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转移性儿童横纹肌肉瘤的系统治疗:挑战历程与对有前景方法的探索

英文原题:Systemic therapy in metastatic pediatric rhabdomyosarcoma: a history of challenges and the search for promising approaches.

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Systemic therapy in metastatic pediatric rhabdomyosarcoma: a history of challenges and the search for promising approaches.

PubMed 2025/03/31(内容时间) Expert Opin Pharmacother Q2 · IF 3.4(JCR 2025)

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研究概要

转移性 RMS 的治疗已有进展,但由于长期生存率低,尽管采用强化化疗和多种优化措施,仍面临挑战。

中文摘要

引言:转移性横纹肌肉瘤(RMS)是一种罕见且侵袭性强的恶性肿瘤,起源于骨骼肌前体细胞,主要影响儿童和青少年。转移性 RMS 预后不佳,因此制定有效治疗策略至关重要。化疗是治疗方案的基础,旨在控制疾病进展、缩小肿瘤并改善生存结局。综述范围:本文探讨化疗在转移性 RMS 管理中的作用,考察标准治疗方案、疗效、挑战及治疗未来方向。专家观点:转移性 RMS 治疗已有进展,但尽管采用强化化疗并尝试多种优化措施,长期生存率仍低,治疗依然困难。靶向治疗、免疫治疗和 CAR-T 细胞疗法等新兴方法带来希望,但仍需持续研究以改善结局并实现个体化治疗策略。

展开英文摘要原文

INTRODUCTION: Metastatic rhabdomyosarcoma (RMS) is a rare and aggressive malignancy that arises from skeletal muscle precursors. It primarily affects children and adolescents. The prognosis for metastatic RMS is poor, making effective treatment strategies crucial. Chemotherapy serves as the backbone of the treatment regimen, aiming to control disease progression, shrink tumors, and improve survival outcomes.

AREAS COVERED: This paper explores the role of chemotherapy in managing metastatic RMS, examining standard therapeutic protocols, their efficacy, challenges, and future directions in treatment. EXPERT OPINION: The treatment of metastatic RMS has evolved but remains challenging due to poor long-term survival rates, despite intensive chemotherapy and various optimization efforts.

Emerging approaches, including targeted therapies, immunotherapy, and CAR-T cell therapy, offer hope, though ongoing research is crucial to improve outcomes and personalize treatment strategies.

论文信息

作者
Di Carlo D、Affinita MC、Poli E、Bisogno G
单位
Department of Women's and Children's Health, University of Padua, Padua, Italy.Italy
文献类型
综述
期刊
Expert opinion on pharmacotherapy2025 Apr
原文标识
PubMed 40129247 · DOI 10.1080/14656566.2025.2484319