Albert Michael H, Becker B, Schuster F R, Klein B, Binder V, Adam K, Nienhoff C, Führer M, Borkhardt A
Department of Pediatric Hematology/Oncology, Dr von Haunersches Kinderspital, Ludwig Maximilians University, Munich, Germany.
Pediatr Transplant. 2007 May;11(3):306-11. doi: 10.1111/j.1399-3046.2006.00666.x.
Oral chronic graft vs. host disease (GVHD) frequently presents in patients with sclerotic features of skin GVHD and is often associated with considerable limitations of oral food intake and decreased quality of life. Systemic tacrolimus is efficacious for prophylaxis and treatment of acute and chronic GVHD and topical tacrolimus has shown activity in chronic GVHD skin lesions. We therefore initiated a pilot study to investigate the safety and efficacy of topical tacrolimus ointment in children with oral GVHD. Six patients suffering from oral GVHD (five chronic and one acute) were included in the study. Tacrolimus ointment 0.1% was applied twice daily using sterile gauze. The only side-effects observed were a slight burning discomfort after the first application in one patient and after food intake in another patient. Tacrolimus was absorbed systemically in four of six patients. Of six patients, we observed a complete response in two, a very good partial response (VGPR) in two, and a PR in two patients, respectively. We conclude that topical application of tacrolimus ointment holds promise as a safe and efficacious treatment for oral GVHD in children. The Food and Drug Administration has recently issued a health advisory about a potential cancer risk associated with topical tacrolimus treatment of the skin; therefore, its benefits should be weighed against its potential risks and diligent long-term follow-up should be carried out especially in children.
口腔慢性移植物抗宿主病（GVHD）常见于有皮肤GVHD硬化特征的患者，常伴有口腔食物摄入量显著受限及生活质量下降。全身应用他克莫司对急性和慢性GVHD的预防和治疗有效，局部应用他克莫司已显示对慢性GVHD皮肤病变有疗效。因此，我们开展了一项初步研究，以调查局部应用他克莫司软膏治疗儿童口腔GVHD的安全性和有效性。6例口腔GVHD患者（5例慢性和1例急性）纳入本研究。使用无菌纱布每天两次涂抹0.1%他克莫司软膏。观察到的唯一副作用是1例患者首次用药后及另1例患者进食后出现轻微烧灼感。6例患者中有4例他克莫司被全身吸收。6例患者中，分别观察到2例完全缓解、2例非常好的部分缓解（VGPR）和2例部分缓解（PR）。我们得出结论，局部应用他克莫司软膏有望成为治疗儿童口腔GVHD的一种安全有效的方法。美国食品药品监督管理局最近发布了一项关于局部应用他克莫司治疗皮肤可能存在癌症风险的健康咨询；因此，应权衡其益处与潜在风险，尤其对儿童应进行长期密切随访。
