Successful autologous stem cell transplantation in two patients with juvenile dermatomyositis
Successful autologous stem cell transplantation in two patients with juvenile dermatomyositis
复制标题
DOI：
10.3109/03009740903096622
复制
发表时间：
2010-01-01
影响因子：
2.1
通讯作者：
Wulffraat, N.
中科院分区：
文献类型：
--
作者：
Holzer, U.;van Royen-Kerkhof, A.;Wulffraat, N.
青少年皮肌炎(JDM)是一种病因不明的慢性炎症性疾病，影响肌肉和皮肤。我们报告了两名严重进展性JDM患者，尽管接受了包括甲氨蝶呤(MTX)、类固醇、免疫球蛋白、环孢菌素A和利妥昔单抗的治疗，但仍出现了痉挛并依赖于轮椅。由于这种难治性疾病，在用氟达拉滨、环磷酰胺和抗胸腺细胞球蛋白免疫消融处理后，使用CD3/CD19耗尽的移植物进行了自体干细胞移植(ASCT)。这导致两名患者的病情显著改善和持续缓解。我们证明ASCT对于严重的、难治性JDM患者是一种低毒的治疗选择。
Juvenile dermatomyositis (JDM) is a chronic inflammatory disorder of unknown aetiology that affects muscle and skin. We report on two patients with severe progressive JDM who developed contractures and were wheelchair dependent despite therapy including rnethotrexate (MTX), steroids, immunoglobulins, cyclosporin A, and rituximab. On account of the refractory disease, autologous stem cell transplantation (ASCT) was performed using a CD3/CD19-depleted graft after immunoablative conditioning with fludarabine, cyclophosphamide, and anti-thymocyte globulin. This induced a dramatic improvement and sustained remission of the disease in both patients. We demonstrate that ASCT is a therapeutic option with low toxicity for patients with severe, refractory JDM.