Combination Therapy with Romiplostim, Danazol, and a Thrombopoietin Receptor Agonist for Immune Restoration in Chronic Refractory Immune Thrombocytopenia: A Case Series.

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.