Department of Anesthesiology, Freiburg University Medical Center, Freiburg, Germany
Department of Nuclear Medicine, Odense University Hospital, Odense, Denmark.
J Nucl Med. 2014 Aug;55(8):1228-34. doi: 10.2967/jnumed.113.127076. Epub 2014 Jun 9.
Since the influential study by van Tinteren et al. published in The Lancet in 2002, there have been an increasing number of diagnostic randomized controlled trials (RCTs) investigating the benefit of PET. If they provide valid and useful information on the benefit, these studies can play an important role in informing guideline developers and policy makers. Our aim was to investigate how far the nuclear medicine community has come on its way from accuracy studies to RCTs and which issues we have to take into account in planning future studies.
We conducted a systematic review of diagnostic randomized trials, in which PET was applied in only one arm. We covered published studies as well as registered unpublished and planned studies. We considered 3 quality indicators related to the usefulness of a trial to generate evidence for a clinical benefit: use of patient-important outcome, sufficient sample size, and current standard as comparator.
Fourteen published and 15 planned studies were identified. Five of the published studies and 12 of the planned studies did not use a patient-important outcome. Sample sizes were often so small that a significant result could be expected only under the assumption of a substantial reduction in the event rate. Comparators typically reflected the current standard.
If we consider the traditional areas of primary diagnosis, staging, and follow-up, then the number and quality of RCTs on PET is currently not sufficient to provide a major source for evidence-based decisions on the clinical benefit of PET. There will also be a future need in these traditional areas to deduce the clinical benefit of PET from the results of accuracy studies. The situation may be more favorable for the areas of treatment planning and response evaluation. Choice of patient-important outcomes and sufficient sample sizes are crucial issues in planning RCTs to demonstrate the clinical benefit of using PET.
自 2002 年 van Tinteren 等人在《柳叶刀》上发表的有影响力的研究以来，越来越多的诊断性随机对照试验（RCT）研究了 PET 的益处。如果这些研究能提供关于益处的有效和有用的信息，那么它们可以在为指南制定者和决策者提供信息方面发挥重要作用。我们的目的是调查核医学领域从准确性研究到 RCT 的进展情况，以及在规划未来研究时我们必须考虑哪些问题。
我们对仅在一个臂中应用 PET 的诊断性 RCT 进行了系统回顾。我们涵盖了已发表的研究以及已注册的未发表和计划中的研究。我们考虑了 3 个与试验产生临床益处证据的有用性相关的质量指标：使用患者重要结局、足够的样本量和当前标准作为对照。
确定了 14 项已发表和 15 项计划中的研究。在已发表的研究中，有 5 项和计划中的研究中，有 12 项没有使用患者重要结局。样本量通常很小，只有在假定事件率大幅降低的情况下，才有可能得出显著的结果。对照通常反映了当前的标准。
如果我们考虑原发性诊断、分期和随访的传统领域，那么目前关于 PET 的 RCT 的数量和质量不足以提供基于证据的关于 PET 临床益处的决策的主要来源。在这些传统领域中，未来还需要从准确性研究的结果中推断出 PET 的临床益处。在治疗计划和反应评估领域，情况可能更为有利。选择患者重要结局和足够的样本量是规划 RCT 以证明使用 PET 的临床益处的关键问题。
