Division of Hematology, Department of Internal Medicine, Cukurova University, 01380 Adana, Turkey.
Medicina (Kaunas). 2023 Mar 2;59(3):498. doi: 10.3390/medicina59030498.
: Relapsed or refractory acute myeloid leukemia (r/r AML) is a disease with a poor prognosis. Limited treatment options are available in r/r AML. Here, we administered gemtuzumab ozogamicin (GO) as salvage therapy in twenty-four patients with r/r AML. The aim of the study was to determine the role of GO in r/r AML in real life. : This retrospective observational study recruited 24 adult patients with diagnosed r/r AML from 2018 to 2022. Twenty-four patients with r/r AML were treated with GO. GO treatment was used as monotherapy in 23 patients and in combination with cytarabine in 1 patient. At the time of diagnosis, the risk status of all patients was determined as favorable, intermediate, or adverse according to the 2017 ELN AML guidelines. : The median follow-up was 44.3 (13-144) months. Fifteen (62.5%) of the twenty-four patients were in the intermediate-risk cytogenetics group and nine (37.5%) were in the favorable cytogenetics group. The most common adverse events included nausea/vomiting in 79.17% ( = 19) of patients, headache in 62.50% ( = 15), elevated LFTs in 37.50% ( = 9), febrile neutropenia in 25% ( = 6), and bleeding in 25% ( = 6). The most common cause of death was infection. The most common causes of mortality were septic shock, accounting for 33.3% ( = 8) of deaths, and opportunistic lung infection, accounting for 12.5% ( = 3) of deaths. Acute infusion-related toxicities associated with GO were usually transient and, in most cases, responded to the standard of care treatment. After treatment with GO, 16.6% ( = 4) of patients achieved MLFS and 37.5% ( = 9) achieved CR. The overall response rate was 54.1%. The median overall survival time of the patients was 44 months (37.8-50.2 months). Disease-free survival was 22 months (0-48.6 months). The 5-year survival rate was 33%. : A low dose of GO improved the overall survival and disease-free survival in r/r AML patients. GO treatment had a positive safety profile in terms of toxicity.
复发性或难治性急性髓系白血病（r/r AML）是一种预后不良的疾病。r/r AML 的治疗选择有限。在这里，我们对 24 例 r/r AML 患者进行了吉妥珠单抗奥佐米星（GO）作为挽救性治疗。本研究的目的是确定 GO 在 r/r AML 中的实际作用。
这项回顾性观察性研究招募了 2018 年至 2022 年间确诊的 24 例成人 r/r AML 患者。24 例 r/r AML 患者接受 GO 治疗。23 例患者单用 GO，1 例患者与阿糖胞苷联合使用。在诊断时，根据 2017 年 ELN AML 指南，所有患者的风险状态均被确定为低危、中危或高危。
中位随访时间为 44.3（13-144）个月。24 例患者中有 15 例（62.5%）为中危细胞遗传学组，9 例（37.5%）为低危细胞遗传学组。最常见的不良事件包括 79.17%（=19）的患者出现恶心/呕吐、62.50%（=15）的患者出现头痛、37.50%（=9）的患者出现肝酶升高、25%（=6）的患者出现发热性中性粒细胞减少症和 25%（=6）的患者出现出血。最常见的死亡原因是感染。导致死亡的最常见原因是感染性休克，占 33.3%（=8），其次是机会性肺部感染，占 12.5%（=3）。与 GO 相关的急性输注相关毒性通常是短暂的，并且在大多数情况下，对标准治疗有反应。GO 治疗后，16.6%（=4）的患者达到 MLFS，37.5%（=9）的患者达到 CR。总缓解率为 54.1%。患者的中位总生存时间为 44 个月（37.8-50.2 个月）。无病生存时间为 22 个月（0-48.6 个月）。5 年生存率为 33%。
低剂量 GO 可改善 r/r AML 患者的总生存和无病生存。GO 治疗在毒性方面具有积极的安全性特征。
