Background The study was aim to assess the efficacy and safety of sunitinib on 14 Chinese patients with locally unresectable or metastatic Alveolar Soft Part Sarcoma (ASPS) at two institutions retrospectively. Methods Patients were treated with 37.5 mg of sunitinib once daily continuously without a scheduled off-treatment period. Dose holds or reductions were recommended for grade 3 AEs but were required for grade 4 AEs. Restarting treatment of sunitinib was allowed when AEs returned back to grade 1 or disappeared. The treatment was continued until progression disease (PD), unacceptable toxicity or death. Results From January 2011 to December 2015, 14 patients with unresectable or metastatic ASPS received sunitinib treatment. Among them, 4 patients achieved partial remission (PR), and 10 patients achieved stable disease (SD). Median progression free survival (PFS) was 41.0 months (95 % CI: 7.7 to 74.4 month). Median overall survival (OS) was not reached. The 1- and 4-year OS rates were 90.0 % and 60.0 % respectively. Two patients with primarily unresectable ASPS received complete surgical resection after neoadjuvant treatment of sunitinib. The majority of toxicities encountered were grade 1 or 2 and manageable. The most common adverse events (AEs) were bleeding (35.7 %), hair & skin color change (37.5 %) and mucositis (28.6 %). Conclusions Sunitinib is effective in locally unresectable or metastatic ASPS with a good safety profile. Neoadjuvant treatment with sunitinib improves the chance of resection for patients with locally advanced ASPS.
Keywords: Alveolar soft part sarcoma; Sunitinib; Target therapy.