Cannella Laura, Caocci Giovanni, Jacobs Marc, Vignetti Marco, Mandelli Franco, Efficace Fabio
Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), Rome, Italy.
Department of Medical Sciences, University of Cagliari, Cagliari, Italy.
Crit Rev Oncol Hematol. 2015 Dec;96(3):542-54. doi: 10.1016/j.critrevonc.2015.07.012. Epub 2015 Aug 1.
Health-related quality of life (HRQOL) and other patient-reported outcomes (PROs) are crucial for a comprehensive evaluation of treatment effectiveness. A systematic review of randomized controlled trials (RCTs) with a PRO endpoint conducted in patients with leukemia and myelodysplastic syndromes (MDS) was performed. Eligible studies were evaluated independently, according to a pre-defined coding scheme, by two reviewers. Thirteen RCTs, enrolling overall 3380 patients were identified. There were four RCTs involving acute myeloid leukemia patients (AML), one with acute lymphoid leukemia (ALL), five with chronic lymphocytic leukemia (CLL) and three with MDS. Six RCTs accurately documented PRO methodology assessment and were thus considered likely to robustly inform clinical decision-making. Of these, three RCTs dealt with AML, two with CLL, one with MDS. A growing number of RCTs in leukemia and MDS have included a PRO component in recent years. Inclusion of PROs in RCTs is feasible and can provide unique information to facilitate clinical decision-making.
健康相关生活质量（HRQOL）及其他患者报告结局（PROs）对于全面评估治疗效果至关重要。我们对在白血病和骨髓增生异常综合征（MDS）患者中开展的以PRO为终点的随机对照试验（RCTs）进行了系统综述。两名评审员根据预先定义的编码方案对符合条件的研究进行独立评估。共识别出13项RCTs，总计纳入3380例患者。其中有4项RCTs涉及急性髓系白血病（AML）患者，1项涉及急性淋巴细胞白血病（ALL），5项涉及慢性淋巴细胞白血病（CLL），3项涉及MDS。6项RCTs准确记录了PRO方法学评估，因此被认为可能为临床决策提供有力依据。其中，3项RCTs针对AML，2项针对CLL，1项针对MDS。近年来，越来越多的白血病和MDS的RCTs纳入了PRO部分。在RCTs中纳入PROs是可行的，并且可以提供独特信息以促进临床决策。
