曲美替尼治疗复杂淋巴畸形的疗效和安全性。

IF 2.3 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer Pub Date : 2025-10-20 DOI:10.1002/pbc.32119

Yulang Xu, Jiangyuan Zhou, Yuru Lan, Yi Ji

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引用次数: 0

摘要

本研究评估了曲美替尼在5例（中位年龄9岁）患有复杂淋巴畸形(如kaposiform淋巴管瘤病（KLA）和广泛性淋巴异常（GLA）的儿科患者中的疗效。所有患者均表现出显著的临床改善：凝血功能消失（血小板增加54%-362%，d -二聚体减少> 91%），80%的放射学达到部分缓解，毒性最小（1例2级皮疹）。在80%的病例中检测到NRAS原癌基因突变，但在没有突变的情况下发生临床获益。曲美替尼是一种有效、安全的KLA/GLA一线治疗药物。

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Efficacy and Safety of Trametinib Therapy for Complex Lymphatic Malformations.

This study evaluated trametinib in five pediatric patients (median age 9 years) with complex lymphatic malformations, such as kaposiform lymphangiomatosis (KLA) and generalized lymphatic anomaly (GLA). All showed significant clinical improvement: coagulopathy resolved (platelet increase 54%-362%, D-dimer decrease > 91%), 80% achieved partial response radiographically, and toxicity was minimal (one Grade 2 rash). NRAS proto-oncogene mutations were detected in 80% of cases, but clinical benefit occurred without mutation. Trametinib is an effective, safe, first-line therapy for KLA/GLA.

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来源期刊

Pediatric Blood & Cancer 医学-小儿科

CiteScore

4.90

自引率

9.40%

发文量

546

审稿时长

1.5 months

期刊介绍： Pediatric Blood & Cancer publishes the highest quality manuscripts describing basic and clinical investigations of blood disorders and malignant diseases of childhood including diagnosis, treatment, epidemiology, etiology, biology, and molecular and clinical genetics of these diseases as they affect children, adolescents, and young adults. Pediatric Blood & Cancer will also include studies on such treatment options as hematopoietic stem cell transplantation, immunology, and gene therapy.