RANDOMIZED CLINICAL-TRIALS IN SINGLE PATIENTS DURING A 2-YEAR PERIOD
RANDOMIZED CLINICAL-TRIALS IN SINGLE PATIENTS DURING A 2-YEAR PERIOD
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DOI：
10.1001/jama.270.22.2708
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发表时间：
1993-12-08
影响因子：
120.7
通讯作者：
OAS, J
中科院分区：
文献类型：
--
作者：
LARSON, EB;ELLSWORTH, AJ;OAS, J
客观。-描述单个患者试验（SPT）服务的可行性，并研究正式SPT对治疗精度的影响。SPT的描述性和评价性研究。所有计划的试验均为双盲、随机、多交叉试验。个体试验的其他关键特征是随机分配顺序和评估预先确定的明确结果。患者和医生在视觉模拟量表上评估SPT前后治疗的置信水平。两年的经验（1988年9月至1990年9月）的SPT试验转诊服务提供给医生在学术医疗中心。计划和完成的SPT的数量;完成的试验产生明确答案的比例;患者和医生对治疗前和治疗后SPT的信心水平;时间运动研究，以估计选定的SPT的资源消耗（成本）。在34项已完成的试验中，17项被判定为给出了明确的结果，而17项仅显示了趋势。在16项试验中，结果支持积极治疗，导致治疗继续（9例患者）或开始（7例患者）。根据18项试验结果，停止治疗（7例患者）或未开始治疗（11例患者），这些结果表明积极治疗无效（7例），有害（2例）或明显等效（9例）。大多数患者（65%）报告说，试验后他们对治疗的信心水平没有变化，而医生对治疗的信心水平在SPT后根据试验结果的方向增加或减少。患者一致认为SPT服务非常有用。时间运动估计表明，每次审判花费16.75个工作人员小时，导致每次审判的直接成本估计约为450至500美元。我们的结论是，一个SPT服务是可行的，试验成本与其他传统的服务相比，毫不逊色，临床医生似乎从SPT获得信心和精度。当患者或临床医生不确定症状性慢性病治疗的价值（包括副作用的可能性）时，我们相信可以向患者提供SPT，并且可能会产生影响后续治疗的结果。
Objective.-To describe the feasibility of a single patient trial (SPT) service and study the influence of formal SPTs on therapeutic precision.Design.-Descriptive and evaluative study of SPTs. All planned trials were double-blind, randomized, multiple crossover trials. Other key features of individual trials were random assignment of order and assessment of predetermined explicit outcomes. Patients and physicians rated level of confidence in treatment before and after the SPT on visual analog scales.Setting.-Two-year experience (September 1988 to September 1990) of an SPT trial referral service available to physicians in an academic medical center.Outcome Measures.-The number of planned and completed SPTs; proportion of completed trials yielding definitive answers; patient- and physician-rated levels of confidence in treatment pre- and post-SPT; time-motion studies to estimate resource consumption (costs) for selected SPTs.Results.-Of 34 completed trials, 17 were judged to give definitive results whereas 17 showed trends only. Results favored active treatment in 16 trials that led to treatment being continued (nine patients) or started (seven patients). Treatment was discontinued (seven patients) or not started (11 patients) based on 18 trial results that demonstrated active treatment was ineffective (seven), harmful (two), or apparently equivalent (nine). Most patients (65%) reported no change in their already high level of confidence in therapy as a result of trials, whereas physicians' confidence levels in therapy either increased or decreased post-SPT depending on the direction of trial results. Patients consistently rated the SPT service as extremely useful. Time-motion estimates indicate that 16.75 staff hours were spent per trial leading to a direct cost estimate of approximately $450 to $500 per trial.Conclusion.-We conclude that an SPT service is feasible, trial costs compare favorably with other conventional services, and clinicians appear to gain confidence and precision from SPTs. When patients or clinicians are uncertain about the value (including the possibility of side effects) of treatment for symptomatic chronic diseases, we believe an SPT can be offered to a patient and will likely yield results that will effect subsequent treatment.