利妥昔单抗在中国超说明书用药的临床研究进展

A review of clinical studies on off-label use of rituximab in China

利妥昔单抗最初批准用于治疗非霍奇金淋巴瘤,在国内超说明书用药情况普遍。随着中国临床研究的深入开展,利妥昔单抗超适应证用法的循证医学证据逐渐完善,已推荐作为原发免疫性血小板减少症、自身免疫性溶血性贫血、膜性肾病、肾病综合征等疾病的一线或二线治疗药物。但国内临床试验的研究时间普遍较短,对于患者远期获益的探索尚不充分。对于系统性红斑狼疮、视神经脊髓炎谱系疾病等仅获得初步阳性结果的病种,有待开展长期、多中心的大规模随机对照双盲试验,以验证先前结论的准确性。现结合国内外获批情况,对国内有关利妥昔单抗的主要超适应证临床研究进展进行综述。

Rituximab is originally approved for the treatment of non-Hodgkin lymphoma and is commonly used offlabelled in China.With the in-depth development of clinical studies in China,the medical evidence of rituximab off-label use has gradually improved,and rituximab has been recommended as the first-line or second-line treatment for idiopathic thrombocytopenic purpura,autoimmune hemolytic anemia,membranous nephropathy,nephrotic syndrome and other diseases.However,the duration of clinical trials is generally short,and the exploration of long-term benefits for patients is insufficient.For other diseases,such as systemic lupus erythematosus and neuromyelitis optica spectrum diseases with only preliminary positive results,large-scale long-term multicenter randomized controlled double-blind trials are needed to verify the validity of previous conclusions.Combined with the approval situation in China and abroad,the major clinical studies on off-label use of rituximab in China have been reviewed in this article.