Division of Hematology, The Ohio State University, 281 W Lane Ave, Columbus, OH, 43210, USA.
Department of Medicine, New York Medical College, Valhalla, NY, USA.
Adv Ther. 2024 Nov;41(11):4049-4064. doi: 10.1007/s12325-024-02947-1. Epub 2024 Sep 6.
The substantial economic burden of acute myeloid leukemia (AML) could be reduced with post-remission maintenance therapies that delay relapse. Real-world healthcare resource utilization (HCRU) data and costs among patients with AML receiving oral azacitidine (Oral-AZA) maintenance therapy or no maintenance are not well understood. We characterize HCRU and costs among these patients in clinical practice in the USA.
Data from IQVIA PharMetrics® Plus (January 1, 2016-June 30, 2022) were used. Patients ≥ 18 years who were newly diagnosed with AML, received first-line systemic induction therapy, and attained disease remission were eligible. Patients receiving Oral-AZA maintenance and those receiving no maintenance ("watch and wait" [W&W]) were matched 1:3 on baseline characteristics using propensity score matching (PSM) and followed until hematopoietic stem cell transplantation or end of continuous insurance enrollment, whichever occurred first. Outcomes included treatment patterns, inpatient and outpatient visits, and costs.
After PSM, the Oral-AZA cohort included 43 patients and the W&W cohort 129. Of the 43 patients receiving Oral-AZA, 88.4% started at the recommended dose of 300 mg and 11.6% at 200 mg. The Oral-AZA cohort had significantly (p = 0.0025) longer median (95% CI) time to relapse from the index maintenance date (median not reached [NR; 9.0 months-NR] vs 3.3 months [0.8 months-NR]), and fewer per person per month (PPPM) hospitalizations (0.23 vs 0.61; p = 0.0005) and overall outpatient visits (5.77 vs 7.58; p = 0.0391) than the W&W cohort. Despite higher AML drug costs PPPM in the Oral-AZA cohort ($16,401 vs $10,651 for W&W), total healthcare costs PPPM were lower ($25,786 vs $38,530 for W&W; p < 0.0001).
Patients with newly diagnosed AML treated with Oral-AZA maintenance in clinical practice had prolonged remission and lower HCRU and costs than patients receiving no maintenance therapy. These findings underscore the clinical and economic value of Oral-AZA in clinical practice.
急性髓系白血病（AML）的大量经济负担可以通过缓解后维持治疗来减轻，这些治疗可以延迟复发。在接受口服阿扎胞苷（Oral-AZA）维持治疗或不接受维持治疗的 AML 患者中，实际的医疗保健资源利用（HCRU）数据和成本尚不清楚。我们描述了美国临床实践中这些患者的 HCRU 和成本。
使用 IQVIA PharMetrics® Plus 数据库（2016 年 1 月 1 日至 2022 年 6 月 30 日）的数据。符合条件的患者为≥18 岁，新诊断为 AML，接受一线系统性诱导治疗，并达到疾病缓解。接受 Oral-AZA 维持治疗的患者和不接受维持治疗的患者（“观察等待”[W&W]）根据基线特征使用倾向评分匹配（PSM）进行 1:3 匹配，随访至造血干细胞移植或连续保险登记结束，以先发生者为准。结果包括治疗模式、住院和门诊就诊和成本。
PSM 后，Oral-AZA 队列包括 43 例患者，W&W 队列包括 129 例患者。在接受 Oral-AZA 治疗的 43 例患者中，88.4%的患者起始剂量为 300mg，11.6%的患者起始剂量为 200mg。Oral-AZA 队列的中位（95%CI）从索引维持日期开始复发时间明显（p=0.0025）延长（中位未达到[NR；9.0 个月-NR] vs 3.3 个月[0.8 个月-NR]），且每人每月（PPPM）住院次数（0.23 vs 0.61；p=0.0005）和总门诊就诊次数（5.77 vs 7.58；p=0.0391）均低于 W&W 队列。尽管 Oral-AZA 队列的 AML 药物成本 PPPM 较高（Oral-AZA 为 16401 美元，W&W 为 10651 美元），但总医疗保健成本 PPPM 较低（Oral-AZA 为 25786 美元，W&W 为 38530 美元；p<0.0001）。
在临床实践中，接受 Oral-AZA 维持治疗的新诊断 AML 患者的缓解时间延长，HCRU 和成本降低。这些发现强调了 Oral-AZA 在临床实践中的临床和经济价值。
