Center for Therapeutic Endoscopy and Endoscopic Oncology, St. Michael's Hospital, University of Toronto, Toronto, Ontario, Canada.
Eur J Gastroenterol Hepatol. 2009 Dec;21(12):1347-50. doi: 10.1097/MEG.0b013e32832c9346.
Mucosal angiodysplasias, either inherited or acquired, can cause gastrointestinal bleeding, sometimes refractory to treatment. From earlier case reports, thalidomide has been described to possess some benefits in this disease, but its benefits and risks nevertheless remain unclear.
This pilot study assesses the efficacy, safety, and side-effect of thalidomide in the treatment of patients with chronic gastrointestinal bleeding from angiodysplasias.
Patients with chronic angiodysplasia bleeding and requiring ongoing transfusion were eligible for this open nonrandomized study. Thalidomide was started with 50 mg/day and then increased incrementally by 50 mg every week up to 200 mg/day, if tolerated, and continued for 6 months. Adverse events, hemoglobin, blood chemistry, and blood transfusion were monitored during the treatment and for 6-months posttreatment.
Seven patients were recruited in this study. Four patients discontinued thalidomide within 3-8 weeks, because of fatigue (two patients), peripheral neuropathy (one patient), and skin rash (one patient). All side-effects resolved when thalidomide was discontinued. These four patients required the same volume of blood transfusions per month as pre-study. In contrast, the three patients who continued 100-200 mg/day of thalidomide for 6 months did not require any transfusions during the 6 months of medication. During 6-months posttreatment of these three patients, one maintained response without any transfusion for 2 months, then required 1 U of blood every 4 weeks, one patient required 2 U of blood every 3-4 weeks, and one patient died from diabetes complications.
Thalidomide should be considered as a therapeutic option in patients who are resistant to conventional therapy, but it has a high discontinuation rate because of its side-effects.
黏膜血管发育不良，无论是遗传性的还是获得性的，都可能导致胃肠道出血，有时治疗效果不佳。从早期的病例报告来看，沙利度胺已被描述为对这种疾病具有一定的疗效，但它的益处和风险仍不清楚。
本研究旨在评估沙利度胺治疗血管发育不良引起的慢性胃肠道出血患者的疗效、安全性和副作用。
本研究为开放性非随机研究，纳入需要持续输血的慢性血管发育不良出血患者。沙利度胺起始剂量为 50mg/天，如可耐受，每周增加 50mg，最高剂量为 200mg/天，持续治疗 6 个月。在治疗期间和治疗结束后 6 个月监测不良反应、血红蛋白、血液化学和输血情况。
本研究共纳入 7 例患者。4 例患者因疲劳（2 例）、周围神经病（1 例）和皮疹（1 例）在 3-8 周内停用沙利度胺。所有不良反应在停药后均得到缓解。这 4 例患者在研究期间每月需要相同量的输血。相比之下，3 例患者继续服用 100-200mg/天沙利度胺 6 个月，在用药期间不需要输血。在这 3 例患者停药后的 6 个月中，1 例患者在未输血的情况下维持缓解 2 个月，随后每 4 周需要输血 1U，1 例患者每 3-4 周需要输血 2U，1 例患者因糖尿病并发症死亡。
沙利度胺应作为常规治疗无效患者的治疗选择，但因其副作用，停药率较高。
