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已发表论文
罗米司亭、达那唑与血小板生成素受体激动剂联合疗法用于慢性难治性免疫性血小板减少症免疫重建：病例系列
Authors Wang SX, Tan GM, Zou ZM, Zou LF, Tu YC, Kong FC, Xie X, Li F
Received 8 July 2025
Accepted for publication 11 October 2025
Published 22 October 2025 Volume 2025:14 Pages 1159—1167
DOI https://doi.org/10.2147/ITT.S543982
Checked for plagiarism Yes
Review by Single anonymous peer review
Peer reviewer comments 2
Editor who approved publication: Professor Michael Shurin
Shi-Xuan Wang,1– 3,* Gen-Mei Tan,1– 3,* Zhi-Ming Zou,1– 3 Li-Fang Zou,1– 3 Ye-Chao Tu,1– 3 Fan-Cong Kong,1– 3 Xing Xie,1 Fei Li1– 3
1Center of Hematology, The First Affiliated Hospital of Nanchang University, Nanchang, People’s Republic of China; 2Jiangxi Clinical Research Center for Hematologic Disease, Nanchang, People’s Republic of China; 3Institute of Lymphoma and Myeloma, Nanchang University, Nanchang, People’s Republic of China
*These authors contributed equally to this work
Correspondence: Fei Li, Center of Hematology, The First Affiliated Hospital of Nanchang University, 17 Yongwai Road, Nanchang, Jiangxi Province, 330000, People’s Republic of China, Email yx021021@sina.com
Background: Chronic refractory primary immune thrombocytopenia is defined as thrombocytopenia refractory to multiple therapies using second-line agents with or without splenectomy. Patients face the threat of severe bleeding, and it is challenging to achieve effective treatment. Although thrombopoietin receptor agonists (TPO-RAs) and immunomodulators have been established as second-line options, their efficacy as monotherapy remains suboptimal.
Methods: Here, we report the early and durable response to a novel triple regimen combining romiplostim, danazol, and either hetrombopag or eltrombopag in two patients with chronic refractory primary immune thrombocytopenia at our center, with a follow-up exceeding 4 months.
Results: The bleeding score decreased from grade 3/4 to grade 0 during the follow-up. No significant treatment-related adverse events were observed during the follow-up.
Conclusion: Romiplostim in combination with danazol and hetrombopag or eltrombopag may be a safe and efficacious therapy for chronic refractory primary immune thrombocytopenia; however, this needs to be further explored.
Keywords: chronic refractory immune thrombocytopenia, TPO receptor agonists, romiplostim, danazol, combination therapy
1Center of Hematology, The First Affiliated Hospital of Nanchang University, Nanchang, People’s Republic of China; 2Jiangxi Clinical Research Center for Hematologic Disease, Nanchang, People’s Republic of China; 3Institute of Lymphoma and Myeloma, Nanchang University, Nanchang, People’s Republic of China
*These authors contributed equally to this work
Correspondence: Fei Li, Center of Hematology, The First Affiliated Hospital of Nanchang University, 17 Yongwai Road, Nanchang, Jiangxi Province, 330000, People’s Republic of China, Email yx021021@sina.com
Background: Chronic refractory primary immune thrombocytopenia is defined as thrombocytopenia refractory to multiple therapies using second-line agents with or without splenectomy. Patients face the threat of severe bleeding, and it is challenging to achieve effective treatment. Although thrombopoietin receptor agonists (TPO-RAs) and immunomodulators have been established as second-line options, their efficacy as monotherapy remains suboptimal.
Methods: Here, we report the early and durable response to a novel triple regimen combining romiplostim, danazol, and either hetrombopag or eltrombopag in two patients with chronic refractory primary immune thrombocytopenia at our center, with a follow-up exceeding 4 months.
Results: The bleeding score decreased from grade 3/4 to grade 0 during the follow-up. No significant treatment-related adverse events were observed during the follow-up.
Conclusion: Romiplostim in combination with danazol and hetrombopag or eltrombopag may be a safe and efficacious therapy for chronic refractory primary immune thrombocytopenia; however, this needs to be further explored.
Keywords: chronic refractory immune thrombocytopenia, TPO receptor agonists, romiplostim, danazol, combination therapy