Stem cell transplantation for congenital dyserythropoietic anemia: an analysis from the European Society for Blood and Marrow Transplantation.
作者信息
Miano Maurizio, Eikema Dirk-Jan, Aljurf Mahmoud, Van't Veer Pieter J, Öztürk Gülyüz, Wölfl Matthias, Smiers Frans, Schulz Angsar, Socié Gerard, Vettenranta Kim, de Heredia Cristina Diaz, Zecca Marco, Maertens Johan, Rovira Montserrat, Sierra Jorge, Uckan-Cetinkaya Duygu, Skorobogatova Elena, Antmen Ali Bülent, Dalle Jean-Hugues, Markiewicz Miroslaw, Hamladji Rose Marie, Kitra-Roussou Vassiliki, La Nasa Giorgio, Kriván Gergely, Al-Seiraihy Amal, Giardino Stefano, Risitano Antonio Maria, de Latour Regis Peffault, Dufour Carlo
机构信息
Hematology Unit, IRCCS Istituto Giannina Gaslini, Genoa, Italy
EBMT Statistical Unit, Leiden, Leiden, the Netherlands.
出版信息
Haematologica. 2019 Aug;104(8):e335-e339. doi: 10.3324/haematol.2018.206623. Epub 2019 Jan 24.
Abstract
摘要
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本文引用的文献
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Non-myeloablative allogeneic stem cell transplant with post-transplant cyclophosphamide cures the first adult patient with congenital dyserythropoietic anemia.非清髓性异基因干细胞移植联合移植后环磷酰胺治愈首例先天性红细胞生成异常性贫血成年患者。
Bone Marrow Transplant. 2017 Jun;52(6):905-906. doi: 10.1038/bmt.2017.17. Epub 2017 Mar 20.
2
Diagnosis and management of congenital dyserythropoietic anemias.先天性红细胞生成异常性贫血的诊断与管理
Expert Rev Hematol. 2016 Mar;9(3):283-96. doi: 10.1586/17474086.2016.1131608. Epub 2016 Jan 6.
3
Successful Allogeneic Hematopoietic Stem Cell Transplantation of a Patient Suffering from Type II Congenital Dyserythropoietic Anemia A Rare Case Report from Western India.一名患有II型先天性红细胞生成异常性贫血患者的异基因造血干细胞移植成功：来自印度西部的罕见病例报告
Case Rep Hematol. 2015;2015:792485. doi: 10.1155/2015/792485. Epub 2015 Jan 26.
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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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Successful hematopoietic stem cell transplantation in a patient with congenital dyserythropoietic anemia type II.一名II型先天性红细胞生成异常性贫血患者成功进行造血干细胞移植。
Pediatr Transplant. 2014 Jun;18(4):E130-3. doi: 10.1111/petr.12254. Epub 2014 Apr 12.
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Successful treatment of an infant with CDA type II by intrauterine transfusions and postnatal stem cell transplantation.通过宫内输血和产后干细胞移植成功治疗 II 型 CDA 婴儿。
Pediatr Blood Cancer. 2014 Apr;61(4):743-5. doi: 10.1002/pbc.24786. Epub 2013 Oct 3.
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Two founder mutations in the SEC23B gene account for the relatively high frequency of CDA II in the Italian population.SEC23B 基因中的两个 founder 突变解释了意大利人群中 CDA II 的相对高发率。
Am J Hematol. 2011 Sep;86(9):727-32. doi: 10.1002/ajh.22096.
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Unrelated hematopoietic stem cell transplantation in a patient with congenital dyserythropoietic anemia and iron overload.先天性红细胞生成异常性贫血合并铁过载患者的非亲缘造血干细胞移植
Pediatr Transplant. 2012 May;16(3):E69-73. doi: 10.1111/j.1399-3046.2010.01414.x. Epub 2010 Nov 26.
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Congenital dyserythropoietic anaemia, type I, in a Caucasian patient with retinal angioid streaks (homozygous Arg1042Trp mutation in codanin-1).一名患有视网膜血管样条纹的白种人患者（编码蛋白1存在纯合子Arg1042Trp突变）的I型先天性红细胞生成异常性贫血。
Eur J Haematol. 2008 Mar;80(3):271-4. doi: 10.1111/j.1600-0609.2007.01004.x. Epub 2007 Dec 7.
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Congenital dyserythropoietic anemia type I with bone abnormalities, mutations of the CDAN I gene, and significant responsiveness to alpha-interferon therapy.伴有骨骼异常、CDAN I基因突变且对α-干扰素治疗有显著反应的I型先天性红细胞生成异常性贫血
Ann Hematol. 2006 Sep;85(9):591-5. doi: 10.1007/s00277-006-0143-z. Epub 2006 Jun 10.
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