SHAM-CONTROLLED TRIAL OF PERCUTANEOUS VERTEBROPLASTY

描述(由申请人提供)：本研究方案的目标是改善患有疼痛的骨质疏松性椎体压缩骨折患者的生活质量。这一目标将通过检查经皮椎体成形术治疗疼痛的骨质疏松性椎体压缩骨折的临床疗效来实现。每年有超过70万名患者发生自发性、非创伤性压缩骨折。与这些骨折相关的疼痛可能是痛苦的，但在经皮椎体成形术出现之前，治疗选择有限。到目前为止，止痛药、卧床休息和支具是主要的治疗方法，但每种治疗方法都有很大的局限性。由于缺乏治疗骨质疏松性椎体压缩骨折的可行方法，经皮椎体成形术(即向骨折的椎体内注射聚甲基丙烯酸甲酯(PMMA)或医用水泥)的实践已迅速普及。然而，经皮椎体成形术有效性的证据目前仅限于非对照、非盲目、小病例系列。尽管报告的成功率很高，高达90%的患者获得了实质性的疼痛缓解，但这些病例系列并没有解释许多重要的潜在偏见。这些偏差包括压缩性骨折自然愈合的倾向，回归平均值(患者在疼痛最严重时寻求医疗护理)，以及安慰剂效应。此外，以前的研究还没有使用专门针对椎体成形术的有效健康结果量表，也没有试图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，以排除混淆变量，如安慰剂效应，以此来解释椎体成形术的明显疗效。此外，我们将验证一种新的、椎体成形术专用的健康结局仪器。最后，我们将确定经皮椎体成形术的成本-效果。试验的成功实施将为经皮椎体成形术的真正疗效提供令人信服的证据，为治疗患有疼痛的骨质疏松性压缩骨折的患者提供合理的决策依据。

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.