The effectiveness and value of betibeglogene autotemcel for the management of transfusion-dependent beta-thalassemia.
作者信息
Lancaster Victoria, Richardson Marina, Beaudoin Francesca L, Synnott Patricia G, Rind David M, Herce-Hagiwara Belén, Campbell Jon D, Pearson Steven D
机构信息
Institute for Clinical and Economic Review, Boston, MA.
Center for the Evaluation of Value and Risk in Health, Tufts Medical Center, Boston, MA.
出版信息
J Manag Care Spec Pharm. 2022 Nov;28(11):1316-1320. doi: 10.18553/jmcp.2022.28.11.1316.
Abstract
摘要
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The effectiveness and value of betibeglogene autotemcel for the management of transfusion-dependent beta-thalassemia.betibeglogene autotemcel用于治疗输血依赖型β地中海贫血的有效性和价值。
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本文引用的文献
1
Betibeglogene Autotemcel Gene Therapy for Non-β/β Genotype β-Thalassemia.贝替贝洛基因自体造血干祖细胞基因治疗非β/β基因型β-地中海贫血。
N Engl J Med. 2022 Feb 3;386(5):415-427. doi: 10.1056/NEJMoa2113206. Epub 2021 Dec 11.
2
β-Thalassemia.β地中海贫血
Genet Med. 2017 Jun;19(6):609-619. doi: 10.1038/gim.2016.173. Epub 2016 Nov 3.
3
Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel.重型地中海贫血和镰状细胞病的造血干细胞移植：国际专家小组的适应证及管理建议
Haematologica. 2014 May;99(5):811-20. doi: 10.3324/haematol.2013.099747.
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