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对于患有出血障碍的儿科患者,在水中使用粉碎的特兰胺酸片

Ahmad Al-Huniti¹, Linda Marshall², Dawn Rusk³

¹Division of Pediatric Hematology/Oncology, Mayo Clinic, Rochester, Minnesota, USA.

Haemophilia : the official journal of the World Federation of Hemophilia

|March 20, 2024

概括

在水中粉碎的松酸片 (cTXAw) 为患有出血障碍的儿童提供了一种安全有效的替代品,可以替代口服的氨基酸溶液 (EACA OS). 这种低成本的选择解决了与EACA OS相关的障碍,改善了儿科患者的可访问性.

科学领域:

儿科血液学儿科血液学
药理学药理学是指药理学的学科.
血液静止和血栓形成

背景情况:

乙氨基酸口服溶液 (EACA OS) 是唯一可用的口服抗纤维素催化剂,用于无法吞药片的儿科患者.
高昂的成本和保险拒绝对EACA OS的可访问性构成重大障碍.

研究的目的:

为了评估水中注射的粉碎特兰酸片 (cTXAw) 治疗儿童出血障碍的安全性和有效性.
将cTXAw与EACA OS作为儿科出血病的治疗选择进行比较.

主要方法:

对于2018年12月至2022年7月期间接受治疗的10岁以下患有出血障碍儿童的医疗记录的回顾性审查.
患者接受了cTXAw或EACA OS,使用国际血栓与血液静止学会 (ISTH) 标准评估出血结果.

主要成果:

其中包括32名儿童,常见的诊断包括·维勒布兰德病和血友病.
cTXAw和EACA OS都在预防和治疗出血事件方面表现出相似的疗效,没有报告的不良影响.
cTXAw治疗成本明显低于EACA OS (94美元与905美元相比),并且42%最初处方EACA OS的患者由于成本或保险问题而没有接受.

结论:

在水中粉碎的特兰胺酸片 (cTXAw) 是EACA OS的安全,有效和经济高效的替代品,适用于患有出血障碍的幼儿.
这种方法可以克服财务和保险相关的障碍,提高儿科患者的治疗可用性.

关键词:

抗纤维素分析剂是一种抗纤维素分析剂.血液凝固障碍血液凝固障碍血小板疾病血小板疾病血友症A是一种血友病.血友病B型血友病B型血友病B型儿科儿科医生儿科血小板细胞减少症 (Thrombocytopenia) 是一种特兰克萨姆酸是什么意思 ·威勒布兰德病是什么?

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