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儿童慢性免疫性血小板减少症的临床结局:泰国一家三级中心38年的经验

Clinical outcome of childhood chronic immune thrombocytopenia: A 38-year experience from a single tertiary center in Thailand.

作者信息

Chotsampancharoen Thirachit, Sripornsawan Pornpun, Duangchoo Sarapee, Wongchanchailert Malai, McNeil Edward

机构信息

Department of Pediatrics, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhla, Thailand.

Epidemiology Unit, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhla, Thailand.

出版信息

Pediatr Blood Cancer. 2017 Nov;64(11). doi: 10.1002/pbc.26598. Epub 2017 Apr 28.

Abstract

BACKGROUND

There is limited information on long-term follow-up and prognostic factors for remission among children diagnosed with chronic immune thrombocytopenia (ITP). The aim of this study was to determine clinical outcomes and factors influencing remission in childhood chronic ITP.

STUDY DESIGN

The hospital records of children aged 0-15 years diagnosed with chronic ITP were retrospectively reviewed. Kaplan-Meier curves were fit to estimate the median time to complete remission with 95% confidence intervals (CIs). Multivariate Cox proportional hazards regression models were used to identify independent factors for remission.

RESULTS

A total of 113 patients were included in the analysis. The number of children achieving complete remission was 49 (46%) and the median time to remission was 7.1 years (95% CI: 4.8-11.0). The remission rates at 3, 5, 10, and 20 years were 25, 43, 60, and 75%, respectively. Factors influencing remission were platelets >60 × 10 /L at the onset of chronic ITP (hazard ratio [HR]: 7.24, 95% CI: 3.0-17.5) and treatment with intravenous immunoglobulin (HR: 0.37, 95% CI: 0.16-0.84). Age, gender, and clinical factors at the time of newly diagnosed ITP including bleeding manifestations, onset of symptoms, and history of preceding infection and vaccination were not predictive of remission.

CONCLUSION

The spontaneous complete remission rates of chronic ITP were 43 and 60% at 5 and 10 years, respectively, and reached 75% at 20 years. A higher platelet level at diagnosis of chronic ITP and form of treatment were statistically significant indicators for achieving complete remission.

摘要

背景

关于慢性免疫性血小板减少症(ITP)患儿的长期随访及缓解的预后因素的信息有限。本研究的目的是确定儿童慢性ITP的临床结局及影响缓解的因素。

研究设计

对0至15岁诊断为慢性ITP的儿童的医院记录进行回顾性分析。采用Kaplan-Meier曲线估计完全缓解的中位时间及95%置信区间(CI)。使用多变量Cox比例风险回归模型确定缓解的独立因素。

结果

共有113例患者纳入分析。实现完全缓解的儿童有49例(46%),缓解的中位时间为7.1年(95%CI:4.8 - 11.0)。3年、5年、10年和20年的缓解率分别为25%、43%、60%和75%。影响缓解的因素为慢性ITP发病时血小板>60×10⁹/L(风险比[HR]:7.24,95%CI:3.0 - 17.5)及静脉注射免疫球蛋白治疗(HR:0.37,95%CI:0.16 - 0.84)。新诊断ITP时的年龄、性别及临床因素,包括出血表现、症状发作、既往感染和疫苗接种史,均不能预测缓解情况。

结论

慢性ITP的自发完全缓解率在5年和10年时分别为43%和60%,20年时达到75%。慢性ITP诊断时较高的血小板水平及治疗方式是实现完全缓解的统计学显著指标。

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