边缘区淋巴瘤（MZL）是一种缓慢型非霍奇金淋巴瘤，通过病理性B细胞受体信号传导发展而来。奥瑞布替尼是一种新一代口服小分子布鲁顿酪氨酸激酶抑制剂，已在复发/难治（r/r）MZL患者中进行评估。中国进行了一项2期、多中心、单臂研究，以奥瑞布替尼每日150毫克的剂量治疗经过先前治疗的r/r MZL患者。主要终点是通过基于Lugano 2014分类进行独立审查委员会（IRC）评估的总体应答率（ORR）。其他疗效、安全性和药代动力学特征则作为次要结果指标进行评估。共招募了111名患者，其中90名患者经中央病理学复核证实为MZL患者，主要为粘膜相关淋巴组织（MALT，46.7%）和淋巴结MZL（35.6%）。大多数患者都处于晚期，其中IV期占75.6%。在中位随访时间为24.3个月后，IRC评估的ORR为58.9%（95%置信区间[CI]，48.0-69.2），完全缓解和部分缓解的比例分别为11.1%和47.8%。IRC评估的应答持续时间的中位数为34.3个月，IRC评估的无进展生存中位期尚未达到，12个月无进展生存率为82.8%（95% CI，72.6-89.5）。12个月总生存率为91.0%（95% CI，82.8-95.4）。常见的所有等级与治疗相关的不良事件（TRAEs）包括贫血（27.9%），中性粒细胞计数下降（23.4%），白细胞计数下降（18.0%），血小板计数下降（17.1%），尿液中出现血（16.2%），皮疹（14.4%）和上呼吸道感染（10.8%）。34名患者（30.6%）出现3级或更高级别的TRAEs。18名患者（16.2%）发生严重的TRAEs，其中肺炎（5.4%）最为常见。7名患者（6.3%）因TRAEs而中断奥瑞布替尼治疗。奥瑞布替尼在中国r/r MZL患者中表现出高应答率和持久的疾病缓解，并且耐受性良好。© 2023 Wiley Periodicals LLC.

Marginal zone lymphoma (MZL) is an indolent type of non-Hodgkin lymphoma that develops through pathological B cell receptor signaling. Orelabrutinib, a new-generation oral small molecule Bruton's tyrosine kinase inhibitor, was evaluated in relapsed/refractory (r/r) MZL patients. Previously treated r/r MZL patients received orelabrutinib 150 mg once daily in a phase 2, multicenter, single-arm study conducted in China. The primary endpoint was overall response rate (ORR) assessed by an Independent Review Committee (IRC) based on the Lugano 2014 classification. Other efficacy, safety, and pharmacokinetic profiles were evaluated as secondary outcome measures. A total of 111 patients were enrolled, of which 90 patients had MZL confirmed by central pathology review, who were mainly with extra-nodal MZL of mucosa-associated lymphoid tissue (MALT, 46.7%) and nodal MZL (35.6%). The majority had late-stage disease, with stage IV accounting for 75.6%. After a median follow-up duration of 24.3 months, the IRC-assessed ORR was 58.9% (95% confidence interval [CI], 48.0-69.2), with rates of complete response and partial response being 11.1% and 47.8%, respectively. The IRC-assessed median duration of response was 34.3 months, and the IRC-assessed median progression-free survival (PFS) was not reached with a 12-month PFS rate of 82.8% (95% CI, 72.6-89.5). The rate of overall survival at 12 months was 91.0% (95% CI, 82.8-95.4). Common all-grade treatment-related adverse events (TRAEs) included anemia (27.9%), neutrophil count decrease (23.4%), white blood cell count decrease (18.0%), platelet count decrease (17.1%), blood present in urine (16.2%), rash (14.4%), and upper respiratory tract infection (10.8%). Thirty-four patients (30.6%) experienced grade 3 or higher TRAEs. Serious TRAEs occurred in 18 patients (16.2%), of which pneumonia (5.4%) was the most common. Seven patients (6.3%) discontinued orelabrutinib due to TRAEs. Orelabrutinib demonstrated high response rates with durable disease remission and was well tolerated in Chinese patients with r/r MZL.© 2023 Wiley Periodicals LLC.