Qian Xiaowen, Zhang Dufei, Li Kai, Chen Weiming, Zhuang Peijun, Wang Hongsheng, Lei Zhixian, Li Yan, Eldridge James, Dong Kuiran, Zhai Xiaowen
Children's Hospital of Fudan University, 399 Wanyuan Road, Minhang District, Shanghai, China.
Hainan Branch of the Children's Hospital of Fudan University, Haikou, China.
Drugs Real World Outcomes. 2025 Mar;12(1):115-123. doi: 10.1007/s40801-024-00468-5. Epub 2024 Dec 20.
The humanized anti-disialoganglioside-2 monoclonal antibody naxitamab was approved in the USA in 2020 for the treatment of patients with relapsed/refractory high-risk neuroblastoma, limited to the bone or bone marrow, in combination with granulocyte-macrophage colony-stimulating factor. Treatment with naxitamab under expanded access was initiated by physicians from the Children's Hospital of Fudan University, Shanghai, China, in August 2021.
We reviewed all suspected adverse reactions (ARs) reported to the Y-mAbs Argus Global Pharmacovigilance Safety Database for patients treated with naxitamab under expanded access in China from 1 August 2021 to 31 July 2022.
We assessed patient demographics and the safety profile of naxitamab over multiple treatment cycles.
At the data cutoff, 41 patients with relapsed/refractory high-risk neuroblastoma had received a total of 150 treatment cycles (451 infusions) of naxitamab. The median number of cycles completed was three; 13 patients (32%) were receiving ongoing naxitamab treatment. The median patient age was 3 years (range 1-9 years) and 63% were female. Overall, ARs were reported in 89/150 cycles (59%); serious ARs were reported in 23/150 cycles (15%). The cumulative reporting rate (ARs/cycle) decreased after 3 versus 12 months of expanded access: all ARs (8.7-4.6), serious ARs (0.9-0.3), hypotension (1.4-1.0), flushing (0.7-0.5), cough (0.6-0.3), pain (0.5-0.2), and hypoxia (0.3-0.2).
During the first 12 months of expanded access treatment in China, 41 patients received naxitamab therapy with a cumulative 451 infusions administered. Over the course of this expanded access program, a reduction in the AR rate, including serious ARs, was observed as more patients were initiated and proceeded to later treatment cycles. While additional research is needed, the observed decrease in the AR rate may be attributed to clinicians' increased knowledge of AR management and hands-on experience with naxitamab-treated patients.
人源化抗二唾液酸神经节苷脂-2单克隆抗体那昔妥单抗于2020年在美国获批，用于治疗复发/难治性高危神经母细胞瘤患者，病变局限于骨骼或骨髓，联合粒细胞-巨噬细胞集落刺激因子使用。2021年8月，中国上海复旦大学附属儿科医院的医生开始在扩大使用途径下用那昔妥单抗进行治疗。
我们回顾了2021年8月1日至2022年7月31日在中国扩大使用途径下接受那昔妥单抗治疗的患者向Y-mAbs Argus全球药物警戒安全数据库报告的所有疑似不良反应（ARs）。
我们评估了患者的人口统计学特征以及那昔妥单抗在多个治疗周期中的安全性概况。
在数据截止时，41例复发/难治性高危神经母细胞瘤患者共接受了150个那昔妥单抗治疗周期（451次输注）。完成的周期数中位数为3个；13例患者（32%）正在接受那昔妥单抗持续治疗。患者年龄中位数为3岁（范围1 - 9岁），63%为女性。总体而言，150个周期中有89个（59%）报告了ARs；150个周期中有23个（15%）报告了严重ARs。扩大使用途径3个月与12个月后累积报告率（ARs/周期）下降：所有ARs（8.7 - 4.6）、严重ARs（0.9 - 0.3）、低血压（1.4 - 1.0）、潮红（0.7 - 0.5）、咳嗽（0.6 - 0.3）、疼痛（0.5 - 0.2）和缺氧（0.3 - 0.2）。
在中国扩大使用途径治疗的前12个月中，41例患者接受了那昔妥单抗治疗，共进行了451次输注。在这个扩大使用途径项目过程中，随着更多患者开始并进入后续治疗周期，观察到AR率有所降低，包括严重ARs。虽然还需要进一步研究，但观察到的AR率下降可能归因于临床医生对AR管理的知识增加以及对那昔妥单抗治疗患者的实际经验。
