Daikeler Thomas, Kötter Ina, Bocelli Tyndall Chiara, Apperley Jane, Attarbaschi Andishe, Guardiola Philippe, Gratwohl Alois, Jantunen Esa, Marmont Alberto, Porretto Ferdinando, Musso Maurizio, Maurer Britta, Rinaldi Nadia, Saccardi Riccardo, Tyndall Alan
Department of Rheumatology, University of Basle, Petersgraben 4, CH-4031 Basle, Switzerland.
Ann Rheum Dis. 2007 Feb;66(2):202-7. doi: 10.1136/ard.2006.056630. Epub 2006 Sep 1.
To evaluate the feasibility of haematopoietic stem cell transplantation (HSCT) in vasculitis.
This is a retrospective analysis of patients who had received HSCT for vasculitic diseases and have been reported to the European League Against Rheumatism autoimmune disease or European Bone Marrow Transplantation ProMISe databases. Information about the disease and outcome was obtained by a questionnaire sent to the referring centres. Response of the disease to HSCT was defined as partial or complete responses according to the ability to reduce immunosuppression after HSCT. In addition, the Medline database was searched for reports on HSCT in patients with vasculitis.
Detailed information was obtained for 15 patients, whose median age at HSCT was 37 years. The diagnoses were cryoglobulinaemia in four patients, Behçet's disease in three patients, Wegener's granulomatosis in three patients, and undifferentiated vasculitis, Churg-Strauss angiitis, polychondritis, Takayasu arteritis and polyarteritis nodosa in one patient each. 14 patients received autologous HSCT and 1 an allogeneic HSCT as the first transplant. In three patients, further transplantation was given because of relapse. The overall response, including all consecutive transplantations (HSCT/patient, n = 1-3, median 1.3) to HSCT, was 93%, with 46% complete responses and 46% partial responses; median (range) duration of response at the time of reporting was 45 (16-84) months. Three patients died, one from advanced disease, one from cancer and one from graft-versus-host disease. The Medline search showed five other patients who were effectively treated with HSCT for vasculitic diseases.
This retrospective study suggests that autologous HSCT is feasible for vasculitis. Its value remains to be tested in prospective controlled studies.
评估造血干细胞移植（HSCT）治疗血管炎的可行性。
这是一项对接受HSCT治疗血管炎性疾病患者的回顾性分析，这些患者的资料已上报至欧洲抗风湿病联盟自身免疫性疾病数据库或欧洲骨髓移植ProMISe数据库。通过向转诊中心发送问卷获取疾病及预后信息。根据HSCT后减少免疫抑制的能力，将疾病对HSCT的反应定义为部分缓解或完全缓解。此外，检索Medline数据库中关于血管炎患者HSCT的报道。
获取了15例患者的详细信息，他们接受HSCT时的中位年龄为37岁。诊断包括4例冷球蛋白血症、3例白塞病、3例韦格纳肉芽肿，以及1例未分化血管炎、1例变应性肉芽肿性血管炎、1例复发性多软骨炎、1例高安动脉炎和1例结节性多动脉炎。14例患者接受自体HSCT，1例接受异基因HSCT作为首次移植。3例患者因复发接受了进一步移植。HSCT的总体反应（包括所有连续移植，HSCT/患者，n = 1 - 3，中位值1.3）为93%，其中46%为完全缓解，46%为部分缓解；报告时反应的中位（范围）持续时间为45（16 - 84）个月。3例患者死亡——1例死于晚期疾病，1例死于癌症，1例死于移植物抗宿主病。Medline检索显示另外5例血管炎患者接受HSCT治疗有效。
这项回顾性研究表明自体HSCT治疗血管炎是可行的。其价值仍有待在前瞻性对照研究中进行验证。
