Phenotyping Responses to Optimize the Management of Pain Treatment in Endometriosis

项目总结/摘要 子宫内膜异位症是一种使人衰弱的进行性疾病，影响10%的育龄妇女。 子宫内膜异位症的特征是子宫外存在子宫内膜病变， 慢性盆腔疼痛、出血和不孕，疼痛是患者寻求的最令人衰弱的症状。 在乎子宫内膜异位症造成巨大的经济和医疗负担，并极大地影响生活质量。 子宫内膜异位症临床实践指南建议进行六个月的保守治疗， 抑制局部或全身雌激素水平;然而，疼痛缓解程度存在实质性差异 从药物治疗中获得并且一些妇女随后需要手术切除病变， 提示疾病和症状表现的异质性可能是治疗反应的基础。此外，本发明还 预测治疗失败和/或成功的机制特征不明确，从而使提供者 处于长期症状管理的位置，并且对于可能实现的治疗几乎没有指导， 最大的功效。量化这种异质性和识别治疗反应特征可能是 这是子宫内膜异位症个体化治疗方法的关键一步，以减少整体疼痛负担。在 根据精准医学倡议，这项前瞻性观察性研究的目的是探索 接受基于指南的保守治疗的患者中与治疗反应相关的机制 子宫内膜异位症的治疗，并描述表型特征与盆腔疼痛严重程度之间的相关性 和疼痛影响轨迹年龄在18至45岁之间的绝经前女性，临床疑似子宫内膜异位症 将招募并在接受保守治疗后随访6个月， 美国妇产科学院临床实践指南。异黄酮前 治疗子宫内膜异位症的症状表现将通过以下措施进行量化：（1） 社会人口统计学，（2）临床，（3）生物医学，（4）疼痛质量，（5）行为，（6）心理和（7） 功能性健康患者将每月随访6个月，以评估盆腔疼痛的严重程度和疼痛影响。 总的来说，这些目标有可能促进我们对表型机制的理解， 子宫内膜异位症妇女的保守治疗反应，可能作为基础的影响， 减少不必要和无效的医疗，缩短正确诊断的时间， 治疗

PROJECT SUMMARY/ABSTRACT Endometriosis is a debilitating and progressive disease that affects 10% of reproductive-aged women. Characterized by the presence of endometrial lesions outside of the uterus, endometriosis is associated with chronic pelvic pain, bleeding, and infertility, with pain being the most debilitating symptom for which patients seek care. Endometriosis results in tremendous economic and healthcare burden and greatly impacts quality of life. Clinical practice guidelines for endometriosis recommend six months of conservative medical treatment for suppression of local or systemic estrogen levels; however, substantial variation exists in the degree of pain relief obtained from pharmacological therapy and some women subsequently require surgical removal of lesions, suggesting that heterogeneity in disease and symptom presentation may underlie treatment response. Further, the mechanisms that predict treatment failure and/or success are poorly characterized, thereby leaving providers in a position of long-term symptom management and with little guidance regarding therapies that may achieve the greatest efficacy. Quantifying this heterogeneity and identifying treatment response characteristics may be a crucial step toward individualizing therapeutic approaches for endometriosis to reduce overall pain burden. In line with the Precision Medicine Initiative, the aims of this prospective observational study are to explore mechanisms associated with treatment response in patients undergoing guideline-based conservative medical therapy for endometriosis, and to characterize associations between phenotypic profiles with pelvic pain severity and pain impact trajectories. Premenopausal women ages 18 to 45 years with clinically suspected endometriosis will be recruited and followed for a period of six months after undergoing conservative treatment according to the American College of Obstetricians and Gynecologists clinical practice guidelines. Heterogeneity in pre- treatment endometriosis symptom presentation will be quantified across the following measures: (1) sociodemographic, (2) clinical, (3) biomedical, (4) pain quality, (5) behavioral, (6) psychological, and (7) functional health. Patients will be followed monthly for six months to assess pelvic pain severity and pain impact. Collectively, these aims have the potential to advance our understanding of phenotypic mechanisms underlying conservative treatment response among women with endometriosis, effects which may serve as a foundation for reducing unnecessary and ineffective medical treatments and lessening the time to proper diagnosis and treatment.