Roose Steven P, Rutherford Bret R, Wall Melanie M, Thase Michael E
Steven P. Roose, MD, Bret R. Rutherford, MD, Melanie M. Wall, PhD, Columbia University College of Physicians and Surgeons, New York State Psychiatric Institute, New York, USA; Michael E. Thase, Departments of Psychiatry, Perelman School of Medicine of the University of Pennsylvania, Philadelphia Veterans Affairs Medical Center and University of Pittsburgh Medical Center, USA
Steven P. Roose, MD, Bret R. Rutherford, MD, Melanie M. Wall, PhD, Columbia University College of Physicians and Surgeons, New York State Psychiatric Institute, New York, USA; Michael E. Thase, Departments of Psychiatry, Perelman School of Medicine of the University of Pennsylvania, Philadelphia Veterans Affairs Medical Center and University of Pittsburgh Medical Center, USA.
Br J Psychiatry. 2016 May;208(5):416-20. doi: 10.1192/bjp.bp.115.163261.
The number needed to treat (NNT) statistic was developed to facilitate the practice of evidence-based medicine. Placebo was assumed to be therapeutically inert when the NNT was originally conceived, but more recent data for conditions such as major depressive disorder (MDD) suggest that the placebo control condition can have considerable therapeutic effects. Complications arise because the NNT calculated from randomised controlled trials (RCTs) reflects a comparison between medication plus clinical management and placebo plus clinical management, whereas, in the clinical setting, physicians choose between prescribing open medication, observing a patient over time with a supportive approach, and doing nothing. Thus, NNTs derived from clinical trials are not directly relevant to clinical decision-making, because they are based on control conditions that do not exist in standard practice. Additional difficulties may arise when using NNTs to compare alternative treatments for MDD, such as medication and psychotherapy, since these comparisons require the control conditions upon which the respective NNTs are based to be similar.Whereas pill placebo conditions include intensive clinical management and elicit expectations of improvement, attention control conditions for psychotherapy research are less well developed. Often the effects of psychotherapy are gauged against a wait-list control condition, which has substantially fewer therapeutic components than a pill placebo control condition. To improve the clinical utility of NNTs for the treatment of MDD, we advocate effectiveness studies that include treatment conditions resembling actual clinical practice, rather than using placebo-controlled RCTs for this purpose. Until such studies are performed, the effect of bias in comparing NNTs across treatments can be controlled by ensuring that the RCT control conditions upon which the NNTs are based are comparable.
需治疗人数(NNT)统计方法的提出是为了促进循证医学的实践。最初构想NNT时,安慰剂被假定为无治疗活性,但最近针对重度抑郁症(MDD)等病症的数据表明,安慰剂对照条件可能具有相当大的治疗效果。问题在于,随机对照试验(RCT)计算得出的NNT反映的是药物加临床管理与安慰剂加临床管理之间的比较,而在临床环境中,医生要在开具开放药物、采用支持性方法长期观察患者以及不采取任何措施之间做出选择。因此,从临床试验得出的NNT与临床决策并无直接关联,因为它们基于标准医疗实践中不存在的对照条件。在使用NNT比较MDD的替代治疗方法(如药物治疗和心理治疗)时,可能会出现更多困难,因为这些比较要求各自NNT所基于的对照条件相似。虽然药丸安慰剂条件包括强化临床管理并引发改善的期望,但心理治疗研究中的注意力对照条件发展得并不完善。心理治疗的效果通常是与等待名单对照条件进行比较,而该条件的治疗成分比药丸安慰剂对照条件少得多。为提高NNT在MDD治疗中的临床实用性,我们提倡开展有效性研究,纳入类似于实际临床实践的治疗条件,而不是为此使用安慰剂对照的RCT。在进行此类研究之前,通过确保NNT所基于的RCT对照条件具有可比性,可以控制跨治疗比较NNT时的偏差影响。