Malignant Hematology, H. Lee Moffitt Cancer Center and Research Institute, SRB 2nd Floor, 12902 USF Magnolia Drive, Tampa, FL, 33612, USA.
Morsani College of Medicine, University of South Florida, Tampa, FL, USA.
Curr Treat Options Oncol. 2018 Oct 27;19(12):67. doi: 10.1007/s11864-018-0581-6.
Chronic myelomonocytic leukemia (CMML) is an aggressive myeloid neoplasm in which treatment strategies with the capacity to improve survival are currently lacking. Clinical features are heterogeneous and although the overall prognosis is poor, survival can vary significantly between individuals. This reflects the need for an individualized treatment approach which incorporates accurate risk stratification. Though numerous prognostic scores exist, newer CMML-specific models incorporating molecular data should be favored. While asymptomatic, low-risk patients should be observed until their disease progresses, the majority of patients will require treatment. Due to a deficiency in treatments with disease-modifying capacity, any patient who requires treatment should be considered for enrollment in clinical trials evaluating novel therapeutic approaches. Allogeneic stem cell transplant (allo-SCT) remains the only current therapy with the potential to cure the disease and should be considered in most patients with intermediate- to high-risk disease. However, substantial risks are involved and, in part, because of advanced age at diagnosis, a minority of patients are candidates. Hypomethylating agents (HMAs) have become a preferred treatment approach, and should be used in those with cytopenias. Patients presenting with proliferative features can be treated with hydroxyurea to manage their symptoms and control leukocytosis, though HMAs can be incorporated as well, particularly in patients with higher risk disease. HMAs should also be considered in patients with a high burden of disease prior to proceeding with allo-SCT. Induction chemotherapy should be reserved for younger, healthy patients who have transformed to acute myeloid leukemia to induce remission prior to transplant. Supportive care utilizing transfusion support, erythropoiesis-stimulating agents, and infection prevention measures should be incorporated into the care of all patients.
慢性髓单核细胞白血病（CMML）是一种侵袭性髓系肿瘤，目前缺乏能够改善生存的治疗策略。临床特征具有异质性，虽然总体预后较差，但个体间的生存差异很大。这反映了需要采用个体化治疗方法，包括准确的风险分层。虽然存在许多预后评分，但应优先考虑纳入分子数据的新型 CMML 特异性模型。虽然无症状、低危患者应在疾病进展前观察，但大多数患者需要治疗。由于缺乏具有疾病修饰能力的治疗方法，任何需要治疗的患者都应考虑参加评估新型治疗方法的临床试验。异基因造血干细胞移植（allo-SCT）仍然是唯一具有治愈疾病潜力的治疗方法，应考虑用于大多数中高危疾病患者。然而，存在很大的风险，部分原因是诊断时年龄较大，少数患者适合。低甲基化剂（HMAs）已成为首选的治疗方法，应在有细胞减少症的患者中使用。有增生特征的患者可以用羟基脲治疗以控制症状和白细胞增多，但也可以合并使用 HMAs，特别是在高危疾病患者中。在进行 allo-SCT 之前，也应考虑在疾病负担较高的患者中使用 HMAs。诱导化疗应保留给年轻、健康的患者，这些患者已经转化为急性髓系白血病，以便在移植前诱导缓解。应将输血支持、促红细胞生成素刺激剂和感染预防措施等支持性护理纳入所有患者的护理中。
