Cai Qiyan, Xu Bushu, Zhang Peng, Ma Jie, Liang Jiahui, Le Liyuan, Li Shining, Yang Jing, Pan Qiuzhong, Peng Ruiqing, Zhang Xing
Melanoma and Sarcoma Medical Oncology Unit, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, 510060, China.
Department of Medical Oncology, Henan Cancer Hospital, Affiliated Cancer Hospital of Zhengzhou University, Zhengzhou, 450000, China.
BMC Cancer. 2025 Jul 1;25(1):1030. doi: 10.1186/s12885-025-14376-6.
Pseudomyogenic hemangioendothelioma (PHE) is a rare, locally aggressive vascular tumor. Less than 200 cases of PHE have been reported. Surgery is the cornerstone of PHE treatment, and the data on systemic treatment is limited.
A retrospective multicenter study was conducted on patients with PHE between January 2016 to June 2024. The clinical characteristics were summarized and the efficacy and safety of the GD regimen (gemcitabine and docetaxel) were evaluated. The primary end point was progression-free survival (PFS). Secondary end points included overall survival (OS), objective response rate (ORR), disease control rate (DCR), and adverse events (AEs).
16 patients were identified, with a male/female ratio of 7:1. The average age at diagnosis was 33 years. 43.8% of cases had multifocal lesions involved. The incidence of metastasis was higher than that of recurrence (43.8% vs. 18.8%). The median OS was not reached. 9 patients received chemotherapy. Among them, 6 patients were treated with GD and most of them (5/6, 83.3%) were palliative. In this palliative subgroup, 1-year PFS rate was 80.0% (4/5), with an ORR of 60% (3/5), and a DCR of 100% (5/5). Grade ≥ 3 AEs were reported in 3 patients, all classified as grade 3, without impacting the continuation of chemotherapy after symptomatic management.
Gemcitabine and docetaxel demonstrated satisfactory efficacy and acceptable safety in PHE patients; however, clinical trials and large-scale studies are needed to validate these results.
假性肌源性血管内皮瘤（PHE）是一种罕见的、具有局部侵袭性的血管肿瘤。已报道的PHE病例不足200例。手术是PHE治疗的基石，关于全身治疗的数据有限。
对2016年1月至2024年6月期间的PHE患者进行了一项回顾性多中心研究。总结临床特征，评估吉西他滨和多西他赛（GD方案）的疗效和安全性。主要终点是无进展生存期（PFS）。次要终点包括总生存期（OS）、客观缓解率（ORR）、疾病控制率（DCR）和不良事件（AE）。
共纳入16例患者，男女比例为7:1。诊断时的平均年龄为33岁。43.8%的病例有多发病灶。转移发生率高于复发率（43.8%对18.8%）。中位OS未达到。9例患者接受了化疗。其中，6例患者接受了GD方案治疗，大多数（5/6，83.3%）为姑息治疗。在这个姑息治疗亚组中，1年PFS率为80.0%（4/5），ORR为60%（3/5），DCR为100%（5/5）。3例患者报告了≥3级AE，均为3级，经对症处理后未影响化疗的继续进行。
吉西他滨和多西他赛在PHE患者中显示出令人满意的疗效和可接受的安全性；然而，需要进行临床试验和大规模研究来验证这些结果。
