要进一步努力, 为这一慢性、昂贵和压倒性的疾病制定有效且有望治愈的治疗方法。

Keywords:

• endometriosis
• hormonal therapy
• pelvic pain
• GnRH antagonists
• SPRMs
• SERMs

关键词:

• 子宫内膜异位症
• 激素治疗
• 盆腔疼痛
• 促性腺激素释放激素拮抗剂
• 选择性孕激素受体调节剂
• 选择性雌激素受体调节剂

Disclosure statement

No potential conflict of interest was reported by the authors.

Funding

The author(s) reported there is no funding associated with the work featured in this article.