Soft tissue sarcoma is treatable using various agents to heal and prevent progress. Those who relapse or have advanced cancers require special care. The dosage and drug combination has to be effective. Pazopanib can be independently used to treat soft tissue sarcoma. But its efficacy may improve when combined with gemcitabine. This study compares the combination therapy with treatment that uses only pazopanib.

A total of 86 patients, with 43 women, were evaluated from a randomized trial of 90 patients. They had a measurable lesion, adequate organ function, and progressed after prior treatment. The combination group took pazopanib and gemcitabine (A), but the single-agent group received pazopanib only (B). The primary endpoint involved a progression-free survival rate (PFSR) at 12 weeks. Quality of life, toxicity, overall survival, and response rate determined the secondary point.

The majority of patients had leiomyosarcoma at 26% or liposarcoma at 19%. The study met the primary endpoint after a median follow-up of 12.4 months. The 12-week PFSR, median, overall survival, and ORR for group A were 74%, 5.6 months, 13.1 months, and 11%, respectively. For arm B, they were 47%, two months, 11.2 months, and 5%. An increase in A group’s toxicity was manageable and mainly hematological.

The phase 2 trial shows tolerability for the combination arm of gemcitabine and pazopanib. The PFSR was higher compared to single-agent pazopanib treatment. The suggested clinical activity requires further confirmation after a phase 3 trial. The new study has to be more homogenous with leiomyosarcoma patients.