Real-world safety and efficacy of rADAMTS13 in the treatment of congenital thrombotic thrombocytopenic purpura in pediatric patients in Poland.

IF 5.5 2区医学 Q1 HEMATOLOGY

Journal of Thrombosis and Haemostasis Pub Date : 2024-11-26 DOI:10.1016/j.jtha.2024.11.008

Pawel Laguna, Maria Szczepanska, Magdalena Wojdalska, Halina Bobrowska, Joanna Kulik, Danuta Pietrys, Walentyna Balwierz, Elzbieta Trembecka-Dubel, Wojciech Mlynarski, Aleksandra Laguna

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引用次数: 0

Abstract

Background: Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultrarare microvascular disease caused by the deficiency of the metalloprotease ADAMTS13 (a disintegrin and metalloprotease with thrombospondin motifs 13). Approximately half of all cases remain undiagnosed until a triggering event in adulthood; therefore, the prevalence rates may be underestimated. The current standard of care is based on regular transfusions of fresh frozen plasma, which often lead to allergic reactions in patients. Recombinant ADAMTS13 (rADAMTS13) is a novel treatment for cTTP, which has been approved for use in the USA, Europe, and Japan.

Objectives: The primary objective of this real-world data collection was to comprehensively analyze the clinical data of pediatric patients with cTTP and to provide real-world evidence of the effectiveness of rADAMTS13 treatment in the pediatric population.

Methods: Nine pediatric patients with cTTP were treated with an intravenous infusion of rADAMTS13 every 2 weeks.

Results: The results showed an increase in platelet count and a decrease in lactate dehydrogenase levels compared with baseline. None of the patients experienced any adverse events or complications as a result of treatment. Patients reported an improved quality of life due to fewer hospital visits and a reduced number of recurrent episodes of cTTP.

Conclusion: Treatment with rADAMTS13 resulted in the normalization of laboratory parameters in all pediatric patients with cTTP.

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rADAMTS13 治疗波兰小儿先天性血栓性血小板减少性紫癜的实际安全性和有效性

背景：先天性血栓性血小板减少性紫癜（cTTP先天性血栓性血小板减少性紫癜（cTTP）是一种超罕见的微血管疾病，由金属蛋白酶 ADAMTS13 缺乏引起。大约有一半的病例在成年后发生诱发事件之前一直未被诊断出来，因此患病率可能被低估。目前的治疗标准是定期输注新鲜冰冻血浆（FFP），这往往会导致患者出现过敏反应。重组 ADAMTS13（rADAMTS13）是一种治疗 cTTP 的新型疗法，已在美国、欧洲和日本获批使用。目的：此次真实世界数据收集的主要目的是全面分析 cTTP 儿科患者的临床数据，并提供 rADAMTS13 治疗在儿科人群中有效性的真实世界证据。方法：在此，我们首次展示了 9 名儿科 cTTP 患者每 2 周接受一次 rADAMTS13 静脉输注治疗的真实世界证据：结果：结果显示，与基线相比，血小板计数增加，乳酸脱氢酶水平下降。所有患者均未因治疗而出现任何不良反应或并发症。由于减少了医院就诊次数和 cTTP 复发次数，患者的生活质量得到了改善：结论：使用 rADAMTS13 治疗可使所有儿童 cTTP 患者的实验室指标恢复正常。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of Thrombosis and Haemostasis 医学-外周血管病

CiteScore

24.30

自引率

3.80%

发文量

321

审稿时长

1 months

期刊介绍： The Journal of Thrombosis and Haemostasis (JTH) serves as the official journal of the International Society on Thrombosis and Haemostasis. It is dedicated to advancing science related to thrombosis, bleeding disorders, and vascular biology through the dissemination and exchange of information and ideas within the global research community. Types of Publications: The journal publishes a variety of content, including: Original research reports State-of-the-art reviews Brief reports Case reports Invited commentaries on publications in the Journal Forum articles Correspondence Announcements Scope of Contributions: Editors invite contributions from both fundamental and clinical domains. These include: Basic manuscripts on blood coagulation and fibrinolysis Studies on proteins and reactions related to thrombosis and haemostasis Research on blood platelets and their interactions with other biological systems, such as the vessel wall, blood cells, and invading organisms Clinical manuscripts covering various topics including venous thrombosis, arterial disease, hemophilia, bleeding disorders, and platelet diseases Clinical manuscripts may encompass etiology, diagnostics, prognosis, prevention, and treatment strategies.