在 EML4::ALK 阳性淋巴畸形中有效使用 ALK 抑制剂。

IF 2.4 3区医学 Q2 HEMATOLOGY Pediatric Blood & Cancer Pub Date : 2024-11-11 DOI:10.1002/pbc.31441

Beth Apsel Winger, Christopher F Dowd, Kristin A Shimano, W Patrick Devine, Erin Mathes, Ilona Frieden, Carrie Schaefer, Alok Kothari

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引用次数: 0

摘要

基因靶向药物正在成为治疗对西罗莫司无反应的淋巴畸形（LM）的重要疗法。我们描述了两名EML4::ALK阳性淋巴畸形患者，其中一名患有Gorham Stout病，另一名患有大型泌尿生殖系统（GU）淋巴畸形，他们都成功接受了ALK抑制剂治疗。该报告为不断增长的LMs分子靶向疗法工具箱增添了ALK抑制剂。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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Effective Use of ALK Inhibitors in EML4::ALK-Positive Lymphatic Malformations.

Genetically targeted medications are emerging as important therapies for lymphatic malformations (LMs) unresponsive to sirolimus. We describe two patients with EML4::ALK-positive LMs, one with Gorham Stout disease and one with a large genitourinary (GU) LM, who were successfully treated with ALK inhibitors. This report adds ALK inhibitors to the growing toolbox of molecularly targeted therapies for LMs.

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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.