PDF(Pubmed)
Abstract：
Autologous hematopoietic stem cell transplantation (aHSCT) represents an effective treatment option in patients with severe forms of systemic sclerosis (SSc) by resetting the immune system. Nevertheless, secondary autoimmune disorders and progressive disease after aHSCT might necessitate renewed immunosuppressive treatments. This is particularly challenging when organ dysfunction, i.e., end-stage kidney failure, is present. In this case report, we present the unique case of a 43-year-old female patient with rapidly progressive diffuse systemic sclerosis who underwent aHSCT despite end-stage renal failure as consequence of SSc-renal crisis. Therefore, conditioning chemotherapy was performed with melphalan instead of cyclophosphamide with no occurrence of severe adverse events during the aplastic period and thereafter. After aHSCT, early disease progression of the skin occurred and was successfully treated with secukinumab. Thereby, to the best of our knowledge, we report the first case of successful aHSCT in a SSc-patient with end-stage kidney failure and also the first successful use of an IL-17 inhibitor to treat early disease progression after aHSCT.
摘要：
自体造血干细胞移植（aHSCT）代表了通过重置免疫系统对患有严重形式的系统性硬化症（SSc）的患者的有效治疗选择。然而，aHSCT后继发性自身免疫性疾病和进行性疾病可能需要重新进行免疫抑制治疗.当器官功能障碍时，这尤其具有挑战性，即，终末期肾衰竭,是present。在这个案例报告中,我们介绍了一例43岁女性患者的独特病例,该患者患有快速进展的弥漫性系统性硬化症,尽管由于SSc-肾危象导致终末期肾功能衰竭,但仍接受了aHSCT治疗.因此,使用美法仑代替环磷酰胺进行条件化疗,在再生障碍期及之后未发生严重不良事件.在aHSCT之后，皮肤发生早期疾病进展,苏金单抗治疗成功.因此，据我们所知,我们报道了首例终末期肾衰竭的SSc患者成功实施aHSCT的病例,也是首例成功使用IL-17抑制剂治疗aHSCT后早期疾病进展的病例.
