LAUSR

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 Table . 1693P Efficacy Alotinib (n = 27) Placebo (n = 14) P-value mPFS (mos) 5.83 1.43 HR(95%CI) 2.85-8.81 1.41-1.45 ORR, n(%) 0(0%) 0(0%) N/A DCR, n(%) 16(59.26%) 2(14.29%) 0.01 The most common AEs, n(%) Hypertension 20(74.07%) 1(7.14%) TSH elevation 19(70.37%) 0(0%) Hypertriglyceridaemia 13(48.15%) 4(28.57%) 0.32 Grade≥3 AEs, n(%) Hypertension 5(18.52%) 0(0%) Gamma glutamyl transferase elevation 2(7.41%) 1(7.14%) 1.0 Hyponatremia 2(7.41%) 0(0%) 0.54 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. Clinical trial identification NCT02449343. Legal entity responsible for the study Chia Tai Tianqing Pharmaceutical Group Co., Ltd. Funding Chia Tai Tianqing Pharmaceutical Group Co., Ltd. Disclosure All authors have declared no conflicts of interest.