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促红细胞生成素:实践指南制定的范例

Erythropoietin: a paradigm for the development of practice guidelines.

作者信息

Rizzo J D, Seidenfeld J, Piper M, Aronson N, Lichtin A, Littlewood T J

机构信息

IBMTR/ABMTR, Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee WI 53226-4801, USA.

出版信息

Hematology Am Soc Hematol Educ Program. 2001:10-30. doi: 10.1182/asheducation-2001.1.10.

DOI:10.1182/asheducation-2001.1.10
PMID:11722976
Abstract

Erythropoietin (EPO) is an endogenous hormone produced in the kidney that regulates red blood cell production within the body. Since the cloning and first clinical introduction of recombinant erythropoietin (epoetin) in the late 1980s indications and usage of epoetin have expanded significantly. It is estimated that as many as one third of patients with substantial anemia (hemoglobin less than 10.0 g/dL) resulting from chemotherapy for cancer are treated with epoetin. Though use of epoetin may avoid the inconvenience and infectious risk of blood transfusions, it is expensive and its benefit in some clinical scenarios has been modest. Like many new technologies, strong evidence suggesting situations where the benefit is high has lagged behind its adoption by patients and practitioners. As well, epoetin is expensive and third party payers do not always reimburse it. Research suggests there is considerable variation in epoetin usage in practice. To provide guidance to hematology/oncology specialists regarding use of epoetin, the American Society of Hematology (ASH) and the American Society of Clinical Oncology (ASCO) proposed that the Agency for Healthcare Research and Quality (AHRQ) fund an evidence review by one of the Evidence-based Practice Centers (EPC) that would be used to develop evidence-based guidelines for members of the society. This review highlights principles of evidence-based medicine, distills and appraises the evidence in the published literature that supports the use of epoetin, and presents evidence-based recommendations for use of epoetin in situations where benefit is substantiated by high-quality studies. As well, this review addresses some of the difficulties of performing clinical research in this area, provocative research findings that will require further study, and suggestions regarding epoetin in those areas where further strong evidence has yet to be developed.

摘要

促红细胞生成素(EPO)是一种在肾脏中产生的内源性激素,它调节体内红细胞的生成。自20世纪80年代末重组促红细胞生成素(依泊汀)克隆成功并首次临床应用以来,依泊汀的适应证和用途已显著扩大。据估计,因癌症化疗导致严重贫血(血红蛋白低于10.0 g/dL)的患者中,多达三分之一接受依泊汀治疗。尽管使用依泊汀可避免输血带来的不便和感染风险,但它价格昂贵,且在某些临床情况下其益处并不显著。与许多新技术一样,有力证据表明其在某些情况下益处显著,但这一证据滞后于患者和从业者对它的采用。此外,依泊汀价格昂贵,第三方支付方并不总是给予报销。研究表明,依泊汀在实际应用中的使用情况存在很大差异。为了给血液学/肿瘤学专家提供关于依泊汀使用的指导,美国血液学会(ASH)和美国临床肿瘤学会(ASCO)提议医疗保健研究与质量局(AHRQ)资助其中一个循证实践中心(EPC)进行证据审查,该审查将用于为学会成员制定循证指南。本综述强调循证医学原则,提炼和评估已发表文献中支持使用依泊汀的证据,并针对高质量研究证实有益的情况下使用依泊汀提出循证建议。此外,本综述还探讨了该领域进行临床研究的一些困难、需要进一步研究的有争议的研究结果,以及在尚未有进一步有力证据的领域中关于依泊汀的建议。

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