A Mulit-Centered Prospective Study of Quality of Life in Adult Scoliosis

描述（由申请人提供）：本研究的目的是获得成人症状性腰椎侧凸（ASLS）患者治疗前后的健康相关生活质量（HRQOL）结局数据，以促进循证治疗指南的制定，改善患者护理，优化医疗资源利用。我们将ASLS定义为Oswestry残疾指数（ODI） e20，脊柱侧凸研究协会（SRS）域评分d4.0和基线Cobb测量e300。这项研究将帮助我们确定目前的治疗方法，无论是非手术还是手术，在多大程度上改善了患有这种病理的人的生活。意义：推动这一关键努力的动力是成人脊柱侧凸造成的重大疾病负担，该疾病影响2.5-4%的北美人口。原发性ASLS患者中最具症状的是那些年龄在40到80岁之间并伴有腰椎畸形的患者，这也是我们研究的目标人群。鉴于我国人口老龄化和手术治疗费用高昂（仅住院费用就至少为8万美元），这种疾病构成了一个重大问题。然而，迄今为止的研究通常是有限的、小规模的、单一机构的和回顾性的。治疗对ASLS患者的益处程度尚未得到很好的确定。初步数据：脊柱畸形研究组/成人畸形结局组（SDSG/ADO）成立于7年前，已经开展了多中心的研究，专门研究成人脊柱侧凸，并建立了收集一致的多中心数据的基础设施。SDSG 1)验证了一种针对成人脊柱畸形的疾病特异性结局工具（SRS患者报告的结局工具）；2)开发和验证疾病特异性放射学工具；3)分析某些干预措施的效果，进行治疗对比；4)收集治疗对患者预后影响的初步数据。方法：该研究将包括一项多中心、前瞻性、随机对照试验（RCT）（30%）和连续队列研究（70%），研究对象为接受当前标准治疗的患者，并将使用经我们小组验证的统一临床和放射学评估来评估这些患者。非手术和手术护理将在5个中心内标准化。生活质量将通过SRS-QOL、ODI、SF-12、数字评定量表（NRS）背痛和腿部疼痛评分进行评估。功能测试将包括一项跑步机测试，以评估患者在基线和治疗后2年的速度和耐力。主要目的：衡量非手术和手术治疗对ASLS患者的影响并比较其结果。创新：目前任何同行评议的文献中都没有结果研究证明非手术和手术治疗对原发性成人脊柱畸形的任何方面的影响。公共卫生相关性：这一关键努力的推动力是成人症状性腰椎侧凸（ASLS）带来的重大疾病负担。鉴于我国人口老龄化和手术治疗费用高昂（仅住院费用就至少为8万美元），这种疾病构成了一个日益严重的问题。我们的研究将促进循证治疗指南的制定，改善患者护理，优化医疗资源利用。

DESCRIPTION (provided by applicant): The goal of this patient-oriented study is to obtain health-related quality of life (HRQOL) outcomes data on patients who present with adult symptomatic lumbar scoliosis (ASLS) pre- and post-treatment in order to facilitate the development of evidence-based treatment guidelines, improve patient care and optimize healthcare resource utilization. We are defining ASLS as Oswestry Disability Index (ODI) e20, Scoliosis Research Society (SRS) domain scores d4.0 and a Cobb measurement of e300 at baseline. This study will help us determine how much the current treatment, either nonoperative or operative, improves the lives of people with this pathology. Significance: The impetus for this critical effort is the significant burden of disease posed by adult scoliosis, which affects 2.5-4% of the North American population. The most symptomatic group of primary presentation ASLS patients are those between the ages of 40 and 80 with lumbar deformities - our target population for this study. This disease constitutes a significant problem in light of our aging population and the high cost of surgical treatment (at least $80,000 in hospital costs alone). Studies to date, however, have typically been limited, small, single institution and retrospective. The extent to which treatment benefits patients with ASLS is not well established. Preliminary data: The Spinal Deformity Study Group/Adult Deformity Outcomes section (SDSG/ADO), which was formed 7 years ago, has developed a multi-center effort specifically to study adult scoliosis and has established an infrastructure for collecting consistent multicenter data. The SDSG has 1) validated a disease-specific outcomes tool (the SRS patient-reported outcomes instrument) for adult spinal deformity; 2) developed and validated disease-specific radiographic tools; 3) analyzed outcomes for certain interventions for comparison of treatments; and 4) collected preliminary data regarding the impact of treatments on patient outcomes. Methods: The study will comprise a multicenter, prospective, randomized controlled trial (RCT) (30%) and consecutive cohort study (70%) of patients receiving current standard-of-care treatments and will use uniform clinical and radiographic assessments that have been validated by our group for assessment of these patients. Both nonoperative and operative care will be standardized within the 5 centers. Quality of life will be assessed by SRS-QOL, ODI, SF-12, numerical rating scale (NRS) back pain and leg pain scores. Functional measures will include a walking treadmill test to assess speed and endurance in patients at baseline and 2 years post-treatment. Primary Aims: Measure the impacts and compare the outcomes of nonoperative and operative treatments in ASLS patients. Innovation: No outcomes study exists in any current peer-reviewed literature to document the effect of both nonoperative and operative treatment on any aspect of primary presentation adult spinal deformity. PUBLIC HEALTH RELEVANCE: The impetus for this critical effort is the significant burden of disease posed by adult symptomatic lumbar scoliosis (ASLS). This disease constitutes a growing problem in light of our aging population and the high cost of surgical treatment (at least $80,000 in hospital costs alone). Our study will facilitate the development of evidence-based treatment guidelines, improve patient care, and optimize healthcare resource utilization.