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ALK 抑制在两个具有代表性的儿科炎症性肌纤维母细胞瘤病例中的应用:疗效和副作用。

ALK inhibition in two emblematic cases of pediatric inflammatory myofibroblastic tumor: Efficacy and side effects.

机构信息

Prinses Maxima Centrum, Center for Pediatric Oncology, Utrecht, the Netherlands.

Department of Pediatric Oncology/Hematology, Erasmus MC-Sophia Children's Hospital, Rotterdam, the Netherlands.

出版信息

Pediatr Blood Cancer. 2019 May;66(5):e27645. doi: 10.1002/pbc.27645. Epub 2019 Jan 29.

Abstract

There is an increasing interest for anaplastic lymphoma kinase (ALK) inhibitors in pediatric oncology for specific entities such as ALK-driven inflammatory myofibroblastic tumor (IMT). IMT treatment can be challenging due to localization of the tumor and in rare cases of metastasis. When standard surgical treatment is not feasible, ALK inhibitors may play an important role, as recently reported for the first-generation ALK inhibitors (crizotinib). However, data on the second-generation ALK inhibitors are limited. We report two emblematic cases of IMT in pediatric patients, treated with the second-generation ALK inhibitor ceritinib in the context of a clinical trial (NCT01742286).

摘要

人们对间变性淋巴瘤激酶(ALK)抑制剂在儿科肿瘤学中的应用越来越感兴趣,特别是在ALK 驱动的炎性肌纤维母细胞瘤(IMT)等特定实体瘤中。由于肿瘤的位置以及在罕见的转移情况下,IMT 的治疗可能具有挑战性。当标准的手术治疗不可行时,ALK 抑制剂可能会发挥重要作用,正如第一代 ALK 抑制剂(克唑替尼)最近报道的那样。然而,关于第二代 ALK 抑制剂的数据有限。我们报告了两例儿科患者的 IMT 典型病例,在临床试验(NCT01742286)中使用第二代 ALK 抑制剂色瑞替尼进行治疗。

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