Evidence- and consensus-based practice guidelines for the therapy of primary myelodysplastic syndromes. A statement from the Italian Society of Hematology.
Evidence- and consensus-based practice guidelines for the therapy of primary myelodysplastic syndromes. A statement from the Italian Society of Hematology.
复制标题
原发性骨髓增生异常综合征治疗的基于证据和共识的实践指南。
作者：
E. Alessandrino;S. Amadori;G. Barosi;M. Cazzola;A. Grossi;L. Liberato;F. Locatelli;M. Marchetti;E. Morra;P. Rebulla;G. Visani;S. Tura
背景和目标
近年来，骨髓增生异常综合征（MDS）的治疗中引入了新的治疗药物和策略。这为MDS患者带来了更多的治疗选择和更好的长期生存机会，但也带来了关于这些治疗的最佳使用和可能的副作用的不确定性。意大利血液学学会委托开展了一个项目，利用循证知识和共识形成技术制定骨髓增生异常综合症治疗指南。
设计和方法
一个咨询理事会（AC）围绕一系列关键的临床问题塑造了该项目，进行了系统的证据搜索，并根据苏格兰校际指南网络（SIGN）对现有证据进行了分级。将临床问题列表邮寄给组成专家小组（EP）的10名高级血液学家：要求小组成员对最相关的问题进行排名，并根据证据表制定问题的答案。随后是设想阶段，以便就三个最重要的问题达成共识。根据兰德技术，要求EP对患者特征进行评分，认为其适合或不适合接受审查的治疗策略。最后，从2001年9月至2002年1月，在意大利米兰举行了四次根据名义团体技术举行的协商一致会议。会议的总体目标是对不确定情景的适当性和对临床问题的不确定反应做出最终决定。
结果
经判断，有足够的证据提供关于使用异基因干细胞移植、白血病样化疗、自体干细胞移植、低剂量化疗、达那唑、免疫抑制治疗、低甲基化剂和造血生长因子的建议。发表了支持性治疗的具体建议，包括铁螯合。异基因干细胞移植被一致认为是MDS患者的唯一治愈性治疗方法，并根据患者的年龄，风险，临床特征和供体可用性对其使用的建议达成一致。AML样化疗也被认为是MDS患者亚群的有价值的治疗选择。自体造血干细胞移植被推荐用于缺乏HLA相同供体且经AML样化疗达到完全缓解的患者。地西他滨、重组人促红细胞生成素和免疫抑制治疗被认为是MDS患者亚群有价值的治疗选择，而低剂量阿糖胞苷则不是。对于年龄小于18岁或大于75岁的受试者，具体的治疗策略和观察等待策略由患者导向的问题决定。
解释和结论
使用证据和共识，提出了MDS治疗的建议。根据支持证据的强度对陈述进行分级，并明确宣布不确定性。
BACKGROUND AND OBJECTIVES
Novel therapeutic agents and strategies have been introduced into the management of myelodysplastic syndromes (MDS) in the last years. This has led to more treatment options and a better chance of long-term survival for MDS patients, but also to uncertainty regarding the optimal use and possible side effects of these treatments. The Italian Society of Hematology commissioned a project to develop guidelines for the therapy of MDS using evidence-based knowledge and consensus-formation techniques.
DESIGN AND METHODS
An Advisory Council (AC) shaped the project around a series of key clinical questions, performed a systematic search for evidence and graded the available evidence according to the Scottish Intercollegiate Guidelines Network (SIGN). A list of clinical questions was mailed to each of 10 senior hematologists composing the Expert Panel (EP): the panelists were asked to rank the most relevant questions, and to formulate answers to the questions according to the tables of evidence. A scenario phase followed, so as to reach a consensus on the three top ranked questions. The EP was asked to score patient profiles as appropriate or not appropriate for the therapeutic strategy under scrutiny, according to the RAND technique. Finally, from September 2001 to January 2002, four Consensus Conferences conducted according to the Nominal Group Technique were held in Milan, Italy. The overall goal of the conferences was to take a final decision upon the appropriateness of the uncertain scenarios and of the uncertain responses to the clinical questions.
RESULTS
Evidence was judged sufficient for providing recommendations on the use of allogeneic stem cell transplantation, leukemia-like chemotherapy, autologous stem cell transplantation, low-dose chemotherapy, danazol, immunosuppressive therapy, hypomethylating agents and hematopoietic growth factors. Specific recommendations for supportive therapy, including iron chelation, were issued. Allogeneic stem cell transplantation was unanimously considered as the only curative treatment for MDS patients, and recommendations on its use were agreed based on patient's age, risk, clinical features and donor availability. AML-like chemotherapy was also considered a valuable therapeutic option for subsets of MDS patients. Autologous stem cell transplantation was recommended for patients who lack an HLA identical donor and have achieved complete remission with AML-like chemotherapy. Decitabine, recombinant human erythropoietin and immunosuppressive therapy were judged valuable therapeutic options for subsets of MDS patients whereas low-dose cytarabine was not. Specific therapeutic strategies for those subjects younger than 18 years or older than 75 years and the strategy of watchful waiting were decided by patient-oriented questions.
INTERPRETATION AND CONCLUSIONS
Using evidence and consensus, recommendations for the treatment of MDS were issued. Statements were graded according to the strength of the supporting evidence and uncertainty was explicitly declared.
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