Shimada E, Nakagawa M, Endo M, Yokoyama N, Nabeshima A, Fujiwara T, Kawai A, Nakashima Y
Department of Orthopaedic Surgery, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan; Department of Orthopedic Surgery, Duke University School of Medicine, Durham, USA.
Department of Orthopaedic Surgery, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan.
ESMO Open. 2025 May;10(5):105097. doi: 10.1016/j.esmoop.2025.105097. Epub 2025 May 12.
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.
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.
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.
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.
“超罕见肉瘤”这一概念的提出是为了提高人们对因其罕见性而带来的临床挑战的认识。鉴于这种分类的新颖性以及随之而来的数据匮乏，本研究旨在调查超罕见肉瘤的流行病学和预后情况。
我们分析了日本骨与软组织肿瘤登记处2001年至2019年的数据，对超罕见和非超罕见肉瘤进行了比较。为评估超罕见肉瘤对预后的影响，我们使用了倾向得分匹配的Kaplan-Meier生存分析、多变量分析以及一种名为随机生存森林的机器学习技术。
在分析的22821例患者中，超罕见肉瘤占病例的18.9%。超罕见骨肉瘤患者的年龄比非超罕见骨肉瘤患者大（平均年龄：57.6岁对39.2岁，P<0.001），而超罕见软组织肉瘤多见于年轻患者（平均年龄：49.4岁对62.2岁，P<0.001）。对于80岁以上的骨肉瘤患者和20岁以下的软组织肉瘤患者，超罕见肉瘤约占病例的一半。生存分析表明，超罕见骨肉瘤与更长的生存期相关（P=0.022），而超罕见软组织肉瘤在总生存期方面无显著差异（P=0.052）。然而，按年龄分层时，40岁以下的超罕见软组织肉瘤患者生存期较短（P<0.001）。多变量分析表明，超罕见骨肉瘤的风险比为0.73，超罕见软组织肉瘤为1.25。随机生存森林显示，与其他参数相比，超罕见肉瘤的重要性相对较低。
超罕见肉瘤在老年骨肉瘤患者和年轻软组织肉瘤患者中更为常见。患有超罕见软组织肉瘤的年轻患者预后明显更差。总体而言，虽然超罕见肉瘤对预后的总体影响较小，但其影响在特定年龄组中更为明显。
