Page 100 - 202016

·循证药学·

地舒单抗治疗骨巨细胞瘤的卫生技术评估 Δ

芮明军 ，唐文熙，李洪超（中国药科大学国际医药商学院，南京 211198)
＊
#
+
中图分类号 R979.1 9 文献标志码 A 文章编号 1001-0408（2020）16-2010-08
DOI 10.6039/j.issn.1001-0408.2020.16.17
摘 要 目的：评价地舒单抗治疗骨巨细胞瘤的有效性、安全性和经济性，为临床治疗和决策提供循证依据。方法：系统检索
PubMed、Cochrane图书馆、ScienceDirect、中国知网、万方数据和维普网等数据库以及各国卫生技术评估（HTA）机构官网，纳入有
关地舒单抗治疗成人和骨骼发育成熟的青少年骨巨细胞瘤患者的HTA报告、随机对照试验（RCT）、单臂试验以及回顾性研究，并
对这些证据进行质量评估。采用描述性分析的方法描述纳入的HTA报告，使用R语言3.6.0软件对单臂临床试验和回顾性研究，
进行Meta分析。结果：从49篇文献中筛选出HTA报告6篇、单臂试验5篇、回顾性研究3篇，没有RCT研究。各国的HTA报告普
遍认为地舒单抗具有良好的有效性和安全性，且法国、澳大利亚等国的HTA报告认为该药具有经济性。在不可手术的骨巨细胞
瘤患者中，地舒单抗可为约81％[95％CI（77％，86％）]的患者带来临床获益，完全缓解率约为14％[95％CI（10％，19％）]，部分缓解
率约为51％[95％CI（32％，70％）]。在可手术的骨巨细胞瘤患者中，地舒单抗可使35％[95％CI（21％，49％）]的患者免于手术，降
低了40％[95％CI（36％，45％）]患者的手术分期；使用地舒单抗后，患者的术后复发率约为19％[95％CI（7％，35％）]，术后中位复
发时间约为6.73个月[95％CI（3.92，9.55）]。该药3～4级或高频发作且需治疗的不良事件主要为背痛、四肢疼痛、低磷血症和颌骨
坏死。结论：基于现有证据，在本研究涉及的研究和地区中，地舒单抗用于骨巨细胞瘤治疗具有良好的有效性、安全性和经济性。
关键词 地舒单抗；骨巨细胞瘤；卫生技术评估；有效性；安全性；经济性

Denosumab in the Treatment of Giant Cell Tumor of Bone：A Health Technology Assessment
RUI Mingjun，TANG Wenxi，LI Hongchao （School of International Pharmaceutical Business， China
Pharmaceutical University，Nanjing 211198，China）

ABSTRACT OBJECTIVE：To provide evidence-based evidence for clinical treatment and decision by evaluating efficacy，safety
and cost-effectiveness of denosumab in the treatment of giant cell tumor of bone（GCTB）. METHODS：Retrieved from PubMed，
the Cochrane Library，ScienceDirect，CNKI，Wanfang database and VIP as well as health technology assessment（HTA）organi-
zation websites，HTA reports，randomized controlled trials（RCTs），single-arm trials and retrospective studies were included about
denosumab in the treatment of GCTB in the adults and adolescents with mature bone，and their qualities were evaluated. HTA
reports were analyzed with descriptive analysis qualitatively；Meta-analysis was conducted for single-arm clinical studies and
retrospective studies by using R version 3.6.0 software. RESULTS：Among 49 screened literatures，there were 6 HTA reports，5
single-arm trials and 3 retrospective studies．No eligible RCTs were retrieved. HTA reports of various countries generally believed
that denosumab possessed good efficacy and safety；HTA reports of France，Austrila and other contries showed that denosumab
possessed economics. For the GCTB patients who was unresectable，denosumab would bring the clinical benefits to about
81％ [95％CI（77％，86％）] of patients. The complete response rate and partial response rate was around 14％[95％CI（10％，19％）]
and 51％[95％CI（32％，70％）]，respectively. For the GCTB patients was resectable，denosumab would prevent some patients from
receiving surgery （35％）[95％ CI（21％ ，49％）] or bring surgical down staging to them （40％）[95％ CI（36％ ，45％）]，the
postoperative recurrence rate after experiencing the denosumab therapy was about 19％[95％CI（7％，35％）]，and median relapse
time was approximately 6.73 months [95％CI（3.92，9.55）] after receiving surgery. Main grade 3-4 or high frequercy ADR requiring
treatment was back pain，limbs pain，hypophosphatemia and jaw osteonecrosis. CONCLUSIONS：Based on the currently available
evidence，among the studies and regions covered in this study，denosumab has favorable efficacy，safety and cost-effectiveness in
the treatment of TCTB.
KEYWORDS Denosumab；Giant cell tumor of bone；Health technology assessment；Efficacy；Safety；Cost-effectiveness

骨巨细胞瘤（Giant cell tumor of bone，GCTB）是一 种罕见的组织学良性但局部呈侵袭性生长的骨肿瘤 。
[1]
该病变可能会导致严重的骨骼并发症，包括病理性骨
Δ 基金项目：国家自然科学基金资助项目（No.71603278）
折、疼痛、关节活动受限、神经压迫以及肿瘤细胞侵及相
＊硕士研究生。研究方向：药物经济学、卫生经济与政策。电话：
[2]
邻结构等 。在我国，GCTB 的发病率占到了所有原发
025-86185038。E-mail：rmj_matthew@163.com
[3]
# 通信作者：讲师，博士。研究方向：药物经济学、卫生经济与政 性骨肿瘤的 11.61％，显著高于西方人群（3％～8％） 。
策。电话：025-86185038。E-mail：lihongchao@cpu.edu.cn GCTB好发于20～40岁的青壮年，且肿瘤细胞在妊娠期
·2010 · China Pharmacy 2020 Vol. 31 No. 16 中国药房 2020年第31卷第16期