Hôpital Saint-Louis, CIC 1427, Paris, France.
HEMORIO, State Institute of Hematology Arthur de Siquiera Cavalcanti, Rio de Janeiro, Brazil.
Leukemia. 2023 May;37(5):1060-1067. doi: 10.1038/s41375-023-01888-y. Epub 2023 Apr 14.
In ASCEMBL, an open-label, randomized Phase 3 study, asciminib demonstrated superior efficacy and better safety profile compared with bosutinib in patients with chronic myeloid leukemia in chronic phase (CML-CP) previously treated with ≥2 tyrosine kinase inhibitors. Health-related quality of life (HRQOL) reported by patients is key to understanding the benefit and impact of treatment on patients' lives, and is becoming increasingly important as the life expectancy of CML-CP patients increases and patients require long-term treatment. In ASCEMBL, patients completed questionnaires to assess CML symptoms and interference with daily life (M.D. Anderson Symptom Inventory - CML [MDASI-CML]), general HRQOL (five-level EQ-5D [EQ-5D-5L], Patient Global Impression of Change - CML [PGIC-CML]), and impact of CML on working life and activity (Work Productivity and Activity Impairment questionnaire - CML [WPAI-CML]). Patients' CML symptoms and HRQOL remained stable during 48 weeks of treatment with asciminib, with a general trend for decreased CML symptom severity, particularly for fatigue, and improvement in HRQOL. A clinically meaningful increase in diarrhea severity was observed in patients treated with bosutinib compared to asciminib. These data provide better understanding of the patient perspective and treatment impact on HRQOL in a later-line setting, where little information has been published to date.
在 ASCEMBL 这项开放标签、随机 III 期研究中,与博舒替尼相比,asciminib 为先前接受过≥2 种酪氨酸激酶抑制剂治疗的慢性髓性白血病慢性期(CML-CP)患者带来了更优的疗效和更好的安全性。患者报告的健康相关生活质量(HRQOL)是了解治疗对患者生活的益处和影响的关键,并且随着 CML-CP 患者预期寿命的延长和患者需要长期治疗,其重要性日益增加。在 ASCEMBL 中,患者完成了调查问卷以评估 CML 症状和对日常生活的干扰(MD 安德森 CML 症状量表[MDASI-CML])、一般 HRQOL(五维健康量表-5 级[EQ-5D-5L]、CML 患者整体印象变化量表[PGIC-CML])以及 CML 对工作生活和活动的影响(CML 工作生产力和活动障碍问卷[WPAI-CML])。在接受 asciminib 治疗的 48 周期间,患者的 CML 症状和 HRQOL 保持稳定,CML 症状严重程度呈总体下降趋势,尤其是疲劳,并且 HRQOL 得到改善。与 asciminib 相比,接受 bosutinib 治疗的患者腹泻严重程度出现了临床意义上的增加。这些数据为我们提供了更好的理解,即患者视角和治疗对后续治疗环境中 HRQOL 的影响,因为迄今为止,这方面的信息很少。