Sasaki Koji, Strom Sara S, O'Brien Susan, Jabbour Elias, Ravandi Farhad, Konopleva Marina, Borthakur Gautam, Pemmaraju Naveen, Daver Naval, Jain Preetesh, Pierce Sherry, Kantarjian Hagop, Cortes Jorge E
Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX, USA; Department of Hematology, Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University, Tokyo, Japan.
Department of Epidemiology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Lancet Haematol. 2015 May;2(5):e186-93. doi: 10.1016/S2352-3026(15)00048-4. Epub 2015 Apr 20.
Tyrosine-kinase inhibitors improve overall survival in patients with chronic myeloid leukaemia in chronic phase (CML-CP). Survival compared with the general population by age, response, and type of tyrosine-kinase inhibitor is not known. With use of data from trials of tyrosine kinase inhibitors, we compared overall survival in patients with newly diagnosed CML-CP to that of general population.
In this cohort analysis, we included data from patients with CML-CP enrolled in six consecutive or parallel prospective clinical trials of tyrosine-kinase inhibitors at a single institution from July 30, 2000, to Sept 17, 2012. We analysed data for response and survival with the Kaplan-Meier method. For estimated overall survival in the general population, we obtained data from national vital statistics reports and matched to patients with CML-CP by age, sex, ethnicity, and year at diagnosis. We assessed numbers and causes of death within 1 year of beginning treatment by age group and by response to therapy. We then did univariate analysis and multivariate analysis to investigate factors associated with survival probability.
Our analysis included 483 patients, 271 received imatinib, 105 received nilotinib, and 107 received dasatinib. Most patients were younger than 65 years, and no patients were older than 85 years. Median follow-up was 99·4 months (IQR 44·9-121·6), by which time 53 (11%) patients had died. The most common causes of death were progression to advanced disease stage, including complications of stem-cell transplantation (17 [4%] patients), secondary malignancies (nine [2%] patients), and cardiovascular causes (nine [2%] patients). 5-year overall survival in patients with CML-CP decreased in older age categories. For the whole population of patients with CML-CP, 5-year survival was only slightly lower than that of the matched general population (relative survival 94·7% [95% 92·1-97·4]). Individuals of all ages with a report of complete cytogenetic response to treatment or deeper within 1 year had a 5-year survival similar to that of the general population.
In the era of treatment with tyrosine-kinase inhibitors, patients diagnosed with CML-CP can expect a 5-year survival that is only slightly lower than that of the general population. With access to tyrosine-kinase inhibitors, most patients with chronic myeloid leukaemia could enjoy a near normal life expectancy.
MD Anderson Cancer Center, National Cancer Institute.
酪氨酸激酶抑制剂可提高慢性期慢性髓性白血病（CML-CP）患者的总生存率。目前尚不清楚按年龄、反应及酪氨酸激酶抑制剂类型与普通人群相比的生存率情况。利用酪氨酸激酶抑制剂试验的数据，我们比较了新诊断的CML-CP患者与普通人群的总生存率。
在这项队列分析中，我们纳入了2000年7月30日至2012年9月17日在单一机构进行的六项连续或平行的酪氨酸激酶抑制剂前瞻性临床试验中登记的CML-CP患者的数据。我们采用Kaplan-Meier方法分析反应和生存数据。对于普通人群的估计总生存率，我们从国家生命统计报告中获取数据，并按年龄、性别、种族和诊断年份与CML-CP患者进行匹配。我们按年龄组和治疗反应评估开始治疗后1年内的死亡人数和死因。然后我们进行单因素分析和多因素分析以研究与生存概率相关的因素。
我们的分析纳入了483例患者，271例接受伊马替尼治疗，105例接受尼罗替尼治疗，107例接受达沙替尼治疗。大多数患者年龄小于65岁，无患者年龄大于85岁。中位随访时间为99.4个月（四分位间距44.9 - 121.6），此时有53例（11%）患者死亡。最常见的死因是进展至疾病晚期，包括干细胞移植并发症（17例[4%]患者）、继发性恶性肿瘤（9例[2%]患者）和心血管原因（9例[2%]患者）。CML-CP患者的5年总生存率在老年类别中降低。对于所有CML-CP患者群体，5年生存率仅略低于匹配的普通人群（相对生存率94.7%[95%CI 92.1 - 97.4]）。所有年龄组中在1年内报告有完全细胞遗传学反应或更深反应的个体的5年生存率与普通人群相似。
在酪氨酸激酶抑制剂治疗时代，诊断为CML-CP的患者预期5年生存率仅略低于普通人群。有了酪氨酸激酶抑制剂，大多数慢性髓性白血病患者可以享有接近正常的预期寿命。
MD安德森癌症中心、美国国立癌症研究所。
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