Real-world evidence reports modest survival outcomes in localized non-uterine leiomyosarcoma, suggesting an urgent need for improved treatment strategies.
17.7%), doxorubicin-ifosfamide (16.4%), gemcitabine-docetaxel (12.9%), and pazopanib (12.9%).The disease-control rate was 74.2%.Median progression-free survival (PFS) and overall survival (OS) were 6.73 and 10.64 months, respectively, with no significant differences across primary or metastatic sites.Notably, less intensive regimens such as single-agent doxorubicin and pazopanib achieved progression-free survival comparable to multi-agent therapies (6.7 vs 6.73 months; p = 0.7 for doxorubicin, and 10.4 vs 6.73 months; p = 0.3 for pazopanib) while demonstrating more favorable tolerability.Grade 3-4 toxicities were predominantly hematological, especially neutropenia.Next-generation sequencing in 27 showed no alteration in 12(44%) patients, TP53 mutations in 8(30%), BRCA2 mutations in two (7%) patients, and isolated CDKN2A, GNAQ, PTEN, MEN1, and FGFR amplifications.Conclusions: This decade-long real-world study provides one of the largest LMIC datasets of metastatic non-uterine LMS.Systemic therapy outcomes remain modest despite diverse treatment strategies.Future efforts must prioritize biomarker-integrated clinical trials, exploration of novel therapeutic targets, and optimization of treatment sequencing to meaningfully advance patient outcomes.
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Addagalla et al. (2026) studied this question.
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