Department of leukemia, The University of Texas MD Anderson Cancer Center , Houston , TX , USA.
Expert Rev Hematol. 2019 Sep;12(9):707-714. doi: 10.1080/17474086.2019.1643231. Epub 2019 Aug 1.
: Hairy cell leukemia is a rare indolent B-cell malignancy, characterized by pancytopenia, recurrent infections, and splenomegaly. After initial therapy with purine nucleoside analogs, up to 50% of patients relapse after several years of remission. The number of relapsed patients is increasing and, until recently, there was no approved therapy with durable responses for hairy cell leukemia patients in the relapsed setting, thus the need for new non-chemotherapy approach with significant efficacy and less myelosuppression. : Moxetumomab pasudotox is a recombinant immunotoxin containing a Fv fragment of an anti-CD22 monoclonal antibody and truncated (PE38). The authors reviewed pre-clinical and clinical studies that led to the FDA approval of the drug in patients with relapsed and/or refractory hairy cell leukemia, who received at least two prior therapies, including at least one purine nucleoside analog. : Moxetumomab pasudotox demonstrated a durable complete remission rate of 30% in heavily pretreated patients with hairy cell leukemia, and MRD eradication in 85% of responding patients. Moxetumomab pasudotox got a global FDA approval in September 2018. The US prescribing information carries boxed warnings regarding the risk of capillary leak syndrome and hemolytic uremic syndrome. Long-term follow-up of the pivotal study is ongoing (NCT01829711).
毛细胞白血病是一种罕见的惰性 B 细胞恶性肿瘤，其特征为全血细胞减少、反复感染和脾肿大。初始接受嘌呤核苷类似物治疗后，多达 50%的患者在缓解数年后复发。复发患者的数量正在增加，直到最近，对于复发毛细胞白血病患者，尚无批准的具有持久缓解作用的治疗方法，因此需要采用非化疗方法，具有显著疗效且骨髓抑制作用较小。莫昔妥莫单抗泊马度胺是一种重组免疫毒素，包含抗 CD22 单克隆抗体的 Fv 片段和截断的（PE38）。作者回顾了导致 FDA 批准该药用于至少接受过两次治疗的复发和/或难治性毛细胞白血病患者的临床前和临床研究，这些患者接受了至少一种嘌呤核苷类似物治疗。莫昔妥莫单抗泊马度胺在接受过多重预处理的毛细胞白血病患者中显示出持久的完全缓解率为 30%，并使 85%的应答患者清除了微小残留病。莫昔妥莫单抗泊马度胺于 2018 年 9 月获得全球 FDA 批准。美国处方信息载有关于毛细血管渗漏综合征和溶血尿毒综合征风险的黑框警告。该关键研究的长期随访正在进行中（NCT01829711）。
