Ojaimi S, O'Connor K, Lin M W, Schifter M, Fulcher D A
Department of Clinical Immunology, Westmead Hospital, Sydney, New South Wales, Australia.
Intern Med J. 2015 Mar;45(3):284-92. doi: 10.1111/imj.12674.
Pemphigus vulgaris (PV) is a rare autoimmune blistering condition. Treatment typically combines corticosteroids with another immunosuppressive agent, such as azathioprine, mycophenolate mofetil (MMF) or rituximab.
This study aims to compare these second agents for their clinical efficacy and steroid-sparing effect.
This was a single-centre, retrospective observational cohort study of 21 patients with oral PV over a 6-year period, 18 of whom were newly diagnosed. Of the latter, the first 13 were initially given azathioprine, progressing to MMF and then rituximab on treatment failure, while the next five patients started directly on MMF.
Of the 13 newly diagnosed patients, 2/13 were intolerant of azathioprine, and only 1/11 was controlled, with a median time to treatment failure (MTTF) of 254 days. MMF was given to 17 patients, either de novo (5) or after azathioprine (12), and was significantly more effective, controlling activity in 4/17 patients, and for a significantly longer time (MTTF 395 days, P = 0.019). All 13 patients failing MMF received rituximab, seven required a second dose, and three, a third dose. All patients responded, with 11/13 able to cease steroids. Control was maintained for a similar time to MMF (MTTF 364 days, P = NS). Rituximab also had the best steroid-sparing effect followed by MMF, then azathioprine. Side-effects were common with azathioprine, while the other two agents were well tolerated.
Rituximab was the most effective of the three immunosuppressives for PV, although repeat dosing was frequently required. These observations have significant implications for the choice of drugs for this condition.
寻常型天疱疮（PV）是一种罕见的自身免疫性水疱病。治疗通常将皮质类固醇与另一种免疫抑制剂联合使用，如硫唑嘌呤、霉酚酸酯（MMF）或利妥昔单抗。
本研究旨在比较这些第二种药物的临床疗效和激素节省效果。
这是一项单中心回顾性观察队列研究，对21例口腔PV患者进行了为期6年的研究，其中18例为新诊断患者。在后者中，前13例最初给予硫唑嘌呤，治疗失败后进展为MMF，然后是利妥昔单抗，而后5例患者直接开始使用MMF。
在13例新诊断患者中，2/13对硫唑嘌呤不耐受，只有1/11得到控制，治疗失败的中位时间（MTTF）为254天。17例患者接受了MMF，其中5例为初治，12例在使用硫唑嘌呤后使用，MMF显著更有效，使4/17的患者病情得到控制，且控制时间显著更长（MTTF 395天，P = 0.019）。所有13例MMF治疗失败的患者均接受了利妥昔单抗治疗，7例需要第二剂，3例需要第三剂。所有患者均有反应，11/13能够停用类固醇。控制时间与MMF相似（MTTF 364天，P = 无显著性差异）。利妥昔单抗的激素节省效果也最好，其次是MMF，然后是硫唑嘌呤。硫唑嘌呤的副作用很常见，而其他两种药物耐受性良好。
利妥昔单抗是三种用于PV的免疫抑制剂中最有效的，尽管经常需要重复给药。这些观察结果对这种疾病的药物选择具有重要意义。
