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相似文献

1
Management of the thalassemias.地中海贫血症的治疗管理。
Cold Spring Harb Perspect Med. 2013 Jun 1;3(6):a011767. doi: 10.1101/cshperspect.a011767.
2
Transparency and access to full information for the fatal or serious toxicity risks, low efficacy and high price of deferasirox, could increase the prospect of improved iron chelation therapy worldwide.对于地拉罗司的致命或严重毒性风险、低疗效和高价格,提高透明度并提供全面信息,可能会增加全球改善铁螯合治疗的前景。
Hemoglobin. 2008;32(6):608-15. doi: 10.1080/03630260802341703.
3
Ethical issues and risk/benefit assessment of iron chelation therapy: advances with deferiprone/deferoxamine combinations and concerns about the safety, efficacy and costs of deferasirox.铁螯合疗法的伦理问题及风险/效益评估:去铁酮/去铁胺联合疗法的进展以及对地拉罗司安全性、有效性和成本的担忧。
Hemoglobin. 2008;32(1-2):1-15. doi: 10.1080/03630260701726533.
4
Current status of iron overload and chelation with deferasirox.铁过载现状及与地拉罗司的螯合作用
Indian J Pediatr. 2007 Aug;74(8):759-64. doi: 10.1007/s12098-007-0134-7.
5
Transfusion-dependent thalassaemia: a new era.输血依赖型地中海贫血:一个新时代。
Med J Aust. 2008 Jan 21;188(2):68-9. doi: 10.5694/j.1326-5377.2008.tb01523.x.
6
Iron chelation therapy in myelodysplastic syndromes.骨髓增生异常综合征的铁螯合疗法。
Am J Health Syst Pharm. 2010 Apr;67(7 Suppl 2):S10-4; quiz S16. doi: 10.2146/ajhp090654.
7
Differential effects of iron chelators on iron burden and long-term morbidity and mortality outcomes in a large cohort of transfusion-dependent β-thalassemia patients who remained on the same monotherapy over 10 years.在一个大型输血依赖型β-地中海贫血患者队列中,铁螯合剂对铁负荷和长期发病率及死亡率结局的影响存在差异,这些患者在 10 多年的时间里一直接受同一种单一疗法治疗。
Blood Cells Mol Dis. 2024 Jul;107:102859. doi: 10.1016/j.bcmd.2024.102859. Epub 2024 May 29.
8
Deferasirox for the treatment of iron overload associated with regular blood transfusions (transfusional haemosiderosis) in patients suffering with chronic anaemia: a systematic review and economic evaluation.地拉罗司用于治疗慢性贫血患者因定期输血导致的铁过载(输血性含铁血黄素沉着症):一项系统评价与经济学评估
Health Technol Assess. 2009 Jan;13(1):iii-iv, ix-xi, 1-121. doi: 10.3310/hta13010.
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Therapeutic efficacy of different iron chelators in Egyptian children with Beta Thalassemia with iron overload.不同铁螯合剂对埃及铁过载β地中海贫血患儿的治疗效果。
Infect Disord Drug Targets. 2015;15(2):98-105. doi: 10.2174/1871526515666150724111721.
10
Iron-chelating therapies in a transfusion-dependent thalassaemia population in Thailand: a cost-effectiveness study.泰国依赖输血的地中海贫血患者群体中的铁螯合疗法:成本效益研究。
Clin Drug Investig. 2011;31(7):493-505. doi: 10.2165/11587120-000000000-00000.

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1
Thalassemia in Sub-Saharan Africa: epidemiology, diagnosis, and management - a narrative review.撒哈拉以南非洲地区的地中海贫血:流行病学、诊断与管理——一篇叙述性综述
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The Optimized γ-Globin Lentiviral Vector GGHI-mB-3D Leads to Nearly Therapeutic HbF Levels In Vitro in CD34 Cells from Sickle Cell Disease Patients.优化的 γ-珠蛋白慢病毒载体 GGHI-mB-3D 在来自镰状细胞病患者的 CD34 细胞中体外导致接近治疗水平的 HbF。
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The transfusion management of beta thalassemia in the United States.美国β地中海贫血的输血管理
Transfusion. 2021 Oct;61(10):3027-3039. doi: 10.1111/trf.16640. Epub 2021 Aug 28.
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Clinical Usefulness of Furosemide to Prevent Volume Overload Among Children and Young Adults with Transfusion-Dependent Thalassemia: A Randomized, Open-Label, Crossover Study.速尿对预防依赖输血的地中海贫血儿童和青年成人容量超负荷的临床效用:一项随机、开放标签、交叉研究。
J Blood Med. 2020 Dec 29;11:503-513. doi: 10.2147/JBM.S285647. eCollection 2020.
5
Challenges in chronic transfusion for patients with thalassemia.地中海贫血患者慢性输血面临的挑战。
Hematology Am Soc Hematol Educ Program. 2020 Dec 4;2020(1):160-166. doi: 10.1182/hematology.2020000102.
6
Thalassemia: Common Clinical Queries in Management.地中海贫血症:管理中的常见临床问题。
Indian J Pediatr. 2020 Jan;87(1):75-81. doi: 10.1007/s12098-019-03065-5. Epub 2019 Oct 16.
7
Managing the need to tell: Triggers and strategic disclosure of thalassemia major in Singapore.管理告知需求:新加坡重型地中海贫血症的触发因素和策略性披露。
Am J Med Genet A. 2019 May;179(5):762-769. doi: 10.1002/ajmg.a.61107. Epub 2019 Mar 1.
8
Measurement of the liver iron concentration in transfusional iron overload by MRI R2* and by high-transition-temperature superconducting magnetic susceptometry.MRI R2* 与高温超导磁强计测量输血致铁过载患者肝铁浓度。
Clin Imaging. 2019 May-Jun;55:65-70. doi: 10.1016/j.clinimag.2019.01.012. Epub 2019 Jan 31.
9
Information Technology-Assisted Treatment Planning and Performance Assessment for Severe Thalassemia Care in Low- and Middle-Income Countries: Observational Study.信息技术辅助的低收入和中等收入国家重型地中海贫血治疗规划与绩效评估:观察性研究
JMIR Med Inform. 2019 Jan 23;7(1):e9291. doi: 10.2196/medinform.9291.
10
Efficacy of Oral Acetaminophen and Intravenous Chlorpheniramine Maleate versus Placebo to Prevent Red Cell Transfusion Reactions in Children and Adolescent with Thalassemia: A Prospective, Randomized, Double-Blind Controlled Trial.口服对乙酰氨基酚和静脉注射马来酸氯苯那敏与安慰剂预防地中海贫血儿童和青少年红细胞输血反应的疗效:一项前瞻性、随机、双盲对照试验。
Anemia. 2018 Oct 1;2018:9492303. doi: 10.1155/2018/9492303. eCollection 2018.

本文引用的文献

1
The prevention of thalassemia.地中海贫血症的预防。
Cold Spring Harb Perspect Med. 2013 Feb 1;3(2):a011775. doi: 10.1101/cshperspect.a011775.
2
Development of gene therapy for thalassemia.地中海贫血症的基因治疗发展。
Cold Spring Harb Perspect Med. 2012 Nov 1;2(11):a011833. doi: 10.1101/cshperspect.a011833.
3
Contemporary approaches to treatment of beta-thalassemia intermedia.β-地中海贫血中间型的当代治疗方法。
Blood Rev. 2012 Apr;26 Suppl 1:S24-7. doi: 10.1016/S0268-960X(12)70008-5.
4
Hematopoietic stem cell transplantation in thalassemia and sickle cell anemia.地中海贫血和镰状细胞贫血的造血干细胞移植。
Cold Spring Harb Perspect Med. 2012 May;2(5):a011825. doi: 10.1101/cshperspect.a011825.
5
Pluripotent stem cells in research and treatment of hemoglobinopathies.多能干细胞在血红蛋白病的研究和治疗中的应用。
Cold Spring Harb Perspect Med. 2012 Apr;2(4):a011841. doi: 10.1101/cshperspect.a011841.
6
Pituitary iron and volume predict hypogonadism in transfusional iron overload.垂体铁含量和体积可预测输血性铁过载中的性腺功能减退症。
Am J Hematol. 2012 Feb;87(2):167-71. doi: 10.1002/ajh.22247. Epub 2011 Dec 27.
7
Pancreatic iron and glucose dysregulation in thalassemia major.地中海贫血症中的胰腺铁和葡萄糖失调。
Am J Hematol. 2012 Feb;87(2):155-60. doi: 10.1002/ajh.22223. Epub 2011 Nov 24.
8
Elevated liver iron concentration is a marker of increased morbidity in patients with β thalassemia intermedia.肝铁浓度升高是中间型β地中海贫血患者发病率增加的标志物。
Haematologica. 2011 Nov;96(11):1605-12. doi: 10.3324/haematol.2011.047852. Epub 2011 Jul 26.
9
Optimal management of β thalassaemia intermedia.β 中间型地中海贫血的最佳管理。
Br J Haematol. 2011 Mar;152(5):512-23. doi: 10.1111/j.1365-2141.2010.08486.x. Epub 2011 Jan 20.
10
Iron-chelating therapy for transfusional iron overload.铁螯合疗法治疗输血引起的铁过载。
N Engl J Med. 2011 Jan 13;364(2):146-56. doi: 10.1056/NEJMct1004810.

地中海贫血症的治疗管理。

Management of the thalassemias.

机构信息

Hemoglobinopathy Research University Health Network, Toronto, Ontario M5G 2C4, Canada.

出版信息

Cold Spring Harb Perspect Med. 2013 Jun 1;3(6):a011767. doi: 10.1101/cshperspect.a011767.

DOI:10.1101/cshperspect.a011767
PMID:23732853
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3662352/
Abstract

During the last 30 years, in addition to the considerable progress made in control and prevention of thalassemias(3), there have also been major advances in their symptomatic management, at least in wealthier countries where appropriate facilities are available. Remarkable improvements in survival in the severe forms of thalassemia have followed the more judicious use of blood transfusion and, in particular, the ability to manage the iron accumulation resulting from transfusion with its severe and ultimately lethal effects on endocrine and cardiac function.

摘要

在过去的 30 年中,除了在地中海贫血症的控制和预防方面取得了相当大的进展(3)之外,在症状管理方面也取得了重大进展,至少在有适当设施的富裕国家是如此。更明智地使用输血,特别是能够管理输血引起的铁积累,以及其对内分泌和心脏功能的严重和最终致命影响,使得严重地中海贫血症的存活率显著提高。