A prospective, multicenter, randomized trial comparing steroids and pulse cyclophosphamide versus steroids and oral cyclophosphamide in the treatment of generalized Wegener's granulomatosis
A prospective, multicenter, randomized trial comparing steroids and pulse cyclophosphamide versus steroids and oral cyclophosphamide in the treatment of generalized Wegener's granulomatosis
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DOI：
10.1002/art.1780401213
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发表时间：
1997-12-01
影响因子：
--
通讯作者：
Longy-Boursier, M
中科院分区：
文献类型：
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作者：
Guillevin, L;Cordier, JF;Longy-Boursier, M
客观的。探讨口服环磷酰胺 (CYC) 与冲击环磷酰胺 (CYC) 联合皮质类固醇 (CS) 治疗系统性韦格纳肉芽肿 (WG) 的有效性和副作用。方法。新诊断的全身性 WG 患者被纳入一项前瞻性随机试验。诊断时，在随机分组之前，每位患者每天注射甲泼尼龙，持续 3 天，然后每天口服泼尼松（1 mg/kg/天）和 0.7 gm/m(2) CYC 脉冲。然后将患者随机分配接受泼尼松加静脉脉冲 CYC（A 组）或泼尼松加口服 CYC（B 组）作为一线治疗，CYC 给予至少 1 年，然后逐渐减量并停药。 结果。该研究纳入了 50 名患者：A 组 27 名，B 组 23 名。6 个月时，A 组 24 名患者（88.9%）病情缓解，B 组 18 名患者（78.3%）缓解。试验结束时，18 名 A 组患者（66.7%）和 13 名 B 组患者（56.5%）处于缓解状态，A 组中 66.7% 的患者出现副作用，B 组为 69.6%。B 组（69.6%）的感染性副作用明显高于 A 组（40.7%）（P < 0.05）。口服CYC治疗患者的卡氏肺孢子虫肺炎发生率（30.4%）高于脉冲CYC治疗患者（11.1%），A组患者9例（33.3%）和B组患者10例（43.5%）死亡。精算曲线显示，A 组（59.2%）的复发频率明显高于 B 组（13%）（P = 0.02）。结论。我们的结果表明，脉冲 CYC 在实现 WG 初步缓解方面与口服 CYC 一样有效，并且副作用较少，死亡率较低。然而，从长远来看，脉冲 CYC 治疗并不能像口服 CYC 那样维持缓解或防止复发。
Objective. To investigate the effectiveness and side effects of oral versus pulse cyclophosphamide (CYC) in combination with corticosteroids (CS) in the treatment of systemic Wegener's granulomatosis (WG).Methods. Patients with newly diagnosed systemic WG were enrolled in a prospective, randomized trial. At the time of diagnosis, prior to randomization, every patient received a daily injection of methylprednisolone for 3 days, followed by daily oral prednisone (1 mg/kg/ day) and a 0.7-gm/m(2) pulse of CYC. Patients were then randomly assigned to receive either prednisone plus intravenous pulse CYC (group A) or prednisone plus oral CYC (group B) as first-line treatment, CYC was given for at least 1 year and was then progressively tapered and discontinued.Results. Fifty patients were included in the study: 27 in group A and 23 in group B. At 6 months, 24 group A patients (88.9%) were in remission, versus 18 group B patients (78.3%). At the end of the trial, 18 group A patients (66.7%) and 13 group B patients (56.5%) were in remission, In group A, 66.7% of the patients experienced side effects, versus 69.6% in group B, Infectious side effects were significantly more frequent in group B (69.6%) than in group A (40.7%) (P < 0.05). The incidence of Pneumocystis carinii pneumonia was higher in oral CYC-treated patients (30.4%) than in pulse CYC-treated patients (11.1%), Nine group A patients (33.3%) and 10 group B patients (43.5%) died. Actuarial curves showed that relapses were significantly more frequent in group A (59.2%) than in group B (13%) (P = 0.02).Conclusion. Our results indicate that pulse CYC is as effective as oral CYC in achieving initial remission of WG and is associated with fewer side effects and lower mortality. However, in the long term, treatment with pulse CYC does not maintain remission or prevent relapses as well as oral CYC.