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Abstract：
OBJECTIVE: Patients with Waldenström macroglobulinemia (WM) have disparate outcomes. Newer therapies have emerged since the development of International Prognostic Scoring System, and MYD88L265P mutation is now frequently assessed at diagnosis, warranting reexamination of the prognostic parameters.
METHODS: We reviewed records of 889 treatment-naïve patients with active WM, consecutively seen between January 01, 1996, and December 31, 2017, to identify clinical predictors of overall survival (OS) in univariate analyses. Patients with complete data for the parameters significant on the univariate analyses (n = 341) were included in a multivariable analysis to derive a prognostic model, subsequently validated in a multi-institutional cohort.
RESULTS: In the derivation cohort (n = 341), age (hazard ratio [HR], 1.9 [95% CI, 1.2 to 2.1]; P = .0009), serum lactate dehydrogenase (LDH) above upper limit of normal (HR, 2.3 [95% CI, 1.3 to 4.5]; P = .007), and serum albumin <3.5 g/dL (HR, 1.5 [95% CI, 0.99 to 2.3]; P = .056) were independently prognostic. By assigning a score of 1 point each to albumin <3.5 g/dL (HR, 1.5) and age 66-75 years (HR 1.4) and 2 points for age >75 years (HR, 2.6) or elevated LDH (HR, 2.3), four groups with distinct outcomes were observed on the basis of the composite scores. Five-year OS was 93% for the low-risk (score 0), 82% for low-intermediate risk (score 1), 69% for intermediate-risk (score 2), and 55% for the high-risk (score ≥3; P < .0001) groups. In the validation cohort (N = 335), the model maintained its prognostic value, with a 5-year OS of 93%, 90%, 75%, and 57% for the four groups, respectively (P < .0001).
CONCLUSIONS: Modified Staging System for WM (MSS-WM), utilizing age, albumin, and LDH is a simple, clinically useful, and externally validated prognostic model that reliably risk-stratifies patients with symptomatic WM into four groups with distinct prognosis.
METHODS: We reviewed records of 889 treatment-naïve patients with active WM, consecutively seen between January 01, 1996, and December 31, 2017, to identify clinical predictors of overall survival (OS) in univariate analyses. Patients with complete data for the parameters significant on the univariate analyses (n = 341) were included in a multivariable analysis to derive a prognostic model, subsequently validated in a multi-institutional cohort.
RESULTS: In the derivation cohort (n = 341), age (hazard ratio [HR], 1.9 [95% CI, 1.2 to 2.1]; P = .0009), serum lactate dehydrogenase (LDH) above upper limit of normal (HR, 2.3 [95% CI, 1.3 to 4.5]; P = .007), and serum albumin <3.5 g/dL (HR, 1.5 [95% CI, 0.99 to 2.3]; P = .056) were independently prognostic. By assigning a score of 1 point each to albumin <3.5 g/dL (HR, 1.5) and age 66-75 years (HR 1.4) and 2 points for age >75 years (HR, 2.6) or elevated LDH (HR, 2.3), four groups with distinct outcomes were observed on the basis of the composite scores. Five-year OS was 93% for the low-risk (score 0), 82% for low-intermediate risk (score 1), 69% for intermediate-risk (score 2), and 55% for the high-risk (score ≥3; P < .0001) groups. In the validation cohort (N = 335), the model maintained its prognostic value, with a 5-year OS of 93%, 90%, 75%, and 57% for the four groups, respectively (P < .0001).
CONCLUSIONS: Modified Staging System for WM (MSS-WM), utilizing age, albumin, and LDH is a simple, clinically useful, and externally validated prognostic model that reliably risk-stratifies patients with symptomatic WM into four groups with distinct prognosis.
摘要：
目的：Waldenström巨球蛋白血症（WM）患者的结局不同。自国际预后评分系统发展以来，出现了较新的疗法，MYD88L265P突变现在经常在诊断时评估，保证对预后参数进行重新检查。
方法：我们回顾了889例未经治疗的活动性WM患者的记录，在1996年1月1日至2017年12月31日期间连续观察，以在单变量分析中确定总生存期（OS）的临床预测因子。在多变量分析中纳入了对参数有意义的完整数据的患者（n=341），以得出预后模型。随后在多机构队列中验证。
结果：在派生队列中（n=341），年龄(危险比[HR]，1.9[95%CI，1.2至2.1]；P=.0009)，血清乳酸脱氢酶（LDH）高于正常上限（HR，2.3[95%CI，1.3至4.5]；P=.007)，和血清白蛋白<3.5g/dL（HR，1.5[95%CI，0.99至2.3]；P=.056）是独立预后。通过给白蛋白<3.5g/dL(HR，1.5）和年龄66-75岁（HR1.4）和年龄>75岁的2分（HR，2.6）或LDH升高（HR，2.3),根据综合评分,观察四组结局不同的患者.低风险患者的五年OS为93%（0分），低-中等风险为82%(得分1)，中等风险为69%(得分为2)，高风险组（评分≥3；P<0.0001）为55%。在验证队列中（N=335），该模型保持了其预后价值，5年OS为93%，90%,75%,四组的57%，分别（P<0.0001）。
结论：改进的WM分期系统（MSS-WM），利用年龄，白蛋白,LDH是一个简单的，临床上有用，和外部验证的预后模型，该模型可靠地将有症状的WM患者风险分层为具有不同预后的四组。
方法：我们回顾了889例未经治疗的活动性WM患者的记录，在1996年1月1日至2017年12月31日期间连续观察，以在单变量分析中确定总生存期（OS）的临床预测因子。在多变量分析中纳入了对参数有意义的完整数据的患者（n=341），以得出预后模型。随后在多机构队列中验证。
结果：在派生队列中（n=341），年龄(危险比[HR]，1.9[95%CI，1.2至2.1]；P=.0009)，血清乳酸脱氢酶（LDH）高于正常上限（HR，2.3[95%CI，1.3至4.5]；P=.007)，和血清白蛋白<3.5g/dL（HR，1.5[95%CI，0.99至2.3]；P=.056）是独立预后。通过给白蛋白<3.5g/dL(HR，1.5）和年龄66-75岁（HR1.4）和年龄>75岁的2分（HR，2.6）或LDH升高（HR，2.3),根据综合评分,观察四组结局不同的患者.低风险患者的五年OS为93%（0分），低-中等风险为82%(得分1)，中等风险为69%(得分为2)，高风险组（评分≥3；P<0.0001）为55%。在验证队列中（N=335），该模型保持了其预后价值，5年OS为93%，90%,75%,四组的57%，分别（P<0.0001）。
结论：改进的WM分期系统（MSS-WM），利用年龄，白蛋白,LDH是一个简单的，临床上有用，和外部验证的预后模型，该模型可靠地将有症状的WM患者风险分层为具有不同预后的四组。
