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未雨绸缪:及时使用 N-乙酰半胱氨酸(NAC)治疗化疗引起的静脉闭塞性疾病(VOD)——是否具有成本效益?

A stitch in time saves nine: timely use of N-acetyl cysteine (NAC) for chemotherapy-induced veno-occlusive disease (VOD)-is it a cost-effective alternative?

机构信息

Department of Pediatric Oncology, Regional Cancer Centre, Trivandrum, Kerala, 695011, India.

出版信息

Support Care Cancer. 2022 Nov;30(11):8611-8614. doi: 10.1007/s00520-022-07321-x. Epub 2022 Aug 13.

DOI:10.1007/s00520-022-07321-x
PMID:35963952
Abstract

Chemotherapy-induced veno-occlusive disease (VOD) is a rare liver dysfunction seen among pediatric cancer patients which could lead to severe morbidity and mortality. Defibrotide is the commonly used antidote in the management of both stem cell transplant and chemotherapy-associated VOD along with liver supportive measures. Defibrotide is costly and generally not accessible to majority of patients treated at resource poor settings. In this report, we describe the successful management of chemotherapy-induced VOD with timely administration of N-acetyl cysteine.

摘要

化疗诱导的肝静脉闭塞病(VOD)是一种罕见的肝功能障碍,见于儿科癌症患者,可导致严重的发病率和死亡率。去纤苷是干细胞移植和化疗相关 VOD 治疗中常用的解毒剂,同时还需要进行肝脏支持治疗。去纤苷价格昂贵,并且在资源匮乏的治疗环境中,大多数患者无法获得。在本报告中,我们描述了及时给予 N-乙酰半胱氨酸成功治疗化疗诱导的 VOD。

相似文献

1
A stitch in time saves nine: timely use of N-acetyl cysteine (NAC) for chemotherapy-induced veno-occlusive disease (VOD)-is it a cost-effective alternative?未雨绸缪:及时使用 N-乙酰半胱氨酸(NAC)治疗化疗引起的静脉闭塞性疾病(VOD)——是否具有成本效益?
Support Care Cancer. 2022 Nov;30(11):8611-8614. doi: 10.1007/s00520-022-07321-x. Epub 2022 Aug 13.
2
Defibrotide for Treatment of Severe Veno-Occlusive Disease in Pediatrics and Adults: An Exploratory Analysis Using Data from the Center for International Blood and Marrow Transplant Research.去纤苷治疗儿童和成人严重肝静脉闭塞病:一项利用国际血液和骨髓移植研究中心数据的探索性分析
Biol Blood Marrow Transplant. 2016 Jul;22(7):1306-1312. doi: 10.1016/j.bbmt.2016.04.011. Epub 2016 Apr 19.
3
Defibrotide for Patients with Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome: Interim Results from a Treatment IND Study.去纤苷治疗肝静脉闭塞病/窦性阻塞综合征患者:一项治疗性 IND 研究的中期结果。
Biol Blood Marrow Transplant. 2017 Jun;23(6):997-1004. doi: 10.1016/j.bbmt.2017.03.008. Epub 2017 Mar 8.
4
BCSH/BSBMT guideline: diagnosis and management of veno-occlusive disease (sinusoidal obstruction syndrome) following haematopoietic stem cell transplantation.BCSH/BSBMT 指南:造血干细胞移植后静脉闭塞病(窦状隙阻塞综合征)的诊断和治疗。
Br J Haematol. 2013 Nov;163(4):444-57. doi: 10.1111/bjh.12558. Epub 2013 Sep 17.
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Defibrotide for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome following nontransplant-associated chemotherapy: Final results from a post hoc analysis of data from an expanded-access program.地塞米松预防非移植相关化疗后肝静脉闭塞病/窦状隙阻塞综合征:扩展使用项目数据事后分析的最终结果。
Pediatr Blood Cancer. 2018 Oct;65(10):e27269. doi: 10.1002/pbc.27269. Epub 2018 Jun 6.
6
Combination of High-Dose Methylprednisolone and Defibrotide for Veno-Occlusive Disease in Pediatric Hematopoietic Stem Cell Transplant Recipients.大剂量甲基泼尼松龙联合去纤维肽治疗儿童造血干细胞移植后肝静脉闭塞病。
Biol Blood Marrow Transplant. 2018 Jan;24(1):91-95. doi: 10.1016/j.bbmt.2017.09.007. Epub 2017 Sep 20.
7
Treatment of hepatic veno-occlusive disease in children with N-acetylcysteine.用 N-乙酰半胱氨酸治疗儿童肝静脉阻塞病。
Pediatr Blood Cancer. 2019 Feb;66(2):e27518. doi: 10.1002/pbc.27518. Epub 2018 Oct 23.
8
Defibrotide sodium for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome.去纤苷钠用于治疗肝静脉闭塞性疾病/窦性阻塞综合征。
Expert Rev Clin Pharmacol. 2018 Feb;11(2):113-124. doi: 10.1080/17512433.2018.1421943. Epub 2018 Jan 5.
9
Earlier defibrotide initiation post-diagnosis of veno-occlusive disease/sinusoidal obstruction syndrome improves Day +100 survival following haematopoietic stem cell transplantation.造血干细胞移植后,在静脉闭塞性疾病/窦性阻塞综合征诊断后更早开始使用去纤苷可提高第100天的生存率。
Br J Haematol. 2017 Jul;178(1):112-118. doi: 10.1111/bjh.14727. Epub 2017 Apr 26.
10
Defibrotide for children and adults with hepatic veno-occlusive disease post hematopoietic cell transplantation.去纤维肽在造血细胞移植后肝静脉闭塞性疾病患儿和成人中的应用。
Expert Rev Gastroenterol Hepatol. 2017 Oct;11(10):885-898. doi: 10.1080/17474124.2017.1370372. Epub 2017 Sep 4.

本文引用的文献

1
Diagnosis and severity criteria for sinusoidal obstruction syndrome/veno-occlusive disease in pediatric patients: a new classification from the European society for blood and marrow transplantation.儿童患者窦状隙阻塞综合征/静脉阻塞性疾病的诊断和严重程度标准:来自欧洲血液和骨髓移植学会的新分类。
Bone Marrow Transplant. 2018 Feb;53(2):138-145. doi: 10.1038/bmt.2017.161. Epub 2017 Jul 31.
2
Revised diagnosis and severity criteria for sinusoidal obstruction syndrome/veno-occlusive disease in adult patients: a new classification from the European Society for Blood and Marrow Transplantation.成人患者窦性阻塞综合征/静脉闭塞性疾病的修订诊断和严重程度标准:欧洲血液和骨髓移植学会的新分类
Bone Marrow Transplant. 2016 Jul;51(7):906-12. doi: 10.1038/bmt.2016.130. Epub 2016 May 16.
3
Evidence for the changing regimens of acetylcysteine.乙酰半胱氨酸治疗方案变化的证据。
Br J Clin Pharmacol. 2016 Mar;81(3):471-81. doi: 10.1111/bcp.12789. Epub 2015 Nov 23.
4
Hepatic sinusoidal obstruction syndrome during chemotherapy for childhood medulloblastoma: report of a case and review of the literature.儿童髓母细胞瘤化疗期间的肝窦阻塞综合征:1例报告及文献复习
J Pediatr Hematol Oncol. 2014 Jan;36(1):76-80. doi: 10.1097/MPH.0b013e3182a8f352.
5
Dactinomycin-induced Hepatic Sinusoidal Obstruction Syndrome Responding to Treatment with N-acetylcysteine.放线菌素 D 诱导的肝窦阻塞综合征对乙酰半胱氨酸治疗有反应。
J Cancer. 2011;2:527-31. doi: 10.7150/jca.2.527. Epub 2011 Oct 25.
6
Sinusoidal obstruction syndrome during induction therapy for acute lymphoblastic leukemia managed with N-acetyl Cysteine.急性淋巴细胞白血病诱导治疗期间的窦性阻塞综合征,采用N-乙酰半胱氨酸治疗。
Pediatr Blood Cancer. 2011 Oct;57(4):700. doi: 10.1002/pbc.23142. Epub 2011 Jun 8.
7
Hepatic veno-occlusive disease: a chemotherapy-related toxicity in children with malignancies.肝静脉闭塞病:儿童恶性肿瘤化疗相关毒性。
Paediatr Drugs. 2010 Oct 1;12(5):277-84. doi: 10.2165/11531840-000000000-00000.
8
A prospective randomized study using N-acetyl-L-cysteine for early liver toxicity after allogeneic hematopoietic stem cell transplantation.一项使用N-乙酰-L-半胱氨酸预防异基因造血干细胞移植后早期肝毒性的前瞻性随机研究。
Bone Marrow Transplant. 2008 May;41(9):785-90. doi: 10.1038/sj.bmt.1705969. Epub 2008 Jan 7.
9
Hepatic veno-occlusive disease after hematopoietic stem cell transplantation: update on defibrotide and other current investigational therapies.造血干细胞移植后肝静脉闭塞病:去纤苷及其他当前研究性治疗方法的最新进展
Bone Marrow Transplant. 2008 Feb;41(3):229-37. doi: 10.1038/sj.bmt.1705899. Epub 2007 Nov 12.
10
Favorable response of pediatric stem cell recipients to human protein C concentrate substitution for veno-occlusive disease.儿科干细胞接受者对人蛋白C浓缩物替代治疗静脉闭塞性疾病的良好反应。
Pediatr Transplant. 2007 Feb;11(1):49-57. doi: 10.1111/j.1399-3046.2006.00612.x.