The efficacy and safety of gemcitabine and docetaxel in pseudomyogenic hemangioendothelioma: a multi-center experience.
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BACKGROUND: 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. METHODS: 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). RESULTS: 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. CONCLUSIONS: 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.