Transient myeloproliferative disorder in children with Down syndrome: clarity to this enigmatic disorder
Summary Children with trisomy 21 have a unique predisposition to develop a megakaryoblastic proliferative disease of varying severity during their first 3 months of life. This disorder exists in no other children or adults without the presence of trisomy 21 and only occurs in the fetal or neonatal p...
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| Veröffentlicht in: | British journal of haematology 2012-11, Vol.159 (3), p.277-287 |
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| container_title | British journal of haematology |
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| creator | Gamis, Alan S. Smith, Franklin O. |
| description | Summary
Children with trisomy 21 have a unique predisposition to develop a megakaryoblastic proliferative disease of varying severity during their first 3 months of life. This disorder exists in no other children or adults without the presence of trisomy 21 and only occurs in the fetal or neonatal period of life. Its spontaneous resolution in most cases further delineates it from otherwise indistinguishable neonatal leukaemias. The identification that GATA1 mutations are the leukaemogenic source along with three recently reported prospective clinical trials now provide a clearer understanding of this haematopoietic disorder. These recent advances in this enigmatic disorder, now known as Transient Myeloproliferative Disorder, are reviewed here in order to bring clarity to the breadth of organ involvement, the range of severity, the risk factors for mortality, the therapeutic options for severe manifestations, the natural course of spontaneous resolution regardless of therapy, and the elucidation of the subsequent risk for myeloid leukaemia. |
| doi_str_mv | 10.1111/bjh.12041 |
| format | Article |
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Children with trisomy 21 have a unique predisposition to develop a megakaryoblastic proliferative disease of varying severity during their first 3 months of life. This disorder exists in no other children or adults without the presence of trisomy 21 and only occurs in the fetal or neonatal period of life. Its spontaneous resolution in most cases further delineates it from otherwise indistinguishable neonatal leukaemias. The identification that GATA1 mutations are the leukaemogenic source along with three recently reported prospective clinical trials now provide a clearer understanding of this haematopoietic disorder. These recent advances in this enigmatic disorder, now known as Transient Myeloproliferative Disorder, are reviewed here in order to bring clarity to the breadth of organ involvement, the range of severity, the risk factors for mortality, the therapeutic options for severe manifestations, the natural course of spontaneous resolution regardless of therapy, and the elucidation of the subsequent risk for myeloid leukaemia.</description><identifier>ISSN: 0007-1048</identifier><identifier>EISSN: 1365-2141</identifier><identifier>DOI: 10.1111/bjh.12041</identifier><identifier>PMID: 22966823</identifier><identifier>CODEN: BJHEAL</identifier><language>eng</language><publisher>Oxford: Blackwell Publishing Ltd</publisher><subject>Biological and medical sciences ; Child ; Child, Preschool ; Chromosome aberrations ; Down syndrome ; Down Syndrome - complications ; GATA1 ; Hematologic and hematopoietic diseases ; Humans ; Incidence ; leukaemia ; Leukemia - etiology ; Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis ; Medical genetics ; Medical sciences ; Myeloproliferative Disorders - diagnosis ; Myeloproliferative Disorders - epidemiology ; Myeloproliferative Disorders - etiology ; Myeloproliferative Disorders - therapy ; newborn ; Risk Factors ; Transient Myeloproliferative Disorder</subject><ispartof>British journal of haematology, 2012-11, Vol.159 (3), p.277-287</ispartof><rights>2012 Blackwell Publishing Ltd</rights><rights>2015 INIST-CNRS</rights><rights>2012 Blackwell Publishing Ltd.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1111%2Fbjh.12041$$EPDF$$P50$$Gwiley$$H</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1111%2Fbjh.12041$$EHTML$$P50$$Gwiley$$H</linktohtml><link.rule.ids>314,777,781,1412,1428,27905,27906,45555,45556,46390,46814</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=26548520$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/22966823$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Gamis, Alan S.</creatorcontrib><creatorcontrib>Smith, Franklin O.</creatorcontrib><title>Transient myeloproliferative disorder in children with Down syndrome: clarity to this enigmatic disorder</title><title>British journal of haematology</title><addtitle>Br J Haematol</addtitle><description>Summary
Children with trisomy 21 have a unique predisposition to develop a megakaryoblastic proliferative disease of varying severity during their first 3 months of life. This disorder exists in no other children or adults without the presence of trisomy 21 and only occurs in the fetal or neonatal period of life. Its spontaneous resolution in most cases further delineates it from otherwise indistinguishable neonatal leukaemias. The identification that GATA1 mutations are the leukaemogenic source along with three recently reported prospective clinical trials now provide a clearer understanding of this haematopoietic disorder. These recent advances in this enigmatic disorder, now known as Transient Myeloproliferative Disorder, are reviewed here in order to bring clarity to the breadth of organ involvement, the range of severity, the risk factors for mortality, the therapeutic options for severe manifestations, the natural course of spontaneous resolution regardless of therapy, and the elucidation of the subsequent risk for myeloid leukaemia.</description><subject>Biological and medical sciences</subject><subject>Child</subject><subject>Child, Preschool</subject><subject>Chromosome aberrations</subject><subject>Down syndrome</subject><subject>Down Syndrome - complications</subject><subject>GATA1</subject><subject>Hematologic and hematopoietic diseases</subject><subject>Humans</subject><subject>Incidence</subject><subject>leukaemia</subject><subject>Leukemia - etiology</subject><subject>Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis</subject><subject>Medical genetics</subject><subject>Medical sciences</subject><subject>Myeloproliferative Disorders - diagnosis</subject><subject>Myeloproliferative Disorders - epidemiology</subject><subject>Myeloproliferative Disorders - etiology</subject><subject>Myeloproliferative Disorders - therapy</subject><subject>newborn</subject><subject>Risk Factors</subject><subject>Transient Myeloproliferative Disorder</subject><issn>0007-1048</issn><issn>1365-2141</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2012</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNpFkctuFDEQRS0EIkNgwQ8gb5DYdOJ3u9mRBCagKEgoCImNZbfLtEM_gt2Tof8ekxmG2lRJde6Vqi5CLyk5oaVO3W13QhkR9BFaUa5kxaigj9GKEFJXlAh9hJ7lfEsI5UTSp-iIsUYpzfgKdTfJjjnCOONhgX66S1MfAyQ7x3vAPuYpeUg4jrjtYu8TjHgb5w5fTNsR52X0aRrgLW57m-K84HnCcxczhjH-GIpHe7B4jp4E22d4se_H6OuH9zfnl9XV5_XH83dXVeRS0Mo3ogkNaST44FRtGaU1JVrr4LnzgXDmpSZBBcmDg-BCDVwo56zlrhWt5sfozc63XPJrA3k2Q8wt9L0dYdpkQ4u3ZpJKUdBXe3TjBvDmLsXBpsX8-04BXu8Bm1vbh_KqNub_nJJCS0YKd7rjtrGH5bCnxPyNx5R4zEM85uzT5cNQFNVOEfMMvw8Km34aVfNamm_Xa7NW9LtS19J84X8AWfySkQ</recordid><startdate>201211</startdate><enddate>201211</enddate><creator>Gamis, Alan S.</creator><creator>Smith, Franklin O.</creator><general>Blackwell Publishing Ltd</general><general>Blackwell</general><scope>BSCLL</scope><scope>IQODW</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>7X8</scope></search><sort><creationdate>201211</creationdate><title>Transient myeloproliferative disorder in children with Down syndrome: clarity to this enigmatic disorder</title><author>Gamis, Alan S. ; Smith, Franklin O.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-i3541-d949f9095edfb67a211710888fd3bdf032d580f6f53fbefbf7e346bbaa3bc4c83</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2012</creationdate><topic>Biological and medical sciences</topic><topic>Child</topic><topic>Child, Preschool</topic><topic>Chromosome aberrations</topic><topic>Down syndrome</topic><topic>Down Syndrome - complications</topic><topic>GATA1</topic><topic>Hematologic and hematopoietic diseases</topic><topic>Humans</topic><topic>Incidence</topic><topic>leukaemia</topic><topic>Leukemia - etiology</topic><topic>Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis</topic><topic>Medical genetics</topic><topic>Medical sciences</topic><topic>Myeloproliferative Disorders - diagnosis</topic><topic>Myeloproliferative Disorders - epidemiology</topic><topic>Myeloproliferative Disorders - etiology</topic><topic>Myeloproliferative Disorders - therapy</topic><topic>newborn</topic><topic>Risk Factors</topic><topic>Transient Myeloproliferative Disorder</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Gamis, Alan S.</creatorcontrib><creatorcontrib>Smith, Franklin O.</creatorcontrib><collection>Istex</collection><collection>Pascal-Francis</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>MEDLINE - Academic</collection><jtitle>British journal of haematology</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Gamis, Alan S.</au><au>Smith, Franklin O.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Transient myeloproliferative disorder in children with Down syndrome: clarity to this enigmatic disorder</atitle><jtitle>British journal of haematology</jtitle><addtitle>Br J Haematol</addtitle><date>2012-11</date><risdate>2012</risdate><volume>159</volume><issue>3</issue><spage>277</spage><epage>287</epage><pages>277-287</pages><issn>0007-1048</issn><eissn>1365-2141</eissn><coden>BJHEAL</coden><abstract>Summary
Children with trisomy 21 have a unique predisposition to develop a megakaryoblastic proliferative disease of varying severity during their first 3 months of life. This disorder exists in no other children or adults without the presence of trisomy 21 and only occurs in the fetal or neonatal period of life. Its spontaneous resolution in most cases further delineates it from otherwise indistinguishable neonatal leukaemias. The identification that GATA1 mutations are the leukaemogenic source along with three recently reported prospective clinical trials now provide a clearer understanding of this haematopoietic disorder. These recent advances in this enigmatic disorder, now known as Transient Myeloproliferative Disorder, are reviewed here in order to bring clarity to the breadth of organ involvement, the range of severity, the risk factors for mortality, the therapeutic options for severe manifestations, the natural course of spontaneous resolution regardless of therapy, and the elucidation of the subsequent risk for myeloid leukaemia.</abstract><cop>Oxford</cop><pub>Blackwell Publishing Ltd</pub><pmid>22966823</pmid><doi>10.1111/bjh.12041</doi><tpages>11</tpages><oa>free_for_read</oa></addata></record> |
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| source | MEDLINE; Wiley Online Library Journals Frontfile Complete; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals; Wiley Free Content |
| subjects | Biological and medical sciences Child Child, Preschool Chromosome aberrations Down syndrome Down Syndrome - complications GATA1 Hematologic and hematopoietic diseases Humans Incidence leukaemia Leukemia - etiology Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis Medical genetics Medical sciences Myeloproliferative Disorders - diagnosis Myeloproliferative Disorders - epidemiology Myeloproliferative Disorders - etiology Myeloproliferative Disorders - therapy newborn Risk Factors Transient Myeloproliferative Disorder |
| title | Transient myeloproliferative disorder in children with Down syndrome: clarity to this enigmatic disorder |
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