A systematic review and meta-analysis of pazopanib efficacy and adverse effects in sarcomas

Pazopanib, a multi-targeted tyrosine kinase inhibitor, is employed in the treatment of various malignancies, including metastatic non-adipocytic soft tissue sarcoma.

This systematic review and meta-analysis adhered to PRISMA guidelines to evaluate the efficacy and toxicity of pazopanib monotherapy in treating sarcomas. A comprehensive search of PubMed/MEDLINE and Scopus/ELSEVIER databases was conducted, covering the period from 2009 to 2025. The included studies were evaluated for quality using the MINORS, Newcastle-Ottawa Scale, and RoB2 tools. The spectrum of toxicities and responses in sarcoma types was described. A meta-analysis was performed to compare the efficacy of pazopanib with non-placebo treatments, using both fixed-effect and random-effect models to calculate pooled hazard ratios (HRs) for progression-free survival (PFS) and overall survival (OS).

A total of 40 studies were included, encompassing a wide range of study designs and quality. The analysis revealed variable objective response rates (ORR) across different sarcoma types, with the highest ORRs by RECIST observed in desmoid tumors (37.0%) and alveolar soft part sarcoma (35.5%). Common toxicities included hypertension, liver function test abnormalities, and fatigue, with significant variability in dose reductions and treatment interruptions across studies. The pooled HRs for PFS and OS were 1.10 (95% CI: 0.69-1.25) and 0.99 (95% CI: 0.63-1.35), respectively, indicating no significant advantage of non-placebo treatments respect to pazopanib.

Pazopanib demonstrated histology-specific efficacy in sarcomas, with a manageable toxicity profile. Furthermore, it does not appear inferior to non-placebo interventions, highlighting the need for further comparative studies to clarify its role in the therapeutic landscape of advanced sarcomas.