Pawel Laguna, Maria Szczepanska, Magdalena Wojdalska, Halina Bobrowska, Joanna Kulik, Danuta Pietrys, Walentyna Balwierz, Elzbieta Trembecka-Dubel, Wojciech Mlynarski, Aleksandra Laguna
{"title":"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","authors":"Pawel Laguna, Maria Szczepanska, Magdalena Wojdalska, Halina Bobrowska, Joanna Kulik, Danuta Pietrys, Walentyna Balwierz, Elzbieta Trembecka-Dubel, Wojciech Mlynarski, Aleksandra Laguna","doi":"10.1016/j.jtha.2024.11.008","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Objectives: </strong>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.</p><p><strong>Methods: </strong>Nine pediatric patients with cTTP were treated with an intravenous infusion of rADAMTS13 every 2 weeks.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusion: </strong>Treatment with rADAMTS13 resulted in the normalization of laboratory parameters in all pediatric patients with cTTP.</p>","PeriodicalId":17326,"journal":{"name":"Journal of Thrombosis and Haemostasis","volume":" ","pages":""},"PeriodicalIF":5.5000,"publicationDate":"2024-11-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Real-world safety and efficacy of rADAMTS13 in the treatment of congenital thrombotic thrombocytopenic purpura in pediatric patients in Poland.\",\"authors\":\"Pawel Laguna, Maria Szczepanska, Magdalena Wojdalska, Halina Bobrowska, Joanna Kulik, Danuta Pietrys, Walentyna Balwierz, Elzbieta Trembecka-Dubel, Wojciech Mlynarski, Aleksandra Laguna\",\"doi\":\"10.1016/j.jtha.2024.11.008\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>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.</p><p><strong>Objectives: </strong>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.</p><p><strong>Methods: </strong>Nine pediatric patients with cTTP were treated with an intravenous infusion of rADAMTS13 every 2 weeks.</p><p><strong>Results: </strong>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. 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Real-world safety and efficacy of rADAMTS13 in the treatment of congenital thrombotic thrombocytopenic purpura in pediatric patients in Poland.
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.
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.