Department of Hematology, National Hospital Organization Mito Medical Center, Ibaraki-machi, Japan.
Blood Disorders Center, Aiiku Hospital, Sapporo, Japan.
Int J Hematol. 2022 Jul;116(1):89-101. doi: 10.1007/s12185-022-03334-8. Epub 2022 Apr 8.
Acute myeloid leukemia (AML) predominantly affects elderly adults, and its prognosis worsens with age. Treatment options for patients in Japan ineligible for intensive chemotherapy include cytarabine/aclarubicin ± granulocyte colony-stimulating factor (CA ± G), azacitidine (AZA), low-dose cytarabine (LDAC), targeted therapy, and best supportive care (BSC). The country's aging population and the evolving treatment landscape are contributing to a need to understand treatment pathways and associated outcomes. This retrospective chart review evaluated outcomes in patients across Japan with primary/secondary AML who were ineligible for intensive chemotherapy and began first-line treatment or BSC between 01/01/2015 and 12/31/2018. The primary endpoint was overall survival (OS); secondary endpoints included progression-free survival (PFS) and healthcare resource utilization (HRU). Of 199 patients (58% > 75 years), 121 received systemic therapy (38 CA ± G, 37 AZA, 7 LDAC, 39 other) and 78 received BSC. Median OS was 5.4, 9.2, 2.2, 3.8, and 2.2 months for CA ± G, AZA, LDAC, other systemic therapy, and BSC, respectively; median PFS was 3.4, 7.7, 1.6, 2.3, and 2.1 months, respectively. HRU rates were uniformly high, with > 80% patients hospitalized in each cohort. The poor clinical outcomes and high HRU among Japanese AML patients who are ineligible for intensive chemotherapy highlight an unmet need for novel therapies.
急性髓系白血病(AML)主要影响老年人,其预后随年龄增长而恶化。日本不适合强化化疗的患者的治疗选择包括阿糖胞苷/阿克拉霉素+粒细胞集落刺激因子(CA+G)、阿扎胞苷(AZA)、低剂量阿糖胞苷(LDAC)、靶向治疗和最佳支持治疗(BSC)。日本的人口老龄化和不断发展的治疗格局促使人们需要了解治疗途径和相关结果。这项回顾性图表审查评估了 2015 年 1 月 1 日至 2018 年 12 月 31 日期间,不适合强化化疗且开始一线治疗或 BSC 的原发性/继发性 AML 日本患者的结局。主要终点是总生存期(OS);次要终点包括无进展生存期(PFS)和医疗资源利用(HRU)。在 199 名患者中(58%年龄>75 岁),121 名接受了系统治疗(38 例 CA+G、37 例 AZA、7 例 LDAC、39 例其他),78 名接受了 BSC。CA+G、AZA、LDAC、其他系统治疗和 BSC 的中位 OS 分别为 5.4、9.2、2.2、3.8 和 2.2 个月;中位 PFS 分别为 3.4、7.7、1.6、2.3 和 2.1 个月。HRU 率均很高,每个队列中有>80%的患者住院。不适合强化化疗的日本 AML 患者的临床结局较差且 HRU 较高,突显了对新型疗法的未满足需求。