[Combination therapy with efgartigimod, fostamatinib, eltrombopag and prednisolone for a patient with refractory immune thrombocytopenia].

Abstract

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.