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病例报告:免疫性血小板减少症患儿 rhTPO 诱导产生中和抗体后的临床表现及治疗选择

Case report:Clinical manifestations and therapeutic options following rhTPO-induced neutralizing antibody production in a child with immune thrombocytopenia.

作者信息

Wang Nan, Wang Zhifa, Dong Shuyue, Ma Jingyao, Cheng Xiaoling, Wu Runhui

机构信息

Department of Pharmacy, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.

School of Basic Medicine and Clinical Pharmacy, China Pharmaceutical University, Nanjing, Jiangsu, China.

出版信息

Ann Hematol. 2024 Dec;103(12):5941-5944. doi: 10.1007/s00277-024-06055-6. Epub 2024 Nov 4.

Abstract

Recombinant human thrombopoietin (rhTPO) is commonly used to improve low platelet status in immune thrombocytopenia (ITP), as one protein product, even with a very low rate, there is still the possibility to produce neutralizing antibodies of thrombopoietin (TPO). We described a 7-year-old boy with ITP and normal TPO levels who had previously received rhTPO for 2 weeks but showed persistent thrombocytopenia and was misdiagnosed as acquired amegakaryocytic thrombocytopenia (AATP) and ineffectively treated with cyclosporine A (CsA) in combination with avatrombopag (AVA). As suspicious the TPO neutralizing antibody development as re-test of TPO level is 0, the CD20 + deletion antibody drug rituximab (RTX) was prescribed and received efficacy. rhTPO serves as one bio-protein drug and should be cautious with developing neutralizing antibodies if the drug effect is lost. The tests for antibody and/or TPO level should be done for the diagnosis, and the antibody eradication medication as an anti-CD20 antibody, RTX, should be prescribed to delete thes e neutralizing antibodies to recover the TPO level to reattain the response of ITP treatment.

摘要

重组人血小板生成素(rhTPO)常用于改善免疫性血小板减少症(ITP)患者的低血小板状态,作为一种蛋白产品,即使产生中和抗体的几率很低,但仍有可能产生血小板生成素(TPO)的中和抗体。我们描述了一名7岁ITP男孩,其TPO水平正常,此前接受rhTPO治疗2周,但血小板减少持续存在,被误诊为获得性无巨核细胞血小板减少症(AATP),并接受环孢素A(CsA)联合阿伐曲泊帕(AVA)治疗但无效。由于怀疑发生了TPO中和抗体,再次检测TPO水平为0,遂给予CD20+清除抗体药物利妥昔单抗(RTX)治疗并取得疗效。rhTPO作为一种生物蛋白药物,如果失去药物疗效,应警惕产生中和抗体。应进行抗体和/或TPO水平检测以明确诊断,并应使用抗CD20抗体RTX等抗体清除药物来清除这些中和抗体,使TPO水平恢复,从而重新获得ITP治疗反应。

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