Goldberg Stuart L
John Theurer Cancer Center at Hackensack University Medical Center, Hackensack NJ; and Cancer Outcomes Tracking and Analysis (COTA), Inc, New York, NY.
Clin Lymphoma Myeloma Leuk. 2015 Dec;15(12):711-4. doi: 10.1016/j.clml.2015.08.088. Epub 2015 Sep 2.
In clinical trials, in which treatment algorithms and monitoring schedules are tightly prescribed by research protocols, outcomes for patients with chronic myeloid leukemia (CML) have been excellent, with > 90% 5-year survival rates. However, outside of clinical trials in the so-called real world, monitoring schedules are more variable, with < 40% of patients undergoing quantitative polymerase chain reaction (qPCR) molecular testing 3 to 4 times during the first year after diagnosis as recommended by National Comprehensive Cancer Network/European Leukemia Net (NCCN/ELN) evidence-based guidelines. Results from chart reviews, claims-based databases, and observational databases suggest that carefully monitored patients with CML are more likely to be adherent to medications, incur fewer hospitalizations, experience lower overall treatment costs, and have better progression-free survival and overall survival compared with patients who are not monitored. Regular monitoring provides valuable early information on treatment responses that physicians can use to modify treatment. Unfortunately, physician-perceived resource barriers, lack of familiarity, and lack of agreement have restricted monitoring guideline adoptions. Multifaceted approaches to encourage appropriate monitoring are needed to improve clinical outcomes and reduce costs in the real world.
在临床试验中，治疗方案和监测计划由研究方案严格规定，慢性髓性白血病（CML）患者的治疗结果非常出色，5年生存率超过90%。然而，在所谓的现实世界中，临床试验之外的监测计划则更为多变，只有不到40%的患者在诊断后的第一年按照美国国立综合癌症网络/欧洲白血病网（NCCN/ELN）循证指南的建议进行3至4次定量聚合酶链反应（qPCR）分子检测。病历审查、基于索赔的数据库和观察性数据库的结果表明，与未接受监测的患者相比，接受仔细监测的CML患者更有可能坚持服药，住院次数更少，总体治疗成本更低，无进展生存期和总生存期也更好。定期监测可提供有关治疗反应的有价值的早期信息，医生可据此调整治疗方案。不幸的是，医生所感知到的资源障碍、缺乏熟悉度以及缺乏共识限制了监测指南的采用。需要采取多方面的方法来鼓励进行适当的监测，以改善现实世界中的临床结果并降低成本。
