Watts Nelson B
Bone Health and Osteoporosis Center, University of Cincinnati College of Medicine, 222 Piedmont Avenue, Suite 4300, Cincinnati, OH 45219, USA.
Curr Rheumatol Rep. 2004 Feb;6(1):79-84. doi: 10.1007/s11926-004-0087-z.
Because there are now effective agents for treatment of osteoporosis, the question is being raised as to whether or not it is ethical to have placebo-controlled trials of new agents. It is ethical for patients who are at low risk of serious or irreversible harm to participate in placebo-controlled trials as long as they provide informed consent. Morbidity, mortality, and future fracture risk correlate with the presence of previous fractures, the number of previous fractures, whether or not the fracture is recent, and whether or not the fracture is clinically recognized. Lower-risk subjects who may be allowed to participate in placebo-controlled trials include those with low bone density but without a previous vertebral fracture, those with a single vertebral deformity that was not clinically recognized, and those with a vertebral fracture more than 2 years before. Higher-risk subjects who do not tolerate proven drugs or who have not responded to proven drugs may also participate. Even though it may be ethical for selected subjects to participate in placebo-controlled trials of new therapies for osteoporosis, steps should be taken to minimize their exposure (eg, unbalanced randomization, integration of outcomes, and powering trials to actual events rather than a projected number over 3 years), and treating patients who fracture or who fail to respond.
由于目前已有治疗骨质疏松症的有效药物，因此对于新型药物进行安慰剂对照试验是否符合伦理道德这一问题正被提出来。对于那些面临严重或不可逆伤害风险较低的患者，只要他们提供知情同意，参与安慰剂对照试验就是符合伦理道德的。发病率、死亡率和未来骨折风险与既往骨折的存在、既往骨折的数量、骨折是否为近期发生以及骨折是否在临床上被识别相关。可能被允许参与安慰剂对照试验的低风险受试者包括骨密度低但无既往椎体骨折的患者、有单个未被临床识别的椎体畸形的患者以及骨折超过2年的患者。不能耐受已证实药物或对已证实药物无反应的高风险受试者也可参与。尽管对于选定的受试者参与骨质疏松症新疗法的安慰剂对照试验可能符合伦理道德，但应采取措施尽量减少他们的暴露（例如，不均衡随机分组、整合结果以及根据实际事件而非预计的3年数量进行试验效能分析），并对发生骨折或无反应的患者进行治疗。
