Roswell Park Comprehensive Cancer Center, Buffalo, NY, USA.
The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Leuk Lymphoma. 2020 Aug;61(8):1965-1973. doi: 10.1080/10428194.2020.1742897. Epub 2020 May 20.
Gemtuzumab ozogamicin (GO) remained available to US clinicians through an open-label expanded-access protocol (NCT02312037) until GO was reapproved. Patients were aged ≥3 months with relapsed/refractory (R/R) acute myeloid leukemia (AML), high-risk myelodysplastic syndrome, or acute promyelocytic leukemia (APL), and had exhausted other treatment options. Three hundred and thirty one patients received GO as monotherapy for R/R AML ( 139), combination therapy for R/R AML ( 183), or treatment for R/R APL ( 9). Corresponding treatment discontinuations occurred in 68, 39, and 33% of patients. All-causality grade 5 AEs occurred in 52, 22, and 22% of patients in the monotherapy, combination, and APL groups, respectively. Corresponding grades 3 and 4 treatment-related AEs were reported in 60, 55 and 78% of patients. Hepatotoxicity occurred in five patients: veno-occlusive disease ( 4) and drug-induced liver injury ( 1). GO was generally well tolerated in patients with R/R AML or APL. Most frequent treatment-related grade ≥3 AEs were hematologic AEs. NCT02312037.
吉妥珠单抗奥佐米星（GO）通过开放标签扩展访问协议（NCT02312037）仍然可供美国临床医生使用，直到 GO 重新获得批准。患者年龄≥3 个月，患有复发/难治性（R/R）急性髓细胞白血病（AML）、高危骨髓增生异常综合征或急性早幼粒细胞白血病（APL），且已用尽其他治疗选择。331 名患者接受 GO 作为 R/R AML 的单药治疗（139 名）、R/R AML 的联合治疗（183 名）或 R/R APL 的治疗（9 名）。分别有 68%、39%和 33%的患者停止了相应的治疗。在单药治疗、联合治疗和 APL 组中，分别有 52%、22%和 22%的患者发生了所有病因的 5 级不良事件。分别有 60%、55%和 78%的患者报告了相应的 3 级和 4 级治疗相关不良事件。5 名患者发生了肝毒性：静脉闭塞病（4 例）和药物性肝损伤（1 例）。GO 在 R/R AML 或 APL 患者中通常具有良好的耐受性。最常见的治疗相关≥3 级不良事件是血液学不良事件。NCT02312037。
