Wierenga K J, Hambleton I R, Lewis N A
Sickle Cell Unit, Tropical Medicine Research Institute, University of the West Indies, Mona, Kingston, Jamaica.
Lancet. 2001 Mar 3;357(9257):680-3. doi: 10.1016/s0140-6736(00)04132-5.
Information about life expectancy of patients with homozygous sickle-cell disease is needed for research and patient counselling. Our aim was to study two Jamaican populations, one clinic-based and one birth cohort and, by careful consideration of data quality and statistical analysis, to identify ways to increase the chances of obtaining valid and generalisable results.
We investigated the survival experience of 3301 patients with homozygous sickle-cell disease attending the Jamaican sickle-cell clinic between Jan 1, 1987, and Dec 31, 1996. We applied and assessed a simulation technique for incorporating early life mortality using a birth cohort, and analysed the precision of this technique. Kaplan-Meier survival estimates are produced.
290 of the 3301 patients died. Median survival calculated with the excess mortality rate simulation data was 53 years (95% CI 49.3-57.0) for men and 58.5 (55.1-67.5) for women.
Our simulation technique, with realistic assumptions based on empirical evidence, offers a new estimate of median survival for patients with homozygous sickle-cell disease. We present the precision of these survival estimates, which introduces an important level of uncertainty. The inherent biases of clinically ascertained populations of patients, and the assumptions underlying analysis techniques are crucial features of survival studies in sickle-cell disease, and can modify summary statistics substantially.
对于研究和患者咨询而言，需要有关纯合子镰状细胞病患者预期寿命的信息。我们的目的是研究两个牙买加人群，一个是基于诊所的人群，另一个是出生队列人群，并通过仔细考虑数据质量和统计分析，确定增加获得有效且可推广结果可能性的方法。
我们调查了1987年1月1日至1996年12月31日期间在牙买加镰状细胞病诊所就诊的3301例纯合子镰状细胞病患者的生存经历。我们应用并评估了一种使用出生队列纳入早期死亡率的模拟技术，并分析了该技术的精确性。生成了Kaplan-Meier生存估计值。
3301例患者中有290例死亡。根据超额死亡率模拟数据计算，男性的中位生存期为53岁（95%可信区间49.3 - 57.0），女性为58.5岁（55.1 - 67.5）。
我们基于经验证据做出实际假设的模拟技术，为纯合子镰状细胞病患者的中位生存期提供了新的估计值。我们展示了这些生存估计值的精确性，但这也带来了重要程度的不确定性。临床确定的患者群体的固有偏差以及分析技术所依据的假设，是镰状细胞病生存研究的关键特征，并且可能会显著改变汇总统计数据。
