Transfusion-dependent congenital dyserythropoietic anemia type I successfully treated with allogeneic stem cell transplantation
Until recently, therapy for patients with severe congenital dyserythropoietic anemia (CDA) has been limited to blood transfusions and chelation therapy. Three children with transfusion-dependent CDA type I underwent allogeneic stem cell transplantation (SCT) from matched sibling donors. Conditioning...
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| Veröffentlicht in: | Bone marrow transplantation (Basingstoke) 2002-04, Vol.29 (8), p.681-682 |
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| container_title | Bone marrow transplantation (Basingstoke) |
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| creator | AYAS, M AL-JEFRI, A BAOTHMAN, A AL-MAHR, M MUSTAFA, M. M KHALIL, S KARAOUI, M SOLH, H |
| description | Until recently, therapy for patients with severe congenital dyserythropoietic anemia (CDA) has been limited to blood transfusions and chelation therapy. Three children with transfusion-dependent CDA type I underwent allogeneic stem cell transplantation (SCT) from matched sibling donors. Conditioning was with cyclophosphamide 50 mg/kg/day for 4 days, busulphan 4 mg/kg/day for 4 days, and antithymocyte globulin (ATG) 30 mg/kg for four doses pre-SCT. All patients engrafted and are alive, and transfusion independent. To our knowledge, this is the first report of successful SCT in the management of CDA type I. |
| doi_str_mv | 10.1038/sj.bmt.1703526 |
| format | Article |
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M ; KHALIL, S ; KARAOUI, M ; SOLH, H</creator><creatorcontrib>AYAS, M ; AL-JEFRI, A ; BAOTHMAN, A ; AL-MAHR, M ; MUSTAFA, M. M ; KHALIL, S ; KARAOUI, M ; SOLH, H</creatorcontrib><description>Until recently, therapy for patients with severe congenital dyserythropoietic anemia (CDA) has been limited to blood transfusions and chelation therapy. Three children with transfusion-dependent CDA type I underwent allogeneic stem cell transplantation (SCT) from matched sibling donors. Conditioning was with cyclophosphamide 50 mg/kg/day for 4 days, busulphan 4 mg/kg/day for 4 days, and antithymocyte globulin (ATG) 30 mg/kg for four doses pre-SCT. All patients engrafted and are alive, and transfusion independent. To our knowledge, this is the first report of successful SCT in the management of CDA type I.</description><identifier>ISSN: 0268-3369</identifier><identifier>EISSN: 1476-5365</identifier><identifier>DOI: 10.1038/sj.bmt.1703526</identifier><identifier>PMID: 12180113</identifier><identifier>CODEN: BMTRE9</identifier><language>eng</language><publisher>Basingstoke: Nature Publishing Group</publisher><subject>Anemia ; Anemia, Dyserythropoietic, Congenital - classification ; Anemia, Dyserythropoietic, Congenital - therapy ; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy ; Antilymphocyte serum ; Biological and medical sciences ; Blood Transfusion ; Bone marrow ; Bone marrow, stem cells transplantation. Graft versus host reaction ; Busulfan ; Chelation ; Child ; Child, Preschool ; Cyclophosphamide ; Female ; Globulins ; Hematopoietic Stem Cell Transplantation ; Humans ; Infant ; Male ; Medical sciences ; Stem cell transplantation ; Stem cells ; Thymocytes ; Transfusion ; Transfusions. Complications. Transfusion reactions. Cell and gene therapy ; Transplantation ; Transplantation Conditioning ; Transplantation, Homologous</subject><ispartof>Bone marrow transplantation (Basingstoke), 2002-04, Vol.29 (8), p.681-682</ispartof><rights>2002 INIST-CNRS</rights><rights>COPYRIGHT 2002 Nature Publishing Group</rights><rights>Copyright Nature Publishing Group Apr 2002</rights><rights>Macmillan Publishers Limited 2002.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c407t-caefda2e92a925c6c422797de36bdda9a3e60a5c4db45950d3202bf86315d05e3</citedby><cites>FETCH-LOGICAL-c407t-caefda2e92a925c6c422797de36bdda9a3e60a5c4db45950d3202bf86315d05e3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>314,776,780,27901,27902</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=13679564$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/12180113$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>AYAS, M</creatorcontrib><creatorcontrib>AL-JEFRI, A</creatorcontrib><creatorcontrib>BAOTHMAN, A</creatorcontrib><creatorcontrib>AL-MAHR, M</creatorcontrib><creatorcontrib>MUSTAFA, M. M</creatorcontrib><creatorcontrib>KHALIL, S</creatorcontrib><creatorcontrib>KARAOUI, M</creatorcontrib><creatorcontrib>SOLH, H</creatorcontrib><title>Transfusion-dependent congenital dyserythropoietic anemia type I successfully treated with allogeneic stem cell transplantation</title><title>Bone marrow transplantation (Basingstoke)</title><addtitle>Bone Marrow Transplant</addtitle><description>Until recently, therapy for patients with severe congenital dyserythropoietic anemia (CDA) has been limited to blood transfusions and chelation therapy. Three children with transfusion-dependent CDA type I underwent allogeneic stem cell transplantation (SCT) from matched sibling donors. Conditioning was with cyclophosphamide 50 mg/kg/day for 4 days, busulphan 4 mg/kg/day for 4 days, and antithymocyte globulin (ATG) 30 mg/kg for four doses pre-SCT. All patients engrafted and are alive, and transfusion independent. To our knowledge, this is the first report of successful SCT in the management of CDA type I.</description><subject>Anemia</subject><subject>Anemia, Dyserythropoietic, Congenital - classification</subject><subject>Anemia, Dyserythropoietic, Congenital - therapy</subject><subject>Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy</subject><subject>Antilymphocyte serum</subject><subject>Biological and medical sciences</subject><subject>Blood Transfusion</subject><subject>Bone marrow</subject><subject>Bone marrow, stem cells transplantation. Graft versus host reaction</subject><subject>Busulfan</subject><subject>Chelation</subject><subject>Child</subject><subject>Child, Preschool</subject><subject>Cyclophosphamide</subject><subject>Female</subject><subject>Globulins</subject><subject>Hematopoietic Stem Cell Transplantation</subject><subject>Humans</subject><subject>Infant</subject><subject>Male</subject><subject>Medical sciences</subject><subject>Stem cell transplantation</subject><subject>Stem cells</subject><subject>Thymocytes</subject><subject>Transfusion</subject><subject>Transfusions. Complications. Transfusion reactions. Cell and gene therapy</subject><subject>Transplantation</subject><subject>Transplantation Conditioning</subject><subject>Transplantation, Homologous</subject><issn>0268-3369</issn><issn>1476-5365</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2002</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>BENPR</sourceid><recordid>eNp1kc2L1TAUxYMozvPp1qUEZdz1mY8mbZbD4OjAgJtxHdLkdl4faVOTFOnKf908pvBAcHU3v3vOvecg9J6SAyW8_ZJOh27MB9oQLph8gXa0bmQluBQv0Y4w2VacS3WF3qR0IoTWNRGv0RVltCWU8h368xjNlPolDWGqHMwwOZgytmF6gmnIxmO3JohrPsYwhwHyYLGZYBwMzusM-B6nxVpIRcL7FecIJoPDv4d8xMb7UFSgrKQMI7bgfSGK3-zNlE0unm_Rq974BO-2uUc_774-3n6vHn58u7-9eahsTZpcWQO9MwwUM4oJK23NWKMaB1x2zhllOEhihK1dVwsliOOMsK5vJafCEQF8jz4_684x_FogZT0O6XxQeSYsSTeMkJaXSPfo0z_gKSxxKrdpJosra1quCvXxvxSVhVNcXDyfjAd9BOPzMQW_nP9O-oYq1SqqJCvg4Rm0MaQUoddzHEYTV02JPtes00mXmvVWc1n4sNkv3Qjugm-9FuB6A0yyxvcldDukC8dlo4Ss-V89qLML</recordid><startdate>20020401</startdate><enddate>20020401</enddate><creator>AYAS, M</creator><creator>AL-JEFRI, A</creator><creator>BAOTHMAN, A</creator><creator>AL-MAHR, M</creator><creator>MUSTAFA, M. M</creator><creator>KHALIL, S</creator><creator>KARAOUI, M</creator><creator>SOLH, H</creator><general>Nature Publishing Group</general><scope>IQODW</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>3V.</scope><scope>7QO</scope><scope>7QP</scope><scope>7T5</scope><scope>7U9</scope><scope>7X7</scope><scope>7XB</scope><scope>88E</scope><scope>8AO</scope><scope>8FD</scope><scope>8FE</scope><scope>8FH</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AZQEC</scope><scope>BBNVY</scope><scope>BENPR</scope><scope>BHPHI</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FR3</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>GNUQQ</scope><scope>H94</scope><scope>HCIFZ</scope><scope>K9.</scope><scope>LK8</scope><scope>M0S</scope><scope>M1P</scope><scope>M7N</scope><scope>M7P</scope><scope>P64</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>7X8</scope></search><sort><creationdate>20020401</creationdate><title>Transfusion-dependent congenital dyserythropoietic anemia type I successfully treated with allogeneic stem cell transplantation</title><author>AYAS, M ; AL-JEFRI, A ; BAOTHMAN, A ; AL-MAHR, M ; MUSTAFA, M. 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Graft versus host reaction</topic><topic>Busulfan</topic><topic>Chelation</topic><topic>Child</topic><topic>Child, Preschool</topic><topic>Cyclophosphamide</topic><topic>Female</topic><topic>Globulins</topic><topic>Hematopoietic Stem Cell Transplantation</topic><topic>Humans</topic><topic>Infant</topic><topic>Male</topic><topic>Medical sciences</topic><topic>Stem cell transplantation</topic><topic>Stem cells</topic><topic>Thymocytes</topic><topic>Transfusion</topic><topic>Transfusions. Complications. Transfusion reactions. Cell and gene therapy</topic><topic>Transplantation</topic><topic>Transplantation Conditioning</topic><topic>Transplantation, Homologous</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>AYAS, M</creatorcontrib><creatorcontrib>AL-JEFRI, A</creatorcontrib><creatorcontrib>BAOTHMAN, A</creatorcontrib><creatorcontrib>AL-MAHR, M</creatorcontrib><creatorcontrib>MUSTAFA, M. 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M</au><au>KHALIL, S</au><au>KARAOUI, M</au><au>SOLH, H</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Transfusion-dependent congenital dyserythropoietic anemia type I successfully treated with allogeneic stem cell transplantation</atitle><jtitle>Bone marrow transplantation (Basingstoke)</jtitle><addtitle>Bone Marrow Transplant</addtitle><date>2002-04-01</date><risdate>2002</risdate><volume>29</volume><issue>8</issue><spage>681</spage><epage>682</epage><pages>681-682</pages><issn>0268-3369</issn><eissn>1476-5365</eissn><coden>BMTRE9</coden><abstract>Until recently, therapy for patients with severe congenital dyserythropoietic anemia (CDA) has been limited to blood transfusions and chelation therapy. Three children with transfusion-dependent CDA type I underwent allogeneic stem cell transplantation (SCT) from matched sibling donors. Conditioning was with cyclophosphamide 50 mg/kg/day for 4 days, busulphan 4 mg/kg/day for 4 days, and antithymocyte globulin (ATG) 30 mg/kg for four doses pre-SCT. All patients engrafted and are alive, and transfusion independent. To our knowledge, this is the first report of successful SCT in the management of CDA type I.</abstract><cop>Basingstoke</cop><pub>Nature Publishing Group</pub><pmid>12180113</pmid><doi>10.1038/sj.bmt.1703526</doi><tpages>2</tpages></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0268-3369 |
| ispartof | Bone marrow transplantation (Basingstoke), 2002-04, Vol.29 (8), p.681-682 |
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| source | MEDLINE; Nature Journals Online; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals; SpringerLink Journals - AutoHoldings |
| subjects | Anemia Anemia, Dyserythropoietic, Congenital - classification Anemia, Dyserythropoietic, Congenital - therapy Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy Antilymphocyte serum Biological and medical sciences Blood Transfusion Bone marrow Bone marrow, stem cells transplantation. Graft versus host reaction Busulfan Chelation Child Child, Preschool Cyclophosphamide Female Globulins Hematopoietic Stem Cell Transplantation Humans Infant Male Medical sciences Stem cell transplantation Stem cells Thymocytes Transfusion Transfusions. Complications. Transfusion reactions. Cell and gene therapy Transplantation Transplantation Conditioning Transplantation, Homologous |
| title | Transfusion-dependent congenital dyserythropoietic anemia type I successfully treated with allogeneic stem cell transplantation |
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