Nagler A, Pines M
Department of Bone Marrow Transplantation, Hadassah University Hospital, Jerusalem, Israel.
Transplantation. 1999 Dec 15;68(11):1806-9. doi: 10.1097/00007890-199912150-00027.
In chronic graft-versus-host disease (cGvHD), skin fibrosis, contractures, and an increase in collagen content form the hallmark. We report a successful treatment of a cGvHD patient by topical application of halofuginone, an inhibitor of collagen alpha1(I) gene expression.
Halofuginone-containing ointment was applied daily on the left side of the neck and shoulder of a cGvHD patient. Collagen alpha1(I) gene expression and collagen content in skin biopsy specimens were evaluated by in situ hybridization and sirius red staining, respectively.
After 3 and 6 months, a marked reduction in skin collagen synthesis was observed, accompanied with increase neck rotation on the treated side. After cessation of treatment, the sclerosis, skin tightness, and collagen alpha1(I) gene expression returned to baseline level. No adverse effects were observed, and no plasma levels of halofuginone could be detected.
Halofuginone may provide a promising novel and safe therapy for cGvHD patients.
在慢性移植物抗宿主病（cGvHD）中，皮肤纤维化、挛缩以及胶原蛋白含量增加是其特征。我们报告了通过局部应用卤夫酮（一种胶原蛋白α1（I）基因表达抑制剂）成功治疗一名cGvHD患者的案例。
将含卤夫酮的药膏每日涂抹于一名cGvHD患者的颈部和肩部左侧。分别通过原位杂交和天狼星红染色评估皮肤活检标本中胶原蛋白α1（I）基因表达和胶原蛋白含量。
3个月和6个月后，观察到皮肤胶原蛋白合成显著减少，同时治疗侧颈部旋转度增加。停止治疗后，硬化、皮肤紧绷及胶原蛋白α1（I）基因表达恢复至基线水平。未观察到不良反应，且未检测到血浆中的卤夫酮水平。
卤夫酮可能为cGvHD患者提供一种有前景的新型安全治疗方法。
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