低剂量达沙替尼（每日 50 毫克）一线治疗新诊断的慢性期慢性髓性白血病：5 年随访结果。

Low-Dose Dasatinib (50 mg Daily) Frontline Therapy in Newly Diagnosed Chronic Phase Chronic Myeloid Leukemia: 5-Year Follow-Up Results.

机构信息

Department of Leukemia, University of Texas MD Anderson Cancer Center, Houston, TX.

出版信息

Clin Lymphoma Myeloma Leuk. 2023 Oct;23(10):742-748. doi: 10.1016/j.clml.2023.05.009. Epub 2023 May 23.

DOI:10.1016/j.clml.2023.05.009

PMID:37308342

Abstract

BACKGROUND

Dasatinib is a BCR::ABL1 tyrosine kinase inhibitor approved as frontline therapy at a 100 mg daily for chronic myeloid leukemia in chronic phase (CML-CP). The use of a lower dose of dasatinib (50 mg daily) has demonstrated better tolerance and improved outcomes compared with the standard dose. Here, we report the updated results in a large cohort with a 5-year follow-up.

PATIENTS AND METHODS

Patients with newly diagnosed CML-CP were eligible. Entry and response-outcome criteria were standard. Dasatinib was given as 50 mg orally daily.

RESULTS

Eighty-three patients were included. At 3 months, 78 (96%) patients achieved BCR::ABL1 transcripts (IS) ≤10%, and at 12 months, 65 (81%) patients achieved BCR::ABL1 transcript (IS) ≤0.1%. The cumulative incidence of complete cytogenetic, major molecular, and deep molecular responses at 5 years were 98%, 95%, and 82%, respectively. Rates of failures due to resistance (n = 4; 5%) and toxicity (n = 4; 5%) were low. The 5-year overall survival was 96% and event-free survival 90%. No transformations to accelerated or blastic phase were observed. Grade 3 to 4 pleural effusions developed in 2% of patients.

CONCLUSION

Dasatinib 50 mg daily is an effective and safe treatment for newly diagnosed CML-CP.

摘要

背景

达沙替尼是一种 BCR::ABL1 酪氨酸激酶抑制剂，批准用于慢性期慢性髓性白血病（CML-CP）的一线治疗，每日剂量为 100mg。与标准剂量相比，较低剂量的达沙替尼（每日 50mg）具有更好的耐受性和改善的疗效。在此，我们报告了一项大样本队列的 5 年随访更新结果。

患者和方法

符合条件的患者为新诊断的 CML-CP。入组和反应结果标准为标准。达沙替尼口服，每日 50mg。

结果

共纳入 83 例患者。3 个月时，78 例（96%）患者达到 BCR::ABL1 转录物（IS）≤10%，12 个月时，65 例（81%）患者达到 BCR::ABL1 转录物（IS）≤0.1%。5 年时完全细胞遗传学、主要分子学和深度分子学反应的累积发生率分别为 98%、95%和 82%。因耐药（n=4；5%）和毒性（n=4；5%）导致的失败率较低。5 年总生存率为 96%，无事件生存率为 90%。未观察到加速或成髓细胞白血病期的转化。2%的患者出现 3-4 级胸腔积液。