艾曲波帕治疗异基因造血干细胞移植后难治性血小板减少的临床研究

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 × 10(9)/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.