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Luspatercept in β-thalassemia: Who and when. Strengths and weaknesses points of a real-world evidence.

作者信息

Torti Lorenza, Ardu Nicolina, Maffei Laura, De Fabritiis Paolo, Sorrentino Francesco

机构信息

Hemoglobinopathies Unit, Hematology Department, S. Eugenio Hospital (ASL Roma 2) Regional Center for the Diagnosis and Treatment of Rare Anemias and Disorders of Iron Metabolism Rome Italy.

出版信息

EJHaem. 2024 Nov 4;5(6):1354-1358. doi: 10.1002/jha2.1032. eCollection 2024 Dec.

DOI:10.1002/jha2.1032
PMID:39691262
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11647712/
Abstract
摘要
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2476/11647712/9d8725919e45/JHA2-5-1354-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2476/11647712/38f5e9371cd1/JHA2-5-1354-g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2476/11647712/224d3e29384b/JHA2-5-1354-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2476/11647712/1d0e06016007/JHA2-5-1354-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2476/11647712/9d8725919e45/JHA2-5-1354-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2476/11647712/38f5e9371cd1/JHA2-5-1354-g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2476/11647712/224d3e29384b/JHA2-5-1354-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2476/11647712/1d0e06016007/JHA2-5-1354-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2476/11647712/9d8725919e45/JHA2-5-1354-g001.jpg

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本文引用的文献

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Luspatercept for transfusion-dependent β-thalassemia: time to get real.卢司帕西普用于输血依赖型β地中海贫血：是时候面对现实了。
Ther Adv Hematol. 2023 Aug 26;14:20406207231195594. doi: 10.1177/20406207231195594. eCollection 2023.
2
Real-life experience with luspatercept in transfusion-dependent β-thalassemia.卢司帕西普治疗输血依赖型β地中海贫血的真实生活经验。
Ann Hematol. 2023 Oct;102(10):2965-2967. doi: 10.1007/s00277-023-05381-5. Epub 2023 Jul 27.
3
Management of luspatercept therapy in patients with transfusion-dependent β-thalassaemia.
luspatercept 治疗依赖输血的β-地中海贫血患者的管理。
Br J Haematol. 2023 Jun;201(5):824-831. doi: 10.1111/bjh.18801. Epub 2023 Apr 10.
4
Neutrophil and platelet increases with luspatercept in lower-risk MDS: secondary endpoints from the MEDALIST trial.在低危骨髓增生异常综合征中，卢司帕西普可使中性粒细胞和血小板增加：MEDALIST试验的次要终点
Blood. 2022 Jan 27;139(4):624-629. doi: 10.1182/blood.2021012589.
5
Luspatercept restores SDF-1-mediated hematopoietic support by MDS-derived mesenchymal stromal cells.Luspatercept 通过 MDS 来源的间充质基质细胞恢复 SDF-1 介导的造血支持。
Leukemia. 2021 Oct;35(10):2936-2947. doi: 10.1038/s41375-021-01275-5. Epub 2021 May 17.
6
The use of luspatercept for thalassemia in adults.成人地中海贫血症中 luspatercept 的应用。
Blood Adv. 2021 Jan 12;5(1):326-333. doi: 10.1182/bloodadvances.2020002725.
7
A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia.一项评估 luspatercept 治疗输血依赖型β地中海贫血患者的 3 期临床试验
N Engl J Med. 2020 Mar 26;382(13):1219-1231. doi: 10.1056/NEJMoa1910182.