Yang Yuan-Ning, Yeh Yun-Hsuan, Chen Jiann-Shiuh, Chen Li-Wen, Lin Yung-Chieh, Cheng Chao-Neng
Department of Pediatrics, College of Medicine, National Cheng Kung University Hospital, National Cheng Kung University, No. 138, Shengli Road, Tainan, 704302, Taiwan.
Department of Pediatrics, College of Medicine, National Cheng Kung University, Tainan, 701401, Taiwan.
Ann Hematol. 2024 Dec;103(12):5181-5189. doi: 10.1007/s00277-024-06056-5. Epub 2024 Oct 27.
This study examined the factors associated with spontaneous remission in children with chronic immune thrombocytopenia (ITP). We retrospectively analyzed the medical records of patients diagnosed with ITP from January 1988 to December 2019 at our institute. A total of 104 children with chronic ITP were identified. The median follow-up time from diagnosis of chronic ITP was 3.6 years (IQR 1.2-8.3, range 0.1-31.4). Fifteen (14.4%) patients with severe symptoms received specific platelet-elevating therapies, including splenectomy, rituximab, and thrombopoietin receptor agonists. Seven of them achieved remission. Among the patients with a platelet count < 30 × 10/L at the time of diagnosis of chronic ITP, those who received specific platelet-elevating therapies had a higher remission rate compared to those who did not (HR: 4.66, 95% CI: 1.36-16.0). Sixteen patients (15.4%) developed systemic lupus erythematosus, 46 (44.2%) still had thrombocytopenia after a median follow-up of 6.8 years, and 42 (40.4%) achieved remission with a median time to remission of 2.0 years (IQR 0.6-4.1, range 0.1-15.7). The two independent predictive factors for spontaneous remission in childhood chronic ITP were platelet counts > 30 × 10/L at the time of diagnosis of chronic ITP (HR: 3.16, 95% CI: 1.51-6.62) and persistently negative ANA at follow-up (HR: 6.12, 95% CI: 1.46-25.7). The cumulative probabilities of spontaneous remission at 10 years post-diagnosis of chronic ITP were 72.2% for patients without risk factor compared to 0% for patients with two risk factors.
本研究探讨了慢性免疫性血小板减少症(ITP)患儿自发缓解的相关因素。我们回顾性分析了1988年1月至2019年12月在我院诊断为ITP的患者的病历。共确定了104例慢性ITP患儿。慢性ITP诊断后的中位随访时间为3.6年(四分位间距1.2 - 8.3,范围0.1 - 31.4)。15例(14.4%)症状严重的患者接受了特定的升血小板治疗,包括脾切除术、利妥昔单抗和血小板生成素受体激动剂。其中7例实现缓解。在慢性ITP诊断时血小板计数<30×10⁹/L的患者中,接受特定升血小板治疗的患者缓解率高于未接受治疗的患者(风险比:4.66,95%置信区间:1.36 - 16.0)。16例患者(15.4%)发生系统性红斑狼疮,46例(44.2%)在中位随访6.8年后仍有血小板减少,42例(40.4%)实现缓解,缓解的中位时间为2.0年(四分位间距0.6 - 4.1,范围0.1 - 15.7)。儿童慢性ITP自发缓解的两个独立预测因素是慢性ITP诊断时血小板计数>30×10⁹/L(风险比:3.16,95%置信区间:1.51 - 6.62)和随访时抗核抗体持续阴性(风险比:6.12,95%置信区间:1.46 - 25.7)。慢性ITP诊断后10年自发缓解的累积概率,无危险因素的患者为72.2%,有两个危险因素的患者为0%。