Kötter I, Daikeler T, Amberger C, Tyndall A, Kanz L
University Hospital, Department of Internal Medicine II (Oncology, Hematology, Immunology, Rheumatology, Pulmology), Tubingen, Germany.
Clin Nephrol. 2005 Dec;64(6):485-9. doi: 10.5414/cnp64485.
Autologous peripheral blood stem cell transplantation (autoPBSCT) is increasingly and successfully applied to patients with treatment-resistant autoimmune diseases, mainly multiple sclerosis and systemic sclerosis, but also juvenile idiopathic arthritis and systemic lupus erythematosus. We intended to analyze the effects of autoPBSCT in patients with treatment-resistant systemic vasculitis by analyzing the outcome of 4 patients from our own hospital, and comparing them to cases reported in the literature.
4 patients with treatment-resistant vasculitis (Wegener granulomatosis, Churg Strauss syndrome, Takayasu arteritis and relapsing polychondritis) received an autologous PBSCT. Stem cell mobilization was performed with cyclophosphamide (CY) and G-CSF, stem cells were purged by positively selecting CD34+ stem cells over a CliniMacs device, and the conditioning was performed with high dose CY and anti-thymocyte globulin (ATG).
AutoPBSCT was well tolerated in all 4 patients. The patient with WG achieved complete remission although cANCA persisted, the other patients are in good partial remissions and respond to maintenance treatments which had been ineffective before PBSCT (CSA, azathioprin). Glucocorticosteroids (GC) could be reduced to a maximum of 10 mg in all patients. Shortly after the procedure, reactivation of viruses from the herpes family occurred in 3 of the patients and had to be treated. In the data base, 25 patients transplanted for severe systemic vasculitis are registered, in the literature, 6 additional vasculitis patients remitting after autoPBSCT are reported.
Autologous PBSCT is feasible and effective in severe, treatment-resistant forms of systemic vasculitis. Data are sparse, further prospective studies are needed. These should also aim at evaluating more optimal regimens for conditioning and purging during PBSCT, as in most of the vasculitis patients reported until now, mostly good partial remissions, but less complete remissions were achieved.
自体外周血干细胞移植（autoPBSCT）越来越多地成功应用于治疗抵抗性自身免疫性疾病患者，主要是多发性硬化症和系统性硬化症，也包括幼年特发性关节炎和系统性红斑狼疮。我们旨在通过分析我院4例患者的治疗结果，并与文献报道的病例进行比较，来分析autoPBSCT对治疗抵抗性系统性血管炎患者的影响。
4例治疗抵抗性血管炎患者（韦格纳肉芽肿、变应性肉芽肿性血管炎、高安动脉炎和复发性多软骨炎）接受了自体PBSCT。采用环磷酰胺（CY）和粒细胞集落刺激因子（G-CSF）进行干细胞动员，通过在CliniMacs设备上阳性选择CD34+干细胞来清除干细胞，并采用高剂量CY和抗胸腺细胞球蛋白（ATG）进行预处理。
所有4例患者对autoPBSCT耐受性良好。韦格纳肉芽肿患者实现了完全缓解，尽管胞浆型抗中性粒细胞胞浆抗体（cANCA）仍然存在，其他患者处于良好的部分缓解状态，并对自体PBSCT前无效的维持治疗（环孢素A、硫唑嘌呤）有反应。所有患者的糖皮质激素（GC）可减至最多10毫克。术后不久，3例患者出现疱疹家族病毒再激活，必须进行治疗。数据库中登记了25例因严重系统性血管炎接受移植的患者，文献中还报道了另外6例自体PBSCT后病情缓解的血管炎患者。
自体PBSCT在严重的、治疗抵抗性的系统性血管炎形式中是可行且有效的。数据稀少，需要进一步的前瞻性研究。这些研究还应旨在评估自体PBSCT期间更优化的预处理和清除方案，因为在目前报道的大多数血管炎患者中，大多实现了良好的部分缓解，但完全缓解较少。
