Medical Oncology, Institute of Cancer, Barts and London School of Medicine, Queen Mary University of London, Charterhouse Square, London EC1M 6BQ, United Kingdom.
Blood Rev. 2010 May;24(3):135-41. doi: 10.1016/j.blre.2010.03.004. Epub 2010 Apr 15.
CLL is extremely heterogeneous in its clinical course, with some patients living decades with no need for treatment whilst others have a rapidly aggressive clinical course. A major focus of research has been to try to identify those biological factors that influence this heterogeneity. The goal of therapy has been to maintain the best quality of life and treat only when patients become symptomatic from their disease. For the majority of patients this means following a "watch and wait" approach to determine the rate of progression of the disease and assess for development of symptoms. Any alteration to this approach will require identification of criteria that define patients sufficiently "high-risk" that they gain benefit by introduction of early therapy. The use of molecular profiling to suggest particular therapies is currently appropriate only in defining the treatment of the minority of patients with 17p deletions or p53 mutations and in all other circumstances remains a clinical trial question.
CLL 在其临床病程中具有极强的异质性，一些患者无需治疗即可生存数十年，而另一些患者则具有快速侵袭性的临床病程。研究的一个主要重点是试图确定影响这种异质性的生物学因素。治疗的目标是保持最佳的生活质量，并且仅在患者因疾病而出现症状时才进行治疗。对于大多数患者来说，这意味着采用“观察和等待”的方法来确定疾病的进展速度，并评估症状的发展。对这种方法的任何改变都需要确定定义患者的标准，这些患者具有足够的“高风险”，通过早期治疗可获益。目前，使用分子谱分析来提示特定的治疗方法仅适用于定义少数 17p 缺失或 p53 突变的患者的治疗，在所有其他情况下，这仍然是临床试验的问题。
