Division of Gastroenterology and Hepatology, Department of Internal Medicine III, Medical University of Vienna, Vienna, Austria; Vienna Hepatic Hemodynamic Lab, Division of Gastroenterology and Hepatology, Department of Internal Medicine III, Medical University of Vienna, Vienna, Austria.
Department of Internal Medicine IV, Ordensklinikum Linz Barmherzige Schwestern, Linz, Austria.
Clin Gastroenterol Hepatol. 2023 Apr;21(4):978-987.e2. doi: 10.1016/j.cgh.2022.04.024. Epub 2022 May 6.
Direct oral anticoagulants (DOACs) may simplify management of Budd-Chiari syndrome (BCS). Here, we report our experience with off-label use of DOACs for anticoagulation in BCS.
The safety of DOAC vs vitamin K antagonist treatment as well as associated clinical outcomes were retrospectively assessed in 47 BCS patients treated at 6 Austrian centers.
Mean age at study inclusion was 37.9 ± 14.0 years and mean Model for End-Stage Liver Disease was 13.1 ± 5.1. Overall, 63.8% (n = 30) of patients had decompensated liver disease, and 87.2% (n = 41) showed clinical signs of portal hypertension. During a median follow-up of 82.5 (interquartile range, 43.1-121.8) months, 43 (91.5%) patients received anticoagulation alone or following interventional treatment, including 22 (46.8%) patients treated with DOACs (edoxaban: 10, apixaban: 4, rivaroxaban: 3, dabigatran: 3, more than one DOAC sequentially: 2) for a median of 24.4 (interquartile range, 5.7-35.1) months. While 72.7% (n = 16 of 22) of patients were switched from low-molecular-weight heparin (n = 12) or vitamin K antagonist (n = 4) to DOAC after disease stabilization or improvement, 27.3% (n = 6 of 22) of BCS patients were initially treated with DOAC. Complete response (European Association for the Study of the Liver criteria) was achieved or maintained in 14 (63.6%) of 22 patients, with ongoing response in 2 patients, while disease progressed in 6 patients (including 2 patients with hepatocellular carcinoma). Four major spontaneous bleedings (18.2%; incidence rate 8.8 per 100 patient-years; n = 2 upper gastrointestinal bleeding, n = 1 lower gastrointestinal bleeding, n = 1 hepatocellular carcinoma rupture), 7 minor bleedings, and 1 major procedure-related bleeding (4.5%; 2.2 per 100 patient-years) occurred during DOAC therapy. Overall transplant-free survival was 91.6% at 5 years.
DOACs seem to be effective and safe for long-term anticoagulation in patients with BCS, but confirmation by larger prospective studies is needed.
直接口服抗凝剂（DOAC）可能简化布加氏综合征（BCS）的管理。在此，我们报告了我们在 6 个奥地利中心使用 DOAC 进行 BCS 抗凝的经验。
回顾性评估了 47 例在 6 个奥地利中心接受治疗的 BCS 患者中 DOAC 与维生素 K 拮抗剂治疗的安全性以及相关临床结局。
研究纳入时的平均年龄为 37.9 ± 14.0 岁，终末期肝病模型平均为 13.1 ± 5.1。总体而言，63.8%（n=30）的患者存在肝功能失代偿，87.2%（n=41）存在门静脉高压的临床征象。中位随访 82.5（四分位间距 43.1-121.8）个月期间，43（91.5%）例患者接受了单独抗凝或介入治疗，其中 22（46.8%）例患者接受了 DOAC 治疗（依度沙班 10 例，阿哌沙班 4 例，利伐沙班 3 例，达比加群 3 例，序贯使用两种 DOAC 各 2 例），中位治疗时间为 24.4（四分位间距 5.7-35.1）个月。在疾病稳定或改善后，72.7%（n=16/22）的患者从低分子肝素（n=12）或维生素 K 拮抗剂（n=4）转换为 DOAC，27.3%（n=6/22）的 BCS 患者初始即接受 DOAC 治疗。22 例患者中，14 例（63.6%）达到或维持完全缓解（欧洲肝脏研究协会标准），2 例患者持续缓解，6 例患者疾病进展（包括 2 例肝细胞癌患者）。在 DOAC 治疗期间，4 例发生自发性大出血（18.2%；发生率 8.8 例/100 患者年；n=2 例上消化道出血，n=1 例下消化道出血，n=1 例肝细胞癌破裂），7 例发生小出血，1 例发生大的与操作相关的出血（4.5%；2.2 例/100 患者年）。5 年时无移植生存为 91.6%。
DOAC 似乎对 BCS 患者的长期抗凝有效且安全，但需要更大规模的前瞻性研究来证实。
