Marcellus D C, Altomonte V L, Farmer E R, Horn T D, Freemer C S, Grant J, Vogelsang G B
Department of Oncology, Johns Hopkins University, Baltimore, MD, USA.
Blood. 1999 Jan 1;93(1):66-70.
Chronic graft-versus-host disease (GVHD) is the most common late complication of allogeneic bone marrow transplantation (BMT). The sclerodermatous form of the disease is often refractory to standard treatment modalities. Based on reports of response to etretinate, a synthetic retinoid, among patients with scleroderma, we have added etretinate to the treatment regimen of 32 patients with refractory sclerodermatous chronic GVHD. This case series is comprised mainly of patients who had chronic GVHD of long duration (median of 30 months before the initiation of etretinate). Most had failed to respond to three or more agents before etretinate treatment was started. Clinical response was assessed after 3 months of therapy. Five patients did not complete a 3-month trial. Among the 27 patients evaluable for response, 20 showed improvement including softening of the skin, flattening of cutaneous lesions, increased range of motion, and improved performance status. Four showed no response after 3 months of therapy and 3 had progression of their sclerosis. Overall, etretinate has been fairly well tolerated in our patients, with skin breakdown and/or ulceration leading to its discontinuation in 6 patients. We believe the results in our patients are encouraging and suggest that further evaluation of etretinate in the treatment of sclerodermatous chronic GVHD is warranted.
慢性移植物抗宿主病（GVHD）是同种异体骨髓移植（BMT）最常见的晚期并发症。该病的硬皮病形式通常对标准治疗方式无效。基于硬皮病患者对合成维甲酸依曲替酯反应的报道，我们在32例难治性硬皮病样慢性GVHD患者的治疗方案中加入了依曲替酯。该病例系列主要由病程较长的慢性GVHD患者组成（开始使用依曲替酯前的中位病程为30个月）。大多数患者在开始依曲替酯治疗前对三种或更多药物治疗无效。治疗3个月后评估临床反应。5例患者未完成3个月的试验。在可评估反应的27例患者中，20例有改善，包括皮肤变软、皮损变平、活动范围增加和功能状态改善。4例在治疗3个月后无反应，3例硬化进展。总体而言，我们的患者对依曲替酯耐受性相当好，6例患者因皮肤破损和/或溃疡而停药。我们认为我们患者的结果令人鼓舞，并表明有必要对依曲替酯治疗硬皮病样慢性GVHD进行进一步评估。
