Aplastic anemia is a syndrome characterized by the decrease in hematopoietic stem cells along with bone marrow hypoplasia and pancytopenia, which is likely to be a T cell-mediated autoimmune disease. Since the response rate to immunosuppressive therapy is higher if started ahead of time, early initiation of treatment is recommended even in non-severe cases. Among treatment approaches in severe cases, immunosuppressive therapy with anti-thymocyte globulin (ATG) plus cyclosporin is the basic approach. However, the effectiveness of thrombopoietin receptor agonists has also been reported, with recovery of hematopoiesis in three blood lineages observed in some patients. Despite no evidence of increased incidence of genetic mutations with thrombopoietin receptor agonist treatment, bone marrow testing is recommended after three to six months of long-term treatment to detect the presence of chromosomal abnormalities. With regard to hematopoietic stem cell transplantation for aplastic anemia, cyclophosphamide is reduced as a pretreatment therapy, and instead, fludarabine is used in combination in order to reduce cardiotoxicity. Since HLA haploidentical hematopoietic stem cell transplantation has been developed and is being reportedly used in patients with no suitable donors, this transplantation approach might also be extended to aplastic anemia patients who were not considered eligible for transplantation in the past.
"},{"@language":"ja","@value":"再生不良性貧血は，造血幹細胞が減少して，骨髄の低形成と汎血球減少を呈する症候群であり，T細胞を介した自己免疫疾患の可能性が有力である。免疫抑制療法は発症早期症例がより奏効率は高く，軽症例でも早期の治療開始が推奨されている。重症例では，造血幹細胞移植以外の治療ではATGとシクロスポリンによる免疫抑制療法が基本であるが，トロンボポエチン受容体作動薬の併用が有効であり，3系統の血球回復が期待できる。治療効果発現には時間がかかることが多く，効果判定は早くても3～6ヶ月後に行う。トロンボポエチン受容体作動薬による遺伝子変異の発生増加は認められていないが，長期間の投与開始後は3～6ヶ月後に骨髄検査を施行し，染色体異常の有無を確認することが勧められる。造血幹細胞移植では心毒性の軽減を期待して，前処置のシクロフォスファミドを減量し，フルダラビンを併用するレジメンが行われつつある。HLA半合致移植が開発され，ドナーが見つからない症例を対象に報告が増えている。
"}],"abstractLicenseFlag":"disallow"}],"creator":[{"@id":"https://cir.nii.ac.jp/crid/1410286426516043904","@type":"Researcher","personIdentifier":[{"@type":"NRID","@value":"9000410658742"}],"foaf:name":[{"@language":"en","@value":"OBARA Naoshi"},{"@language":"ja","@value":"小原 直"}],"jpcoar:affiliationName":[{"@language":"ja","@value":"筑波大学医学医療系 血液内科"},{"@language":"en","@value":"Department of Hematology, Faculty of Medicine, University of Tsukuba"}]}],"publication":{"publicationIdentifier":[{"@type":"PISSN","@value":"04851439"},{"@type":"LISSN","@value":"04851439"},{"@type":"EISSN","@value":"18820824"},{"@type":"NDL_BIB_ID","@value":"000000024253"},{"@type":"ISSN","@value":"04851439"},{"@type":"NCID","@value":"AN00252940"}],"prism:publicationName":[{"@language":"en","@value":"Rinsho Ketsueki"},{"@language":"ja","@value":"臨床血液"},{"@language":"en","@value":"Rinsho Ketsueki"},{"@language":"en","@value":"Jpn. J. Clin. Hematol."},{"@language":"en","@value":"The Japanese Journal of Clinical Hematology"},{"@language":"ja","@value":"臨床血液"}],"dc:publisher":[{"@language":"en","@value":"The Japanese Society of Hematology"},{"@language":"ja","@value":"一般社団法人 日本血液学会"}],"prism:publicationDate":"2020","prism:volume":"61","prism:number":"9","prism:startingPage":"1071","prism:endingPage":"1079"},"url":[{"@id":"http://id.ndl.go.jp/bib/030678988"},{"@id":"https://ndlsearch.ndl.go.jp/books/R000000004-I030678988"},{"@id":"https://search.jamas.or.jp/link/ui/2021033086"}],"availableAt":"2020","foaf:topic":[{"@id":"https://cir.nii.ac.jp/all?q=Immunosuppressive%20therapy","dc:title":"Immunosuppressive therapy"},{"@id":"https://cir.nii.ac.jp/all?q=Clonal%20hematopoiesis","dc:title":"Clonal hematopoiesis"},{"@id":"https://cir.nii.ac.jp/all?q=TPO-R%20agonist","dc:title":"TPO-R agonist"},{"@id":"https://cir.nii.ac.jp/all?q=Haploidentical%20hematopoietic%20cell%20transplantation","dc:title":"Haploidentical hematopoietic cell transplantation"},{"@id":"https://cir.nii.ac.jp/all?q=Immunosuppressive%20therapy","dc:title":"Immunosuppressive therapy"},{"@id":"https://cir.nii.ac.jp/all?q=Clonal%20hematopoiesis","dc:title":"Clonal hematopoiesis"},{"@id":"https://cir.nii.ac.jp/all?q=TPO-R%20agonist","dc:title":"TPO-R agonist"},{"@id":"https://cir.nii.ac.jp/all?q=Haploidentical%20hematopoietic%20cell%20transplantation","dc:title":"Haploidentical hematopoietic cell transplantation"}],"dataSourceIdentifier":[{"@type":"JALC","@value":"oai:japanlinkcenter.org:2008124160"},{"@type":"NDL_SEARCH","@value":"oai:ndlsearch.ndl.go.jp:R000000004-I030678988"},{"@type":"PUBMED","@value":"33162501"},{"@type":"CIA","@value":"130007937134"},{"@type":"OPENAIRE","@value":"pmid_dedup__::785f609a0e7264653916400b7fc4408d"}]}