Eltrombopag Treatment for Patients with Thrombocytopenia Post-Transplantation Showed a Good Chance of Platelet Recovery

Allogeneic hematopoietic stem cell transplantation (Allo-HSCT) dramatically improves the outcomes of patients with hematologic disorders. However, thrombocytopenia post-transplantation is a common and severe complication, which not only diminishes the quality of life but also leads to fatal bleeding events. Blood transfusion could not be a permanent solution and it seldom achieves satisfactory results. Eltrombopag (EPAG), a novel oral thrombopoietin receptor agonist (TPO-RA), has been shown promising effects in thrombocytopenia caused by immune thrombocytopenic purpura (ITP) and severe aplastic anemia (SAA). Yet, the effectiveness of EPAG for patients with thrombocytopenia post-transplantation still needs to be discovered. From September 2017 to April 2019, twenty-two patients were diagnosed as thrombocytopenia post-transplantation in our center, including 10 patients with poor graft function (PGF) and 12 patients with secondary failure of platelet recovery (SFPR). Since insensitive to traditional treatment such as thrombopoietin and intravenous immunoglobin, all these patients received the EPAG treatment. So far, 16 (72.7%) patients achieved hematopoietic overall recovery (OR) and 11(50.0%) patients were complete recovery (CR). 12(75.0%) of the responded patients discontinued or tapered the drug and 9 patients (56.3%) successfully maintained their best response. Among 16 responded patients, the median time to achieve PR status was 33 (range, 7-105) days and the median time of CR was 67 (range, 40-175) days. Interestingly, 11 (91.7%) patients from SFPR subgroup achieved OR, while only 5 (50%) patients from PGF subgroup achieved OR. Besides, patients with normal spleen size (n=16) experience a higher CR rate than splenomegaly patients (n=8) (10 vs 1, 71.4% vs 12.5%). On univariate and multivariate analysis, PGF was identified as the independent risk factor of OR (P=0.050) and splenomegaly was identified as the independent risk factor of CR (P=0.019). Interestingly, 3 patients with enlarged spleen received ruxolitinib to minimize the spleen size during the EPAG treatment. Accompanied by the reduction in spleen size, the platelet counts increased gradually and all these 3 patients achieved OR. In contrast, only 2 of the other 5 splenomegaly patients who without ruxolitinib achieved OR. In conclusion, EPAG treatment for patients with thrombocytopenia post-transplantation showed a good chance of platelet recovery.