Segel G B, Simon W, Lichtman M A
Blood. 1986 Nov;68(5):1055-64.
The prognosis for patients with chronic myelogenous leukemia (CML) has improved only for patients who can receive marrow transplantation from a histocompatible sibling. The timing of the marrow transplant is made difficult by the high peritransplant mortality of 20% to 35% and a group of patients with a prolonged chronic phase of CML, which can be identified on the basis of prognostic indexes (age, percent blood myeloblasts, spleen size, and platelet count). We have developed a mathematic model and computer program that consider age, prognostic index, and projected survival rate by transplantation to balance the risk of peritransplant mortality against the risk of delaying the transplantation of patients with Philadelphia chromosome-positive CML. The computation assesses the risk of delaying transplantation; it does not offer the option of avoiding transplantation, since long-term survival ultimately requires transplantation. Three prognostic groups were considered as described by Sokal and co-workers (Blood 63:789, 1984) (I, best; II, intermediate; III, worst prognosis). The computation used the projected survival rates of transplantation from the Seattle experience and from the International Bone Marrow Transplant Registry. As an example of the model's utility, we have determined the ratio of the calculated life expectancy to the normal life expectancy for hypothetical patients up to 50 years of age in each of the three prognostic categories. A value of 20% is used for patients who successfully receive transplants after the onset of the accelerated phase. The analysis allows assessment of the risk of delaying transplantation for a finite time in patients with CML. The importance of the method rests in its consideration of multiple variables, including the peritransplant mortality, transplant projected survival before and upon entering the accelerated phase, age, prognostic group, and other risk factors. The program permits a change in these parameters as new information or advances in treatment occur. This analysis does not replace the diagnostic deliberations of the clinician. Rather, it provides a numeric framework for prognosis based on the currently available data. The physician in conjunction with the patient, not the algorithm, makes the decisions of whether and when to transplant.
慢性粒细胞白血病（CML）患者的预后仅在能够接受来自组织相容性同胞的骨髓移植的患者中有所改善。骨髓移植的时机因移植围手术期高达20%至35%的死亡率以及一组慢性期延长的CML患者而变得困难，这组患者可根据预后指标（年龄、血中原始粒细胞百分比、脾脏大小和血小板计数）来识别。我们开发了一个数学模型和计算机程序，该模型和程序考虑年龄、预后指标以及移植后的预计生存率，以平衡移植围手术期死亡风险与延迟费城染色体阳性CML患者移植的风险。该计算评估延迟移植的风险；它不提供避免移植的选择，因为长期生存最终需要移植。按照索卡尔及其同事所描述的（《血液》63:789，1984年），考虑了三个预后组（I组，预后最佳；II组，中等；III组，预后最差）。该计算使用了来自西雅图经验以及国际骨髓移植登记处的移植预计生存率。作为该模型实用性的一个例子，我们确定了三个预后类别中每一类50岁及以下假设患者的计算预期寿命与正常预期寿命的比率。对于加速期开始后成功接受移植的患者，使用20%的值。该分析允许评估CML患者在有限时间内延迟移植的风险。该方法的重要性在于它考虑了多个变量，包括移植围手术期死亡率、进入加速期之前和之时的移植预计生存率、年龄、预后组以及其他风险因素。随着新信息或治疗进展的出现，该程序允许改变这些参数。这种分析并不能取代临床医生的诊断思考。相反，它基于当前可用数据提供了一个预后的数值框架。医生与患者一起，而不是算法，做出是否以及何时进行移植的决定。
