A phase II study of bortezomib in patients with relapsed or refractory aggressive adult T-cell leukemia/lymphoma.
A phase II study of bortezomib in patients with relapsed or refractory aggressive adult T-cell leukemia/lymphoma.
复制标题
DOI：
10.1111/cas.12735
复制
发表时间：
2015-09
期刊：
影响因子：
5.7
通讯作者：
Tamura K
中科院分区：
文献类型：
--
作者：
Ishitsuka K;Utsunomiya A;Katsuya H;Takeuchi S;Takatsuka Y;Hidaka M;Sakai T;Yoshimitsu M;Ishida T;Tamura K
成人T细胞白血病/淋巴瘤(ATL)是一种预后较差的外周T淋巴细胞的恶性肿瘤。这项多中心、两阶段、单臂II期研究评估了波特佐米在至少接受一种化疗方案的复发/难治性ATL患者中的有效性和安全性。主要终点是最佳总体缓解率(ORR)，次要终点包括安全性、对病变的最佳反应和无进展生存期(PFS)。本研究的第一阶段共入选15名患者。1例部分缓解(PR)和5例病情稳定(SD)的总有效率为6.7%(95%可信区间为0.17~31.95%)。根据病变部位的不同，外周血有1例完全缓解(CR)，可测量的靶点皮损有2例完全缓解，皮肤皮损有2例完全缓解。无进展生存期(PFS)为38天(95%可信区间：18~106天)。所有患者均出现≥-1不良事件(AEs)，80%的患者出现≥-1 3/4级不良事件；然而，没有新的安全性发现。虽然这些结果满足了进入第二阶段的计划设置，但协调委员会决定终止这项研究，因为单一药物活动似乎对这群患者来说并不是很有希望。
Adult T-cell leukemia/lymphoma (ATL) is a malignancy of peripheral T-lymphocytes with a poor prognosis. This multicenter, two-stage, single-arm, phase II study assessed the efficacy and safety of bortezomib in patients with relapsed/refractory ATL who received at least one regimen of chemotherapy. The primary endpoint was the best overall response rate (ORR), and secondary endpoints included safety, the best response by lesions, and progression-free survival (PFS). Fifteen patients were enrolled in the first stage of this study. One partial remission (PR) and five stable disease (SD) were observed as the best overall responses, and ORR was 6.7% (95% confidence interval (C.I.) 0.17-31.95%). Responses according to disease sites were one complete remission (CR) in peripheral blood, two PR in measurable targeted lesions, and two PR in skin lesions. Progression-free survival (PFS) was 38 (95% CI; 18–106) days. All patients developed ≥1 adverse events (AEs), and 80% of patients had ≥1 grade 3/4 AEs; however, no new safety findings were obtained. Although these results fulfilled the planned settings to proceed to the second stage, the coordinating committee decided to terminate this study because single agent activity did not appear to be very promising for this cohort of patients.