Wei Guoxu, Wu Min, Zhu He, Han Sheng, Chen Jing, Zhai Chenchen, Shi Luwen
Department of Pharmacy Administration and Clinical Pharmacy, School of Pharmaceutical Sciences, Peking University, Beijing, China.
International Research Center for Medicinal Administration, Peking University Health Science Center, Beijing, China.
Front Pharmacol. 2021 Apr 8;12:616453. doi: 10.3389/fphar.2021.616453. eCollection 2021.
Cancer is a leading cause of morbidity and mortality worldwide. Off-label (OL) use of antineoplastic drugs to treat malignancies is prevalent. In this study, we quantified and characterized OL use of antineoplastic drugs to treat malignancies in China. This was a retrospective study using nationwide data collected from 2008 to 2010. Use of antineoplastic drugs was considered OL if they were used for indications not reflected in the package insert published by the National Medical Products Administration at the time of prescription. Descriptive analysis and Spearman rank correlation were used to evaluate the frequency and pattern of OL drug use. In total, 51,382 patients with malignancies, 24 categories of antineoplastic drugs, and 77 types of malignancies treated with OL drugs were included in this study. Twenty commonly used antineoplastic drugs (ICD encoded as L01) were used OL in 10-61% of cases, and four commonly used endocrine therapy antineoplastic drugs (ICD encoded as L02) were used OL in 10-19% of cases. There was a significant negative correlation between the disease constituent ratio and the average OL use rate of antineoplastic drugs for various malignancies. In contrast, there was a significant positive correlation between the average OL use rate of antineoplastic drugs and the number of malignancies treated with OL drugs. This study provided information regarding OL use of antineoplastic drugs for treatment of malignancies, and showed that OL use was prevalent. In addition, uncommon malignancies were more likely to be treated with OL antineoplastic drugs. Furthermore, more commonly used antineoplastic drugs were more likely to be used OL.
癌症是全球发病和死亡的主要原因。使用抗肿瘤药物进行恶性肿瘤的超说明书（OL）治疗很普遍。在本研究中，我们对中国使用抗肿瘤药物进行恶性肿瘤OL治疗的情况进行了量化和特征分析。这是一项回顾性研究，使用了2008年至2010年收集的全国性数据。如果抗肿瘤药物用于国家药品监督管理局在处方时发布的药品说明书中未体现的适应症，则其使用被视为OL。采用描述性分析和Spearman等级相关性分析来评估OL药物使用的频率和模式。本研究共纳入了51382例恶性肿瘤患者、24类抗肿瘤药物以及接受OL药物治疗的77种恶性肿瘤。20种常用抗肿瘤药物（ICD编码为L01）的OL使用率为10%至61%，4种常用内分泌治疗抗肿瘤药物（ICD编码为L02）的OL使用率为10%至19%。各种恶性肿瘤的疾病构成比与抗肿瘤药物的平均OL使用率之间存在显著负相关。相反，抗肿瘤药物的平均OL使用率与接受OL药物治疗的恶性肿瘤数量之间存在显著正相关。本研究提供了关于使用抗肿瘤药物进行恶性肿瘤OL治疗的信息，并表明OL治疗很普遍。此外，罕见恶性肿瘤更有可能接受OL抗肿瘤药物治疗。此外，更常用的抗肿瘤药物更有可能被用于OL治疗。
