Jena University Hospital, Jena, Germany.
Hematology Am Soc Hematol Educ Program. 2011;2011:128-35. doi: 10.1182/asheducation-2011.1.128.
Elucidation of the pathogenesis of chronic myeloid leukemia (CML) and the introduction of tyrosine kinase inhibitors (TKIs) has transformed this disease from being invariably fatal to being the type of leukemia with the best prognosis. Median survival associated with CML is estimated at > 20 years. Nevertheless, blast crisis occurs at an incidence of 1%-2% per year, and once this has occurred, treatment options are limited and survival is short. Due to the overall therapeutic success, the prevalence of CML is gradually increasing. The optimal management of this disease includes access to modern therapies and standardized surveillance methods for all patients, which will certainly create challenges. Furthermore, all available TKIs show mild but frequent side effects that may require symptomatic therapy. Adherence to therapy is the key prerequisite for efficacy of the drugs and for long-term success. Comprehensive information on the nature of the disease and the need for the continuous treatment using the appropriate dosages and timely information on efficacy data are key factors for optimal compliance. Standardized laboratory methods are required to provide optimal surveillance according to current recommendations. CML occurs in all age groups. Despite a median age of 55-60 years, particular challenges are the management of the disease in children, young women with the wish to get pregnant, and older patients. The main challenges in the long-term management of CML patients are discussed in this review.
阐明慢性髓性白血病(CML)的发病机制和引入酪氨酸激酶抑制剂(TKI)已经将这种疾病从必然致命转变为预后最好的白血病类型。CML 的中位生存期估计超过 20 年。然而,每年有 1%-2%的患者发生急变期,一旦发生这种情况,治疗选择有限,生存期短。由于整体治疗成功,CML 的患病率逐渐增加。这种疾病的最佳管理包括为所有患者提供现代治疗方法和标准化的监测方法,这肯定会带来挑战。此外,所有可用的 TKI 都有轻微但频繁的副作用,可能需要对症治疗。对治疗的坚持是药物疗效和长期成功的关键前提。全面了解疾病性质和持续使用适当剂量和及时了解疗效数据的治疗需求是最佳依从性的关键因素。根据当前建议,需要标准化的实验室方法来提供最佳监测。CML 发生在所有年龄段。尽管中位年龄为 55-60 岁,但儿童、有怀孕愿望的年轻女性和老年患者的疾病管理是特别的挑战。本文讨论了 CML 患者长期管理中的主要挑战。