THALIDOMIDE FOR THE TREATMENT OF CHRONIC GRAFT-VERSUS-HOST DISEASE
THALIDOMIDE FOR THE TREATMENT OF CHRONIC GRAFT-VERSUS-HOST DISEASE
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DOI：
10.1056/nejm199204163261604
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发表时间：
1992-04-16
影响因子：
158.5
通讯作者：
SANTOS, GW
中科院分区：
文献类型：
--
作者：
VOGELSANG, GB;FARMER, ER;SANTOS, GW
背景 异基因骨髓移植是治疗血液肿瘤、再生障碍性贫血和遗传性免疫缺陷的公认疗法。慢性移植物抗宿主病（GVHD）是存活超过100天的患者的主要并发症。沙利度胺已被实验证明是有效的治疗GVHD。 我们治疗了23例常规治疗无效的慢性GVHD患者和21例“高危”慢性GVHD患者（被鉴定为具有以下三种风险因素中的至少两种：从急性GVHD演变而来的慢性GVHD、苔藓样皮肤或粘膜变化和肝功能障碍;这类患者具有高死亡率）与沙利度胺一起使用，所述沙利度胺的剂量在给药后两小时产生5 μ g/ml的血浆水平。完全缓解后继续治疗3个月，部分缓解后继续治疗6个月。 所有入选患者的总体精算生存率为64%。在接受难治性GVHD挽救治疗的患者中，生存率为76%，在高危GVHD患者中，生存率为48%。14例患者观察到完全缓解，12例患者观察到部分缓解，18例患者观察到无缓解。副作用很小，最明显的是几乎所有患者的镇静作用。 在这项初步试验中，沙利度胺似乎是安全和有效的治疗慢性GVHD。将需要一项在新诊断的慢性GVHD患者中比较沙利度胺与泼尼松的试验来证明其相对疗效。
Background. Allogeneic bone marrow transplantation is an accepted therapy for hematologic cancer, aplastic anemia, and inherited immunodeficiencies. Chronic graft-versus-host disease (GVHD) is the principal complication in patients surviving more than 100 days. Thalidomide has been shown experimentally to be effective in treating GVHD.Methods. We treated 23 patients with chronic GVHD refractory to conventional treatment and 21 patients with "high-risk" chronic GVHD (identified as having at least two of the following three risk factors: chronic GVHD that has evolved from acute GVHD, lichenoid skin or mucous-membrane changes, and hepatic dysfunction; such patients have a high mortality rate) with thalidomide in a dose that produced a plasma level of 5-mu-g per milliliter two hours after administration. Therapy was continued for three months after a complete response or for six months after a partial response.Results. The overall actuarial survival of all enrolled patients was 64 percent. Survival was 76 percent among the patients receiving salvage therapy for refractory GVHD and 48 percent among those with high-risk GVHD. A complete response was observed in 14 patients, a partial response in 12 patients, and no response in 18. Side effects were minor, most notably sedation in almost all patients.Conclusions. In this preliminary trial, thalidomide appeared to be safe and effective for the treatment of chronic GVHD. A trial comparing thalidomide with prednisone in patients with newly diagnosed chronic GVHD will be required to demonstrate its relative efficacy.