Prevention of Fractures in Patients with Parkinson's Disease

帕金森病患者骨折的预防

基本信息

批准号：
10617169
负责人：
STEVEN RON CUMMINGS
金额：
$ 319.24万
依托单位：
CALIFORNIA PACIFIC MED CTR RES INSTITUTE
依托单位国家：
美国
项目类别：
财政年份：
2018
资助国家：
美国
起止时间：
2018-09-30 至 2025-02-28
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10617169
关键词：
Acids Address Age Binding Blinded Bone Density Bone remodeling Calcium Cessation of life Clinic Visits Clinical Communities Data Diagnosis Documentation Enrollment Evaluation Foundations Fracture Guidelines Health system Hip Fractures Home Infusion procedures Institutional Review Boards Intravenous Medical Monitor Movement Disorders Neurologist Nursing Research Oral Osteoporosis Parkinson Disease Patient Care Patients Pharmaceutical Preparations Placebo Control Placebos Prevention Recovery of Function Research Design Risk Risk Reduction Specialist Subgroup Surveys Telemedicine Testing Uncertainty Visit Vitamin D supplementation Woman Zoledronic Acid barrier to care bisphosphonate bone bone loss clinical diagnosis design diaries economic evaluation efficacy testing efficacy trial experience fall risk falls follow-up fracture risk health economics high risk human old age (65+)improved men mortality non-compliance older patient pelvis fracture pharmacologic population based preservation prevent primary endpoint randomized placebo controlled trial randomized trial recruit safety assessment screening secondary endpoint treatment response

项目摘要

Patients with Parkinson's disease (PD) have a very high risk of fracture: about 2 to 3-fold increased risk of all fractures and 4-fold increased risk of hip fracture that may be due in large part to their very high risk of multiple falls. Compared with other patients who suffer fractures, PD patients have more severe consequences of fractures: they have twice the risk of dying following hip and pelvic fractures and longer and incomplete functional recovery, with worsening of Parkinson's disease. Crucially, men and women age 65 or older have a fracture risk that is so high, that even those with normal bone density (BMD) for age would warrant a therapy that has been proven to reduce fracture risk in patients with PD. However, very few PD patients age 65 of older receive a treatment for osteoporosis. There are several major barriers to treatment of PD patients with drugs for osteoporosis. 1) There is a lack of evidence that treatments reduce fracture risk in patients with PD whose risk of fractures may be due to multiple and more severe falls. 2) Standard osteoporosis screening and treatment may also be a barrier. Medical visits to assess fracture risk, test BMD, prescribe treatment, generally an oral drug, and then follow-up to monitor compliance and response to treatment, are particularly burdensome for patients with PD. 3) Most patients with PD do not continue taking oral osteoporosis drugs even for 1 year. We propose a randomized trial that addresses all of these barriers. 1) We will test the anti-fracture efficacy of Zoledronic acid (ZA) a very potent bisphosphonate that inhibits bone loss and decreased fracture risk in women with osteoporosis and patients with hip fractures. 2) We will give ZA intravenously at home without clinic visits. 3) ZA inhibits bone loss for at least 2 years overcoming noncompliance with oral drugs. We will enroll 3,500 men and women PD age 65 years or older from large health systems, patient communities, and referrals from neurologists in the Parkinson's Study Group. A movement disorders specialist will confirm the diagnosis of PD by telemedicine. We will then provide calcium and vitamin D supplements. A research nurse will visit the patient at home, check for potential contraindications to ZA and then administer IV ZA or placebo. We will then follow patients for 2 years to ascertain and confirm fractures. A successful result may revolutionize the care of patients with PD. It may lead to guidelines promoting home-based ZA treatment of older PD patients. ZA, rather than oral drugs, would become the 1st line treatment for patients with PD. The trial would demonstrate that home-based treatment with ZA, without other assessments, could reach all older PD patients without the need for burdensome medical visits. Thus, a positive result may lead to treatment of many more PD patients and a substantial reduction in the occurrence of disabling fractures in patients with PD.

帕金森病 (PD) 患者骨折的风险非常高：所有患者的风险增加约 2 至 3 倍骨折和髋部骨折的风险增加 4 倍，这在很大程度上可能是由于其多发性骨折的风险非常高瀑布。与其他骨折患者相比，PD患者的后果更为严重骨折：髋部和骨盆骨折以及更长且不完全骨折后死亡的风险是其两倍功能恢复，但帕金森病恶化。至关重要的是，65 岁或以上的男性和女性拥有骨折风险如此之高，即使是那些骨密度 (BMD) 正常的人也需要接受治疗已被证明可以降低 PD 患者骨折的风险。然而，很少有 65 岁以上的 PD 患者接受骨质疏松症治疗。主要有几个 PD患者骨质疏松症药物治疗的障碍。 1）缺乏证据表明治疗降低 PD 患者的骨折风险，这些患者的骨折风险可能是由于多次且更严重的跌倒造成的。 2）标准骨质疏松症筛查和治疗也可能是一个障碍。就诊以评估骨折风险，测试 BMD，开出治疗方案，通常是口服药物，然后随访以监测依从性和反应对于 PD 患者来说，治疗尤其是负担。 3) 大多数PD患者不会继续服用口服骨质疏松药连1年。我们提出了一项随机试验来解决所有这些障碍。 1）我们将测试抗骨折功效唑来膦酸 (ZA) 是一种非常有效的双膦酸盐，可抑制骨质流失并降低骨折风险患有骨质疏松症的女性和髋部骨折的患者。 2）我们会在家静脉注射ZA，无需诊所就诊。 3) ZA 可以抑制骨质流失至少 2 年，克服口服药物的不依从性。我们将招募 3,500 名年龄在 65 岁或以上的男性和女性 PD，来自大型卫生系统、患者社区和帕金森病研究组神经科医生的推荐。运动障碍专家将确认通过远程医疗诊断PD。然后我们将提供钙和维生素 D 补充剂。研究护士将拜访患者家中，检查 ZA 的潜在禁忌症，然后静脉注射 ZA 或安慰剂。然后，我们将对患者进行两年的随访，以确定并确认骨折。成功的结果可能会彻底改变帕金森病患者的护理。它可能会导致指导方针促进老年 PD 患者的家庭 ZA 治疗。 ZA，而不是口服药物，将成为一线治疗对于 PD 患者。该试验将证明 ZA 的家庭治疗，无需其他药物评估，可以覆盖所有老年帕金森病患者，而无需进行繁重的医疗就诊。因此，一个阳性结果可能会导致更多帕金森病患者得到治疗并大幅减少帕金森病的发生率 PD 患者致残性骨折的影响。