A 50-year-old man with immune thrombocytopenic purpura (ITP) was initially treated with prednisolone after 10 years of observation, but did not respond. Treatment with the thrombopoietin receptor agonist (TPO-RA) eltrombopag failed as well. After a transient partial response with fostamatinib, platelet counts decreased again, and the patient showed a severe bleeding tendency. Additional treatment with rituximab or TPO-RAs also failed, and thus efgartigimod was added to fostamatinib. The patient finally maintained a partial response with a combination of efgartigimod, fostamatinib, eltrombopag and prednisolone. Although new agents for ITP (fostamatinib and efgartigimod) have recently been introduced in Japan, there is little clinical experience with combination therapies incorporating these agents. This case suggests that four-drug combination might be beneficial.
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[Combination therapy with efgartigimod, fostamatinib, eltrombopag and prednisolone for a patient with refractory immune thrombocytopenia].
Rinsho Ketsueki
March 2025
Department of hematology, Toyonaka Municipal Hospital.
A 50-year-old man with immune thrombocytopenic purpura (ITP) was initially treated with prednisolone after 10 years of observation, but did not respond. Treatment with the thrombopoietin receptor agonist (TPO-RA) eltrombopag failed as well. After a transient partial response with fostamatinib, platelet counts decreased again, and the patient showed a severe bleeding tendency.
View Article and Find Full Text PDFArticle Synopsis
- Japanese guidelines recommend two thrombopoietin receptor agonists (eltrombopag and romiplostim), rituximab, or splenectomy for treating glucocorticoid-resistant ITP.
- Fostamatinib and efgartigimod were approved in Japan in 2023 and 2024, offering new options for refractory ITP patients.
- Recent clinical trials show promise for new treatments like avatrombopag, rilzabrutinib, and sutimlimab, signaling significant advancements in ITP management.
Novel therapeutics and future directions for refractory immune thrombocytopenia.
Br J Haematol
October 2023
Department of Hematology, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
Article Synopsis
- Immune thrombocytopenia (ITP) is an autoimmune disorder affecting blood platelet levels, with about 1 in 20,000 people impacted, and some patients are resistant to standard therapies, known as refractory ITP.
- There is a need for new treatments, with several novel agents currently in clinical trials, targeting new mechanisms in ITP that current medications do not address.
- The manuscript discusses these promising therapeutics, their development stages, innovative trial designs, the importance of assessing patients' quality of life, and weighing drug benefits against potential side effects.
Efgartigimod alfa for the treatment of primary immune thrombocytopenia.
Ther Adv Hematol
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Lombardi Comprehensive Cancer Center, MedStar Georgetown University Hospital, 3800 Reservoir Road, Lombardi Cancer Center Podium D, Washington, DC 20007, USA.
Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by isolated thrombocytopenia. Most patients with ITP have antiplatelet antibodies of the immunoglobulin G (IgG) subtype which through interaction with platelet and megakaryocyte glycoproteins result in increased platelet destruction and inhibition of platelet production. There are a variety of therapeutic options available for the treatment of ITP including corticosteroids, IVIgG, TPO-RA, rituximab, fostamatinib, and splenectomy.
View Article and Find Full Text PDFRecent progress in ITP treatment.
Int J Hematol
March 2023
Hematology Project Foundation, Affiliated to the Department of Hematology, "S. Bortolo" Hospital, Contrà San Francesco 41, 36100, Vicenza, Italy.
In this review, the recently approved drugs avatrombopag and fostamatinib, which were not extensively covered within 2019 international recommendations for ITP, will be discussed in some detail. Avatrombopag appears more convenient than eltrombopag as it does not require dietary restrictions or subcutaneous administration like romiplostim. However, data on quality of life (QoL) are lacking and the rate of thromboembolic events in exposed patients is not negligible.
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