Simona Pagliuca, Annalisa Ruggeri, Régis Peffault de Latour
{"title":"Cord blood transplantation for bone marrow failure syndromes: state of art.","authors":"Simona Pagliuca, Annalisa Ruggeri, Régis Peffault de Latour","doi":"10.21037/sci.2019.10.04","DOIUrl":null,"url":null,"abstract":"<p><p>Hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST) represent the milestones of the treatment algorithm for idiopathic and inherited bone marrow failure (BMF) disorders. However, patients lacking a suitable donor or failing IST still have a poor prognosis. Cord blood transplantation (CBT) has extended the possibility of HSCT for many patients in case of the absence of an eligible donor, and although in the last years, this procedure is less used in several hematological diseases, it remains an option for the treatment of patients with BMF syndromes. Nevertheless, optimization of conditioning regimen and cord blood unit selection is warranted to reduce the risk of graft failure and transplant-related mortality. This review summarizes the state of art of CBT in the field of BMF diseases, focusing on historical and recent issues in idiopathic aplastic anemia and inherited disorders.</p>","PeriodicalId":21938,"journal":{"name":"Stem cell investigation","volume":" ","pages":"39"},"PeriodicalIF":0.0000,"publicationDate":"2019-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.21037/sci.2019.10.04","citationCount":"10","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Stem cell investigation","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.21037/sci.2019.10.04","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2019/1/1 0:00:00","PubModel":"eCollection","JCR":"Q1","JCRName":"Biochemistry, Genetics and Molecular Biology","Score":null,"Total":0}
引用次数: 10
Abstract
Hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST) represent the milestones of the treatment algorithm for idiopathic and inherited bone marrow failure (BMF) disorders. However, patients lacking a suitable donor or failing IST still have a poor prognosis. Cord blood transplantation (CBT) has extended the possibility of HSCT for many patients in case of the absence of an eligible donor, and although in the last years, this procedure is less used in several hematological diseases, it remains an option for the treatment of patients with BMF syndromes. Nevertheless, optimization of conditioning regimen and cord blood unit selection is warranted to reduce the risk of graft failure and transplant-related mortality. This review summarizes the state of art of CBT in the field of BMF diseases, focusing on historical and recent issues in idiopathic aplastic anemia and inherited disorders.
期刊介绍：
The Stem Cell Investigation (SCI; Stem Cell Investig; Online ISSN: 2313-0792) is a free access, peer-reviewed online journal covering basic, translational, and clinical research on all aspects of stem cells. It publishes original research articles and reviews on embryonic stem cells, induced pluripotent stem cells, adult tissue-specific stem/progenitor cells, cancer stem like cells, stem cell niche, stem cell technology, stem cell based drug discovery, and regenerative medicine. Stem Cell Investigation is indexed in PubMed/PMC since April, 2016.
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