On TRacK With Larotrectinib in a Neonate With a Giant Congenital ETV6::NTRK3 Fusion-Positive Infantile Fibrosarcoma of the Head and Neck.

IF 2.3 3区医学 Q1 OTORHINOLARYNGOLOGY

Head and Neck-Journal for the Sciences and Specialties of the Head and Neck Pub Date : 2024-12-30 DOI:10.1002/hed.28058

Teresa M Cardesa-Salzmann, Monika Sparber-Sauer, Peter Hingst, Andreas Erbersdobler, Bjoern Schneider, Maja Hühns, Andre Jakob, Friederike Terpe, Christian Spang, Dorothea Stalmann, Claudia Bierwirth, Christina Hauenstein, Stefanie Märzheuser, Manfred Ballmann, Carl Friedrich Classen

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引用次数: 0

Abstract

Background: Infantile fibrosarcoma (IFS) is a rare pediatric tumor of intermediate malignancy with high local aggressiveness that typically presents in young infants. Its occurrence in the head and neck region is rare. Complete non-mutilating surgical resection is often not possible, requiring multimodal treatment. IFS frequently harbors neurotrophic receptor tyrosine kinase (NTRK) fusions. Targeted therapy with NTRK inhibitors is modifying treatment paradigms of IFS.

Methods: Herein, we report the case of a neonate with a giant unresectable congenital ETV6::NTRK3 (+) IFS of the head and neck region without rapid response to chemotherapy who was treated with larotrectinib oral suspension.

Results: Larotrectinib was well tolerated and induced an impressive clinical and radiologic response.

Conclusions: This case illustrates an example of pediatric precision oncology in a neonate with an ETV6::NTRK3 (+) congenital IFS of the head and neck region and provides further reference for the use of larotrectinib in the neonatal period.

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用 Larotrectinib 治疗患有巨大先天性 ETV6::NTRK3 融合阳性婴儿头颈部纤维肉瘤的新生儿的 TRacK。

背景：婴儿纤维肉瘤（IFS）是一种罕见的小儿中度恶性肿瘤，具有高度的局部侵袭性，通常出现在年幼的婴儿中。它发生在头颈部是罕见的。完全的非致残性手术切除通常是不可能的，需要多模式治疗。IFS经常包含神经营养受体酪氨酸激酶（NTRK）融合。NTRK抑制剂靶向治疗正在改变IFS的治疗模式。方法：在此，我们报告了一例头颈部巨大的不可切除的先天性ETV6::NTRK3 (+) IFS，对化疗无快速反应的新生儿，使用larorectinib口服混悬液治疗。结果：larorectinib耐受性良好，并诱导了令人印象深刻的临床和放射反应。结论：本病例为新生儿ETV6::NTRK3（+）先天性头颈部IFS患儿提供了儿科精准肿瘤学治疗的范例，为新生儿期larorectinib的应用提供了进一步的参考。

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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来源期刊

Head and Neck-Journal for the Sciences and Specialties of the Head and Neck 医学-耳鼻喉科学

CiteScore

7.00

自引率

6.90%

发文量

278

审稿时长

1.6 months

期刊介绍： Head & Neck is an international multidisciplinary publication of original contributions concerning the diagnosis and management of diseases of the head and neck. This area involves the overlapping interests and expertise of several surgical and medical specialties, including general surgery, neurosurgery, otolaryngology, plastic surgery, oral surgery, dermatology, ophthalmology, pathology, radiotherapy, medical oncology, and the corresponding basic sciences.