Hind T. Hatoum & Company and Pharmacy Administration, The University of Illinois at Chicago, IL 60647, USA.
Curr Med Res Opin. 2011 Jun;27(6):1255-62. doi: 10.1185/03007995.2011.576236. Epub 2011 May 10.
The purpose of this study is to describe patterns of hypomethylating agents (HMA) use and to compare treatment outcomes of decitabine (DAC) and azacitidine (AZA) with respect to transfusion dependence and the use of erythropoiesis-stimulating agents (ESA) treatment in commercially-insured patients with Myelodysplastic Syndromes (MDS).
A retrospective study using MarketScan Research Data, a large claims database studied patients who received DAC, AZA, or Supportive Care (SC) with at least two claims for MDS between January 1, 2006 and December 31, 2008. Poisson regressions were used to compare DAC and AZA on post-index number of red blood cell/platelet (RBC/PLT) transfusions and ESA treatment, controlling for age, gender, Charlson Comorbidity Index (CCI), time to HMA initiation, number of HMA cycles, and pretreatment RBC/PLT or ESA claims. No other adjustment for disease severity was made.
Approximately 48% of the patients were males with a mean age of 73 years (N = 2525). There were 37 DAC-treated and 60 AZA-treated patients. The length of follow-up did not significantly differ between the DAC- and AZA-treated groups (DAC = 349.2; AZA = 350.5 days); however, the number of days from MDS diagnosis to HMA therapy initiation was longer in the DAC cohort than in the AZA cohort (mean 93.7 days vs. 50.8 days, respectively, p = 0.029). Both DAC- and AZA-treated patients received similar number of treatment cycles (mean: 4.8 vs. 5.6 in DAC vs. AZA, p > 0.05), with means of 4.6 days per cycle for patients receiving DAC and 7.4 days for those receiving AZA (p = 0.003). Following treatment with HMA using Poisson regression analysis, DAC-treated patients had significantly lower use of RBC/PLT transfusions (RR 0.206, p = 0.034) and similar use of ESAs compared with AZA-treated patients. Limitations of the study included the small sample size, and the fact that the majority of patients were unspecified regarding their International Prognostic Scoring System (IPSS) risk category, which did not allow for accounting for differences in disease severity.
In MDS patients treated with an HMA, treatment with DAC was associated with less frequent transfusions than with AZA treatment. Further studies with the ability to control for disease severity are warranted.
本研究旨在描述低甲基化药物(HMA)的使用模式,并比较去甲基化药物(DAC)和阿扎胞苷(AZA)在输血依赖和红细胞生成刺激剂(ESA)治疗方面的治疗结果,以评估患有骨髓增生异常综合征(MDS)的商业保险患者。
这是一项使用 MarketScan Research Data 的回顾性研究,该大型理赔数据库纳入了 2006 年 1 月 1 日至 2008 年 12 月 31 日期间至少有两次 MDS 理赔的接受 DAC、AZA 或支持性治疗(SC)的患者。采用泊松回归比较 DAC 和 AZA 治疗后的红细胞/血小板(RBC/PLT)输注次数和 ESA 治疗情况,同时控制年龄、性别、Charlson 合并症指数(CCI)、HMA 起始时间、HMA 周期数以及治疗前 RBC/PLT 或 ESA 理赔情况。未对疾病严重程度进行其他调整。
约 48%的患者为男性,平均年龄为 73 岁(N=2525)。37 例患者接受 DAC 治疗,60 例患者接受 AZA 治疗。DAC 和 AZA 治疗组的随访时间无显著差异(DAC=349.2;AZA=350.5 天);然而,DAC 队列的 MDS 诊断至 HMA 治疗开始的时间长于 AZA 队列(分别为 93.7 天和 50.8 天,p=0.029)。DAC 和 AZA 治疗的患者接受的治疗周期数相似(DAC 为 4.8 个,AZA 为 5.6 个,p>0.05),接受 DAC 治疗的患者每个周期的平均天数为 4.6 天,接受 AZA 治疗的患者为 7.4 天(p=0.003)。使用泊松回归分析,与 AZA 治疗相比,接受 HMA 治疗的 DAC 治疗患者 RBC/PLT 输注量显著减少(RR 0.206,p=0.034),ESA 使用率相似。该研究的局限性包括样本量小,且大多数患者未明确其国际预后评分系统(IPSS)风险类别,这无法说明疾病严重程度的差异。
在接受 HMA 治疗的 MDS 患者中,与 AZA 治疗相比,DAC 治疗与输血频率较低相关。需要进一步的研究来控制疾病的严重程度。