Treatment of Inherited Platelet Disorders: Current Status and Future Options

被引:5
作者
Bargehr, Caroline [1 ,3 ]
Knoefler, Ralf [2 ]
Streif, Werner [1 ,3 ]
机构
[1] Med Univ Innsbruck, Dept Paediat 1, Innsbruck, Austria
[2] Univ Hosp Carl Gustav Carus Dresden, Dept Paediat Haemostaseol, Dresden, Germany
[3] Med Univ Innsbruck, Dept Paediat 1, Anichstr 35, A-6020 Innsbruck, Austria
来源
HAMOSTASEOLOGIE | 2023年 / 43卷 / 04期
关键词
blood platelet disorder; therapy; inherited platelet disorder; STEM-CELL TRANSPLANTATION; WISKOTT-ALDRICH-SYNDROME; BONE-MARROW-TRANSPLANTATION; RECOMBINANT FACTOR VIIA; GLANZMANNS-THROMBASTHENIA; GENE-THERAPY; TRANEXAMIC ACID; MANAGEMENT; THROMBOCYTOPENIA; PREDISPOSITION;
D O I
10.1055/a-2080-6602
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Inherited platelet disorders (IPDs) comprise a heterogeneous group of entities that manifest with variable bleeding tendencies. For successful treatment, the underlying platelet disorder, bleeding severity and location, age, and sex must be considered in the broader clinical context. Previous information from the AWMF S2K guideline #086-004 ( www.awmf.org ) is evaluated for validity and supplemented by information of new available and future treatment options and clinical scenarios that need specific measures. Special attention is given to the treatment of menorrhagia and risk management during pregnancy in women with IPDs. Established treatment options of IPDs include local hemostatic treatment, tranexamic acid, desmopressin, platelet concentrates, and recombinant activated factor VII. Hematopoietic stem cell therapy is a curative approach for selected patients. We also provide an outlook on promising new therapies. These include autologous hematopoietic stem cell gene therapy, artificial platelets and nanoparticles, and various other procoagulant treatments that are currently tested in clinical trials in the context of hemophilia.
引用
收藏
页码:261 / 270
页数:10
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