Department of Orthopedic Oncology Surgery, Beijing Jishuitan Hospital, Capital Medical University, Beijing, China.
Department of Orthopedics, West China Hospital, Sichuan University, Chengdu, China.
Nat Commun. 2024 Nov 5;15(1):9541. doi: 10.1038/s41467-024-53686-4.
This was a multicenter, single-arm, open-label, phase Ib/II study (NCT04255576), aimed to evaluate the efficacy and safety of JMT103 in patients with unresectable or surgically-challenging giant cell tumor of bone (GCTB). JMT103 (2 mg/kg) was administered subcutaneously every four weeks, with loading doses on days 8 and 15. The primary endpoint was the objective tumor response rate (OTR) based on best response, defined as the proportion of patients who achieved elimination of at least 90% of the giant cells or radiologic complete or partial response per the modified Inverse Choi density/size (mICDS) or modified European Organization for Research and Treatment of Cancer (mEORTC) within 12 weeks. Secondary endpoints included objective response rate (ORR) per mICDS and mEORTC, and safety. A total of 139 patients were enrolled, and 135 were analyzed for efficacy. OTR, determined by the independent review committee (IRC) was 93.3% (95% CI 87.7-96.9). Treatment-related adverse events occurred in 90 (64.7%) patients, with hypophosphatemia and hypocalcemia being the most common. No serious treatment-related adverse events were observed. Thus, JMT103 demonstrates potential as a therapeutic option for GCTB.
这是一项多中心、单臂、开放标签、Ib/II 期研究（NCT04255576），旨在评估 JMT103 治疗不可切除或手术挑战性巨大细胞瘤（GCTB）患者的疗效和安全性。JMT103（2mg/kg）每四周皮下给药一次，第 8 天和第 15 天给予负荷剂量。主要终点是根据最佳反应评估的客观肿瘤反应率（ORR），定义为在 12 周内至少有 90%的巨细胞消除或影像学完全或部分缓解的患者比例，按改良 Inverse Choi 密度/大小（mICDS）或改良欧洲癌症研究与治疗组织（mEORTC）标准。次要终点包括按 mICDS 和 mEORTC 评估的客观反应率（ORR）和安全性。共纳入 139 例患者，135 例患者进行了疗效分析。IRC 评估的 OTR 为 93.3%（95%CI 87.7-96.9）。90 例（64.7%）患者发生与治疗相关的不良事件，最常见的是低磷血症和低钙血症。未观察到严重的治疗相关不良事件。因此，JMT103 有望成为 GCTB 的一种治疗选择。
