Department of Haematology, University College London Hospitals NHS Foundation Trust, London, UK.
Cardiometabolic Programme-NIHR UCLH/UC BRC, London, UK.
Eur J Haematol. 2020 Sep;105(3):344-351. doi: 10.1111/ejh.13456. Epub 2020 Jun 19.
Primary immune thrombocytopenia (ITP) is a bleeding disorder characterised by an isolated thrombocytopenia in the absence of an alternative diagnosis. The condition is highly heterogeneous with some patients requiring multiple of therapy before achieving response. In this study, we collected data on a large cohort of primary ITP patients with the objective of identifying variables which may predict treatment requirements.
We collected data on 379 patients, 275 with a confirmed diagnosis of primary ITP included demographics, baseline laboratory results and treatments. These were compared against treatment responses and lines of therapy.
Patients who presented with a platelet count of <30 × 10 /L or bleeding symptoms were observed to require more subsequent lines of therapy (P-value <0.001). 32% of patients (n = 87) received no treatment, and these patients had a significantly higher median count compared to those with required >2 lines of therapy (P-value <0.001). Superior response rates were demonstrated with thrombopoietin receptor agonists when compared with other agents irrespective of baseline characteristics.
Platelet counts at diagnosis are a potentially strong predictive indicator of subsequent lines of therapy. Patients with bleeding symptoms at diagnosis were more likely to have lower median platelets counts.
原发性免疫性血小板减少症(ITP)是一种以孤立性血小板减少为特征的出血性疾病,无其他明确诊断。该疾病具有高度异质性,部分患者在获得缓解之前需要接受多次治疗。本研究收集了大量原发性 ITP 患者的数据,旨在确定可能预测治疗需求的变量。
我们收集了 379 名患者的数据,其中 275 名患者被确诊为原发性 ITP,包括人口统计学、基线实验室结果和治疗情况。将这些数据与治疗反应和治疗线进行比较。
血小板计数<30×10 /L 或有出血症状的患者观察到需要更多后续治疗线(P 值<0.001)。32%的患者(n=87)未接受治疗,与需要>2 线治疗的患者相比,这些患者的中位血小板计数明显更高(P 值<0.001)。与其他药物相比,血小板生成素受体激动剂无论基线特征如何,均显示出更高的缓解率。
诊断时的血小板计数是后续治疗线的潜在强预测指标。诊断时有出血症状的患者更可能有较低的中位血小板计数。