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The early and rapid response to daratumumab in children with chronic refractory immune thrombocytopenia from a referral single centre of China

达拉图穆马 医学 脾切除术 细胞减少 内科学 耐火材料(行星科学) CD38 免疫性血小板减少症 免疫系统 免疫学 肿瘤科 胃肠病学 抗体 单克隆抗体 血小板 骨髓 干细胞 物理 天体生物学 生物 遗传学 脾脏 川地34
作者
Yu Hu,Zhifa Wang,Jingyao Ma,Nan Wang,Jinxi Meng,Shuyue Dong,Zhenping Chen,Xiaoling Cheng,Runhui Wu
出处
期刊:British Journal of Haematology [Wiley]
卷期号:205 (1): 300-305 被引量:2
标识
DOI:10.1111/bjh.19553
摘要

Summary Chronic refractory primary immune thrombocytopenia (CRITP) is currently defined as refractory to multiple therapeutic of second‐line agents with or without splenectomy, faced with the threat of severe bleeding and challenging to obtain effective treatment. Although stable and effective drug therapy is needed, it is tough to find one. Daratumumab (Dara), an anti‐CD38 monoclonal antibody presented the target cloned plasma cells in multiple myeloma, has also been reported to be effective in refractory autoimmune cytopenia in some case or series reports and ongoing clinical trials for adult patients with CRITP. Here, we report the early and durable response of Dara combination with avatrombopag in three CRITP patients (2 male and 1 female aged 12, 5 and 7 years, respectively) in our centre, with a follow‐up period of more than 25 weeks. Before Dara, the duration of immune thrombocytopenia was 9, 1.4 and 4 years, respectively, a baseline platelet count of 4, 6, 9 × 10 9 /L, the bleeding score was all above level 2 and the number of previous drugs was >3. The time to response (R: Plt ≥30 × 10 9 /L with at least a twofold increase in the baseline count) of Dara was on Day 45, 6 and 4 and achieved complete response (CR: Plt ≥100 × 10 9 /L) on Day 51, 6 and 8, the sustained response (SR: Plt >30 × 10 9 /L following Dara at ≥75% of the platelet count assessment at follow‐up end‐point since the patient achieved response) was 48, 175 and 204 days with the follow‐up time of 39.1, 25.9 and 29.7 weeks. The bleeding score decreased from grade 3 to grade 0 during follow‐up. No significant treatment‐related adverse events were found during follow‐up. Dara combination with avatrombopag may be a safe and efficacious therapy for children with CRITP, but it needs to be further explored.

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