Comparing focused ultrasound and uterine artery embolization for uterine fibroids

描述(由申请人提供)：本申请涉及挑战领域#15：翻译科学和特定挑战主题，15-HD-102：骨盆疼痛。我们建议描述子宫肌瘤患者在基线和微创治疗后的痛经和非周期性盆腔疼痛的特征。我们的意见书还涉及挑战领域#5：比较有效性研究和特定挑战主题，05-EB-103非侵入性超声技术的比较有效性。我们提出了一项随机临床试验，比较了子宫肌瘤两种微创治疗方法的症状缓解情况：子宫动脉栓塞术(UAE)和MRI引导的聚焦超声(MRgFUS)。子宫肌瘤(肌瘤或肌瘤)在四分之一的女性和高达75%的非裔美国女性中被诊断出。每年可归因于肌瘤的直接医疗费用超过21亿美元1。然而，我们的工作报告称，对于患有临床显著子宫肌瘤的女性来说，间接成本(残疾和旷工)相当于直接成本(11,720美元对11,752美元/女性/年)2。与控制人口和病例组合因素的对照女性相比，患有症状性子宫肌瘤的女性的间接成本显著增加(11,752美元对8,083美元，p&lt；0.0001)2。因此，了解和解决临床显著子宫肌瘤的症状，尤其是疼痛，对于从社会角度优化治疗结果和控制医疗成本非常重要。尽管肌瘤的发病率很高，但对其症状的了解仍不完全。据报道，肌瘤会引起一系列症状，包括大量或长时间的月经出血、盆腔压迫、月经痛(痛经)以及肠道和膀胱功能障碍。然而，肌瘤疼痛从来没有用评估和量化疼痛的标准工具麦吉尔疼痛问卷(MPQ)3进行研究。而且，大多数肌瘤研究没有区分月经期疼痛和非周期性盆腔疼痛。一篇对子宫肌瘤的专家综述表明，非周期性盆腔疼痛的存在应促使对其他疾病过程的研究4。然而，对子宫动脉栓塞术(UAE)后3年结果的研究表明，83%的女性盆腔疼痛有长期改善5。这项研究还将跟踪使用FDA批准的两种肌瘤微创治疗技术子宫动脉栓塞术(UAE)和磁共振引导聚焦超声手术(MRgFUS)治疗后盆腔疼痛和其他症状结果的变化。UAE和MRgFUS在治疗方法、作用机制和副作用方面存在显著差异，而且它们从未在随机临床试验中进行过比较。阿联酋采用血管造影术阻断子宫动脉作为导致缺血性坏死的子宫治疗方法。在UAE后，女性在术后即刻似乎有更多的疼痛，但研究表明，在长期的随访中，盆腔疼痛有所减轻。相反，MRgFUS靶向于单个肌瘤的间质，并产生热融性凝固性坏死。这种专门针对肌瘤的方法术后疼痛较少，允许门诊治疗，但不全面，可能导致长期症状改善较少。因此，我们提出了PAM试验：肌瘤疼痛评估，这是一项随机临床试验，用UAE或MRgFUS治疗220名患有症状性子宫肌瘤的不同种族的女性，并对她们进行至少6个月的随访。这一筹资机制将涵盖每个主题从基线到6个月的费用，我们正在积极寻求公私伙伴关系，以便进行长期的后续行动。通过梅奥诊所和杜克大学的招聘，将实现不同种族的学科队列。 公共卫生相关性：子宫肌瘤或子宫肌瘤是育龄妇女，尤其是非裔美国妇女的一种极其常见的疾病。子宫肌瘤是子宫切除术的主要原因，但它们引起的盆腔疼痛程度尚未明确记录。这项研究将描述患有子宫肌瘤的妇女在月经期间和月经其他时间的盆腔疼痛程度，以及子宫动脉栓塞术(UAE)和磁共振引导的聚焦超声(MRgFUS)这两种治疗肌瘤的微创疗法是否能减轻疼痛。

DESCRIPTION (provided by applicant): This application addresses Challenge Area #15:Translational Science and Specific Challenge Topic, 15-HD-102: Pelvic Pain. We propose to characterize the pain with menses and noncyclic pelvic pain in women with uterine leiomyomas at baseline and following minimally-invasive treatment. Our submission also addresses Challenge Area #5: Comparative Effectiveness Research and Specific Challenge Topic, 05- EB-103 Comparative Effectiveness of Non-Invasive Ultrasound Techniques. We propose a randomized clinical trial comparing symptomatic relief following two minimally invasive treatments for uterine leiomyomas: uterine artery embolization (UAE) and MRI guided focused ultrasound (MRgFUS). Uterine leiomyomas (fibroids or myomas) are diagnosed in one quarter of all women and up to 75 % of African-American women. The direct health care costs attributable to leiomyomas exceed $2.1 billion annually1. However, our work reported that indirect costs (disability and absenteeism) are equivalent to direct costs for women with clinically significant fibroids ($11,720 vs. $11,752/woman/year) 2. This is a significant increase in indirect costs for women with symptomatic fibroids compared to control women when controlled for demographic and case mix factors ($11,752 vs. $8083, p<0.0001)2. Thus, understanding and addressing the symptoms of clinically-significant uterine fibroids and particularly pain is important in optimizing treatment outcomes and controlling health care costs from a societal perspective. Despite their prevalence, fibroid symptomatology is still incompletely understood. Myomas are reported to cause a number of symptoms including heavy or prolonged menstrual bleeding, pelvic pressure, pain with menses (dysmenorrhea) and bowel and bladder dysfunction. However leiomyoma pain has never been studied with the standard instrument for assessing and quantitating pain, the McGill Pain Questionnaire (MPQ) 3. Moreover, most leiomyoma studies have not differentiated between pain with menses and non cyclic pelvic pain. One expert review of leiomyomas suggests the presence of noncyclic pelvic pain should prompt investigation of other disease processes 4. Yet studies of 3 year outcomes following uterine artery embolization treatment (UAE) suggest that 83% of women have long term improvement in pelvic pain 5. This study will also track changes in pelvic pain and other symptomatic outcomes following treatment with two minimally-invasive FDA-approved techniques for leiomyoma treatment uterine artery embolization (UAE) and MRI-guided focused ultrasound surgery (MRgFUS). UAE and MRgFUS differ significantly in approach, mechanism of action and side effects and they have never been compared in a randomized clinical trial. UAE employs angiographic occlusion of the uterine arteries as a global uterine treatment leading to ischemic necrosis. Following UAE, women appear to have more pain in the immediate postoperative period, but studies document decreased pelvic pain during long term follow-up. In contrast, MRgFUS targets the interstitium of individual fibroids and produces thermoablative coagulative necrosis. This myoma-specific approach causes less postoperative pain and allows for outpatient treatment but is less comprehensive and may lead to less long term symptomatic improvement. We therefore propose the PAM Trial: Pain Assessment for Myomas, a randomized clinical trial treating 220 racially-diverse women with symptomatic uterine leiomyomas with UAE or MRgFUS and following them for a minimum of 6 months. This funding mechanism will cover costs from baseline to 6 months for each subject and we are actively pursuing public/private partnerships to allow long term follow up. A racially diverse cohort of subjects will be achieved by recruiting through Mayo Clinic and Duke University. PUBLIC HEALTH RELEVANCE: Uterine leiomyomas or fibroids are an extremely common disease in reproductive age women and particularly African-American women. Fibroids are the leading cause of hysterectomy, yet the amount of pelvic pain they cause has not been clearly documented. This study will characterize the amount of pelvic pain women with fibroids have with their menstrual periods and at other times in the month and whether pain is decreased by uterine artery embolization (UAE) and magnetic resonance guided focused ultrasound (MRgFUS), two minimally-invasive treatments for fibroids