Department of Dermatology and Venereology, All India Institute of Medical Sciences, New Delhi, India.
Clin Exp Dermatol. 2013 Aug;38(6):659-64. doi: 10.1111/ced.12073.
Corticosteroids are the mainstay of treatment for pemphigus. However, despite the introduction of adjuvant therapy for PV, the mortality rate has remained static over the past 2 decades, and consequently, a new adjuvant therapy that is both safe and effective is needed. Intravenous pulse cyclophosphamide has shown encouraging results in few studies, with a better safety profile than the oral formulation, and with better treatment compliance. We undertook a randomized, prospective, non-blinded trial to assess the efficacy of cyclophosphamide pulse therapy (CPT) as an adjuvant to oral corticosteroid in pemphigus vulgaris (PV). We enrolled 60 patients with mild to moderate active disease to receive either daily oral prednisolone or intravenous CPT prednisolone for 1 year, and they were then followed up for another year. At the end of the study period, the time taken to initiate response was similar in both groups, but in the group who received pulse cyclophosphamide, there was a reduced time to remission, a greater proportion of cases who achieved remission, a lower number of patients who relapsed while on treatment or after stopping treatment, and a lower cumulative dose of corticosteroid. Hence, the overall trend was in favour of CPT, although this was not significant. CPT also had a good safety profile. Based on the results of this trial, CPT may be a useful adjuvant in PV in terms of reducing time to remission, relapse rates and cumulative dose of steroid, and it has a good safety profile.
皮质类固醇是天疱疮治疗的主要药物。然而,尽管 PV 的辅助治疗已经引入,但在过去的 20 年中,死亡率一直保持不变,因此需要一种安全有效的新辅助治疗方法。静脉注射脉冲环磷酰胺在少数研究中显示出令人鼓舞的结果,与口服制剂相比具有更好的安全性,且治疗依从性更好。我们进行了一项随机、前瞻性、非盲试验,以评估环磷酰胺脉冲治疗(CPT)作为天疱疮寻常型(PV)口服皮质类固醇辅助治疗的疗效。我们招募了 60 名患有轻度至中度活动性疾病的患者,他们分别接受每日口服泼尼松龙或静脉内 CPT 泼尼松龙治疗 1 年,然后再随访 1 年。在研究结束时,两组开始反应的时间相似,但在接受脉冲环磷酰胺治疗的组中,缓解时间缩短,达到缓解的比例更高,治疗期间或停药后复发的患者更少,累积的皮质类固醇剂量更低。因此,总体趋势有利于 CPT,尽管这并不显著。CPT 也具有良好的安全性。基于这项试验的结果,CPT 可能在减少缓解时间、复发率和类固醇累积剂量方面对 PV 有辅助作用,且具有良好的安全性。