Klein John P, Shu Youyi
Division of Biostatistics, Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee, WI 53226, USA.
Stat Methods Med Res. 2002 Apr;11(2):117-39. doi: 10.1191/0962280202sm277ra.
High-dose chemotherapy followed by stem cell recovery, more commonly called a bone marrow transplant, is a common treatment for a number of diseases. This article examines four problems commonly encountered when dealing with bone marrow transplant studies. First, we look at the problem of competing causes of failure and at methods based on a multi-state model to estimate meaningful probabilities for these risks. Second, we examine methods for estimating the prevalence of an intermediate condition, here the prevalence of chronic GVHD. Third, we look at the problem of modeling the post transplant recovery process and we provide two examples of how these estimates can be used to assess dynamically a patient's prognosis or how these probabilities can be used to design trials of new therapy. Finally, we present an estimate of a new measure of treatment efficiency, the current leukemia free survival function, which is derived from a multi-state model approach.
大剂量化疗后进行干细胞恢复，更常见的说法是骨髓移植，是多种疾病的常见治疗方法。本文探讨了处理骨髓移植研究时通常遇到的四个问题。首先，我们研究失败的竞争原因问题以及基于多状态模型来估计这些风险的有意义概率的方法。其次，我们研究估计中间状态患病率的方法，这里指慢性移植物抗宿主病的患病率。第三，我们研究移植后恢复过程的建模问题，并提供两个例子，说明这些估计如何用于动态评估患者的预后，或者这些概率如何用于设计新疗法的试验。最后，我们给出一种新的治疗效率衡量指标——当前无白血病生存函数的估计值，它源自多状态模型方法。
