Objective: To analyze the clinical characteristics, treatment and prognosis of 16 allogeneic hematopoietic stem cell transplantation (allo- HSCT)- associated thrombotic microangiopathy (TA- TMA) patients.
Methods: The clinical data of 16 TA- TMA cases in 852 patients following allo- HSCT from Jan. 2013 to Jun. 2015 in the First Affiliated Hospital of Soochow University were retrospectively analyzed.
Results: Of all the 852 allo-HSCT recipients, 16 patients were diagnosed as TA-TMA and the 1-year cumulative incidence of TA-TMA was (2.3±0.6)%. Among them, there were 9 males and 7 females, the median age was 41-year-old (12-54), and the median times of diagnosis of TA-TMA were 72 (21- 525) days after HSCT. Additionally, the median platelet counts, hemoglobin, percentage of schistocytes and Lactate dehydrogenase (LDH) levels were 20(11-36) ×10(9)/L, 74(56-99) g/L, 3% (2%- 13%) and 762(309-1 049) U/L, respectively. All 16 cases have normal ADAMTS13 level (over 60%), 10 patients had neurologic dysfunction and elevated creatinine were seen in 7. The major treatment of TA-TMA was withdrawn of calcineurin inhibitors, plasma exchange and corticosteroids. Finally, 8 patients achieved response after treatment and the other patients died of poor response. Compared with TA- TMA who achieved remission after therapy, those who got no response after interventions presented acute GVHD and they had higher schistocytes (62.5% cases>5% vs all cases ≤4%), LDH [826 (674-1 310) U/L vs 636 (309- 941) U/L] and serum creatinine levels [127 (70- 215) μmol/L vs 56 (22- 101) μmol/L].
Conclusion: TA-TMA was a severe complication after allo-HSCT, it could progress to multi-organ injury and was associated with poor outcome, the therapeutic efficacy depends on disease severity and coexisted complications.
目的: 分析异基因造血干细胞移植（allo-HSCT）相关血栓性微血管病（TA-TMA）患者临床特征、疗效及转归。
方法: 回顾性分析2013年1月至2015年6月于苏州大学附属第一医院接受allo-HSCT后发生TA-TMA的16例患者临床资料。
结果: 纳入研究的852例allo-HSCT患者，16例（1.9%）发生TA-TMA，中位随访时间14个月，1年累计发生率为（2.3±0.6）%。16例患者中，男7例，女9例，中位年龄41(12~54）岁，中位发病时间为移植后72(21~525) d, PLT中位数为20(11~36）×109/L，HGB中位数为74(56~99）g/L，LDH中位水平为762(309~1 049) U/L，外周血破碎红细胞比例中位数为3%（2%~13%），所有患者ADAMTS13活性均>60%。10例出现精神症状，7例肌酐水平升高。TA-TMA确诊后的主要治疗措施为钙调磷酸酶体抑制剂的减停、激素及血浆置换疗法，8例经治疗后病情得以控制，治疗有效；8例患者治疗无效死亡。治疗无效组8例患者中5例合并急性肠道移植物抗宿主病（GVHD），治疗有效组患者无一例合并肠道GVHD；8例治疗无效患者中5例外周血破碎红细胞比例>5%，而治疗有效组破碎红比例最高为4%；治疗无效患者LDH及肌酐中位水平均高于治疗有效组，分别为826(674~1 310) U/L对636(309~941）U/L及127(70~215）µmol/L对56(22~101）µmol/L。
结论: TA-TMA是allo-HSCT后的一类严重并发症，可导致全身多器官功能损伤，即使采取治疗早期病死率仍较高，疗效与病情轻重及有无并发症有关。