期刊
BRITISH JOURNAL OF HAEMATOLOGY
卷 192, 期 2, 页码 366-374出版社
WILEY
DOI: 10.1111/bjh.17174
关键词
Wiskott– Aldrich syndrome; thrombocytopenia; bleeding; thrombopoietin receptor agonist; romiplostim
类别
This study demonstrates that romiplostim treatment can effectively increase platelet counts and reduce the risk of bleeding in WAS patients, with a significant decrease in hemorrhagic events even in non-responder group after the first month of treatment. The treatment responses tend to be durable and stable over time, with no significant fluctuations in platelet counts.
Wiskott-Aldrich syndrome (WAS) is a life-threatening primary immunodeficiency associated with bleeding of variable severity due to thrombocytopenia. Correction of the thrombocytopenia is of paramount importance for most WAS patients. We report a retrospective analysis of the safety and efficacy of romiplostim treatment in reducing thrombocytopenia and bleeding tendency in 67 children (median age 1 center dot 3 years) with genetically confirmed WAS, followed in eight months (range, 1-12 months). Complete or partial primary responses regarding platelet counts were observed in 22 (33%) and 18 (27%) subjects, respectively. Yet, even in the non-responder group, the risk of haemorrhagic events decreased significantly, to 21%, after the first month of treatment. The responses tended to be durable and stable over time, with no significant fluctuations in platelets counts. The results of this retrospective study of a large cohort of WAS patients demonstrates that romiplostim can be used to increase platelet counts and reduce the risks of life-threatening bleeding in WAS patients awaiting haematopoietic stem cell transplantation or forgoing the procedure for various reasons.
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