关键词: 骨髓增生异常综合征, 血小板减少, 血小板生成素, 巨核细胞

Abstract:  Myelodysplastic syndromes (MDS) is a kind of bone marrow failure disease. Thrombocytopenia in patients with MDS is a frequent causes of mortality in MDS with 37% to 67% incidence. Thrombocytopenia in MDS is an independent factor predicting worse prognosis associated with increased risk of acute leukemic transformation (AML) and reduces overall survival. In addition, thrombocytopenia in MDS limits the therapeutic efficacy of disease-modifying therapies, such as azacitidine or lenalidomide. Mechanisms of thrombocytopenia in MDS are complicated, involving suppression of megakaryocytic differentiation, enhanced apoptosis, and increased platelet destruction. Platelet transfusion is still the standard treatment option for MDS combined with thrombocytopenia. Recently, novel thrombopoietin (TPO) receptor agonists have showed curative effect in MDS patients in many clinical trials, including reducing bleeding events, decreasing dependency on platelet transfusions and increasing clinical benefits of disease-modifying therapies. Several clinical trials are ongoing to assess the efficacy and safety of novel TPO receptor agonists; the results would further help guide treatment for thrombocytopenia in MDS.

Key words: myelodysplastic syndromes, thrombocytopenia, thrombopoietin, megakaryocytes