Bearman S I, Anderson G L, Mori M, Hinds M S, Shulman H M, McDonald G B
Division of Clinical Research, Fred Hutchinson Cancer Research Center, Seattle, WA.
J Clin Oncol. 1993 Sep;11(9):1729-36. doi: 10.1200/JCO.1993.11.9.1729.
Hepatic venoocclusive disease (VOD) is a common complication of cytoreductive therapy for marrow transplantation. Only 25% of patients who develop VOD have severe disease. We tested the hypothesis that early clinical signs of VOD would predict which patients would recover and which would die.
We evaluated 355 consecutive patients who had transplants between August 6, 1987 and July 21, 1988 for occurrence of VOD and whether it was reversible within 100 days of transplant. Total serum bilirubin and weight gain from day -7 through day +16 posttransplant were compared among patients with no, severe, or nonsevere VOD. Logistic regression models were developed to estimate probabilities of severe VOD at each of six time intervals. The accuracy of these models was tested by applying them to 392 consecutive patients who underwent transplantation between July 22, 1988 and July 20, 1989.
As early as day -1, bilirubin and weight gain were significantly different between patients whose VOD proved to be severe and patients with reversible VOD or no disease. Regression models were used to generate coefficients (beta 0, beta 1, beta 2) for the equation P = 1/(1 + e-z), where P is the probability of severe VOD and z = beta 0 + beta 1 (In total serum bilirubin [mg/dL]) + beta 2 (percent weight gain). Application of this equation to the next 392 patients allowed us to calculate sensitivity, specificity, and positive predictive value for a range of probabilities.
The course of VOD after cytoreductive therapy can be predicted by knowing the serum bilirubin and weight gained within 1 to 2 weeks of transplantation. Probability estimates derived from patient data are highly specific and moderately sensitive. Such probability estimates may be useful when considering potentially risky interventions to treat VOD, such as recombinant human tissue plasminogen activator.
肝静脉闭塞病（VOD）是骨髓移植细胞减灭疗法的常见并发症。发生VOD的患者中只有25%患有严重疾病。我们检验了这样一个假设，即VOD的早期临床体征可以预测哪些患者会康复，哪些患者会死亡。
我们评估了1987年8月6日至1988年7月21日期间连续进行移植的355例患者，观察VOD的发生情况以及在移植后100天内是否可逆。比较了无VOD、严重VOD或非严重VOD患者从移植前第7天到移植后第16天的总血清胆红素和体重增加情况。建立了逻辑回归模型，以估计六个时间间隔中每个间隔发生严重VOD的概率。通过将这些模型应用于1988年7月22日至1989年7月20日期间连续进行移植的392例患者，检验了这些模型的准确性。
早在移植前第1天，VOD被证明为严重的患者与可逆性VOD或无疾病的患者之间，胆红素和体重增加就有显著差异。回归模型用于生成方程P = 1/(1 + e-z)的系数（β0、β1、β2），其中P是严重VOD的概率，z = β0 + β1（血清总胆红素[mg/dL]的自然对数）+ β2（体重增加百分比）。将该方程应用于接下来的392例患者，使我们能够计算一系列概率的敏感性、特异性和阳性预测值。
通过了解移植后1至2周内的血清胆红素和体重增加情况，可以预测细胞减灭疗法后VOD的病程。从患者数据得出的概率估计具有高度特异性和中等敏感性。在考虑治疗VOD的潜在风险干预措施（如重组人组织纤溶酶原激活剂）时，这种概率估计可能会有所帮助。
