Maurer B, Hensel M, Max R, Fiehn C, Ho A D, Lorenz H M
Department of Internal Medicine, Unit V (Haematology, Oncology and Rheumatology), University of Heidelberg, Germany.
Ann Rheum Dis. 2006 Jan;65(1):127-9. doi: 10.1136/ard.2005.035410. Epub 2005 May 26.
Myeloablative chemotherapy and autologous haematopoietic stem cell transplantation (HSCT) may provide a therapeutic option in severe Behçet's disease (BD) with pulmonary involvement.
Two patients with BD with pulmonary involvement refractory to conventional immunosuppressive treatment underwent HSCT 1999. Stem cells were mobilised with cyclophosphamide (2 and 4 g/m(2)) and subsequently enriched ex vivo for CD34+ cells. The conditioning regimen used was melphalan (200 mg/m(2)). Outcome was measured by improvement of clinical features, function of affected organs, serological markers, need for immunosuppressive chemotherapy after transplant, and relapse. In both cases HSCT was successful, with good response and without serious complications. After 5 years of follow up one patient is in complete remission without immunosuppressive drugs and one has partial remission, needing low dose corticosteroids (8 mg/day).
In these two patients myeloablative chemotherapy, followed by HSCT could be performed safely with marked improvement. In comparison with other observational studies the duration of more than 5 years of remission is remarkable, and its full duration is still unknown.
清髓性化疗及自体造血干细胞移植（HSCT）可能为重症伴有肺部受累的白塞病（BD）提供一种治疗选择。
两名伴有肺部受累且对传统免疫抑制治疗无效的BD患者于1999年接受了HSCT。采用环磷酰胺（2和4 g/m²）动员干细胞，随后在体外富集CD34⁺细胞。所采用的预处理方案为美法仑（200 mg/m²）。通过临床特征改善情况、受累器官功能、血清学标志物、移植后免疫抑制化疗需求及复发情况来衡量治疗结果。在这两例患者中，HSCT均获成功，反应良好且无严重并发症。经过5年随访，一名患者在未使用免疫抑制药物的情况下完全缓解，另一名患者部分缓解，需要低剂量皮质类固醇（8 mg/天）。
在这两名患者中，清髓性化疗后行HSCT可安全进行且有显著改善。与其他观察性研究相比，超过5年的缓解期颇为显著，其完整时长仍未知。
