Whiteley Jennifer, Iyer Shrividya, Candrilli Sean D, Kaye James A
Pfizer Inc. , New York, NY , USA.
Curr Med Res Opin. 2015 Feb;31(2):299-314. doi: 10.1185/03007995.2014.991817. Epub 2015 Jan 30.
Given the multiple options for treatment of chronic-phase chronic myeloid leukemia (CML) with tyrosine kinase inhibitors, our objective was to understand treatment patterns in routine practice and prognostic indicators of response.
We conducted a retrospective medical record review of 681 patients with CML in Australia, Canada, and South Korea. Eligible patients had a diagnosis of chronic-phase CML, were Philadelphia chromosome and/or BCR-ABL positive, were aged 18 years or older, and had been treated with first-line imatinib therapy between January 2005 and September 2010. Data on patient demographics, medical history (e.g., comorbidities, Sokal score), and treatment characteristics (e.g., time to initiation, therapy duration) were abstracted. Descriptive analyses were stratified by country and therapy line. Prognostic indicators of response to imatinib were evaluated using multivariable logistic regression, adjusting for country, patient demographics, medical history, treatment characteristics, and side effects.
Hematologic, cytogenetic, and molecular responses at 3, 6, 12, and 18 months following initiation of each therapy line.
Patients' average age was 57 years, and 59% were male. Overall, imatinib was initiated approximately 4 months following CML diagnosis. Complete or major molecular response (C/MMR) at 6 months following imatinib initiation was 54% in Australia, 22% in Canada, and 38% in South Korea. At 18 months, over 60% of patients achieved C/MMR. Approximately 30% of patients discontinued imatinib primarily due to intolerance and lack of response. Among patients who received second-line treatment, dasatinib was used more frequently than nilotinib. Multivariable regression results indicated Sokal score was identified as a prognostic indicator of response to imatinib therapy at several time points.
There are several limitations to this study. First, we selected a convenience sample of patients and physicians and therefore results may not be representative of the true population of patients with chronic-phase CML. Second, data were entered by the selected physician and could be subject to data entry errors or inaccuracies. Third, limited information was collected from the patient records, and it is possible that we did not capture additional prognostic or confounding factors related to the measured outcomes. Next, because this was an analysis of previously documented data (i.e., retrospective), we were unable to provide a priori definitions of response. Finally, multivariable analyses were limited to imatinib-related outcomes.
Treatment patterns and prognostic indicators differed by country. Health care providers, payers, and patients can utilize these results to inform treatment and policies aimed at improving the effectiveness of care for patients with chronic-phase CML.
鉴于慢性期慢性髓性白血病（CML）酪氨酸激酶抑制剂治疗方案多样，我们的目的是了解常规治疗模式及反应的预后指标。
我们对澳大利亚、加拿大和韩国的681例CML患者进行了回顾性病历审查。符合条件的患者诊断为慢性期CML，费城染色体和/或BCR-ABL阳性，年龄18岁及以上，且在2005年1月至2010年9月期间接受一线伊马替尼治疗。提取了患者人口统计学、病史（如合并症、索卡尔评分）和治疗特征（如开始治疗时间、治疗持续时间）的数据。描述性分析按国家和治疗线分层。使用多变量逻辑回归评估伊马替尼反应的预后指标，并对国家、患者人口统计学、病史、治疗特征和副作用进行调整。
各治疗线开始后3、6、12和18个月时的血液学、细胞遗传学和分子反应。
患者平均年龄57岁，59%为男性。总体而言，CML诊断后约4个月开始使用伊马替尼。伊马替尼开始治疗后6个月时，澳大利亚的完全或主要分子反应（C/MMR）为54%，加拿大为22%，韩国为38%。18个月时，超过60%的患者达到C/MMR。约30%的患者主要因不耐受和无反应而停用伊马替尼。在接受二线治疗的患者中，达沙替尼的使用频率高于尼罗替尼。多变量回归结果表明，索卡尔评分在几个时间点被确定为伊马替尼治疗反应的预后指标。
本研究存在若干局限性。首先，我们选择了方便样本的患者和医生，因此结果可能不代表慢性期CML患者的真实总体情况。其次，数据由选定的医生录入，可能存在数据录入错误或不准确。第三，从患者记录中收集的信息有限，我们可能未捕捉到与测量结果相关的其他预后或混杂因素。接下来，由于这是对先前记录数据的分析（即回顾性分析），我们无法提供反应的先验定义。最后，多变量分析仅限于与伊马替尼相关的结果。
治疗模式和预后指标因国家而异。医疗保健提供者、支付方和患者可利用这些结果为旨在提高慢性期CML患者护理效果的治疗和政策提供参考。
