[Eltrombopag for refractory thrombocytopenia in patients with allogeneic hematopoietic stem cell transplantation]

Objective: To evaluate the efficacy and safety of eltrombopag in post-HSCT thrombocytopenia. Methods: A total of 10 patients who underwent post-HSCT thrombocytopenia at Peking University center, who had been treated with eltrombopag, were retrospectively evaluated. Results: Of the 10 cases, 5 males and 5 females with a median of 34 years old (range, 17-54 years), 5 patients were acute myeloid leukemia, 3 with acute lymphoid leukemia and 2 with severe aplastic anemia. Nine patients had undergone haplo-identical donor transplantation, and one patient was a matched related recipient. All patients had failed prior treatment for thrombocytopenia before eltrombopag started. The median time when eltrombopag started was 221 days (range, 73-917 days) after transplantation. Five patients (50%) had achieved CR. The cumulative incidence of 30-day CR was 35.7%. The median time to platelet recovery ≥ 50 × 10⁹/L without transfusion support was 16 days (range, 10-56 days). At the last follow-up, three of the patients with CR had withdrawal eltrombopag and remained normal platelet counts. No patients experienced drug-related adverse events. Conclusion: Eltrombopag is effective and well tolerated in patients with refractory post-HSCT thrombocytopenia.