Design of a pragmatic approach to evaluate the effectiveness of concurrent treatment for the prevention of osteoporotic fractures
Design of a pragmatic approach to evaluate the effectiveness of concurrent treatment for the prevention of osteoporotic fractures
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DOI：
10.1007/s00774-010-0188-x
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发表时间：
2011-01-01
影响因子：
3.3
通讯作者：
Orimo, Hajime
中科院分区：
文献类型：
--
作者：
Shiraki, Masataka;Kuroda, Tatsuhiko;Orimo, Hajime
骨质疏松症治疗的目的是预防未来的骨折。虽然在临床实践中，骨质疏松症经常采用并行治疗，但在骨质疏松症患者中，并行治疗预防骨折的准确性和有效性尚无数据证实。为了明确并发治疗的临床有效性，日本骨质疏松学会授权成立A-TOP（骨质疏松症的充分治疗）研究组。本研究的目的是建立一项临床试验设计，以证明在预防骨折方面，阿法骨化醇（1- α -羟基胆骨化醇）和阿仑膦酸钠联合治疗是否比单独使用阿仑膦酸钠更有效。目前的研究被命名为JOINT（日本骨质疏松症干预试验），基于一种全国性、前瞻性、随机、开放标记、盲法终点的方法，关注绝经后骨质疏松症的高风险骨折。患者主要由执业医师选择，采用中央登记系统随机分为两组，一组单独给予阿仑膦酸钠5mg /d，另一组在阿仑膦酸钠基础上给予1- α -羟基胆骨化醇（阿法骨化醇）1mu g/d。终点主要关注骨折预防，其次评估患者的生活质量（QOL）和身高变化，以及治疗的依从性和不良事件。为了在2年观察期的事件中获得足够的统计效力，我们选择了预期风险较高的患者参与本研究，并决定最终方案为每组890例患者（双侧α = 0.05，功率= 0.8）。数据收集始于2003年11月。从公共卫生研究基金会到从业人员通过网络系统进行了有关研究者注册和研究进展的通信。
The aim of osteoporosis treatment is to prevent future fractures. Although concurrent treatment has been used very frequently for osteoporosis in clinical practice, there are no data on accurate and verified effectiveness of concurrent treatment for fracture prevention in patients with osteoporosis. To clarify the clinical usefulness of concurrent treatment, the Japan Osteoporosis Society has authorized the establishment of the A-TOP (Adequate Treatment of Osteoporosis) research group. The objective of this research is to establish a design for a clinical trial to prove whether concurrent treatment using both alfacalcidol (1-alpha-hydroxycholecalciferol) and alendronate is more effective as compared to treatment using alendronate alone in terms of fracture prevention. The present study was named JOINT (Japanese Osteoporosis Intervention Trial) and is based on a method using national, prospective, randomized, open-labeled, blinded endpoints focusing on postmenopausal osteoporosis with a high risk for fracture. The patients were mainly selected by practitioners and allocated randomly by a central registration system into two groups, of which one received 5 mg/day of alendronate alone, and the other received 1 mu g/day of 1-alpha-hydroxycholecalciferol (alfacalcidol) in addition to the alendronate. The endpoints focused primarily on fracture prevention, and the patients' quality of life (QOL) and change in body height, as well as adherence and the adverse events of the treatments were evaluated secondarily. To obtain sufficient statistical power in the events during a 2-year observation period, the patients who are expected to have higher risk were selected to participate in this study, and it was decided that the final plan would involve 890 patients per group (two-sided alpha = 0.05, power = 0.8). Data collection began in November 2003. Correspondence regarding the registration of the investigator and the progress of the study was conducted through a web system from the Public Health Research Foundation to practitioners.