Dieckgraefe Brian K, Korzenik Joshua R
Division of Gastroenterology, Department of Internal Medicine, Washington University School of Medicine, 660 South Euclid Avenue, Box 8124, St Louis, MO 63110, USA.
Lancet. 2002 Nov 9;360(9344):1478-80. doi: 10.1016/S0140-6736(02)11437-1.
Treatment for Crohn's disease is aimed at immunosuppression. Yet inherited disorders associated with defective innate immunity often lead to development of a Crohn's-like disease. We performed an open-label dose-escalation trial (4-8 microg/kg per day) to investigate the safety and possible benefit of granulocyte-macrophage colony-stimulating factor (GM-CSF) in the treatment of 15 patients with moderate to severe Crohn's disease. No patients had worsening of their disease. Adverse events were negligible and included minor injection site reactions and bone pain. Patients had a significant decrease in mean Crohn's disease activity index (CDAI) score during treatment (p<0.0001). After 8 weeks of treatment, mean CDAI had fallen by 190 points. Overall, 12 patients had a decrease in CDAI of more than 100 points, and eight achieved clinical remission. Retreatment was effective, and treatment was associated with increased quality-of-life measures. GM-CSF may offer an alternative to traditional immunosuppression in treatment of Crohn's disease.
克罗恩病的治疗旨在免疫抑制。然而，与先天性免疫缺陷相关的遗传性疾病常常会导致类克罗恩病的发展。我们开展了一项开放标签剂量递增试验（每天4 - 8微克/千克），以研究粒细胞巨噬细胞集落刺激因子（GM - CSF）治疗15例中重度克罗恩病患者的安全性和可能的益处。没有患者病情恶化。不良事件可忽略不计，包括轻微的注射部位反应和骨痛。患者在治疗期间克罗恩病活动指数（CDAI）评分显著降低（p<0.0001）。治疗8周后，平均CDAI下降了190分。总体而言，12例患者的CDAI下降超过100分，8例实现临床缓解。再次治疗有效，且治疗与生活质量指标的改善相关。GM - CSF在克罗恩病治疗中可能是传统免疫抑制治疗的一种替代方法。
