Kentouche Karim, Zintl Felix, Angerhaus Dorothea, Fuchs Dietlinde, Hermann Johann, Schneppenheim Reinhard, Budde Ulrich
Department of Hematology, Children's Hospital, Friedrich Schiller University, Jena, Germany.
Semin Thromb Hemost. 2006 Mar;32(2):98-104. doi: 10.1055/s-2006-939765.
Transplantation-associated microangiopathy (TAM) is a severe complication of stem cell transplantation. Although TAM shares many features with idiopathic thrombotic thrombocytopenic purpura or hemolytic uremic syndrome, the prognosis of TAM is worse. Clinical similarities and the observation that uncleaved ultralarge von Willebrand factor (ULVWF) multimers are found in the circulation of patients suffering from TAM suggest a defect in VWF proteolysis that may be due to a deficiency in ADAMTS13 activity. In this study the course of 28 consecutive patients, who received an allogeneic stem cell transplant was correlated to ADAMTS13 activity. Before stem cell transplantation, mean ADAMTS13 activity was within normal range. Within the first 8 weeks, mean activity declined to less than half the baseline activity. Furthermore, most of the patients showed normalization of ADAMTS13 activity. Low levels of ADAMTS13 activity were not correlated with clinical signs of thrombotic microangiopathy. However, two patients with clinical TAM had the lowest activity of all patients when suffering a severe bout of microangiopathy. Plasma exchange was not able to normalize ADAMTS13 deficiency in these patients, suggesting inactivation or consumption of ADAMTS13 activity in TAM.
移植相关微血管病（TAM）是干细胞移植的一种严重并发症。尽管TAM与特发性血栓性血小板减少性紫癜或溶血尿毒综合征有许多共同特征，但TAM的预后更差。临床相似性以及在TAM患者循环中发现未裂解的超大血管性血友病因子（ULVWF）多聚体这一观察结果表明，VWF蛋白水解存在缺陷，这可能是由于ADAMTS13活性不足所致。在本研究中，28例接受异基因干细胞移植的连续患者的病程与ADAMTS13活性相关。在干细胞移植前，ADAMTS13平均活性在正常范围内。在最初的8周内，平均活性降至基线活性的一半以下。此外，大多数患者的ADAMTS13活性恢复正常。低水平的ADAMTS13活性与血栓性微血管病的临床体征无关。然而，两名临床诊断为TAM的患者在发生严重微血管病发作时，其活性在所有患者中最低。血浆置换未能使这些患者的ADAMTS13缺乏恢复正常，这表明TAM中ADAMTS13活性被灭活或消耗。
