Center for Evidence-Based Medicine and Health Outcome Research, University of South Florida, USA.
Acta Haematol. 2011;125(1-2):8-22. doi: 10.1159/000318880. Epub 2010 Dec 8.
Individual studies rarely provide definitive answers to questions related to the effects of treatments. Whether the treatment is associated with more good than harm is best answered by considering the totality of evidence on the topic through the methodology of systematic reviews. The objective of this overview is to summarize all existing systematic reviews on treatments in multiple myeloma (MM), which accounts for 14% of new cases of hematological malignancies each year. Therefore, MEDLINE and the Cochrane Database of Systematic Reviews were systematically searched to identify systematic reviews of interventions. Data were extracted on patients, interventions, control and outcomes. Methodological quality of the systematic reviews was assessed using the AMSTAR assessment tool. Eleven systematic reviews on treatment of MM were included in the overview. Ten addressed seven unique questions and also performed a meta-analysis. One addressed 21 clinical questions related to treatment decisions in myeloma. The quality of systematic reviews varied. The results from the overview show that early treatment does not offer survival benefit. Thalidomide is associated with improved survival when added to standard chemotherapy regimens as induction or maintenance therapy but at the expense of an increased risk of serious adverse events, such as venous thromboembolism. High-dose therapy with single autologous hematopoietic stem cell transplant (AHCT) is associated with superior event-free but not overall survival compared to chemotherapy. Tandem AHCT does not prolong survival but is associated with better event-free survival in comparison to single AHCT. In addition, combination treatment with bisphosphonates reduces pathological vertebral fractures and pain, but does not prolong survival. We found no systematic review evaluating the effects of other novel agents, such as bortezomib or lenalidomide, as single agents or in combinations. Several key clinical questions have been successfully answered by conducting systematic reviews. However, currently many questions of importance for the management of patients with myeloma continue to be dealt with in individual studies instead of synthesized evidence. There is urgent need to perform research synthesis of data related to the effects of novel agents.
个体研究很少能对与治疗效果相关的问题提供明确的答案。治疗是否比伤害更有益，最好通过系统评价的方法，综合考虑该主题的所有证据来回答。本综述的目的是总结多发性骨髓瘤（MM）治疗的所有现有系统评价，每年约有 14%的新血液系统恶性肿瘤患者患有 MM。因此，系统地检索了 MEDLINE 和 Cochrane 系统评价数据库，以确定干预措施的系统评价。提取了患者、干预措施、对照和结局的数据。使用 AMSTAR 评估工具评估系统评价的方法学质量。本综述共纳入了 11 项关于 MM 治疗的系统评价。其中 10 项针对 7 个独特的问题进行了系统评价和荟萃分析。1 项针对与骨髓瘤治疗决策相关的 21 个临床问题。系统评价的质量参差不齐。本综述的结果表明，早期治疗并不能带来生存获益。沙利度胺作为诱导或维持治疗与标准化疗方案联合应用可提高生存率，但增加了静脉血栓栓塞等严重不良事件的风险。与化疗相比，高剂量治疗联合单倍体自体造血干细胞移植（AHCT）可提高无事件生存率，但不能提高总生存率。与单倍体 AHCT 相比，串联 AHCT 不能延长生存时间，但可提高无事件生存率。此外，联合使用双膦酸盐可减少病理性椎体骨折和疼痛，但不能延长生存时间。我们没有发现评估硼替佐米或来那度胺等新型药物单药或联合应用效果的系统评价。通过进行系统评价，已经成功回答了几个关键的临床问题。然而，目前许多对骨髓瘤患者管理重要的问题仍在进行个体化研究，而不是综合证据。迫切需要对与新型药物效果相关的数据进行研究综合。
