Mycophenolate mofetil:: A new therapeutic option in the treatment of blistering autoimmune diseases
Mycophenolate mofetil:: A new therapeutic option in the treatment of blistering autoimmune diseases
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DOI：
10.1016/s0190-9622(99)70084-8
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发表时间：
1999-06-01
影响因子：
13.8
通讯作者：
Peter, RU
中科院分区：
文献类型：
--
作者：
Grundmann-Kollmann, M;Korting, HC;Peter, RU
背景：霉酚酸酯(MMF)是霉酚酸(MPA)的一种酯，于1995年被美国食品和药物管理局批准，目前主要用于预防肾移植患者的排斥反应。这种药物似乎在治疗牛皮癣和风湿性关节炎方面也有价值。近年来，已有6例霉酚酸酯联合大剂量强的松治疗水泡性自身免疫性疾病的报道获得成功。目的：在此基础上，我们对几例水泡性自身免疫性疾病患者应用了这种新的治疗方案。除了联合使用霉酚酸酯和大剂量强的松外，我们还想评价霉酚酸酯单药治疗水疱性自身免疫性疾病是否也有效。方法：我们用霉酚酸酯治疗5例患有严重寻常型天疱疮或大疱性类天疱疮的患者。2例患者接受MMF联合大剂量强的松治疗，3例患者接受MMF单一治疗。据我们所知，这是第一个用MMF单一疗法成功治疗寻常型天疱疮和大疱性类天疱疮的报告。结果：所有患者在治疗8~11周内症状完全消失。接受MMF单一治疗的患者与接受MMF和大剂量强的松联合治疗的患者的疗效相同。结论：我们的经验强烈表明，即使是严重的寻常型天疱疮和大疱性类天疱疮患者，MMF单一治疗也可能有效。此外，与免疫抑制联合疗法相比，MMF单一疗法，至少在短期内，提供了副作用较少的优势，并被我们的患者很好地耐受。
Background: Mycophenolate mofetil (MMF), an ester of mycophenolic acid (MPA), was approved by the Food and Drug Administration in 1995 and is currently primarily indicated for the prophylaxis of rejection in renal transplant patients. The drug seems also to be of value in the treatment of psoriasis and rheumatic arthritis. Recently there have been 6 reported cases of successful treatment of blistering autoimmune diseases with MMF in combination with high dose prednisone therapy.Objective: On the basis of these: reports we: administered this new treatment regimen to several patients with blistering autoimmune diseases. Besides using a combination of MMF and high-dose prednisone we wanted to evaluate whether MMF monotherapy is also effective in the treatment of blistering autoimmune diseases.Methods: We administered MMF to 5 patients who had severe pemphigus vulgaris or bullous pemphigoid. Two patients received MMF in combination with high-dose prednisone therapy and 3 patients received MMF monotherapy. To our knowledge, this is the first report of successful treatment of pemphigus vulgaris and bullous pemphigoid with MMF monotherapy.Results: All patients were completely free of symptoms within 8 to 11 weeks of therapy. Patients who had received MMF monotherapy responded as well to treatment as those who received a combination of MMF and high-dose prednisone.Conclusion: Our experiences strongly suggest that MMF monotherapy may be effective for patients even with severe pemphigus vulgaris and bullous pemphigoid. In addition, MMF monotherapy, at least over the short term, offers the advantage of fewer side effects in comparison to immunosuppressive combination therapy and was well tolerated by our patients.