Chen Huan, Liu Kai-Yan, Liu Dai-Hong, Xu Lan-Ping, Han Wei, Chen Yu-Hong, Huang Xiao-Jun
Institute of Hematology, People's Hospital, Peking University, Beijing 100044, China.
Zhonghua Yi Xue Za Zhi. 2008 Aug 5;88(30):2127-30.
To retrospectively investigate the safety and efficacy of mycofenolate mofetil (MMF) in combination with cyclosporine A (CSA) or tacrolimus (FK506) in the treatment of graft-versus-host disease(GVHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT).
44 patients with various hematological diseases underwent allo-HSCT: 23 received transplant from HLA-matched siblings and 21 from mismatched related donors. The conditioning regimens included modified busulphan plus cyclophosphamide (Cy) regimen for the patients diagnosed as with leukemia and myelodysplastic syndrome (MDS), and Cy plus antihuman thymocyte globulin (ATG) for severe aplastic anemia (sAA). Cyclosporine A (CsA), short term methotrexate (MTX), and MMF were used for the prophylaxis of GVHD for all patients. Acute GVHD (aGVHD) and chronic GVHD (cGVHD) were diagnosed according to the international commonly accepted GVHD diagnostic criteria. MMF was added to the standard C-sA/FK506 with prednisone (PSE) as either the first line therapy, or the second line salvage therapy.
15 patients were diagnosed as with aGVHD, and 29 with cGVHD. MMF was administrated as a first-line therapy drug in 19 patients and as a second-line therapy drug in 25 patients. For aGVHD the overall response rate was 80% with a complete response (CR) rate of 33.33% and a partial response (PR) rate of 46.66%. The response rates were 70% and 100% respectively for the first line and second line therapy. In 58.3% of the responsive patients the daily dose of PSE could be reduced. For chronic GVHD the overall response rate was 86.2% with a CR rate of 41.37% and a PR rate of 44.83%, and the response rate were 100% and 84.21% for the first and second line therapy respectively. PSE taper was realized in 70.83% of the responsive patients. Side effects occurred in 7 patients, all reversible. The infectious complication rate was 34.09%, only one patient died of interstitial pneumonia. The follow-up of 22 months (10 approximately 65 months) showed that 36 patients (81.82%) survived.
MMF combined with CSA/FK506 is effective and well-tolerated in treatment for aGVHD and cGVHD.
回顾性研究霉酚酸酯（MMF）联合环孢素A（CSA）或他克莫司（FK506）治疗异基因造血干细胞移植（allo-HSCT）后移植物抗宿主病（GVHD）的安全性和有效性。
44例患有各种血液病的患者接受了allo-HSCT：23例接受了HLA匹配同胞的移植，21例接受了不匹配相关供者的移植。预处理方案包括，对于诊断为白血病和骨髓增生异常综合征（MDS）的患者采用改良白消安加环磷酰胺（Cy）方案，对于重型再生障碍性贫血（sAA）患者采用Cy加抗人胸腺细胞球蛋白（ATG）方案。所有患者均使用环孢素A（CsA）、短期甲氨蝶呤（MTX）和MMF预防GVHD。急性移植物抗宿主病（aGVHD）和慢性移植物抗宿主病（cGVHD）根据国际公认的GVHD诊断标准进行诊断。MMF被添加到以泼尼松（PSE）为标准的C-sA/FK506方案中，作为一线治疗或二线挽救治疗。
15例患者被诊断为aGVHD，29例为cGVHD。MMF作为一线治疗药物用于19例患者，作为二线治疗药物用于25例患者。对于aGVHD，总缓解率为80%，完全缓解（CR）率为33.33%，部分缓解（PR）率为46.66%。一线和二线治疗的缓解率分别为70%和100%。在58.3%的缓解患者中，PSE的每日剂量可以减少。对于慢性移植物抗宿主病，总缓解率为86.2%，CR率为41.37%，PR率为44.83%，一线和二线治疗的缓解率分别为100%和84.21%。70.83%的缓解患者实现了PSE减量。7例患者出现副作用，均为可逆性。感染并发症发生率为34.09%，仅1例患者死于间质性肺炎。22个月（10至65个月）的随访显示，36例患者（81.82%）存活。
MMF联合CSA/FK506治疗aGVHD和cGVHD有效且耐受性良好。
