Brady Urological Institute, Johns Hopkins Medical Institutions, Baltimore, MD, USA.
J Clin Oncol. 2011 Feb 1;29(4):464-7. doi: 10.1200/JCO.2010.30.6373. Epub 2010 Dec 28.
The European Randomized Study of Screening for Prostate Cancer (ERSPC) reported a 20% mortality reduction with prostate-specific antigen (PSA) screening. However, they estimated a number needed to screen (NNS) of 1,410 and a number needed to treat (NNT) of 48 to prevent one prostate cancer death at 9 years. Although NNS and NNT are useful statistics to assess the benefits and harms of an intervention, in a survival study setting such as the ERSPC, NNS and NNT are time specific, and reporting values at one time point may lead to misinterpretation of results. Our objective was to re-examine the effect of varying follow-up times on NNS and NNT using data extrapolated from the ERSPC report.
On the basis of published ERSPC data, we modeled the cumulative hazard function using a piecewise exponential model, assuming a constant hazard of 0.0002 for the screening and control groups for years 1 to 7 of the trial and different constant rates of 0.00062 and 0.00102 for the screening and control groups, respectively, for years 8 to 12. Annualized cancer detection and drop-out rates were also approximated based on the observed number of individuals at risk in published ERSPC data.
According to our model, the NNS and NNT at 9 years were 1,254 and 43, respectively. Subsequently, NNS decreased from 837 at year 10 to 503 at year 12, and NNT decreased from 29 to 18.
Despite the seemingly simplistic nature of estimating NNT, there is widespread misunderstanding of its pitfalls. With additional follow-up in the ERSPC, if the mortality difference continues to grow, the NNT to save a life with PSA screening will decrease.
欧洲前列腺癌筛查随机研究(ERSPC)报告称,前列腺特异性抗原(PSA)筛查可降低 20%的死亡率。然而,他们估计,需要筛查的人数(NNS)为 1410 人,需要治疗的人数(NNT)为 48 人,才能在 9 年内预防一例前列腺癌死亡。尽管 NNS 和 NNT 是评估干预措施益处和危害的有用统计数据,但在 ERSPC 这样的生存研究环境中,NNS 和 NNT 是特定于时间的,报告一个时间点的值可能会导致对结果的误解。我们的目的是使用从 ERSPC 报告中推断的数据重新检查不同随访时间对 NNS 和 NNT 的影响。
根据已发表的 ERSPC 数据,我们使用分段指数模型模拟累积危险函数,假设筛查组和对照组在试验的第 1 年至第 7 年的危险率为 0.0002,而筛查组和对照组在第 8 年至第 12 年的危险率分别为 0.00062 和 0.00102。根据已发表的 ERSPC 数据中观察到的风险人数,还近似估算了每年的癌症检出率和脱落率。
根据我们的模型,9 年时的 NNS 和 NNT 分别为 1254 和 43。随后,NNS 从第 10 年的 837 降至第 12 年的 503,NNT 从第 29 年降至第 18 年。
尽管估计 NNT 的性质看似简单,但人们对其陷阱仍存在广泛的误解。随着 ERSPC 的进一步随访,如果死亡率差异继续增大,PSA 筛查挽救生命的 NNT 将降低。