SHAM-CONTROLLED TRIAL OF PERCUTANEOUS VERTEBROPLASTY

描述（由申请方提供）：本研究方案的目的是改善疼痛性、退行性椎体压缩性骨折患者的生活质量。通过检查经皮椎体成形术治疗疼痛性椎体压缩性骨折的临床疗效，将达到这一目的。每年有超过70万例患者发生自发性非创伤性压缩性骨折。与这些骨折相关的疼痛可能是难以忍受的，但在经皮椎体成形术出现之前，治疗选择有限。止痛药物、卧床休息和支具迄今为止一直是治疗的主要手段，但这些治疗方法都有很大的局限性。由于缺乏可行的治疗方案，椎体压缩性骨折，经皮椎体成形术的做法，其中包括注射聚甲基丙烯酸甲酯（PMMA），或医疗水泥，到骨折的椎骨，已迅速传播。然而，经皮椎体成形术的有效性证据目前仅限于非对照、非盲、小病例系列。尽管报告的成功率很高，高达90%的患者实现了显著的疼痛缓解，但这些病例系列并未考虑到许多重要的潜在偏倚。这些偏差包括压缩性骨折自然愈合的趋势，向平均值的回归（其中患者在疼痛最严重时寻求医疗护理）和安慰剂效应。此外，以前的研究没有使用经过验证的椎体成形术的健康结局量表，也没有尝试确定该手术的成本效益。我们将进行一项经皮椎体成形术的假对照前瞻性试验，以排除混杂变量，如安慰剂效应，作为椎体成形术明显疗效的解释。此外，我们将验证一种新的、针对椎体成形术的健康结果工具。最后，我们将确定经皮椎体成形术的成本效益。该试验的成功实施将为经皮椎体成形术的真实疗效提供令人信服的证据，从而为治疗疼痛性椎体压缩性骨折患者提供合理的决策。

DESCRIPTION (provided by applicant): The objective of this research protocol is to improve the quality of life for patients suffering from painful, osteoporotic vertebral compression fractures. This objective will be met by examining the clinical efficacy of percutaneous vertebroplasty for treatment of painful, osteoporotic vertebral compression fractures. Spontaneous, atraumatic compression fractures occur in more than 700,000 patients per year. Pain associated with these fractures may be excruciating, but treatment options, before the advent of percutaneous vertebroplasty, were limited. Analgesic medications, bedrest, and bracing have heretofore represented the mainstay of treatment, but each of these therapies has substantial limitations. Because of the dearth of viable treatment options for osteoporotic vertebral compression fractures, the practice of percutaneous vertebroplasty, which involves injection of polymethylmethacrylate (PMMA), or medical cement, into the fractured vertebra, has disseminated rapidly. However, evidence for the efficacy of percutaneous vertebroplasty currently is limited to uncontrolled, non-blinded, small case series. Even though high rates of success are reported, with up to 90% of patients achieving substantial pain relief, these case series have not accounted for numerous important potential biases. These biases include the natural tendency for compression fractures to heal spontaneously, regression toward the mean (wherein patients seek medical attention when pain is maximum), and the placebo effect. Also, previous studies have not used validated health outcomes scales specific for vertebroplasty, and no attempt has been made to determine the cost effectiveness of the procedure. We will perform a sham-controlled, prospective trial of percutaneous vertebroplasty in order to exclude confounding variables, such as the placebo effect, as the explanation for the apparent efficacy of vertebroplasty. Furthermore, we will validate a new, vertebroplasty-specific health outcomes instrument. Last, we will determine the cost-effectiveness of percutaneous vertebroplasty. Successful implementation of the trial will provide compelling evidence regarding the true efficacy of percutaneous vertebroplasty, to allow rationale decision-making in caring for patients suffering from painful, osteoporotic compression fractures.