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Use of Deferasirox Film-Coated Tablets in Pediatric Patients with Transfusion Dependent Thalassemia: A Single Center Experience.去铁酮薄膜包衣片在输血依赖型地中海贫血患儿中的应用:单中心经验
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Kidney Tubular Damage Secondary to Deferasirox: Systematic Literature Review.地拉罗司继发的肾小管损伤:系统文献综述
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本文引用的文献

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Acute hyperammonemia in children under deferasirox treatment: cutting the Gordian knot.接受地拉罗司治疗的儿童急性高氨血症:快刀斩乱麻。
Clin Toxicol (Phila). 2019 May;57(5):375-377. doi: 10.1080/15563650.2018.1523425. Epub 2018 Nov 16.
2
Jadenu Substituting Exjade in Iron Overloaded β-Thalassemia Major (BTM) Patients: A Preliminary Report of the Effects on the Tolerability, Serum Ferritin Level, Liver Iron Concentration and Biochemical Profiles.Jadenu替代去铁斯若用于重度β地中海贫血(BTM)铁过载患者:对耐受性、血清铁蛋白水平、肝脏铁浓度及生化指标影响的初步报告
Mediterr J Hematol Infect Dis. 2018 Nov 1;10(1):e2018064. doi: 10.4084/MJHID.2018.064. eCollection 2018.
3
Optimising management of deferasirox therapy for patients with transfusion-dependent thalassaemia and lower-risk myelodysplastic syndromes.优化依赖输血的地中海贫血和低危骨髓增生异常综合征患者的地拉罗司治疗管理。
Eur J Haematol. 2018 Sep;101(3):272-282. doi: 10.1111/ejh.13111. Epub 2018 Jul 27.
4
Acute Liver Failure During Deferasirox Chelation: A Toxicity Worth Considering.去铁胺螯合治疗期间的急性肝衰竭:一种值得关注的毒性反应。
J Pediatr Hematol Oncol. 2017 Apr;39(3):217-222. doi: 10.1097/MPH.0000000000000786.
5
New film-coated tablet formulation of deferasirox is well tolerated in patients with thalassemia or lower-risk MDS: Results of the randomized, phase II ECLIPSE study.去铁斯若新的薄膜包衣片新剂型在β地中海贫血或低危骨髓增生异常综合征患者中耐受性良好:随机II期ECLIPSE研究结果
Am J Hematol. 2017 May;92(5):420-428. doi: 10.1002/ajh.24668. Epub 2017 Feb 18.
6
Fulminant Liver Failure in a Child With β-Thalassemia on Deferasirox: A Case Report.接受地拉罗司治疗的β地中海贫血患儿发生暴发性肝衰竭:一例报告
J Pediatr Hematol Oncol. 2017 Apr;39(3):235-237. doi: 10.1097/MPH.0000000000000654.
7
Acute Liver Failure in a Pediatric Patient with Congenital Dysery-Thropoietic Anemia Type I Treated with Deferasirox.一名患有I型先天性异常红细胞生成性贫血的儿科患者使用地拉罗司治疗后出现急性肝衰竭
Hematol Rep. 2015 Sep 23;7(3):5987. doi: 10.4081/hr.2015.5987.
8
A case report of deferasirox-induced kidney injury and Fanconi syndrome.地拉罗司致肾损伤及范科尼综合征一例报告
WMJ. 2013 Aug;112(4):177-80.
9
Deferasirox: appraisal of safety and efficacy in long-term therapy.地拉罗司:长期治疗中的安全性与有效性评估
J Blood Med. 2013 Aug 5;4:101-10. doi: 10.2147/JBM.S35478. eCollection 2013.
10
Efficacy and safety of deferasirox compared with deferoxamine in sickle cell disease: two-year results including pharmacokinetics and concomitant hydroxyurea.比较去铁酮与去铁胺在镰状细胞病中的疗效和安全性:包括药代动力学和同时使用羟基脲的两年结果。
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地拉罗司致肝损伤及范可尼综合征 1 例:β 地中海贫血男性患者

Deferasirox-induced liver injury and Fanconi syndrome in a beta-thalassemia major male.

机构信息

Gastroenterology and Hepatology Department, Austin Health, Heidelberg, Victoria, Australia

Gastroenterology and Hepatology Department, Northern Health, Epping, Victoria, Australia.

出版信息

BMJ Case Rep. 2020 Jul 9;13(7):e234542. doi: 10.1136/bcr-2020-234542.

DOI:10.1136/bcr-2020-234542
PMID:32646935
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7351284/
Abstract

A 33-year-old male presenting with subacute abdominal pain was found to have hyperbilirubinaemia, hypokalaemia and hyponatraemia. This was in the setting of transitioning between deferasirox iron chelator formulations, from dispersible tablets to film-coated tablets for ongoing treatment of chronic iron overload secondary to transfusion requirement for beta-thalassemia major. A liver biopsy demonstrated acute cholestasis with patchy confluent hepatocellular necrosis and mild to moderate microvesicular steatosis. Based on the histological, biochemical and clinical findings, the diagnosis of hepatotoxicity and Fanconi-like syndrome was made. The patient improved clinically and biochemically with cessation of the deferasirox film-coated tablets and supportive management. To our knowledge, this is the first case report of hepatotoxicity and Fanconi-like syndrome occurring due to deferasirox film-coated tablets with previous tolerance of dispersible deferasirox tablets. It is important to raise clinical awareness of this potentially severe complication.

摘要

一位 33 岁男性因亚急性腹痛就诊,发现高胆红素血症、低钾血症和低钠血症。这是在他从分散片转换为薄膜包衣片的过程中发生的,目的是继续治疗因重型β地中海贫血需要输血引起的慢性铁过载。肝活检显示急性胆汁淤积,伴有斑片状融合性肝细胞坏死和轻度至中度微泡性脂肪变性。根据组织学、生化和临床发现,诊断为肝毒性和范可尼样综合征。患者在停用地拉罗司薄膜包衣片并接受支持性治疗后临床和生化情况均有所改善。据我们所知,这是首例因先前耐受分散片而发生的地拉罗司薄膜包衣片导致的肝毒性和范可尼样综合征的病例报告。提高对此种潜在严重并发症的临床认识非常重要。