Magro L, Catteau B, Coiteux V, Bruno B, Jouet J-P, Yakoub-Agha I
Service des Maladies du Sang, Lille, France.
Bone Marrow Transplant. 2008 Dec;42(11):757-60. doi: 10.1038/bmt.2008.252. Epub 2008 Sep 1.
Treatment of sclerodermatous chronic GVHD (cGVHD) remains disappointing. Imatinib mesylate enables selective, dual inhibition of the transforming growth factor beta (TGFbeta) and PDGF pathways. Recently, the drug's effects on fibroblasts have been reported in both in vitro and in vivo studies. The inhibition of fibroblast growth and decreased collagen production in dermal fibroblasts is thus a logical therapeutic approach. Two patients who developed refractory sclerodermatous cGVHD following allo-SCT received imatinib mesylate at the dose of 400 mg/day. In both patients, the scleroderma symptoms disappeared within 3 months of initiation of the treatment. At the time of this report, the two patients were both alive and had a very good skin response. This report shows that imatinib is effective in patients with refractory sclerodermatous cGVHD. Considering its well-documented clinical profile in other diseases, imatinib is a promising candidate for the treatment of sclerodermatous cGVHD.
硬皮病样慢性移植物抗宿主病（cGVHD）的治疗效果仍然令人失望。甲磺酸伊马替尼能够选择性地双重抑制转化生长因子β（TGFβ）和血小板衍生生长因子（PDGF）信号通路。最近，该药物对成纤维细胞的作用已在体外和体内研究中得到报道。因此，抑制成纤维细胞生长并减少真皮成纤维细胞中的胶原蛋白产生是一种合理的治疗方法。两名异基因造血干细胞移植（allo-SCT）后发生难治性硬皮病样cGVHD的患者接受了每日400毫克剂量的甲磺酸伊马替尼治疗。两名患者在治疗开始后的3个月内硬皮病症状均消失。在撰写本报告时，两名患者均存活，且皮肤反应良好。本报告表明，伊马替尼对难治性硬皮病样cGVHD患者有效。鉴于其在其他疾病中已得到充分记录的临床情况，伊马替尼是治疗硬皮病样cGVHD的一个有前景的候选药物。
