Prophylaxis versus Episodic Treatment to Prevent Joint Disease in Boys with Severe Hemophilia
This randomized trial involving young boys with severe hemophilia showed that prophylaxis with regular infusions of recombinant factor VIII was associated with clinically and statistically significant reductions in joint damage, as compared with episodic infusions at the time of a clinically evident...
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| Veröffentlicht in: | The New England journal of medicine 2007-08, Vol.357 (6), p.535-544 |
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| creator | Manco-Johnson, Michael L Manco-Johnson, Marilyn J Abshire, Thomas C Shapiro, Amy D Riske, Brenda Hacker, Michele R Kilcoyne, Ray Ingram, J. David Funk, Sharon Jacobson, Linda Valentino, Leonard A Hoots, W. Keith Buchanan, George R DiMichele, Donna Recht, Michael Brown, Deborah Leissinger, Cindy Bleak, Shirley Cohen, Alan Mathew, Prasad Matsunaga, Alison Medeiros, Desiree Nugent, Diane Thomas, Gregory A Thompson, Alexis A McRedmond, Kevin Soucie, J. Michael Austin, Harlan Evatt, Bruce L |
| description | This randomized trial involving young boys with severe hemophilia showed that prophylaxis with regular infusions of recombinant factor VIII was associated with clinically and statistically significant reductions in joint damage, as compared with episodic infusions at the time of a clinically evident hemarthrosis. Because of the high cost of recombinant factor VIII, its widespread use for prophylaxis may be impractical.
Prophylaxis with regular infusions of recombinant factor VIII was associated with clinically and statistically significant reductions in joint damage, as compared with episodic infusions.
Before the development of cryoprecipitate, a plasma fraction that contains concentrated factor VIII, boys with severe hemophilia A had a diminished life expectancy.
1
–
3
These children are at risk for many types of hemorrhages, but the predominant source of chronic coexisting disease is crippling, painful arthritis due to hemarthrosis.
4
Small trials were conducted in the 1960s to determine whether routine administration of factor VIII concentrate was effective as prophylaxis against hemophilic arthropathy.
5
–
8
Clinically effective prophylactic schedules were developed empirically, without the benefit of data from controlled trials,
9
and many clinicians began to recommend prophylaxis with factor VIII.
10
In the . . . |
| doi_str_mv | 10.1056/NEJMoa067659 |
| format | Article |
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Prophylaxis with regular infusions of recombinant factor VIII was associated with clinically and statistically significant reductions in joint damage, as compared with episodic infusions.
Before the development of cryoprecipitate, a plasma fraction that contains concentrated factor VIII, boys with severe hemophilia A had a diminished life expectancy.
1
–
3
These children are at risk for many types of hemorrhages, but the predominant source of chronic coexisting disease is crippling, painful arthritis due to hemarthrosis.
4
Small trials were conducted in the 1960s to determine whether routine administration of factor VIII concentrate was effective as prophylaxis against hemophilic arthropathy.
5
–
8
Clinically effective prophylactic schedules were developed empirically, without the benefit of data from controlled trials,
9
and many clinicians began to recommend prophylaxis with factor VIII.
10
In the . . .</description><identifier>ISSN: 0028-4793</identifier><identifier>EISSN: 1533-4406</identifier><identifier>DOI: 10.1056/NEJMoa067659</identifier><identifier>PMID: 17687129</identifier><identifier>CODEN: NEJMAG</identifier><language>eng</language><publisher>Boston, MA: Massachusetts Medical Society</publisher><subject>Biological and medical sciences ; Child ; Child, Preschool ; Diseases of the osteoarticular system ; Drug Administration Schedule ; Factor VIII - administration & dosage ; Follow-Up Studies ; General aspects ; Hemarthrosis - complications ; Hemarthrosis - drug therapy ; Hemarthrosis - prevention & control ; Hemophilia A - complications ; Hemophilia A - drug therapy ; Hemorrhage ; Hemorrhage - prevention & control ; Humans ; Infant ; Infusions, Intravenous ; Joint Diseases - etiology ; Joint Diseases - prevention & control ; Male ; Medical research ; Medical sciences ; Miscellaneous. Osteoarticular involvement in other diseases ; Prevention and actions ; Public health. Hygiene ; Public health. Hygiene-occupational medicine ; Treatment Outcome</subject><ispartof>The New England journal of medicine, 2007-08, Vol.357 (6), p.535-544</ispartof><rights>Copyright © 2007 Massachusetts Medical Society. All rights reserved.</rights><rights>2007 INIST-CNRS</rights><rights>Copyright 2007 Massachusetts Medical Society.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c543t-36a8240d6b34dff94e11f27965512ee980b0bcd00da9a45cbf2ed4d3ae144fd3</citedby><cites>FETCH-LOGICAL-c543t-36a8240d6b34dff94e11f27965512ee980b0bcd00da9a45cbf2ed4d3ae144fd3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://www.nejm.org/doi/pdf/10.1056/NEJMoa067659$$EPDF$$P50$$Gmms$$H</linktopdf><linktohtml>$$Uhttps://www.nejm.org/doi/full/10.1056/NEJMoa067659$$EHTML$$P50$$Gmms$$H</linktohtml><link.rule.ids>314,776,780,2746,2747,26080,27901,27902,52357,54039</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=18977165$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/17687129$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Manco-Johnson, Michael L</creatorcontrib><creatorcontrib>Manco-Johnson, Marilyn J</creatorcontrib><creatorcontrib>Abshire, Thomas C</creatorcontrib><creatorcontrib>Shapiro, Amy D</creatorcontrib><creatorcontrib>Riske, Brenda</creatorcontrib><creatorcontrib>Hacker, Michele R</creatorcontrib><creatorcontrib>Kilcoyne, Ray</creatorcontrib><creatorcontrib>Ingram, J. David</creatorcontrib><creatorcontrib>Funk, Sharon</creatorcontrib><creatorcontrib>Jacobson, Linda</creatorcontrib><creatorcontrib>Valentino, Leonard A</creatorcontrib><creatorcontrib>Hoots, W. Keith</creatorcontrib><creatorcontrib>Buchanan, George R</creatorcontrib><creatorcontrib>DiMichele, Donna</creatorcontrib><creatorcontrib>Recht, Michael</creatorcontrib><creatorcontrib>Brown, Deborah</creatorcontrib><creatorcontrib>Leissinger, Cindy</creatorcontrib><creatorcontrib>Bleak, Shirley</creatorcontrib><creatorcontrib>Cohen, Alan</creatorcontrib><creatorcontrib>Mathew, Prasad</creatorcontrib><creatorcontrib>Matsunaga, Alison</creatorcontrib><creatorcontrib>Medeiros, Desiree</creatorcontrib><creatorcontrib>Nugent, Diane</creatorcontrib><creatorcontrib>Thomas, Gregory A</creatorcontrib><creatorcontrib>Thompson, Alexis A</creatorcontrib><creatorcontrib>McRedmond, Kevin</creatorcontrib><creatorcontrib>Soucie, J. Michael</creatorcontrib><creatorcontrib>Austin, Harlan</creatorcontrib><creatorcontrib>Evatt, Bruce L</creatorcontrib><title>Prophylaxis versus Episodic Treatment to Prevent Joint Disease in Boys with Severe Hemophilia</title><title>The New England journal of medicine</title><addtitle>N Engl J Med</addtitle><description>This randomized trial involving young boys with severe hemophilia showed that prophylaxis with regular infusions of recombinant factor VIII was associated with clinically and statistically significant reductions in joint damage, as compared with episodic infusions at the time of a clinically evident hemarthrosis. Because of the high cost of recombinant factor VIII, its widespread use for prophylaxis may be impractical.
Prophylaxis with regular infusions of recombinant factor VIII was associated with clinically and statistically significant reductions in joint damage, as compared with episodic infusions.
Before the development of cryoprecipitate, a plasma fraction that contains concentrated factor VIII, boys with severe hemophilia A had a diminished life expectancy.
1
–
3
These children are at risk for many types of hemorrhages, but the predominant source of chronic coexisting disease is crippling, painful arthritis due to hemarthrosis.
4
Small trials were conducted in the 1960s to determine whether routine administration of factor VIII concentrate was effective as prophylaxis against hemophilic arthropathy.
5
–
8
Clinically effective prophylactic schedules were developed empirically, without the benefit of data from controlled trials,
9
and many clinicians began to recommend prophylaxis with factor VIII.
10
In the . . .</description><subject>Biological and medical sciences</subject><subject>Child</subject><subject>Child, Preschool</subject><subject>Diseases of the osteoarticular system</subject><subject>Drug Administration Schedule</subject><subject>Factor VIII - administration & dosage</subject><subject>Follow-Up Studies</subject><subject>General aspects</subject><subject>Hemarthrosis - complications</subject><subject>Hemarthrosis - drug therapy</subject><subject>Hemarthrosis - prevention & control</subject><subject>Hemophilia A - complications</subject><subject>Hemophilia A - drug therapy</subject><subject>Hemorrhage</subject><subject>Hemorrhage - prevention & control</subject><subject>Humans</subject><subject>Infant</subject><subject>Infusions, Intravenous</subject><subject>Joint Diseases - etiology</subject><subject>Joint Diseases - prevention & control</subject><subject>Male</subject><subject>Medical research</subject><subject>Medical sciences</subject><subject>Miscellaneous. Osteoarticular involvement in other diseases</subject><subject>Prevention and actions</subject><subject>Public health. Hygiene</subject><subject>Public health. Hygiene-occupational medicine</subject><subject>Treatment Outcome</subject><issn>0028-4793</issn><issn>1533-4406</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2007</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>BEC</sourceid><sourceid>BENPR</sourceid><sourceid>GUQSH</sourceid><sourceid>M2O</sourceid><recordid>eNpt0MFP2zAUBnBr2jQK240zsibgRDY7dpz4CKUbVGxDWq8ocuIX4SqJi18C9L_HVSsxIXywffjp8_NHyCFn3znL1I8_s_lvb5jKVaY_kAnPhEikZOojmTCWFonMtdgj-4hLFheX-jPZ47kqcp7qCbm7DX51v27Ns0P6CAFHpLOVQ29dTRcBzNBBP9DB09sAj5vr3Lu4XzoEg0BdTy_8GumTG-7pvygC0CvoYqZrnflCPjWmRfi6Ow_I4udsMb1Kbv7-up6e3yR1JsWQCGWKVDKrKiFt02gJnDdprlWW8RRAF6xiVW0Zs0YbmdVVk4KVVhjgUjZWHJDTbewq-IcRcCg7hzW0renBj1iqIn5bMBbhtzdw6cfQx9HKNBVaFFyJiM62qA4eMUBTroLrTFiXnJWbysv_K4_8aJc5Vh3YV7zrOIKTHTBYm7YJpq8dvrpC5zlXWXTHW9d1WPaw7N5_7wUf8JS-</recordid><startdate>20070809</startdate><enddate>20070809</enddate><creator>Manco-Johnson, Michael L</creator><creator>Manco-Johnson, Marilyn J</creator><creator>Abshire, Thomas C</creator><creator>Shapiro, Amy D</creator><creator>Riske, Brenda</creator><creator>Hacker, Michele R</creator><creator>Kilcoyne, Ray</creator><creator>Ingram, J. David</creator><creator>Funk, Sharon</creator><creator>Jacobson, Linda</creator><creator>Valentino, Leonard A</creator><creator>Hoots, W. Keith</creator><creator>Buchanan, George R</creator><creator>DiMichele, Donna</creator><creator>Recht, Michael</creator><creator>Brown, Deborah</creator><creator>Leissinger, Cindy</creator><creator>Bleak, Shirley</creator><creator>Cohen, Alan</creator><creator>Mathew, Prasad</creator><creator>Matsunaga, Alison</creator><creator>Medeiros, Desiree</creator><creator>Nugent, Diane</creator><creator>Thomas, Gregory A</creator><creator>Thompson, Alexis A</creator><creator>McRedmond, Kevin</creator><creator>Soucie, J. Michael</creator><creator>Austin, Harlan</creator><creator>Evatt, Bruce L</creator><general>Massachusetts Medical Society</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>0TZ</scope><scope>7RV</scope><scope>7X7</scope><scope>7XB</scope><scope>8AO</scope><scope>8C1</scope><scope>8FE</scope><scope>8FH</scope><scope>8FI</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AN0</scope><scope>AZQEC</scope><scope>BBNVY</scope><scope>BEC</scope><scope>BENPR</scope><scope>BHPHI</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>GNUQQ</scope><scope>GUQSH</scope><scope>HCIFZ</scope><scope>K0Y</scope><scope>LK8</scope><scope>M0R</scope><scope>M0T</scope><scope>M1P</scope><scope>M2M</scope><scope>M2O</scope><scope>M2P</scope><scope>M7P</scope><scope>MBDVC</scope><scope>NAPCQ</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>PSYQQ</scope><scope>Q9U</scope><scope>7X8</scope></search><sort><creationdate>20070809</creationdate><title>Prophylaxis versus Episodic Treatment to Prevent Joint Disease in Boys with Severe Hemophilia</title><author>Manco-Johnson, Michael L ; Manco-Johnson, Marilyn J ; Abshire, Thomas C ; Shapiro, Amy D ; Riske, Brenda ; Hacker, Michele R ; Kilcoyne, Ray ; Ingram, J. David ; Funk, Sharon ; Jacobson, Linda ; Valentino, Leonard A ; Hoots, W. Keith ; Buchanan, George R ; DiMichele, Donna ; Recht, Michael ; Brown, Deborah ; Leissinger, Cindy ; Bleak, Shirley ; Cohen, Alan ; Mathew, Prasad ; Matsunaga, Alison ; Medeiros, Desiree ; Nugent, Diane ; Thomas, Gregory A ; Thompson, Alexis A ; McRedmond, Kevin ; Soucie, J. Michael ; Austin, Harlan ; Evatt, Bruce L</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c543t-36a8240d6b34dff94e11f27965512ee980b0bcd00da9a45cbf2ed4d3ae144fd3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2007</creationdate><topic>Biological and medical sciences</topic><topic>Child</topic><topic>Child, Preschool</topic><topic>Diseases of the osteoarticular system</topic><topic>Drug Administration Schedule</topic><topic>Factor VIII - administration & dosage</topic><topic>Follow-Up Studies</topic><topic>General aspects</topic><topic>Hemarthrosis - complications</topic><topic>Hemarthrosis - drug therapy</topic><topic>Hemarthrosis - prevention & control</topic><topic>Hemophilia A - complications</topic><topic>Hemophilia A - drug therapy</topic><topic>Hemorrhage</topic><topic>Hemorrhage - prevention & control</topic><topic>Humans</topic><topic>Infant</topic><topic>Infusions, Intravenous</topic><topic>Joint Diseases - etiology</topic><topic>Joint Diseases - prevention & control</topic><topic>Male</topic><topic>Medical research</topic><topic>Medical sciences</topic><topic>Miscellaneous. 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Michael</au><au>Austin, Harlan</au><au>Evatt, Bruce L</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Prophylaxis versus Episodic Treatment to Prevent Joint Disease in Boys with Severe Hemophilia</atitle><jtitle>The New England journal of medicine</jtitle><addtitle>N Engl J Med</addtitle><date>2007-08-09</date><risdate>2007</risdate><volume>357</volume><issue>6</issue><spage>535</spage><epage>544</epage><pages>535-544</pages><issn>0028-4793</issn><eissn>1533-4406</eissn><coden>NEJMAG</coden><abstract>This randomized trial involving young boys with severe hemophilia showed that prophylaxis with regular infusions of recombinant factor VIII was associated with clinically and statistically significant reductions in joint damage, as compared with episodic infusions at the time of a clinically evident hemarthrosis. Because of the high cost of recombinant factor VIII, its widespread use for prophylaxis may be impractical.
Prophylaxis with regular infusions of recombinant factor VIII was associated with clinically and statistically significant reductions in joint damage, as compared with episodic infusions.
Before the development of cryoprecipitate, a plasma fraction that contains concentrated factor VIII, boys with severe hemophilia A had a diminished life expectancy.
1
–
3
These children are at risk for many types of hemorrhages, but the predominant source of chronic coexisting disease is crippling, painful arthritis due to hemarthrosis.
4
Small trials were conducted in the 1960s to determine whether routine administration of factor VIII concentrate was effective as prophylaxis against hemophilic arthropathy.
5
–
8
Clinically effective prophylactic schedules were developed empirically, without the benefit of data from controlled trials,
9
and many clinicians began to recommend prophylaxis with factor VIII.
10
In the . . .</abstract><cop>Boston, MA</cop><pub>Massachusetts Medical Society</pub><pmid>17687129</pmid><doi>10.1056/NEJMoa067659</doi><tpages>10</tpages><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0028-4793 |
| ispartof | The New England journal of medicine, 2007-08, Vol.357 (6), p.535-544 |
| issn | 0028-4793 1533-4406 |
| language | eng |
| recordid | cdi_proquest_miscellaneous_68149300 |
| source | MEDLINE; EZB-FREE-00999 freely available EZB journals; New England Journal of Medicine |
| subjects | Biological and medical sciences Child Child, Preschool Diseases of the osteoarticular system Drug Administration Schedule Factor VIII - administration & dosage Follow-Up Studies General aspects Hemarthrosis - complications Hemarthrosis - drug therapy Hemarthrosis - prevention & control Hemophilia A - complications Hemophilia A - drug therapy Hemorrhage Hemorrhage - prevention & control Humans Infant Infusions, Intravenous Joint Diseases - etiology Joint Diseases - prevention & control Male Medical research Medical sciences Miscellaneous. Osteoarticular involvement in other diseases Prevention and actions Public health. Hygiene Public health. Hygiene-occupational medicine Treatment Outcome |
| title | Prophylaxis versus Episodic Treatment to Prevent Joint Disease in Boys with Severe Hemophilia |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2025-02-10T07%3A57%3A05IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-proquest_cross&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Prophylaxis%20versus%20Episodic%20Treatment%20to%20Prevent%20Joint%20Disease%20in%20Boys%20with%20Severe%20Hemophilia&rft.jtitle=The%20New%20England%20journal%20of%20medicine&rft.au=Manco-Johnson,%20Michael%20L&rft.date=2007-08-09&rft.volume=357&rft.issue=6&rft.spage=535&rft.epage=544&rft.pages=535-544&rft.issn=0028-4793&rft.eissn=1533-4406&rft.coden=NEJMAG&rft_id=info:doi/10.1056/NEJMoa067659&rft_dat=%3Cproquest_cross%3E68149300%3C/proquest_cross%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_pqid=223938163&rft_id=info:pmid/17687129&rfr_iscdi=true |