Efficacy and safety of anlotinib in advanced leiomyosarcoma: Subgroup analysis of a phase IIB trial (ALTER0203)

Abstract

Background: Leiomyosarcoma (LMS) is one of the most common pathologic subtypes of soft tissue sarcoma (STS) with limited treatment options. An earlier analysis in ALTER0203 showed efficacy and safety of anlotinib in overall subtype of STS. Here we report subgroup analysis of the patients with Leiomyosarcoma in ALTER0203. Methods: Key inclusion criteria: aged from 18 to 70, confirmed histological diagnosis of advanced LMS, angiogenesis inhibitor naive, progressing after anthracycline‐contained chemotherapy, measurable disease (RECIST 1.1), ECOG performance status (PS) 1‐2. Anlotinib 12 mg per day 2 weeks on and 1 week off or placebo was given after 2:1 randomization. Primary endpoint: progression‐free survival (PFS). Secondary endpoints: overall response rate (ORR), disease control rate (DCR) and so on. Results: 41 eligible LMS patients, 9 males (21.95%), median age 49 (range 28‐66), received either anlotinib (n=27) or placebo (n=14). The median PFS was 1.43 months for placebo and 5.83 months for anlotinib (P<0.0001). CR or PR was not observed for both placebo and anlotinib. SD was 2/14 for placebo versus 16/27 for anlotinib (P=0.01). The most common adverse events (AEs) were hypertension, elevated TSH, hypertriglyceridaemia. The most common grade 3 or higher AEs were hypertension, gamma glutamyl transferase elevation, hyponatremia. Conclusions: Anlotinib not only improves PFS and DCR significantly, but also presents good safety in patients with LMS, which suggests that anlotinib could be an option for LMS patients. (Table Presented) .