Health Economics & Outcomes Research, Jazz Pharmaceuticals, Palo Alto, CA, USA.
Biostatistics, Jazz Pharmaceuticals, Philadelphia, PA, USA.
J Med Econ. 2020 Jul;23(7):714-720. doi: 10.1080/13696998.2020.1744613. Epub 2020 Apr 10.
Treatment of acute myeloid leukemia (AML) requires significant healthcare resource utilization (HRU), including lengthy hospitalizations. In a phase 3 study (NCT01696084), CPX-351 (Vyxeos) showed significant benefits to overall survival and complete remission versus conventional 7 + 3 cytarabine/daunorubicin. This analysis evaluated HRU in patients aged 60-75 years with newly diagnosed high-risk/secondary AML treated with CPX-351 versus 7 + 3 in the phase 3 study. Patients were randomized to receive up to two induction cycles with CPX-351 or 7 + 3. Responders could receive up to two cycles of consolidation. To normalize HRU to length of treatment, patients were assessed on a per patient-year (PPY) basis. HRU analyses included hospital and intensive care unit (ICU) stays, anti-infective use, transfusions, and white blood cell colony-stimulating factor (CSF). The median (range) total duration of hospitalization was 39 (3-110) days with CPX-351 ( = 153) and 32 (2-83) days with 7 + 3 ( = 151); the estimated durations of hospitalization PPY were 198.4 and 240.5 days, respectively. The median (range) total duration of ICU stays was 0 (0-45) days with CPX-351 and 0 (0-17) days with 7 + 3; the estimated durations of ICU stays PPY were 6.7 and 10.5 days, respectively. When comparing supportive care use during CPX-351 and 7 + 3 treatment, the estimated number PPY of bags of platelets used (24.6 vs 26.9, respectively), bags of packed red blood cells used (13.0 vs 13.9), days of anti-infectives (162.0 vs 159.2), and days of CSF (4.0 vs 2.4) were not notably different. This clinical study analysis may not represent real-world HRU patterns or be generalizable to a broader AML population. These PPY data, showing shorter durations of hospitalization and similar use of supportive care with CPX-351 versus 7 + 3, suggest CPX-351 is not associated with increased HRU in older patients with newly diagnosed high-risk/secondary AML.
治疗急性髓细胞白血病（AML）需要大量的医疗资源利用（HRU），包括长时间住院。在一项 3 期研究（NCT01696084）中，CPX-351（Vyxeos）在总生存和完全缓解方面与传统的 7+3 阿糖胞苷/柔红霉素相比具有显著优势。这项分析评估了 60-75 岁新诊断为高危/继发性 AML 患者接受 CPX-351 与 3 期研究中 7+3 治疗的 HRU。患者被随机分配接受最多两个诱导周期的 CPX-351 或 7+3。应答者可以接受最多两个周期的巩固治疗。为了将 HRU 归一化为治疗时间，患者按每人每年（PPY）进行评估。HRU 分析包括住院和重症监护病房（ICU）停留、抗感染使用、输血和白细胞集落刺激因子（CSF）。CPX-351（ = 153）的总住院时间中位数（范围）为 39（3-110）天，7+3（ = 151）的总住院时间中位数（范围）为 32（2-83）天；估计的住院 PPY 分别为 198.4 和 240.5 天。CPX-351 的总 ICU 停留时间中位数（范围）为 0（0-45）天，7+3 的总 ICU 停留时间中位数（范围）为 0（0-17）天；估计的 ICU 停留 PPY 分别为 6.7 和 10.5 天。在比较 CPX-351 和 7+3 治疗期间支持性护理的使用时，估计的 PPY 血小板袋数（分别为 24.6 和 26.9）、袋装浓缩红细胞数（分别为 13.0 和 13.9）、抗感染天数（分别为 162.0 和 159.2）和 CSF 天数（分别为 4.0 和 2.4）无明显差异。这项临床研究分析可能无法代表真实世界的 HRU 模式，也可能无法推广到更广泛的 AML 人群。这些 PPY 数据表明，与 7+3 相比，CPX-351 治疗的住院时间更短，支持性护理的使用相似，这表明 CPX-351 不会增加新诊断为高危/继发性 AML 的老年患者的 HRU。
