Clinical observation of the combination therapy of azacitidine and venetoclax in newly diagnosed, elderly patients with acute myeloid leukemia

doi:10.16138/j.1673-6087.2021.03.008

Abstract

Abstract:

Objective To observe the clinical efficacy and safety of azacitidine combined with venetoclax in the treatment of newly diagnosed, elderly patients with acute myeloid leukemia (AML). Methods Fourteen elderly patients admitted in Ruijin Hospital from March 2018 to September 2020, who were newly diagnosed with AML and not suitable for intensive chemotherapy, were enrolled and a retrospective analysis was conducted. All patients received 75 mg/m² azacitidine subcutaneously from day 1 to day 7, and venetoclax orally once a day, total 28 d(the first cause of venetoclax treatment: 100 mg in day 1, 200 mg in day 2, 400 mg from day 3 to day 28). The primary end points were composite complete remission rate (CR+CRi), overall response rate (ORR), time to first response(CR/CRi ) and duration of response (DOR), and the secondary end points were overall survival(OS) and safety. Results The median follow-up time was 12.5 (2.0-24.0) months, the composite complete remission rate (CR+CRi) was 71%, ORR was 79%, the median time to first response was 1.1 (1.0-2.1) months, the median DOR was 16.0 months [95% confidence interval (CI): 4.0-not reached(NR)], and the median OS was 14.0 months (95% CI: 4.0-NR). Among 12 patients with median and high danger of poor cytogenetics, 8 patients (67%) achieved CR+CRi, the median DOR was 9.0 months (95% CI: 2.0-NR), and the median OS was 11.0 months (95% CI: 2.0-NR). Among 10 patients with CR + CRi, 8 patients(80%) achieved MRD<0.01% at least in one assessment, the median DOR was 17.0 months (95% CI: 4.0-NR) and the median OS did not reach (95% CI: 6.0-NR）。All patients showed cytopenia in different degree, including neutropenia of grade 3 or above (100%), thrombocytopenia (7 cases, 50%), febrile neutropenia (6 cases, 43%) and anemia (5 cases, 36%). The other common events occurred in gastrointestinal tract, which were all controllable response of grade 1-2. One patient showed tumor lysis syndrome. Most patients were tolerant to treatment. Conclusions The preliminary results show that the combination therapy of azacitidine and venetoclax is effective and safe, which provides a treatment option for elderly, untreated AML patients who are not suitable for intensive chemotherapy.

Key words: Acute myeloid leukemia, Elderly, Newly diagnosed, Azacitidine, Venetoclax

CLC Number:

R733.71

HUANG Lei, YE Chenjing, WU Chao, XU Wenbin, YU Qing, LI Junmin, YAN Hua. Clinical observation of the combination therapy of azacitidine and venetoclax in newly diagnosed, elderly patients with acute myeloid leukemia[J]. Journal of Internal Medicine Concepts & Practice, 2021, 16(03): 178-182.

Figures/Tables 2

References 15

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特征	n（%）	复合完全缓解率(CR+CRi)[n(%）]	中位DOR（月）（95%CI）	中位OS（月）（95%CI）
所有患者(n）	14	(8+2)[10(71)]	16.0(4.0~NR）	14.0(2.0~NR)
ECOG评分[n(%）]
1分	5(36）
2分	8(57）
3分	1(7）
细胞遗传学[n(%）]
低危	2(14）
中高危	12(86）	(7+1)[8(67)]	9.0(2.0~NR)	11.0(2.0~NR)
基因突变[n(%）]
NPM1	5(36）	(3+1)[4(80)]	17.5(7.0~NR)	NR(6.0~NR)
TP53	4(29）	(2+0)[2(50)]	NR(2.0~NR)	11.0(2.0~NR)
FLT3-ITD	1(7）
基线骨髓原始细胞数[n(%）]
<30%	3(21）
≥30%且<50%	2(14）
≥50%	9(64）
CR+CRi（n）	8+2(10)
MRD<0.01%,n(%)	8(80）		17.0(4.0~NR)	NR(6.0~NR)