Journal Article

Efficacy and safety of anamorelin in patients with metastatic urothelial carcinoma receiving systemic chemotherapy: a randomized controlled study

Naiki, Taku, Aoki, Maria, Yasui, Takahiro, Mimura, Yoshihisa, Gonda, Masakazu, Odagiri, Kunihiro, Nagai, Takashi, Morikawa, Toshiharu, Furukawa-Hibi, Yoko, Naiki-Ito, Aya, Etani, Toshiki, Sugiyama, Yosuke, Tasaki, Yoshihiko, Shimizu, Nobuhiko

2025

Overview

Abstract Background Cancer cachexia reduces chemotherapy efficacy in patients with metastatic urothelial carcinoma (mUC). Anamorelin improves cancer cachexia by increasing serum insulin-like growth factor 1 (IGF-1). The aim of this study was to evaluate the efficacy and safety of anamorelin in patients with mUC. Methods A total of 38 patients with mUC who received platinum-based chemotherapy were enrolled in an interventional prospective study. Patients were randomized to anamorelin (Ana; n = 20) or control (n = 18) groups. The primary endpoint was the change in prealbumin. Secondary endpoints were changes in albumin, IGF-1, body weight, skeletal muscle mass index (SMI), interleukin-6 (IL-6) and median overall survival (mOS). Results Changes in prealbumin, albumin, body weight, and SMI were similar between groups. Compared to control, serum IGF-1 levels in the anamorelin-treated group were higher after 1 week (P < .05). Changes in IGF-1 and SMI positively correlated in the anamorelin group (R = 0.61, P < .05). When the anamorelin group was divided into 2 groups by the median serum IGF-1 level after 1 week (Ana-IGF-1 high and Ana-IGF-1 low groups), the SMI in the latter group showed a decreased tendency (P = .14). The Ana-IGF-1 low group showed significantly higher IL-6 levels (P < .05) at baseline and significantly shorter mOS compared to the Ana-IGF-1 high group (P < .05). Treatment-related adverse events were comparable between the 2 groups (any grade: 100% vs 100%; grade ≥3: 88.9% vs 90.0%). Conclusions Anamorelin was well tolerated in patients with mUC. A higher serum IGF-1 level might be associated with anamorelin efficacy; however, a larger study is needed to confirm this.

• Publisher: Oxford University Press
• Subject: Adult / Aged / Antineoplastic Combined Chemotherapy Protocols - adverse effects / Antineoplastic Combined Chemotherapy Protocols - therapeutic use / Appetite stimulants / Bladder cancer / Cachexia / Cachexia - drug therapy / Cachexia - pathology / Cancer / Causes of / Chemotherapy / Complications and side effects / Dosage and administration / Drug therapy / Female / Genitourinary Cancer / Humans / Insulin-Like Growth Factor I - metabolism / Insulin-like growth factors / Interleukin-6 - blood / Male / Metastasis / Middle Aged / Neoplasm Metastasis / Patient outcomes / Prospective Studies / Treatment Outcome