Medical Oncology, Leiden University Medical Center, Leiden, The Netherlands.
Oral and Maxillofacial Surgery, Amsterdam UMC Location University of Amsterdam, Amsterdam, The Netherlands.
Oncologist. 2023 Nov 2;28(11):1005-e1104. doi: 10.1093/oncolo/oyad196.
Since giant cell tumors of bone (GCTB) and other giant cell rich tumors of bone (GCRTB) share the histological presence of osteoclastic giant cells and expression of RANK/RANKL, we hypothesized that GCRTB will respond similarly to denosumab as GCTB. The primary objective of this study was to determine the efficacy of denosumab in patients with GCRTB that have recurred or require morbid surgery.
In this open-label, multicenter, phase II trial, patients with GCRTB were included (June 2018-March 2020). Recruitment was stopped because of low accrual. Patients received denosumab (120 mg) subcutaneously (SC) on day 1 of every 4-week cycle with a loading dose of 120 mg SC on days 8 and 15.
Three patients were enrolled. One withdrew consent before start of study. The remaining patients had central giant cell granuloma of the jawbone (CGCG). Median treatment duration was 15 cycles (range 12-18). In both subjects, improvement in ossification of lesions was seen. Median follow-up was 28.5 months (range 20-37). One patient developed a recurrence for which surgery was performed.
Due to critical emerging real-world data of denosumab in GCRTBs, the study was prematurely stopped and not supportive of use of denosumab for this indication. (ClinicalTrials.gov Identifier: NCT03605199).
由于骨巨细胞瘤（GCTB）和其他富含巨细胞的骨肿瘤（GCRTB）具有破骨细胞性巨细胞的组织学存在和 RANK/RANKL 的表达，我们假设 GCRTB 对地舒单抗的反应与 GCTB 相似。本研究的主要目的是确定地舒单抗在复发或需要进行恶性手术的 GCRTB 患者中的疗效。
在这项开放标签、多中心、II 期试验中，纳入了 GCRTB 患者（2018 年 6 月至 2020 年 3 月）。由于入组人数较少，试验提前停止。患者每 4 周周期接受皮下注射地舒单抗（120mg）一次，第 8 天和第 15 天给予负荷剂量 120mg 皮下注射。
共纳入 3 例患者，其中 1 例在开始研究前撤回了同意。其余患者患有颌骨中央性巨细胞肉芽肿（CGCG）。中位治疗持续时间为 15 个周期（范围 12-18）。在这 2 例患者中，均可见病变的骨化改善。中位随访时间为 28.5 个月（范围 20-37）。1 例患者复发，行手术治疗。
由于地舒单抗在 GCRTB 中的关键真实世界数据，该研究提前停止，不支持将地舒单抗用于该适应证。（临床试验标识符：NCT03605199）。
