Stephens R J, Langley R E, Mulvenna P, Nankivell M, Vail A, Parmar M K B
MRC Clinical Trials Unit, Aviation House, 125 Kingsway, London WC2B 6NH, United Kingdom.
MRC Clinical Trials Unit, Aviation House, 125 Kingsway, London WC2B 6NH, United Kingdom.
Lung Cancer. 2014 Aug;85(2):116-8. doi: 10.1016/j.lungcan.2014.05.012. Epub 2014 May 22.
Guidelines for the conduct of clinical trials emphasize the importance of keeping the interim results from the main endpoints confidential, in order to maintain the integrity of the trial and to safeguard patients' interests. However, is this essential in every situation?
We review the evidence for these guidelines and consider recent randomised trials that have released interim results, to assess their impact on the success of the trial. However, because the strength of opinion to keep interim results confidential is so strong, there are limited examples of such trials.
In the QUARTZ trial (which is assessing the value of whole brain radiotherapy in patients with brain metastases from non-small cell lung cancer) the decision to release interim results was taken in response to threatened closure due to poor accrual, whereas in the GRIT trial (which compared two obstetric strategies for the delivery of growth retarded pre-term fetuses) the regular release of interim results was pre-planned. Nevertheless there are a number of common factors between these two trials. In particular, the trial treatments were already in wide use, with no reliable randomised evidence on which treatment should be used for which patients, and there was diverse clinical opinion, which meant that accrual was likely to be challenging. In a situation where a quarter to a third of trials do not accrue their required number of patients, the QUARTZ trial continues to accrue patients, and the GRIT trial successfully accrued its target of nearly 600 babies.
This article therefore argues that there is a need to re-consider whether it is always essential to keep the interim results of randomized clinical trials confidential, and suggests some criteria that may help groups planning or running challenging trials decide whether releasing interim results would be a useful strategy.
临床试验实施指南强调对主要终点的中期结果保密的重要性，以维持试验的完整性并保护患者利益。然而，在每种情况下这都是必不可少的吗？
我们回顾了这些指南的证据，并考量了近期公布中期结果的随机试验，以评估其对试验成功的影响。然而，由于对中期结果保密的观点非常强烈，此类试验的例子有限。
在QUARTZ试验（评估全脑放疗对非小细胞肺癌脑转移患者的价值）中，公布中期结果的决定是因入组不佳可能导致试验提前结束而做出的；而在GRIT试验（比较两种针对生长受限早产胎儿的产科策略）中，定期公布中期结果是预先计划好的。尽管如此，这两项试验有一些共同因素。特别是，试验中的治疗方法已被广泛使用，但对于哪些患者应使用哪种治疗方法，尚无可靠的随机证据，而且临床观点多样，这意味着入组可能具有挑战性。在四分之一到三分之一的试验无法招募到所需患者数量的情况下，QUARTZ试验继续招募患者，GRIT试验成功招募到了近600名婴儿的目标数量。
因此，本文认为有必要重新考虑对随机临床试验的中期结果保密是否总是必不可少的，并提出一些标准，可能有助于计划或开展具有挑战性试验的团队决定公布中期结果是否是一种有用的策略。
