Blinatumomab 在日本复发/难治性 B 细胞前体急性淋巴细胞白血病成年和儿科患者中的安全性和疗效：扩展队列的最终结果。

Safety and Efficacy of Blinatumomab in Japanese Adult and Pediatric Patients with Relapsed/Refractory B-Cell Precursor Acute Lymphoblastic Leukemia: Final Results from an Expansion Cohort.

机构信息

Division of Hematology/Oncology, Kanagawa Children's Medical Center, Yokohama, Japan.

Department of Pediatric Oncology, National Cancer Center Hospital, Tokyo, Japan.

出版信息

Acta Haematol. 2022;145(6):592-602. doi: 10.1159/000525835. Epub 2022 Jul 5.

DOI:10.1159/000525835

PMID:35790143

Abstract

INTRODUCTION

The safety and efficacy of blinatumomab, a CD19/CD3 bispecific T-cell engager (BiTE®) molecule, was evaluated in an expansion cohort of the phase 1b/2 study (NCT02412306) in Japanese adult (n = 14) and pediatric (n = 17) patients with relapsed/refractory Philadelphia-negative B-cell precursor (BCP) acute lymphoblastic leukemia (ALL).

MATERIALS AND METHODS

Globally recommended blinatumomab doses were administered to adult (9-28 μg/day) and pediatric (5-15 μg/m2/day) patients. Primary endpoint was the incidence of treatment-emergent adverse events (TEAEs) and treatment-related AEs.

RESULTS

All adult and pediatric patients experienced ≥1 TEAE. Grade ≥3 TEAEs were observed in 11 (79%) adult and 15 (88%) pediatric patients. Blinatumomab was discontinued in 1 (6%) pediatric patient due to treatment-related grade 4 cytokine release syndrome. Fatal AEs such as disease progression and multiple-organ dysfunction syndrome, which were not treatment-related, were reported in 2 (12%) pediatric patients. Eleven (79%) adults achieved complete remission (CR)/CR with partial hematological recovery (CRh) within the first two blinatumomab cycles. Nine of 10 adult patients with CR/CRh and evaluable minimal residual disease (MRD) achieved MRD response. CR/CRh was achieved by 5 (29%) pediatric patients, of which two had MRD response.

CONCLUSION

In conclusion, blinatumomab was safe and efficacious in Japanese patients with relapsed/refractory BCP ALL.

摘要

简介

blinatumomab 是一种 CD19/CD3 双特异性 T 细胞衔接器（BiTE®）分子，在复发/难治性费城染色体阴性 B 细胞前体（BCP）急性淋巴细胞白血病（ALL）的 1b/2 期研究（NCT02412306）扩展队列中，对日本成年（n=14）和儿科（n=17）患者进行了安全性和疗效评估。

材料和方法

全球推荐的 blinatumomab 剂量用于成年（9-28μg/天）和儿科（5-15μg/m2/天）患者。主要终点是治疗后出现的不良事件（TEAEs）和治疗相关不良事件（AEs）的发生率。

结果

所有成年和儿科患者均经历了≥1 次 TEAEs。11 例（79%）成年和 15 例（88%）儿科患者出现≥3 级 TEAEs。1 例（6%）儿科患者因治疗相关的 4 级细胞因子释放综合征而停用 blinatumomab。2 例（12%）儿科患者报告了与治疗无关的致命不良事件（AE），如疾病进展和多器官功能障碍综合征。11 例（79%）成年患者在前两个 blinatumomab 周期内达到完全缓解（CR）/CR 伴部分血液学恢复（CRh）。10 例有 CR/CRh 和可评估微小残留病（MRD）的成年患者中有 9 例达到了 MRD 应答。5 例（29%）儿科患者达到 CR/CRh，其中 2 例有 MRD 应答。