Pilot Randomized Trial of Antibiotics vs Surgery for Treating Acute Appendicitis

描述(申请人提供)：几乎十分之一的人患有阑尾炎，这是一种肠道的一小部分称为阑尾的疾病，变得发炎和疼痛。通常的治疗方法是住院、全身麻醉和手术切除阑尾。人们需要大约两周的时间才能恢复。阑尾炎的病因尚不完全清楚。一种解释是，阑尾变得阻塞，导致扩张和不可逆转的血流不足，因此需要手术切除才能治愈。另一种解释是，阑尾炎是由感染引起的，这表明抗生素也可能有效。之前的非美国研究表明，许多患者可以用抗生素代替阑尾切除术进行治疗，多达70%的患者避免了手术，通常痛苦更少，恢复更快。然而，由于这些研究涉及的患者太少，而且没有使用理想的方法，医生目前没有提供这种选择。我们的总目标是进行第一次 美国进行了一项大型试验，以确定抗生素疗法是否是手术的安全有效的替代方案。 我们的主要目标是证明抗生素治疗产生的患者结果不比阑尾切除术更差。这一目标将通过进行一项大型试验来实现，在试验中，阑尾炎患者被随机分配接受抗生素或手术，并检验这样一种假设，即最初接受抗生素治疗的患者在30天内不会比接受阑尾切除术的患者出现更多重大并发症。次要目标将是在两年的时间内将这些结果与其他以患者为中心的结果进行比较，如疼痛持续时间和恢复正常活动的时间。我们还将进行研究，进一步探索阑尾炎的原因，包括研究患者基因，以确定谁有患阑尾炎的风险，检查阑尾炎组织的微生物学，以及测试血液化学物质，称为生物标志物，这可能有助于确定哪些患者最有可能对抗生素产生反应。在全面试验之前，我们建议对30名受试者进行试点调查，以证明进行全面试验的可行性。这将是一项关键的临床试验，将增加患者的选择，为没有准备好获得手术护理的服务不足的人提供一个安全的选择，同时总体上提高患者的健康、满意度和生产力，并促进我们对疾病的科学理解。

DESCRIPTION (provided by applicant): Almost one in ten people experience appendicitis, a disease in which a small part of the intestine called the appendix becomes inflamed and painful. The usual treatment is hospitalization and general anesthesia and surgery to remove the appendix. It takes people about two weeks to recover. The cause of appendicitis is not entirely clear. One explanation is that the appendix becomes obstructed leading to distention and irreversible lack of blood flow, thus requiring surgical removal to cure. Another explanation is that appendicitis is due to infection, and this suggests that antibiotics may also be effective. Previous non-U.S. studies indicate that many patients can be treated with antibiotics instead of appendectomy, with as many as 70% avoiding surgery and generally experiencing less pain and faster recovery. However, because these studies involved too few patients and did not use ideal methods, doctors do not currently offer this option. Our overall goal is to conduct the first large U.S. trial to determine if antibiotic therapy is a safe and effective alternative to surgery. Our primary aim is to demonstrate that antibiotic treatment produces patient outcomes that are no worse than appendectomy. This aim will be accomplished by conducting a large trial in which patients with appendicitis are assigned by chance to get antibiotics or surgery and testing the hypothesis that those treated initially with antibiotics will experience no more major complications at 30 days than those treated with appendectomy. Secondary aims will be to compare these and other patient-centered outcomes, like duration of pain and time to return to normal activities, over a two-year period. We will also conduct studies to further explore the cause of appendicitis including looking at patient genes to determine who is at risk of the disease, examining the microbiology of appendicitis tissue, and testing blood chemicals, called biomarkers, that may help determine which patients are most likely to respond to antibiotics. Prior to the full- scale trial, we propose a pilot investigation of 30 subjects to demonstrate the feasibility of conducting the full- scale trial. This will be a pivotal clinical trial that will inrease patient choice and provide a safe option for the underserved who do not have ready access to surgical care, while generally enhancing patient health, satisfaction, and productivity, and advancing our scientific understanding of the disease.