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达雷妥尤单抗与罗米司亭联合治疗长期难治性原发性免疫性血小板减少症——病例报告

Daratumumab and Romiplostim Combined Therapy for a Long-Standing Refractory Primary Immune Thrombocytopenia - Case Report.

作者信息

Zoubi Ibrahim, Warwar Amir, Perek Shoshan, Preis Meir

机构信息

Institute of Hematology, Lady Davis Carmel Medical Center, Haifa, Israel.

Bruce and Ruth Rappaport Faculty of Medicine, Technion- Israel Institute of Technology, Haifa, Israel.

出版信息

Immunotargets Ther. 2025 Jan 6;14:1-5. doi: 10.2147/ITT.S487895. eCollection 2025.

DOI:10.2147/ITT.S487895
PMID:39802214
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11721704/
Abstract

Multi-refractory immune thrombocytopenia (ITP) is not uncommon and associated with high morbidity and mortality rates. Although the precise mechanism of ITP is not yet fully understood, a therapeutic approach that relies on using a single agent in each treatment line may not be sufficient in this population. We report the case of a 67-year-old female with long-standing multi-refractory ITP treated with a combination of Daratumumab and Romiplostim who achieved a durable response for more than 42 weeks. Owing to the presentation of chronic and refractory disease, we used a dual-agent approach to address early immune destruction and promote megakaryocyte platelet production. Three doses of Daratumumab were administered during the induction phase (weeks 0,1,5) and then at less frequent intervals - every 4-12 weeks for total of 4 doses during the maintenance phase. Romiplostim was administered weekly, with dose modification depending on the platelet count. We hypothesize that when combined with thrombopoietin receptor agonists (TPO-RAs), daratumumab administered at less frequent intervals over an extended period can be safely used, resulting in a prolonged response.

摘要

多重难治性免疫性血小板减少症(ITP)并不罕见,且发病率和死亡率较高。尽管ITP的确切机制尚未完全明确,但在这一人群中,每次治疗阶段仅使用单一药物的治疗方法可能并不足够。我们报告了一例67岁患有长期多重难治性ITP的女性患者,使用达雷妥尤单抗和罗米司亭联合治疗后获得了超过42周的持久缓解。由于该患者呈现慢性难治性疾病,我们采用了双药联合方法来应对早期免疫破坏并促进巨核细胞血小板生成。在诱导期(第0、1、5周)给予三剂达雷妥尤单抗,然后在维持期以较低频率给药——每4 - 12周给药一次,共给药4剂。罗米司亭每周给药,剂量根据血小板计数进行调整。我们推测,与血小板生成素受体激动剂(TPO - RAs)联合使用时,在较长时间内以较低频率给药的达雷妥尤单抗可以安全使用,并能产生持久缓解。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6698/11721704/2098ad4dcbbe/ITT-14-1-g0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6698/11721704/2098ad4dcbbe/ITT-14-1-g0001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6698/11721704/2098ad4dcbbe/ITT-14-1-g0001.jpg

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本文引用的文献

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A Novel Anti-CD38 Monoclonal Antibody for Treating Immune Thrombocytopenia.一种用于治疗免疫性血小板减少症的新型抗 CD38 单克隆抗体。
N Engl J Med. 2024 Jun 20;390(23):2178-2190. doi: 10.1056/NEJMoa2400409.
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Clonal haematopoiesis and dysregulation of the immune system.克隆性造血与免疫系统失调。
Nat Rev Immunol. 2023 Sep;23(9):595-610. doi: 10.1038/s41577-023-00843-3. Epub 2023 Mar 20.
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Daratumumab as a novel treatment option in refractory ITP.达雷木单抗作为难治性 ITP 的一种新型治疗选择。
Blood Cells Mol Dis. 2023 Mar;99:102724. doi: 10.1016/j.bcmd.2023.102724. Epub 2023 Jan 13.
4
Long-term complete remission of refractory severe idiopathic immune thrombocytopenia (ITP) treated with daratumumab.使用达雷妥尤单抗治疗难治性重度特发性免疫性血小板减少症(ITP)的长期完全缓解
Ann Hematol. 2023 Jan;102(1):245-247. doi: 10.1007/s00277-022-05035-y. Epub 2022 Nov 17.
5
Current therapeutic strategies and perspectives in refractory ITP: What have we learned recently?难治性 ITP 的当前治疗策略和观点:最近我们学到了什么?
Front Immunol. 2022 Aug 8;13:953716. doi: 10.3389/fimmu.2022.953716. eCollection 2022.
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Clonal hematopoiesis in primary immune thrombocytopenia.原发性免疫性血小板减少症中的克隆性造血
Blood Cancer J. 2022 Mar 15;12(3):40. doi: 10.1038/s41408-022-00641-5.
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Daratumumab for immune thrombotic thrombocytopenic purpura.达雷妥尤单抗治疗免疫性血栓性血小板减少性紫癜。
Blood Adv. 2022 Feb 8;6(3):993-997. doi: 10.1182/bloodadvances.2021005124.
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Daratumumab, an original approach for treating multi-refractory autoimmune cytopenia.达雷妥尤单抗,一种治疗多重难治性自身免疫性血细胞减少症的全新方法。
Haematologica. 2021 Dec 1;106(12):3198-3201. doi: 10.3324/haematol.2021.279232.
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Daratumumab in rituximab-refractory autoimmune haemolytic anaemia.达雷妥尤单抗治疗利妥昔单抗难治性自身免疫性溶血性贫血
Br J Haematol. 2021 Sep;194(5):931-934. doi: 10.1111/bjh.17655. Epub 2021 Jun 28.
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Ann Hematol. 2021 May;100(5):1351-1353. doi: 10.1007/s00277-020-04063-w. Epub 2020 May 14.