超罕见肉瘤的景观：一项全国性的流行病学和预后研究

IF 7.1 2区医学 Q1 ONCOLOGY

ESMO Open Pub Date : 2025-05-01 DOI:10.1016/j.esmoop.2025.105097

E. Shimada , M. Nakagawa , M. Endo , N. Yokoyama , A. Nabeshima , T. Fujiwara , A. Kawai , Y. Nakashima

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引用次数: 0

摘要

“超罕见肉瘤”的概念是为了提高人们对其罕见性所带来的临床挑战的认识而建立的。鉴于这种分类的新颖性和随之而来的数据缺乏，本研究旨在调查超罕见肉瘤的流行病学和预后。我们分析了2001年至2019年日本骨和软组织肿瘤登记处的数据，比较了超罕见和非超罕见的肉瘤。为了评估超罕见肉瘤的预后影响，我们使用了Kaplan-Meier生存分析与倾向评分匹配、多变量分析和随机生存森林的机器学习技术。结果22 821例患者中，超罕见肉瘤占18.9%。超罕见骨肉瘤患者比非超罕见骨肉瘤患者年龄大(平均年龄：57.6岁比39.2岁，P <；0.001)，而超罕见的软组织肉瘤出现在年轻患者中(平均年龄：49.4岁对62.2岁，P <；0.001)。在80岁的骨肉瘤患者和20岁的软组织肉瘤患者中，超罕见的肉瘤约占病例的一半。生存分析显示，超罕见骨肉瘤与较长的生存期相关（P = 0.022），而超罕见软组织肉瘤与总生存期无显著差异（P = 0.052）。然而，当按年龄分层时，40岁的超罕见软组织肉瘤患者的生存期较短(P <；0.001)。多因素分析表明，超罕见骨肉瘤的风险比为0.73，超罕见软组织肉瘤的风险比为1.25。随机生存森林表明，与其他参数相比，超罕见肉瘤的重要性相对较低。结论超罕见肉瘤多见于老年骨肉瘤和年轻软组织肉瘤。患有超罕见软组织肉瘤的年轻患者预后明显较差。总的来说，虽然超罕见肉瘤通常对预后的影响较小，但它们在特定年龄组的影响更为明显。

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Landscape of ultra-rare sarcomas: a nationwide study for epidemiology and prognosis

Background

The concept of ‘ultra-rare sarcoma’ was established to raise awareness of the clinical challenges resulting from its rarity. Given the novelty of this classification and the consequent paucity of data, this study aimed to investigate the epidemiology and prognosis of ultra-rare sarcomas.

Design

We analyzed data from the Bone and Soft Tissue Tumor Registry in Japan from 2001 to 2019, comparing ultra-rare and non-ultra-rare sarcomas. To assess the prognostic impact of ultra-rare sarcomas, we used Kaplan–Meier survival analysis with propensity score matching, multivariate analysis, and a machine learning technique known as random survival forest.

Results

Among the 22 821 patients analyzed, ultra-rare sarcomas accounted for 18.9% of the cases. Ultra-rare bone sarcomas were older than non-ultra-rare bone sarcomas (mean age: 57.6 versus 39.2 years, P < 0.001), while ultra-rare soft tissue sarcomas appeared in younger patients (mean age: 49.4 versus 62.2 years, P < 0.001). For patients >80 years old with bone sarcomas and those <20 years old with soft tissue sarcomas, ultra-rare sarcomas constituted approximately half of the cases. Survival analysis indicated that ultra-rare bone sarcomas were associated with longer survival (P = 0.022), whereas ultra-rare soft tissue sarcomas showed no significant difference in overall survival (P = 0.052). When stratified by age, however, patients <40 years old with ultra-rare soft tissue sarcomas had shorter survival (P < 0.001). Multivariate analysis indicated hazard ratios of 0.73 for ultra-rare bone and 1.25 for ultra-rare soft tissue sarcomas. Random survival forest showed that the importance of ultra-rare sarcomas was relatively low compared with other parameters.

Conclusion

Ultra-rare sarcomas are more common among older bone sarcoma patients and younger soft tissue sarcoma patients. Young patients with ultra-rare soft tissue sarcomas have a significantly worse prognosis. Overall, while ultra-rare sarcomas have a generally minor impact on prognosis, their effects are more pronounced in specific age groups.

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来源期刊

ESMO Open Medicine-Oncology

CiteScore

11.70

自引率

2.70%

发文量

255

审稿时长

10 weeks

期刊介绍： ESMO Open is the online-only, open access journal of the European Society for Medical Oncology (ESMO). It is a peer-reviewed publication dedicated to sharing high-quality medical research and educational materials from various fields of oncology. The journal specifically focuses on showcasing innovative clinical and translational cancer research. ESMO Open aims to publish a wide range of research articles covering all aspects of oncology, including experimental studies, translational research, diagnostic advancements, and therapeutic approaches. The content of the journal includes original research articles, insightful reviews, thought-provoking editorials, and correspondence. Moreover, the journal warmly welcomes the submission of phase I trials and meta-analyses. It also showcases reviews from significant ESMO conferences and meetings, as well as publishes important position statements on behalf of ESMO. Overall, ESMO Open offers a platform for scientists, clinicians, and researchers in the field of oncology to share their valuable insights and contribute to advancing the understanding and treatment of cancer. The journal serves as a source of up-to-date information and fosters collaboration within the oncology community.