Rast Jasmin, Schramm Theresa, Mehic Dino, Fillitz Michael, Drexel Tanja, Neusiedler-Nicolas Veronika, Ay Cihan, Pabinger Ingrid, Gebhart Johanna
Division of Hematology and Hemostaseology, Department of Medicine I, Medical University of Vienna, Vienna, Austria.
Department of Internal Medicine, Hanusch Hospital, Vienna, Austria.
Hamostaseologie. 2024 Nov 5. doi: 10.1055/a-2404-0306.
Treatment sequence in primary immune thrombocytopenia (ITP) is based on national and international recommendations, treatment availability, and physician expertise.
This article aimed to provide real-world data on treatment sequence and responses to first- and second-line treatments in newly diagnosed and relapsed adult ITP patients.
We analyzed a cohort of 46 adult ITP patients from the Vienna ITP Biobank, who started first-line therapy within 1 week before their first study visit between February 2016 and March 2023. We investigated clinical patient characteristics and patient management in our specialized center and examined the impact of the international ASH guidelines on ITP treatment.
Forty-six primary ITP patients, 27 (58.7%) with newly diagnosed ITP and 19 (41.3%) with relapsed ITP, were investigated. Most patients were female (65.2%) with a median platelet count of 9 × 10/L, and 31 patients (67.4%) had bleeding symptoms. All patients received first-line treatment with oral prednisolone; 15 patients received oral prednisolone combined with intravenous immunoglobulins (IVIGs), which were more commonly administered in newly diagnosed than in relapsed ITP patients. First-line therapy resulted an overall response in 82.6% of patients after a median (interquartile range [IQR]) time of 10 (5-25) days. There was no difference in treatment responses between newly diagnosed and relapsed ITP patients, but newly diagnosed patients had a shorter time to response (median [IQR]: 8 [5-14] and 14 [8-27], = 0.02). Twenty-three (50%) of the patients (11/27 newly diagnosed [40.7%], 12/19 relapsed [63.2%]) required second-line ITP therapy. Thrombopoietin-receptor agonists (TPO-RAs) were the most commonly used second-line therapy with a response rate of 73.7%, and a median (IQR) time to treatment response of 15 (12-20) days. Overall response rates to TPO-RA treatment did not differ between newly diagnosed and relapsed ITP. Following the publication of novel guidelines in 2019, the median (IQR) duration of corticosteroid treatment shortened (100-52 days, = 0.01), as did the time to second-line treatment (160-47 days, = 0.01), and the median number of first-line therapies decreased from 2 (1-3) to 1 (1-2).
Initial treatment with corticosteroids was effective in the majority of newly diagnosed and relapsed ITP. Response rates to initial corticosteroid treatment in ITP patients are consistent with previous data, but only 50% achieve sustained remission. TPO-RAs, which are well tolerated and effective, are the most commonly used second-line therapy in our study population. International guidelines have led to faster treatment transitions and reduced splenectomy rates. Integration of real-life experience, expert consensus, and guidelines optimizes ITP patient management.
原发性免疫性血小板减少症（ITP）的治疗顺序基于国家和国际指南、治疗的可及性以及医生的专业知识。
本文旨在提供关于新诊断和复发的成年ITP患者治疗顺序以及一线和二线治疗反应的真实世界数据。
我们分析了来自维也纳ITP生物样本库的46例成年ITP患者队列，这些患者在2016年2月至2023年3月的首次研究访视前1周内开始一线治疗。我们在我们的专业中心调查了患者的临床特征和患者管理情况，并研究了国际ASH指南对ITP治疗的影响。
共调查了46例原发性ITP患者，其中27例（58.7%）为新诊断的ITP患者，19例（41.3%）为复发的ITP患者。大多数患者为女性（65.2%），血小板计数中位数为9×10⁹/L，31例患者（67.4%）有出血症状。所有患者均接受口服泼尼松龙一线治疗；15例患者接受口服泼尼松龙联合静脉注射免疫球蛋白（IVIG）治疗，新诊断患者比复发患者更常使用IVIG。一线治疗后，中位（四分位间距[IQR]）时间为10（5 - 25）天，82.6%的患者获得总体反应。新诊断和复发的ITP患者在治疗反应上无差异，但新诊断患者的反应时间较短（中位数[IQR]：8[5 - 14]天和14[8 - 27]天，P = 0.02）。23例（50%）患者（11/27例新诊断患者[40.7%]，12/19例复发患者[63.2%]）需要二线ITP治疗。血小板生成素受体激动剂（TPO - RAs）是最常用的二线治疗药物，反应率为73.7%，治疗反应的中位（IQR）时间为15（12 - 20）天。新诊断和复发的ITP患者对TPO - RA治疗的总体反应率无差异。2019年新指南发布后，糖皮质激素治疗的中位（IQR）持续时间缩短（100 - 52天，P = 0.01），二线治疗时间也缩短（160 - 47天，P = 0.01），一线治疗的中位数从2（1 - 3）次降至1（1 - 2）次。
皮质类固醇初始治疗对大多数新诊断和复发的ITP有效。ITP患者对初始皮质类固醇治疗的反应率与既往数据一致，但只有50%的患者实现持续缓解。TPO - RAs耐受性良好且有效，是我们研究人群中最常用的二线治疗药物。国际指南已促使治疗转换更快且脾切除率降低。结合现实生活经验、专家共识和指南可优化ITP患者管理。
