Bone marrow transplantation in severe Glanzmann's thrombasthenia with antiplatelet alloimmunization
Glanzmann's thrombasthenia is an autosomal recessive disorder characterized by a lack of platelet aggregation due to the absence of platelet glycoprotein IIb and IIIa. Usually, the disease leads to mild hemorrhage but sometimes bleeding is severe enough to be life-threatening. We report the cas...
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| Veröffentlicht in: | Bone marrow transplantation (Basingstoke) 2000-02, Vol.25 (3), p.327-330 |
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| container_title | Bone marrow transplantation (Basingstoke) |
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| creator | BELLUCCI, S DAMAJ, G GLUCKMAN, E BOVAL, B ROCHA, V DEVERGIE, A AGHA, I. Y GARDERET, L RIBAUD, P TRAINEAU, R SOCIE, G |
| description | Glanzmann's thrombasthenia is an autosomal recessive disorder characterized by a lack of platelet aggregation due to the absence of platelet glycoprotein IIb and IIIa. Usually, the disease leads to mild hemorrhage but sometimes bleeding is severe enough to be life-threatening. We report the case of a 16-year-old girl, presenting with very severe type 1 Glanzmann's thrombasthenia, successfully treated with an HLA-identical sibling bone marrow transplant (BMT). We also update the clinical and laboratory data of her brother, who had received a BMT 16 years ago for the same disease. In the light of these two cases and two others published in the literature, we discuss the indications for BMT from HLA-identical sibling donors in Glanzmann's thrombasthenia. Alloimmunization against the missing platelet GPIIb/IIIa complex and severity of bleeding episodes may constitute sufficient criteria for allogeneic BMT after careful assessment of the risk-benefit of such a procedure, although this remains exceptional in this disease. Bone Marrow Transplantation (2000) 25, 327-330. |
| doi_str_mv | 10.1038/sj.bmt.1702139 |
| format | Article |
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Y ; GARDERET, L ; RIBAUD, P ; TRAINEAU, R ; SOCIE, G</creator><creatorcontrib>BELLUCCI, S ; DAMAJ, G ; GLUCKMAN, E ; BOVAL, B ; ROCHA, V ; DEVERGIE, A ; AGHA, I. Y ; GARDERET, L ; RIBAUD, P ; TRAINEAU, R ; SOCIE, G</creatorcontrib><description>Glanzmann's thrombasthenia is an autosomal recessive disorder characterized by a lack of platelet aggregation due to the absence of platelet glycoprotein IIb and IIIa. Usually, the disease leads to mild hemorrhage but sometimes bleeding is severe enough to be life-threatening. We report the case of a 16-year-old girl, presenting with very severe type 1 Glanzmann's thrombasthenia, successfully treated with an HLA-identical sibling bone marrow transplant (BMT). We also update the clinical and laboratory data of her brother, who had received a BMT 16 years ago for the same disease. In the light of these two cases and two others published in the literature, we discuss the indications for BMT from HLA-identical sibling donors in Glanzmann's thrombasthenia. Alloimmunization against the missing platelet GPIIb/IIIa complex and severity of bleeding episodes may constitute sufficient criteria for allogeneic BMT after careful assessment of the risk-benefit of such a procedure, although this remains exceptional in this disease. Bone Marrow Transplantation (2000) 25, 327-330.</description><identifier>ISSN: 0268-3369</identifier><identifier>EISSN: 1476-5365</identifier><identifier>DOI: 10.1038/sj.bmt.1702139</identifier><identifier>PMID: 10673707</identifier><identifier>CODEN: BMTRE9</identifier><language>eng</language><publisher>Basingstoke: Nature Publishing Group</publisher><subject>Adolescent ; Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy ; Biological and medical sciences ; Bleeding ; Blood Platelets - immunology ; Bone marrow ; Bone Marrow Transplantation ; Bone marrow, stem cells transplantation. Graft versus host reaction ; Child ; Female ; Glycoproteins ; Hemorrhage ; Hereditary diseases ; Histocompatibility antigen HLA ; HLA Antigens - immunology ; Humans ; Isoantibodies - blood ; Isoimmunization ; Male ; Medical sciences ; Nuclear Family ; Pedigree ; Platelet aggregation ; Platelet Glycoprotein GPIIb-IIIa Complex - immunology ; Stem cell transplantation ; Thrombasthenia - blood ; Thrombasthenia - immunology ; Thrombasthenia - therapy ; Transfusions. Complications. Transfusion reactions. Cell and gene therapy ; Transplantation</subject><ispartof>Bone marrow transplantation (Basingstoke), 2000-02, Vol.25 (3), p.327-330</ispartof><rights>2000 INIST-CNRS</rights><rights>Copyright Nature Publishing Group Feb 2000</rights><rights>Macmillan Publishers Limited 2000.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c415t-cc272bf1567f7cbb9125b5a98fe138c29ee402ed0a21ec44dd268b0a61904a0c3</citedby><cites>FETCH-LOGICAL-c415t-cc272bf1567f7cbb9125b5a98fe138c29ee402ed0a21ec44dd268b0a61904a0c3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>314,780,784,27924,27925</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=1268574$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/10673707$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>BELLUCCI, S</creatorcontrib><creatorcontrib>DAMAJ, G</creatorcontrib><creatorcontrib>GLUCKMAN, E</creatorcontrib><creatorcontrib>BOVAL, B</creatorcontrib><creatorcontrib>ROCHA, V</creatorcontrib><creatorcontrib>DEVERGIE, A</creatorcontrib><creatorcontrib>AGHA, I. Y</creatorcontrib><creatorcontrib>GARDERET, L</creatorcontrib><creatorcontrib>RIBAUD, P</creatorcontrib><creatorcontrib>TRAINEAU, R</creatorcontrib><creatorcontrib>SOCIE, G</creatorcontrib><title>Bone marrow transplantation in severe Glanzmann's thrombasthenia with antiplatelet alloimmunization</title><title>Bone marrow transplantation (Basingstoke)</title><addtitle>Bone Marrow Transplant</addtitle><description>Glanzmann's thrombasthenia is an autosomal recessive disorder characterized by a lack of platelet aggregation due to the absence of platelet glycoprotein IIb and IIIa. Usually, the disease leads to mild hemorrhage but sometimes bleeding is severe enough to be life-threatening. We report the case of a 16-year-old girl, presenting with very severe type 1 Glanzmann's thrombasthenia, successfully treated with an HLA-identical sibling bone marrow transplant (BMT). We also update the clinical and laboratory data of her brother, who had received a BMT 16 years ago for the same disease. In the light of these two cases and two others published in the literature, we discuss the indications for BMT from HLA-identical sibling donors in Glanzmann's thrombasthenia. Alloimmunization against the missing platelet GPIIb/IIIa complex and severity of bleeding episodes may constitute sufficient criteria for allogeneic BMT after careful assessment of the risk-benefit of such a procedure, although this remains exceptional in this disease. Bone Marrow Transplantation (2000) 25, 327-330.</description><subject>Adolescent</subject><subject>Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy</subject><subject>Biological and medical sciences</subject><subject>Bleeding</subject><subject>Blood Platelets - immunology</subject><subject>Bone marrow</subject><subject>Bone Marrow Transplantation</subject><subject>Bone marrow, stem cells transplantation. Graft versus host reaction</subject><subject>Child</subject><subject>Female</subject><subject>Glycoproteins</subject><subject>Hemorrhage</subject><subject>Hereditary diseases</subject><subject>Histocompatibility antigen HLA</subject><subject>HLA Antigens - immunology</subject><subject>Humans</subject><subject>Isoantibodies - blood</subject><subject>Isoimmunization</subject><subject>Male</subject><subject>Medical sciences</subject><subject>Nuclear Family</subject><subject>Pedigree</subject><subject>Platelet aggregation</subject><subject>Platelet Glycoprotein GPIIb-IIIa Complex - immunology</subject><subject>Stem cell transplantation</subject><subject>Thrombasthenia - blood</subject><subject>Thrombasthenia - immunology</subject><subject>Thrombasthenia - therapy</subject><subject>Transfusions. Complications. Transfusion reactions. 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Graft versus host reaction</topic><topic>Child</topic><topic>Female</topic><topic>Glycoproteins</topic><topic>Hemorrhage</topic><topic>Hereditary diseases</topic><topic>Histocompatibility antigen HLA</topic><topic>HLA Antigens - immunology</topic><topic>Humans</topic><topic>Isoantibodies - blood</topic><topic>Isoimmunization</topic><topic>Male</topic><topic>Medical sciences</topic><topic>Nuclear Family</topic><topic>Pedigree</topic><topic>Platelet aggregation</topic><topic>Platelet Glycoprotein GPIIb-IIIa Complex - immunology</topic><topic>Stem cell transplantation</topic><topic>Thrombasthenia - blood</topic><topic>Thrombasthenia - immunology</topic><topic>Thrombasthenia - therapy</topic><topic>Transfusions. Complications. Transfusion reactions. 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Y</au><au>GARDERET, L</au><au>RIBAUD, P</au><au>TRAINEAU, R</au><au>SOCIE, G</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Bone marrow transplantation in severe Glanzmann's thrombasthenia with antiplatelet alloimmunization</atitle><jtitle>Bone marrow transplantation (Basingstoke)</jtitle><addtitle>Bone Marrow Transplant</addtitle><date>2000-02-01</date><risdate>2000</risdate><volume>25</volume><issue>3</issue><spage>327</spage><epage>330</epage><pages>327-330</pages><issn>0268-3369</issn><eissn>1476-5365</eissn><coden>BMTRE9</coden><abstract>Glanzmann's thrombasthenia is an autosomal recessive disorder characterized by a lack of platelet aggregation due to the absence of platelet glycoprotein IIb and IIIa. Usually, the disease leads to mild hemorrhage but sometimes bleeding is severe enough to be life-threatening. We report the case of a 16-year-old girl, presenting with very severe type 1 Glanzmann's thrombasthenia, successfully treated with an HLA-identical sibling bone marrow transplant (BMT). We also update the clinical and laboratory data of her brother, who had received a BMT 16 years ago for the same disease. In the light of these two cases and two others published in the literature, we discuss the indications for BMT from HLA-identical sibling donors in Glanzmann's thrombasthenia. Alloimmunization against the missing platelet GPIIb/IIIa complex and severity of bleeding episodes may constitute sufficient criteria for allogeneic BMT after careful assessment of the risk-benefit of such a procedure, although this remains exceptional in this disease. Bone Marrow Transplantation (2000) 25, 327-330.</abstract><cop>Basingstoke</cop><pub>Nature Publishing Group</pub><pmid>10673707</pmid><doi>10.1038/sj.bmt.1702139</doi><tpages>4</tpages><oa>free_for_read</oa></addata></record> |
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| subjects | Adolescent Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy Biological and medical sciences Bleeding Blood Platelets - immunology Bone marrow Bone Marrow Transplantation Bone marrow, stem cells transplantation. Graft versus host reaction Child Female Glycoproteins Hemorrhage Hereditary diseases Histocompatibility antigen HLA HLA Antigens - immunology Humans Isoantibodies - blood Isoimmunization Male Medical sciences Nuclear Family Pedigree Platelet aggregation Platelet Glycoprotein GPIIb-IIIa Complex - immunology Stem cell transplantation Thrombasthenia - blood Thrombasthenia - immunology Thrombasthenia - therapy Transfusions. Complications. Transfusion reactions. Cell and gene therapy Transplantation |
| title | Bone marrow transplantation in severe Glanzmann's thrombasthenia with antiplatelet alloimmunization |
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