摘要
目的评价克拉屈滨超说明书用药治疗儿童复发/难治性朗格汉斯细胞组织细胞增生症的合理性。方法查阅国内外网站克拉屈滨的最新版药品说明书、儿童朗格汉斯细胞组织细胞增生症的诊治指南或诊疗规范,收集克拉屈滨的全部适应证。计算机检索PubMed、Embase、Cochrane Library、Clinical trials、CNKI、Sino-Med、VIP和万方数据库,检索时限为1993年1月1日至2020年03月27日。收集含克拉屈滨方案治疗儿童复发/难治性朗格汉斯细胞组织细胞增生症的研究文献,并对其治疗儿童复发/难治性朗格汉斯细胞组织细胞增生症的有效性和安全性进行系统评价。结果国内外药品说明书均未批准克拉屈滨用于治疗朗格汉斯细胞组织细胞增生症的适应证;有2篇指南包含克拉屈滨治疗儿童复发/难治性朗格汉斯细胞组织细胞增生症内容,其中1篇指南为高质量;有13篇病例系列研究纳入分析,4项研究为设计良好的高质量研究报道,9篇为低质量病例系列报道。结论克拉屈滨单药在治疗非危险器官累及的复发/难治性朗格汉斯细胞组织细胞增生症患者方面具有较高治疗反应率,但并不足以改变重症患者的最终预后。克拉屈滨联合阿糖胞苷在治疗复发/难治性朗格汉斯细胞组织细胞增生症方面表现出较高的反应率和优良的5年预期生存率,但患者需耐受更高强度的药物毒性反应及更高水平的支持治疗。
OBJECTIVE To evaluate the rationality of off-label drug use of cladribine in treatment of recurrent/refractory Langerhans cell histiocytosis in children.METHODS The latest edition of drug labels,clinical practice guideline about Langerhans cell histiocytosis(LCH)and rules for clinical medical were retrieved,to collect all indications of cladribine.And PubMed,Embase,Cochrane Library,Clinical trials,CNKI,CBM,VIP and Wanfang databases were searched from January 1,1993 to March 27,2020 to collect all public literatures involved cladribine in treatment of relapsed/refractory LCH in children.And then the efficacy and safety of the drug in treatment of relapsed/refractory LCH were reviewed systematically.RESULTS The latest instruction of cladribine was not approved that the drug could be used to treat LCH.However,two clinical guidelines recommending cladribine was used to treatment of recurrent/refractory LCH as second line regimen or salvage therapy.Although there were no randomized controlled trials(RCT)or well-designed nonrandomized controlled trials were found,there were 13 case series study articles were retrieved for efficacy and safety analysis.CONCLUSION Cladribine as monotherapy is significantly more effective in the treatment of patients with non-risk organ involved,but it is not sufficient to change the final fate of the patients with most severe disease.While,to date,is the best available salvage option for patients with recurrent/refractory diseases cladribine combined with cytarabine which showed extremely response rate and excellent 5-year predicted survival rate besides high treatment toxicity and required highest level of supportive therapy.
作者
邢亚兵
马姝丽
张胜男
张淼
王晓玲
XING Ya-bing;MA Shu-li;ZHANG Sheng-nan;ZHANG Miao;WANG Xiao-ling(Department of Pharmacy,Children's Hospital Affiliated to Zhengzhou University,Zhengzhou 450018,China;Department of Pharmacy,Beijing Children fs Hospital Affiliated to the Capital Medical University,Beijing 100045,China)
出处
《中国药学杂志》
CAS
CSCD
北大核心
2020年第22期1899-1905,共7页
Chinese Pharmaceutical Journal
基金
国家科技部重大专项课题资助(2018ZX09721003)
儿童用药处方审核规范的建立项目资助[国家卫健委医管中心药械处(2019)053号]。
