Analysis Group, Inc., Boston, MA, USA.
Taiho Oncology, Inc., Princeton, NJ, USA.
Hematology. 2021 Dec;26(1):261-270. doi: 10.1080/16078454.2021.1889161.
To evaluate hypomethylating agent (HMA) persistence in patients with myelodysplastic syndromes (MDS), and examine its association with healthcare resource utilization (HRU) and progression to acute myeloid leukemia (AML).
A total of 2,400 adults diagnosed with MDS initiating HMAs were included from IBM MarketScan databases during 1/1/2011-3/31/2018. The index date was HMA initiation following MDS diagnosis. Patients were classified according to their persistence status by the end of a fixed 'landmark period' of 4 months post-index.
Median persistence to HMAs was 5.6 months (95% CI: 5.2, 6.1); HMA non-persistence increased with time. Non-persistent patients had a significantly higher non-HMA-related HRU burden than persistent patients [adjusted incidence rate ratios, outpatient visits: 1.12 (95% CI: 1.10, 1.14); inpatient visits: 1.48 (95% CI: 1.30, 1.69); emergency department visits 1.30 (95% CI: 1.12, 1.50); all -values < 0.001]. All-cause and HMA-related outpatient visits were lower among non-persistent patients, likely because of fewer HMA administration-related visits. The incidence rate of AML was numerically, although not significantly, higher in non-persistent patients, when starting follow-up at the end of the landmark period. When follow-up began at the index date, non-persistent patients had a significantly higher rate of AML [adjusted hazard ratio, 1.88 (95% CI: 1.53, 2.32); -value < 0.001].
HMA non-persistence, which increased over time, was associated with significantly higher non-HMA-related HRU, and numerically higher AML progression in MDS patients initiating HMAs. Future studies should evaluate predictors of HMA non-persistence in this patient population.
评估骨髓增生异常综合征（MDS）患者接受低甲基化药物（HMA）治疗的持续性，并研究其与医疗资源利用（HRU）和进展为急性髓系白血病（AML）的关系。
本研究纳入了 2011 年 1 月 1 日至 2018 年 3 月 31 日期间 IBM MarketScan 数据库中诊断为 MDS 并开始接受 HMA 治疗的 2400 名成年人。索引日期为 MDS 诊断后开始使用 HMA。根据索引日期后 4 个月的固定“基准期”结束时的持续性情况，患者被分为不同的分类。
HMA 的中位持续时间为 5.6 个月（95%CI：5.2，6.1）；随着时间的推移，HMA 的非持续性增加。与持续性患者相比，非持续性患者的非 HMA 相关 HRU 负担明显更高[调整后的发病率比，门诊就诊：1.12（95%CI：1.10，1.14）；住院就诊：1.48（95%CI：1.30，1.69）；急诊就诊：1.30（95%CI：1.12，1.50）；所有 P 值均<0.001]。非持续性患者的全因和 HMA 相关门诊就诊次数较低，可能是因为 HMA 给药相关就诊次数较少。在基准期结束时开始随访时，非持续性患者的 AML 发生率虽然没有显著增加，但数值上更高。当从索引日期开始随访时，非持续性患者的 AML 发生率明显更高[调整后的风险比，1.88（95%CI：1.53，2.32）；P 值<0.001]。
随着时间的推移，HMA 的非持续性增加与 MDS 患者接受 HMA 治疗的非 HMA 相关 HRU 显著增加和 AML 进展的数值增加有关。未来的研究应评估该患者人群中 HMA 非持续性的预测因素。
