■骨巨细胞瘤(GCTB)是一种相对良性的,而是局部侵袭性骨的破骨细胞间质瘤.尽管denosumab已被批准作为抗RANK配体的单克隆抗体用于治疗GCTB,很少有关于肿瘤反应获益的临床试验来证明中国人群的有效性。
■在这个多中心,随机对照,临床试验,纳入160例患者,以比较denosumab和唑来膦酸治疗对手术无法挽救的GCTB患者的疗效和安全性。
■在2015年1月2日至2018年1月1日之间,160名成年人(年龄≥18岁)患有①手术不可挽救的GCTB,②这项随机临床试验包括可手术挽救的GCTB和预期导致严重发病率的计划手术。患者接受皮下denosumab(DB组;每4周一次120mg,在第8天和第15天皮下给予120mg负荷剂量;n=80)或静脉注射唑来膦酸(ZA组;每4周一次4mg;n=80),共6个周期。疾病状态,临床效益,治疗引起的不良反应,总生存率,在随访期间评估治疗费用。使用95%置信区间确定统计显著性。
■Denosumab和唑来膦酸具有相似的肿瘤反应(p=0.118)和临床益处(p=0.574)。与ZA组(15.0%)相比,DB组(12.5%)中观察到更少患者的疾病进展。Denosumab引起疲劳(p=0.001)和背痛(p<0.0001),而唑来膦酸引起低钙血症(p<0.0001),流感样症状(p=0.059)和低血压(p=0.059)。Denosumab治疗比唑来膦酸治疗明显更昂贵(p<0.0001)。ZA组和DB组管理治疗引起的不良反应的成本相同(p=0.425)。DB组4年随访时累积无复发生存率较高(p=0.035)。
■Denosumab是一种安全但昂贵的唑来膦酸替代品,用于治疗手术无法挽救的GCTB。
UNASSIGNED: Giant-cell tumor of bone (GCTB) is a relatively benign, but locally aggressive osteoclastogenic stromal tumour of the bone. Although denosumab has been approved as an monoclonal antibody against RANK ligand for the treatment of GCTB, few clinical trials of the benefit in tumor response have been conducted to prove the efficiency in Chinese population.
UNASSIGNED: In this multicentric, random controlled, clinical trial, 160 patients were enrolled to compare the therapeutic efficacy and safety of denosumab and zoledronic acid treatment in patients with surgically unsalvageable GCTB.
UNASSIGNED: Between 2nd Jan 2015 and 1st Jan 2018, 160 adults (aged ≥ 18 years) with ①surgically unsalvageable GCTB, ②surgically salvageable GCTB with planned surgery expected to result in severe morbidity were included in this randomized clinical trial. Patients received either subcutaneous denosumab (DB group; 120 mg once every 4 weeks with loading doses of 120 mg subcutaneously admininstered on days 8 and 15; n = 80) or intravenous zoledronic acid (ZA group; 4 mg once every 4 weeks; n = 80) for six cycles. Disease status, clinical benefits, treatment-emergent adverse effects, overall survival, and cost of treatment were evaluated during the follow-up period. Statistical significance was determined using 95% confidence intervals.
UNASSIGNED: Denosumab and zoledronic acid had similar tumor responses (p = 0.118) and clinical benefits (p = 0.574). Disease progression was observed in fewer patients in the DB group (12.5%) than ZA group (15.0%). Denosumab caused fatigue (p = 0.001) and back pain (p < 0.0001), while zoledronic acid caused hypocalcemia (p < 0.0001), flu-like symptoms (p = 0.059) and hypotension (p = 0.059). Denosumab treatment was markedly more expensive than zoledronic acid treatment (p < 0.0001). The cost to manage treatment-emergent adverse effects was the same for the ZA group and the DB group (p = 0.425). The accumulate recurrence-free survival rate at 4-year follow-up is higher in DB group (p = 0.035).
UNASSIGNED: Denosumab is a safe but costly alternative to zoledronic acid for treatment of surgically unsalvageable GCTB.