Department of Nephrology, Royal Berkshire Hospital, Reading, Berkshire RG1 5AN, UK.
Rheumatology (Oxford). 2010 Mar;49(3):556-62. doi: 10.1093/rheumatology/kep411. Epub 2009 Dec 23.
To determine the safety and efficacy of prolonged treatment with 15-deoxyspergualin (DSG, gusperimus) in patients with relapsing WG.
Patients with relapsing WG treated with DSG were studied. Other immunosuppressants except corticosteroids were withdrawn and DSG, 0.5 mg/kg/day, self-administered subcutaneously for up to 21 days, in 28-day cycles. The cycle was terminated early for white blood cell count <4 x 10(9)/l. The prednisolone dose was adjusted according to the clinical state. End points were disease remission, relapse, Birmingham Vasculitis Activity Score (BVAS), prednisolone dose and safety.
Eleven patients, five (45%) of whom were female, received a total of 15 treatment periods with DSG. The median (range) duration of each treatment period was 6.8 (3.3-15.9) months. Ten (90.9%) patients responded in 13/15 courses after a median of 1.7 (0.7-2.7) months and six (54.5%) achieved remission after 7.7 (1.9-13.5) months. Two (18.2%) patients relapsed while continuing to receive DSG. Remission was maintained in other patients while DSG was continued. However, 7/8 relapsed after DSG withdrawal. The median BVAS fell from 10 (3-22) at baseline to 3 (0-16) at the end of each treatment period (P = 0.002). Median prednisolone doses were reduced from 20 (5-30) mg/day at baseline to 10 (5-25) mg/day at the end of each treatment period (P = 0.052). Three severe adverse events occurred in two patients.
Extended treatment with DSG was effective in the majority of patients with relapsing WG and permitted prednisolone reduction. There was no unexpected toxicity associated with prolonged DSG administration.
确定延长使用 15-脱氧精胍菌素（DSG，古柏西普）治疗复发性韦格纳肉芽肿（WG）患者的安全性和疗效。
研究接受 DSG 治疗的复发性 WG 患者。除皮质类固醇外，其他免疫抑制剂均停用，DSG 0.5mg/kg/天，皮下自我给药，28 天为 1 个周期。白细胞计数<4x10(9)/l 时提前终止周期。根据临床情况调整泼尼松龙剂量。终点为疾病缓解、复发、伯明翰血管炎活动评分（BVAS）、泼尼松龙剂量和安全性。
11 名患者，其中 5 名（45%）为女性，共接受了 15 个 DSG 治疗期。每个治疗期的中位数（范围）持续时间为 6.8（3.3-15.9）个月。13/15 个疗程中有 10 名（90.9%）患者在中位数 1.7（0.7-2.7）个月后出现反应，6 名（54.5%）在 7.7（1.9-13.5）个月后达到缓解。2 名（18.2%）患者在继续接受 DSG 治疗时复发。在继续使用 DSG 的情况下，其他患者的缓解得以维持。然而，DSG 停药后，7/8 例患者复发。BVAS 中位数从基线时的 10（3-22）降至每个治疗期末的 3（0-16）（P=0.002）。泼尼松龙剂量中位数从基线时的 20（5-30）mg/天降至每个治疗期末的 10（5-25）mg/天（P=0.052）。两名患者出现 3 例严重不良事件。
延长 DSG 治疗对大多数复发性 WG 患者有效，并允许减少泼尼松龙的剂量。与延长 DSG 给药相关的毒性没有意外发生。
