从发病机制到患者床边:骨骼外黏液样软骨肉瘤的全面回顾。

IF 2.8 3区 医学 Q3 ONCOLOGY
Piotr Remiszewski, Sławomir Falkowski, Anna Szumera-Ciećkiewicz, Mateusz J Spałek, Piotr Rutkowski, Anna M Czarnecka
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引用次数: 0

摘要

骨外黏液样软骨肉瘤(EMC)以复发性NR4A3基因重排为特征,最常见的是EWSR1::NR4A3,约占软组织肉瘤(STS)的1-3%。它通常发生在下肢近端深层软组织,特别是大腿。诊断最好通过结合形态学和免疫表型与分子确认来确定;特别是,NR4A3分离荧光原位杂交(FISH)提供了一种实用的单一检测方案。对于局部疾病,完全手术切除仍然是治疗的基石。当肿瘤边缘较近或肿瘤较大时,放射治疗(RT)可改善局部控制。无复发生存率(RFS)各不相同:在所有研究中,局部复发(LR)率从13%到42%不等,约35-45%的患者发生远处转移,主要发生在肺部。转移的中位时间约为28个月。总生存率(OS)反映了典型的惰性但转移的病程:5年生存率为66-88%,10年疾病特异性生存率约为85%。在晚期疾病中,基于蒽环类药物的化疗产生较低的客观缓解率(ORR),尽管偶尔会出现部分缓解。相比之下,在一项多中心2期研究(NCT02066285)中,抗血管生成酪氨酸激酶抑制剂pazopanib的ORR为18%,中位无进展生存期(PFS)为19个月。没有临床验证的药物直接靶向NR4A3。这篇综述总结了当代的诊断和治疗,强调高质量的手术,选择性RT,以及对晚期疾病抗血管生成酪氨酸激酶抑制剂的考虑。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
From pathogenesis to the patient's bedside: a comprehensive review of extraskeletal myxoid chondrosarcoma.

Extraskeletal myxoid chondrosarcoma (EMC) is characterised by recurrent NR4A3 gene rearrangements, most commonly EWSR1::NR4A3, and accounts for approximately 1-3% of soft-tissue sarcomas (STS). It typically arises in the deep soft tissues of the proximal lower limb, particularly the thigh. Diagnosis is best established by integrating morphology and immunophenotype with molecular confirmation; in particular, NR4A3 break-apart fluorescence in situ hybridisation (FISH) provides a practical single-assay solution. For localised disease, complete surgical excision remains the cornerstone of treatment. Radiotherapy (RT) improves local control when margins are close or tumours are large. Recurrence-free survival (RFS) varies: local recurrence (LR) rates range from 13 to 42% across studies, and distant metastases develop in around 35-45% of patients, primarily in the lungs. The median time to metastasis is approximately 28 months. Overall survival (OS) reflects the typically indolent yet metastatic course: 5-year OS 66-88%, and 10-year disease-specific survival approximately 85%. In advanced disease, anthracycline-based chemotherapy yields a low objective response rate (ORR), although occasional partial responses occur. By contrast, the anti-angiogenic tyrosine kinase inhibitor pazopanib produced an ORR of 18% and a median progression-free survival (PFS) of 19 months in a multicentre phase 2 study (NCT02066285). No clinically validated agents directly target NR4A3. This review summarises contemporary diagnostics and treatment, emphasising high-quality surgery, selective RT, and consideration of anti-angiogenic tyrosine kinase inhibitors in advanced disease.

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来源期刊
CiteScore
4.00
自引率
2.80%
发文量
577
审稿时长
2 months
期刊介绍: The "Journal of Cancer Research and Clinical Oncology" publishes significant and up-to-date articles within the fields of experimental and clinical oncology. The journal, which is chiefly devoted to Original papers, also includes Reviews as well as Editorials and Guest editorials on current, controversial topics. The section Letters to the editors provides a forum for a rapid exchange of comments and information concerning previously published papers and topics of current interest. Meeting reports provide current information on the latest results presented at important congresses. The following fields are covered: carcinogenesis - etiology, mechanisms; molecular biology; recent developments in tumor therapy; general diagnosis; laboratory diagnosis; diagnostic and experimental pathology; oncologic surgery; and epidemiology.
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