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的循证药学评价

邢亚兵, 马姝丽, 张胜男, 张淼, 王晓玲

中国药学杂志 ›› 2020, Vol. 55 ›› Issue (22) : 1899-1905.

PDF(1151 KB)
中国药学杂志
PDF(1151 KB)
中国药学杂志 ›› 2020, Vol. 55 ›› Issue (22) : 1899-1905. DOI: 10.11669/cpj.2020.22.012
论著

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的循证药学评价

  • 邢亚兵1, 马姝丽1, 张胜男1, 张淼1, 王晓玲2*
作者信息 +

Evidence-based Pharmacy Evaluation of 2-Chlorodeoxyadenosine in the Therapy of Pediatric Recurrent/Refractory Langerhans Cell Histiocytosis

  • XING Ya-bing1, MA Shu-li1, ZHANG Sheng-nan1, ZHANG Miao1, WANG Xiao-ling2*
Author information +
文章历史 +

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。

Abstract

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.

关键词

克拉屈滨 / 儿童 / 朗格汉斯细胞组织细胞增生症 / 循证药学

Key words

Cladribine / children / Langerhans cell histiocytosis / evidence-based pharmacy

引用本文

EndNote

Ris (Procite)

Bibtex

导出引用
邢亚兵, 马姝丽, 张胜男, 张淼, 王晓玲. 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的循证药学评价[J]. 中国药学杂志, 2020, 55(22): 1899-1905 https://doi.org/10.11669/cpj.2020.22.012
XING Ya-bing, MA Shu-li, ZHANG Sheng-nan, ZHANG Miao, WANG Xiao-ling. Evidence-based Pharmacy Evaluation of 2-Chlorodeoxyadenosine in the Therapy of Pediatric Recurrent/Refractory Langerhans Cell Histiocytosis[J]. Chinese Pharmaceutical Journal, 2020, 55(22): 1899-1905 https://doi.org/10.11669/cpj.2020.22.012
中图分类号： R979.1+2   

参考文献

[1] ZHENG R, PENG X, ZENG H, et al. Incidence, mortality and survival of childhood cancer in China during 2000-2010 period: a population-based study[J]. Cancer Lett, 2015,363(2):176-180.
[2] HOWLADER N, NOONE A, KRAPCHO M, et al. SEER Cancer Statistics Review, 1975-2010[M]. Bethesda: National Cancer Institute Updated June 14, 2013.
[3] MA J, WANG Y, LU B, et al. Investigation and analysis of medication information labeling in 157 package inserts of anticancer drugs [J]. J China Pharm(中国药房),2019,30(7):985-990.
[4] ZHANG L L, LIANG Y, HU D, et al. Definition and literature of evidence-based pharmacy: a systematic review[J]. Chin J Evid-based Med(中国循证医学杂志),2011,11(1):7-13.
[5] FANG H, ZHANG J. Technological process with cochrane library of reasonability assessment of off-label uses of drugs found in comments on prescription [J]. Chin Pharm J(中国药学杂志),2015,50(23):2090-2094.
[6] ABLA O, EGELER R M, WEITZMAN S. Langerhans cell histiocytosis: current concepts and treatments[J]. Cancer Treat Rev, 2010, 36(4):354-359.
[7] MICHALINA J, JOANNA S, GRZEGORZ R, et al. Langerhans cell histiocytosis in children-a disease with many faces. Recent advances in pathogenesis, diagnostic examinations and treatment[J]. Postepy Dermatol Alergol, 2018, 35(1):6-17.
[8] SAVEN A, FIGUEROA M L, PIRO L D, et al. 2-Chlorodeoxyadenosine to treat refractory histiocytosis X[J]. New Engl J Med, 1993, 329(10):734-735.
[9] SAVEN A, BURIAN C. Cladribine activity in adult Langerhans-cell histiocytosis[J]. Blood, 1999, 93(12):4125-4130.
[10] RODRIGUEZ-GALINDO C, JENG M, KHUU P, et al. Clofarabine in refractory Langerhans cell histiocytosis[J]. Pediatr Blood Cancer, 2008, 51(5):703-706.
[11] IMAMURA T, SATO T, SHIOTA Y, et al. Outcome of pediatric patients with Langerhans cell histiocytosis treated with 2 chlorodeoxyadenosine: a nationwide survey in Japan[J]. Int J Hematol, 2010, 91(4):646-651.
[12] WEITZMAN S,BRAIER J,DONADIEU J,et al.2′-Chlorodeoxyadenosine (2-CdA) as salvage therapy for Langerhans cell histiocytosis (LCH):results of the LCH-S-98 protocol of the Histiocyte Society[J]. Pediatr Blood Cancer, 2009, 53(7):1271-1276.
[13] HU Z Q,QI T T,JIANG Q,et al. Appraisal of guidelines on drug prevention for cardiotoxicity of cancer therapy with the AGREE Ⅱ tool[J]. Chin J Pharmacoepidemiol(药物流行病学杂志), 2020, 29(4):268-275.
[14] WANG X Q, CHEN Y L, QU Q Y, et al. Interpretation of the methodological quality assessment tool in case series study[J]. Chin J Evid Based Pediatr(中国循证儿科杂志),2015,10(5):381-385.
[15] ZENG X T, CUI Y M, FENG J J, et al. The basic methods and procedures of development/revision of the evidence-based evaluation of off-label drug use[J]. J Chin Res Hosp(中国研究型医院), 2017, 4(5):35-39.
[16] ZHAO R S, ZHAI S D. Hospital pharmacy and evidence-based pharmacy[J]. Chin Pharm J(中国药学杂志),2007,42(24):1922-1924.
[17] HAUPT R, MINKOV M, ASTIGARRAGA I, et al. Langerhans cell histiocytosis (LCH): guidelines for diagnosis, clinical work-up, and treatment for patients till the age of 18 years[J]. Pediatr Blood Cancer, 2013, 60(2):175-184.
[18] HISTIOCYTE SOCIETY. Langerhans cell histiocytosis evaluation and treatment guideline. April,2009. Available from: http://www.heamatologie-amc.nl/odijk/bijlagen.
[19] STINE K C, SAYLORS R L, WILLIAMS L L, et al. 2-Chlorodeoxyadenosine (2-CDA) for the treatment of Langerhans cell histiocytosis (LCH) in pediatric patients[J]. Med Pediatr Oncol, 1997, 29(4):288-292.
[20] WEITZMAN S, WAYNE A S, ARCECI R, et al. Nucleoside analogues in the therapy of Langerhans cell histiocytosis: a survey of members of the histiocyte society and review of the literature[J]. Med Pediatr Oncol, 1999, 33(5):476-481.
[21] RODRIGUEZ-GALINDO C, KELLY P, JENG M, et al. Treatment of children with Langerhans cell histiocytosis with 2-chlorodeoxyadenosine[J]. A J Hematol, 2002, 69(3):179-184.
[22] STINE K C, SAYLORS R L, SACCENTE S, et al. Efficacy of continuous infusion 2-CDA (cladribine) in pediatric patients with Langerhans cell histiocytosis[J]. Pediatr Blood Cancer, 2004, 43(1):81-84.
[23] BERNARD F, THOMAS C, BERTRAND Y, et al. Multi-center pilot study of 2-chlorodeoxyadenosine and cytosine arabinoside combined chemotherapy in refractory Langerhans cell histiocytosis with hematological dysfunction[J]. Eur J Cancer, 2005, 41(17):2682-2689.
[24] MOTTL H, STARY J, CHANOVA M, et al. Treatment of recurrent Langerhans cell histiocytosis in children with 2-chlorodeoxyadenosine[J]. Leuk Lymphoma, 2006,47(9):1881-1884.
[25] BISWAS G, KHADWAL A, ARORA B, et al. Activity and toxicity of 2-CDA in Langerhans cell histiocytosis: a single institutional experience[J]. Indian J Cancer, 2007,44(4):137-141.
[26] DHALL G, FINLAY J L, DUNKEL I J, et al. Analysis of outcome for patients with mass lesions of the central nervous system due to Langerhans cell histiocytosis treated with 2-chlorodeoxyadenosine[J]. Pediatr Blood Cancer, 2008,50(1):72-79.
[27] HU T, LIU R, LI J H, et al. Cladribine for 13 cases refractory high-risk children Langerhans cell histiocytosis[J]. Chin J Hematol(中华血液学杂志), 2014, 35(11):985-989.
[28] DONADIEU J, BERNARD F, NOESEL M, et al. Cladribine and cytarabine in refractory multisystem Langerhans cell histiocytosis: results of an international phase 2 study[J]. Blood, 2015,126(12):1415-1423.
[29] ROSSO D A, AMARAL D, LATELLA A, et al. Reduced doses of cladribine and cytarabine regimen was effective and well tolerated in patients with refractory-risk multisystem Langerhans cell histiocytosis[J]. Br J Haematol, 2016,172(2):287-290.
[30] CHEN Y L, LI Y P, DU L, et al. Evolution of levels of evidence and strength of recommendations in medical research[J]. Chin J Evid-based Med(中国循证医学杂志),2008,8(2):127-133.
[31] GADNER H, GROIS N, POTSCHGER U, et al. Improved outcome in multisystem Langerhans cell histiocytosis is associated with therapy intensification[J]. Blood, 2008, 111(5):2556-2562.
[32] GADNER H, GROIS N, ARICO M, et al. A randomized trial of treatment for multisystem Langerhans′ cell histiocytosis[J]. J Pediatr, 2001,138(5):728-734.
[33] BRAIER J L, ROSSO D, LATELLA A, et al. Importance of multi-lineage hematologic involvement and hypoalbuminemia at diagnosis in patients with "risk-organ" multi-system Langerhans cell histiocytosis[J]. J Pediatr Hematol Oncol, 2010,32(4):e122-e125.
[34] HUANG Z Z, ZHAO W H. Langerhans cell histiocytosis treated by nucleoside analogues in children[J]. J China Pediatr Blood Cancer(中国小儿血液与肿瘤杂志),2014,19(6):283-286.
[35] ZHAO S, SONG Y Q, WANG X F, et al. Analysis of pediatric use information in package inserts of antineoplastic drugs[J]. Chin J Pharmacov(中国药物警戒),2019,16(9):555-560.
[36] MALLEN C, PEAT G, CROFT P. Quality assessment of observational studies is not commonplace in systematic reviews[J]. J Clin Epidemiol, 2006,59(8):765-769.

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]
PDF(1151 KB)

Accesses

Citation

Detail
段落导航
相关文章

/

〈 〉