Chams-Davatchi Cheyda, Esmaili Nafiseh, Daneshpazhooh Maryam, Valikhani Mahin, Balighi Kamran, Hallaji Zahra, Barzegari Masoumeh, Akhyani Maryam, Ghodsi S Zahra, Seirafi Hassan, Nazemi Mohammad-Javad Tabrizi, Mortazavi Hossein, Mirshams-Shahshahani Mostafa
Pemphigus Research Unit, Department of Dermatology, Tehran University for Medical Sciences, Razi Hospital, Tehran, Iran.
J Am Acad Dermatol. 2007 Oct;57(4):622-8. doi: 10.1016/j.jaad.2007.05.024. Epub 2007 Jun 21.
Pemphigus is a severe autoimmune blistering disease affecting the skin and mucosa. Mortality is high in the absence of treatment. Nowadays, treatment is based mainly on corticosteroids and cytotoxic drugs; however, because of the rarity of the disease worldwide, there is not yet a standard treatment based on randomized controlled trials, and the treatment used is based mainly on the experience of experts.
The aim of this study was to compare the efficacy and safety of 4 treatment regimens for pemphigus vulgaris: prednisolone alone, prednisolone plus azathioprine, prednisolone plus mycophenolate mofetil, and prednisolone plus intravenous cyclophosphamide pulse therapy.
One hundred twenty new cases of pemphigus vulgaris were enrolled. These patients were randomly allocated into 1 of 4 treatment groups (each comprising 30 patients) and received prednisolone (P), prednisolone and azathioprine (P/A), prednisolone and mycophenolate mofetil (P/MM), and prednisolone and intravenous cyclophosphamide pulse therapy (P/PC). They were followed up for 1 year at the Pemphigus Research Unit.
In groups P, P/A, P/MM, and P/PC, 23 (76.5%), 24 (80%), 21 (70%), and 22 (73.3%) of the patients, respectively, followed the regimen for the full 1-year period. The mean total dose of prednisolone administered in groups P, P/A, P/MM, and P/PC was 11631 mg (standard deviation [SD] = 7742), 7712 mg (SD = 955), 9798 mg (SD = 3995), and 8276 mg (SD = 810), respectively. The mean total dose of prednisolone in group P (prednisolone alone) was 11,631 mg, The mean total dose of prednisolone in the 3 cytotoxic groups was 8652 mg. By using analysis of variance, the difference was statistically significant (P = .047). In the cytotoxic groups, there was a significant difference between the P/A and P/MM groups (P = .007), but not between P/A and P/PC (P = .971), and P/MM and P/PC (P = .670). Side effects were not significantly different among the 4 groups.
Larger sample sizes and blind design are suggested for future studies.
The efficacy of prednisolone is enhanced when it is combined with a cytotoxic drug. The most efficacious cytotoxic drug to reduce steroid was found to be azathioprine, followed by cyclophosphamide (pulse therapy), and mycophenolate mofetil.
天疱疮是一种严重的自身免疫性水疱病，可累及皮肤和黏膜。若不进行治疗，死亡率很高。如今，治疗主要基于糖皮质激素和细胞毒性药物；然而，由于该病在全球范围内较为罕见，目前尚无基于随机对照试验的标准治疗方案，所采用的治疗方法主要基于专家经验。
本研究旨在比较4种寻常型天疱疮治疗方案的疗效和安全性：单独使用泼尼松龙、泼尼松龙加硫唑嘌呤、泼尼松龙加霉酚酸酯以及泼尼松龙加静脉注射环磷酰胺脉冲疗法。
纳入120例新诊断的寻常型天疱疮患者。这些患者被随机分为4个治疗组之一（每组30例患者），分别接受泼尼松龙（P）、泼尼松龙和硫唑嘌呤（P/A）、泼尼松龙和霉酚酸酯（P/MM）以及泼尼松龙和静脉注射环磷酰胺脉冲疗法（P/PC）。在天疱疮研究单位对他们进行了1年的随访。
在P组、P/A组、P/MM组和P/PC组中，分别有23例（76.5%）、24例（80%）、21例（70%）和22例（73.3%）患者完整地遵循治疗方案达1年。P组、P/A组、P/MM组和P/PC组泼尼松龙的平均总剂量分别为11631mg（标准差[SD]=7742）、7712mg（SD=955）、9798mg（SD=3995）和827mg（SD=810）。P组（单独使用泼尼松龙）泼尼松龙的平均总剂量为11631mg，3个细胞毒性药物组泼尼松龙的平均总剂量为8652mg。通过方差分析，差异具有统计学意义（P=0.047）。在细胞毒性药物组中，P/A组和P/MM组之间存在显著差异（P=0.007），但P/A组和P/PC组之间（P=0.971）以及P/MM组和P/PC组之间（P=0.670）无显著差异。4组之间的副作用无显著差异。
建议未来研究采用更大样本量和盲法设计。
泼尼松龙与细胞毒性药物联合使用时疗效增强。发现最有效的减少类固醇用量的细胞毒性药物是硫唑嘌呤，其次是环磷酰胺（脉冲疗法）和霉酚酸酯。
