Efficacy and Immunomodulating Properties of Eltrombopag in Aplastic Anemia following Autologous Stem Cell Transplant: Case Report and Review of the Literature

Thrombopoietin receptor agonists (TPO-RA) are currently indicated for the treatment of chronic immune thrombocytopenia and relapsed refractory aplastic anemia. However, the offlabel use of these drugs is more and more frequent, including in the setting of aplasia secondary to chemotherapy and hemopoietic stem cell transplant (SCT). Growing evidence suggests that mechanisms of action of TPO-RA go beyond the TPO-receptor stimulation and point at the immunomodulating properties of these drugs. Here, we present a case of prolonged bone marrow aplasia secondary to autologous SCT treated with eltrombopag. We describe the clinical efficacy and the immunomodulating effect of this drug on inflammatory cytokine profile and bone marrow histology. Furthermore, we provide a review of the most recent literature highlighting the efficacy and safety of TPO-RA after SCT and chemotherapy for hematologic conditions.

Automated summary

Thrombopoietin receptor agonists (TPO-RA) are used to treat chronic immune thrombocytopenia and relapsed refractory aplastic anemia. However, their off-label use is becoming more common, especially in cases of aplasia after chemotherapy and stem cell transplant. Recent evidence suggests that TPO-RA have immunomodulating properties in addition to stimulating platelet production. In this case study, eltrombopag was effective in treating prolonged bone marrow aplasia caused by autologous stem cell transplant. The drug also had an effect on inflammatory cytokine levels and bone marrow histology. This review of literature highlights the efficacy and safety of TPO-RA after stem cell transplant and chemotherapy for hematologic conditions.