获得性低巨核细胞性血小板减少性紫癜临床观察

Clinical Study of Acquired Amegakryocyte Thrombocytopenic Purpura

目的通过对获得性低巨核细胞性血小板减少性紫癜(AATP)患者的临床观察和分析,探讨AATP的诊断、发病机制和治疗方法。方法采集16例临床诊断为AATP患者的临床和实验室资料,结合患者的既往史、疾病进程和疗效进行分析,比较不同疗效组间年龄、血小板数、骨髓巨核细胞数、血小板相关抗体或自身抗体阳性率的差别。结果16例患者中既往有免疫性血小板减少史者4例。4例用皮质激素治疗有效,1例切脾,4例加用环孢素A治疗有效,1例加用环磷酰胺有效,6例用上述治疗无效。3例转为再生障碍性贫血(AA),1例转为骨髓增生异常综合征(MDS)。激素的疗效与患者年龄、血小板数、巨核细胞数、血小板相关抗体或自身抗体阳性率无关。结论多数患者激素治疗无效,激素疗效与上述临床指标无关。激素治疗无效者加用环孢素A或其他免疫抑制剂治疗有效。首次发现部分AATP患者由免疫性血小板减少性紫癜转化而来,提示免疫因素在发病机制中的作用。长期随访发现部分病例可转化为其他血液系统疾病。

Objective To explore the diagnosis, pathogenesis and treatment strategy of acquired amegakryocyte thromboeytopenie purpura （AATP） through analysis of the clinical data of patients with AATP. Methods The clinical and laboratory materials together with the past history, disease progress and treatment outcomes of 16 cases diagnosed with AATP were collected and analyzed. Patient＇s age, peripheral platelet count and megakryocyte numbers at presentation and the presence of anti-platelet antibodies or auto-antibodies in different treatment groups were compared. Results 4 patients have a history of immune-induced thrombocytopenia. 4 cases were responsive to cortieosteroid therapy, 1 was responsive to splenectomy. Among the remaining patients, 4 responded to cyclosporine A, 1 responded to cyelophosphamide and 6 were non-responsive to the above therapies. Response to corticosteroid was not related with any of the above parameters. 3 of our patients progressed to aplastic anemia and 1 to myelodysplastie syndrome. Conclusion Only a few AATP patients responded to corticosteroid. Others were responsive to additional cyclosporine A or CTX therapy. We are the first to find that some AATP cases progressed from immune-induced thrombocytopenia which implicated the role of immunological factors in the pathogenesis of the disease. Long- term follow- up of patients are necessary for some cases may progress to other types of hematologic diseases.