Lopez Francisco, Parker Pablo, Nademanee Auayporn, Rodriguez Roberto, Al-Kadhimi Zaid, Bhatia Ravi, Cohen Sandra, Falk Peter, Fung Henry, Kirschbaum Mark, Krishnan Amrita, Kogut Neil, Molina Arturo, Nakamura Ryotaro, O'Donnell Margaret, Popplewell Leslie, Pullarkat Vinod, Rosenthal Joseph, Sahebi Firoozeh, Smith Eileen, Snyder David, Somlo George, Spielberger Ricardo, Stein Anthony, Sweetman Robert, Zain Jasmine, Forman Stephen
Division of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, California 91010, USA.
Biol Blood Marrow Transplant. 2005 Apr;11(4):307-13. doi: 10.1016/j.bbmt.2005.01.011.
Current treatment of chronic graft-versus-host disease (cGVHD) with prednisone (PSE) alone or with added cyclosporine or tacrolimus still has a very high failure and complication rate, and new treatment approaches are needed for both primary and salvage therapy. Mycophenolate mofetil (MMF) is an immunosuppressive agent currently in use for acute graft-versus-host disease prophylaxis. To determine whether MMF had activity in the treatment of cGVHD, we added MMF to standard cyclosporine, tacrolimus, and/or PSE as salvage/second-line (n = 24) or first-line (n = 10) therapy in 34 patients. Nine (90%) of 10 patients receiving first-line and 18 (75%) of 24 receiving second-line MMF therapy responded. Twelve (35%) patients had a complete remission, 15 (44%) had a partial remission, 5 (15%) had stable disease, and only 2 (6%) had progressive disease. Out of 30 patients receiving PSE, 22 (73%) were able to decrease PSE doses (median decrease of 50%; range, 25%-100%). With a median follow-up of 24 months (range, 6-28 months), 29 (85%) patients are alive. Three patients had to discontinue MMF because of abdominal cramps within 3 months of starting treatment. These data suggest that MMF is an active, well-tolerated agent in the treatment of cGVHD and may have a beneficial effect on the survival of patients with this complication.
目前，单独使用泼尼松（PSE）或联合环孢素或他克莫司治疗慢性移植物抗宿主病（cGVHD）的失败率和并发症发生率仍然很高，因此无论是一线治疗还是挽救治疗都需要新的治疗方法。霉酚酸酯（MMF）是一种目前用于预防急性移植物抗宿主病的免疫抑制剂。为了确定MMF在治疗cGVHD中是否有效，我们将MMF添加到标准的环孢素、他克莫司和/或PSE中，作为34例患者的挽救/二线治疗（n = 24）或一线治疗（n = 10）。接受一线MMF治疗的10例患者中有9例（90%）有反应，接受二线MMF治疗的24例患者中有18例（75%）有反应。12例（35%）患者完全缓解，15例（44%）部分缓解，5例（15%）病情稳定，只有2例（6%）病情进展。在30例接受PSE治疗的患者中，22例（73%）能够降低PSE剂量（中位降低50%；范围25%-100%）。中位随访24个月（范围6-28个月），29例（85%）患者存活。3例患者在开始治疗后3个月内因腹部绞痛而不得不停用MMF。这些数据表明，MMF在治疗cGVHD方面是一种有效的、耐受性良好的药物，可能对患有这种并发症的患者的生存有有益影响。
