Prognostic factors of early outcome in pediatric hemophagocytic lymphohistiocytosis: an analysis of 116 cases
Prognostic factors of early outcome in pediatric hemophagocytic lymphohistiocytosis: an analysis of 116 cases
复制标题
DOI：
10.1007/s00277-016-2727-6
复制
发表时间：
2016-09-01
影响因子：
3.5
通讯作者：
Luo, Jian-Ming
中科院分区：
文献类型：
--
作者：
Bin, Qiong;Gao, Jin-Hong;Luo, Jian-Ming
早期死亡率仍然是治疗噬血细胞性淋巴组织细胞增生症（HLH）的主要挑战，这就需要在疾病的早期进行及时的风险分层。我们回顾性分析了2005年至2015年在中国南方一家三级医院接受治疗的儿科患者队列的临床特征。共纳入116例主要继发性HLH患者（中位年龄27.5个月）。在多变量考克斯回归模型中，中性粒细胞< 0.5 x 10（9）/L（风险比（RR）= 5.01; 95%置信区间（CI）1.55-16.20; P = 0.007），总胆红素超过正常值上限的两倍（RR = 2.86; 95% CI 0.83-9.88; P = 0.097）和白蛋白尿征20 g/L（RR = 5.79; 95% CI 1.70-19.73; P = 0.005）是30 d死亡的独立危险因素。有3个危险因素的患者的30天总生存率（OS）显著低于无~ 2个危险因素的患者（0 vs 90.7%; P < 0.001）。有3个危险因素的患者发生早期不良结局的可能性是无~ 2个危险因素的患者的64倍（RR = 64.45; 95% CI 18.35-226.33; P < 0.001）。2周内血小板计数正常化是初始治疗后消退的独立预测因素，比值比（OR）为18.4（95%CI 2.7-122.9; P = 0.003）。我们的研究结果表明，严重的中性粒细胞减少和肝功能损害是HLH早期死亡的预后因素，2周内血小板计数正常化是初始治疗后解决的关键预测因素。
Early mortality remains a major challenge for the treatment of hemophagocytic lymphohistiocytosis (HLH), which warrants the need for prompt risk stratification in the early phase of the disease. We retrospectively analyzed clinical features of a cohort of pediatric patients managed at a tertiary hospital in southern China from 2005 to 2015. A total of 116 patients (median age 27.5 months) with predominantly secondary HLH were included. In a multivariate Cox regression model, neutrophils < 0.5 x 10(9)/L (risk ratio (RR) = 5.01; 95 % confidence interval (CI) 1.55-16.20; P = 0.007), total bilirubin over twofold upper limit of normal value (RR = 2.86; 95 % CI 0.83-9.88; P = 0.097), and albumin aecurrency sign20 g/L (RR = 5.79; 95 % CI 1.70-19.73; P = 0.005) at diagnosis were independent risk factors for 30-day mortality. The 30-day overall survival rate (OS) of patients with three risk factors was significantly lower than that of patients with zero to two risk factors (0 vs 90.7 %; P < 0.001). Patients with three risk factors were 64-fold more likely to have early adverse outcome as compared to patients with zero to two risk factors (RR = 64.45; 95 % CI 18.35-226.33; P < 0.001). Platelet count normalization in 2 weeks was an independent predictor for resolution after initial therapy with an odds ratio (OR) of 18.4 (95 % CI 2.7-122.9; P = 0.003). Our results indicate that severe neutropenia and liver function damage are prognostic factors for early death in HLH and platelet count normalization in 2 weeks is a critical predictor for resolution after initial therapy.