统计问题第127问：生存分析中的截尾时间概念

Question

Researchers assessed the effectiveness of low dose high frequency ultrasound when combined with standard care in healing venous leg ulcers that had previously been hard to heal. Ultrasound was given for 12 weeks, after which participants returned to standard care alone. A multicentre randomised controlled trial was performed. Control treatment was standard care.

Trial participants were recruited if they had at least one venous leg ulcer of greater than six months’ duration or greater than 5 cm² in area, and an ankle brachial pressure index greater than 0.8. In total, 168 people were randomised to ultrasound and standard care, with 169 allocated to standard care alone. Each trial participant was treated for a total of 12 months. Outcome measures included the time from randomisation until complete healing of all leg ulcers and recurrence of ulcers within the study period of 12 months. No statistically significant difference existed between treatment groups in the time until complete healing of all ulcers or ulcer recurrence.

Which of the following, if any, describe a trial participant with a censored observation?

·a) A person with complete healing of ulcers within 12 months of randomisation.

·b) Someone whose ulcers had not completely healed within the study period.

·c) A trial participant who withdrew from the study before his or her ulcers had completely healed.

·d) Someone with complete healing of ulcers but who subsequently withdrew from the study within 12 months of starting treatment.

提示：正确答案不止一个。

Answer

Answers b and c describe a trial participant with a censored observation.

The length of time from randomisation until complete healing of all ulcers is referred to as time to event data or survival data. The term survival data can be misleading because the end point does not have to be death or an adverse event. The end point may be positive, such as the complete healing of ulcers, as in the above study. The researchers reported complete healing of all ulcers within the 12 month study period for 72 participants in the ultrasound group and 78 in the standard care group. Because the length of time until complete healing of all ulcers for these people is known, their survival times are referred to as exact (a is false). Some of the trial participants did not experience complete healing of all ulcers during the trial. Exactly how long it took for these people to reach the end point, if at all, is not known. Their survival times are called right censored or simply censored. All survival times, whether exact or censored, are included in the subsequent statistical analysis because it is important to make use of all available data.

For people who were observed for the entire 12 month study period and whose ulcers had not completely healed when the study concluded, their survival times would be censored at 12 months (b is true). Complete healing of ulcers would have occurred after 12 months for these trial participants, if at all. People may have left the trial because they withdrew their consent to participate or were lost to follow-up after moving away. If any of these people left the trial before complete healing of their ulcers, their survival time would have been censored at the time they were last observed (c is true). The survival time of anybody who withdrew from the trial after complete healing of ulcers would be recorded as exact (d is false). However, for any such person it would obviously not have been possible to record recurrence of ulcers.

所以答案是选择  b c

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