Long-Term Treatment of Osteopetrosis with Recombinant Human Interferon Gamma
Congenital osteopetrosis is a group of disorders resulting in decreased osteoclastic function and hence decreased bone resorption. The accumulation of sclerotic bone compromises marrow space and cranial-nerve foramina and predisposes patients to pathologic fractures. Most patients become blind or an...
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| Veröffentlicht in: | The New England journal of medicine 1995-06, Vol.332 (24), p.1594-1599 |
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| container_title | The New England journal of medicine |
| container_volume | 332 |
| creator | Key, L. Lyndon Rodriguiz, Ramona Marie Willi, Steven M Wright, Nancy M Hatcher, Heather C Eyre, David R Cure, Joel K Griffin, Paul P Ries, William L |
| description | Congenital osteopetrosis is a group of disorders resulting in decreased osteoclastic function and hence decreased bone resorption. The accumulation of sclerotic bone compromises marrow space and cranial-nerve foramina and predisposes patients to pathologic fractures. Most patients become blind or anemic before six months of age and die because of poor resistance to infection, neurologic deficits, or bone marrow failure during the first decade — often the first two years — of life.
1
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Bone marrow transplantation is curative therapy,
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but an acceptable donor can be found for only about 40 percent of patients. Overall survival 23 months after transplantation was . . . |
| doi_str_mv | 10.1056/NEJM199506153322402 |
| format | Article |
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1
–
4
Bone marrow transplantation is curative therapy,
5
but an acceptable donor can be found for only about 40 percent of patients. Overall survival 23 months after transplantation was . . .</description><identifier>ISSN: 0028-4793</identifier><identifier>EISSN: 1533-4406</identifier><identifier>DOI: 10.1056/NEJM199506153322402</identifier><identifier>PMID: 7753137</identifier><identifier>CODEN: NEJMAG</identifier><language>eng</language><publisher>United States: Massachusetts Medical Society</publisher><subject>Adult ; Biochemical markers ; Biopsy ; Body weight ; Bone imaging ; Bone marrow ; Bone Resorption ; Bone turnover ; Cancellous bone ; Child, Preschool ; Drug dosages ; Endocrinology ; Hemoglobin ; Humans ; Infections ; Injections, Subcutaneous ; Interferon ; Interferon-gamma - administration & dosage ; Interferon-gamma - therapeutic use ; Leukocytes (granulocytic) ; Macrophages ; Orthopedics ; Osteoclasts ; Osteopetrosis ; Osteopetrosis - congenital ; Osteopetrosis - pathology ; Osteopetrosis - therapy ; Patients ; Pediatrics ; Recombinant Proteins - administration & dosage ; Recombinant Proteins - therapeutic use ; Sulfur ; Superoxide ; Transplants & implants</subject><ispartof>The New England journal of medicine, 1995-06, Vol.332 (24), p.1594-1599</ispartof><rights>Copyright © 1995 Massachusetts Medical Society. All rights reserved.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c527t-479ecbb1ac0aa1f45b52cacb635177deb093ad2ba9c00b60864f3628cc9c1bb03</citedby><cites>FETCH-LOGICAL-c527t-479ecbb1ac0aa1f45b52cacb635177deb093ad2ba9c00b60864f3628cc9c1bb03</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/NEJM199506153322402$$EPDF$$P50$$Gmms$$H</linktopdf><linktohtml>$$Uhttps://www.nejm.org/doi/full/10.1056/NEJM199506153322402$$EHTML$$P50$$Gmms$$H</linktohtml><link.rule.ids>314,776,780,2746,2747,26080,27901,27902,52357,54039</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/7753137$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Key, L. Lyndon</creatorcontrib><creatorcontrib>Rodriguiz, Ramona Marie</creatorcontrib><creatorcontrib>Willi, Steven M</creatorcontrib><creatorcontrib>Wright, Nancy M</creatorcontrib><creatorcontrib>Hatcher, Heather C</creatorcontrib><creatorcontrib>Eyre, David R</creatorcontrib><creatorcontrib>Cure, Joel K</creatorcontrib><creatorcontrib>Griffin, Paul P</creatorcontrib><creatorcontrib>Ries, William L</creatorcontrib><title>Long-Term Treatment of Osteopetrosis with Recombinant Human Interferon Gamma</title><title>The New England journal of medicine</title><addtitle>N Engl J Med</addtitle><description>Congenital osteopetrosis is a group of disorders resulting in decreased osteoclastic function and hence decreased bone resorption. The accumulation of sclerotic bone compromises marrow space and cranial-nerve foramina and predisposes patients to pathologic fractures. Most patients become blind or anemic before six months of age and die because of poor resistance to infection, neurologic deficits, or bone marrow failure during the first decade — often the first two years — of life.
1
–
4
Bone marrow transplantation is curative therapy,
5
but an acceptable donor can be found for only about 40 percent of patients. Overall survival 23 months after transplantation was . . .</description><subject>Adult</subject><subject>Biochemical markers</subject><subject>Biopsy</subject><subject>Body weight</subject><subject>Bone imaging</subject><subject>Bone marrow</subject><subject>Bone Resorption</subject><subject>Bone turnover</subject><subject>Cancellous bone</subject><subject>Child, Preschool</subject><subject>Drug dosages</subject><subject>Endocrinology</subject><subject>Hemoglobin</subject><subject>Humans</subject><subject>Infections</subject><subject>Injections, Subcutaneous</subject><subject>Interferon</subject><subject>Interferon-gamma - administration & dosage</subject><subject>Interferon-gamma - therapeutic use</subject><subject>Leukocytes (granulocytic)</subject><subject>Macrophages</subject><subject>Orthopedics</subject><subject>Osteoclasts</subject><subject>Osteopetrosis</subject><subject>Osteopetrosis - congenital</subject><subject>Osteopetrosis - pathology</subject><subject>Osteopetrosis - therapy</subject><subject>Patients</subject><subject>Pediatrics</subject><subject>Recombinant Proteins - administration & dosage</subject><subject>Recombinant Proteins - therapeutic use</subject><subject>Sulfur</subject><subject>Superoxide</subject><subject>Transplants & implants</subject><issn>0028-4793</issn><issn>1533-4406</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>1995</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>BEC</sourceid><sourceid>BENPR</sourceid><sourceid>GUQSH</sourceid><sourceid>M2O</sourceid><recordid>eNp9kVtLJDEQhYO46Hj5BSI0Cr5Ir5WkO5k8yuBtmVWQ8blJMtXawyQZk24W__1mmMEHcbdeqqC-czhUEXJC4SeFWlw93vz6TZWqQdCac8YqYDtktJ7LqgKxS0YAbFxWUvF9cpDSAnLRSu2RPSlrTrkckek0-NdyhtEVs4i6d-j7IrTFU-oxrLCPIXWp-NP1b8Uz2uBM53Um7genffHge4wtxuCLO-2cPiI_Wr1MeLzth-Tl9mY2uS-nT3cPk-tpaWsm-3UgtMZQbUFr2la1qZnV1gheUynnaEBxPWdGKwtgBIxF1XLBxtYqS40BfkguNr6rGN4HTH3jumRxudQew5AaKZkUgtIMnn0BF2GIPmdrGONKAnCeofN_QVSNuaKMS5EpvqFsPkmK2Dar2DkdPxoKzfodzTfvyKrTrfdgHM4_Ndv75_3lZu9cajwu3H_d_gLNgpBQ</recordid><startdate>19950615</startdate><enddate>19950615</enddate><creator>Key, L. Lyndon</creator><creator>Rodriguiz, Ramona Marie</creator><creator>Willi, Steven M</creator><creator>Wright, Nancy M</creator><creator>Hatcher, Heather C</creator><creator>Eyre, David R</creator><creator>Cure, Joel K</creator><creator>Griffin, Paul P</creator><creator>Ries, William L</creator><general>Massachusetts Medical Society</general><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>PHGZM</scope><scope>PHGZT</scope><scope>PJZUB</scope><scope>PKEHL</scope><scope>PPXIY</scope><scope>PQEST</scope><scope>PQGLB</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>PSYQQ</scope><scope>Q9U</scope><scope>7X8</scope></search><sort><creationdate>19950615</creationdate><title>Long-Term Treatment of Osteopetrosis with Recombinant Human Interferon Gamma</title><author>Key, L. Lyndon ; Rodriguiz, Ramona Marie ; Willi, Steven M ; Wright, Nancy M ; Hatcher, Heather C ; Eyre, David R ; Cure, Joel K ; Griffin, Paul P ; Ries, William L</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c527t-479ecbb1ac0aa1f45b52cacb635177deb093ad2ba9c00b60864f3628cc9c1bb03</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>1995</creationdate><topic>Adult</topic><topic>Biochemical markers</topic><topic>Biopsy</topic><topic>Body weight</topic><topic>Bone imaging</topic><topic>Bone marrow</topic><topic>Bone Resorption</topic><topic>Bone turnover</topic><topic>Cancellous bone</topic><topic>Child, Preschool</topic><topic>Drug dosages</topic><topic>Endocrinology</topic><topic>Hemoglobin</topic><topic>Humans</topic><topic>Infections</topic><topic>Injections, Subcutaneous</topic><topic>Interferon</topic><topic>Interferon-gamma - administration & dosage</topic><topic>Interferon-gamma - therapeutic use</topic><topic>Leukocytes (granulocytic)</topic><topic>Macrophages</topic><topic>Orthopedics</topic><topic>Osteoclasts</topic><topic>Osteopetrosis</topic><topic>Osteopetrosis - congenital</topic><topic>Osteopetrosis - pathology</topic><topic>Osteopetrosis - therapy</topic><topic>Patients</topic><topic>Pediatrics</topic><topic>Recombinant Proteins - administration & dosage</topic><topic>Recombinant Proteins - therapeutic use</topic><topic>Sulfur</topic><topic>Superoxide</topic><topic>Transplants & implants</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Key, L. 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Lyndon</au><au>Rodriguiz, Ramona Marie</au><au>Willi, Steven M</au><au>Wright, Nancy M</au><au>Hatcher, Heather C</au><au>Eyre, David R</au><au>Cure, Joel K</au><au>Griffin, Paul P</au><au>Ries, William L</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Long-Term Treatment of Osteopetrosis with Recombinant Human Interferon Gamma</atitle><jtitle>The New England journal of medicine</jtitle><addtitle>N Engl J Med</addtitle><date>1995-06-15</date><risdate>1995</risdate><volume>332</volume><issue>24</issue><spage>1594</spage><epage>1599</epage><pages>1594-1599</pages><issn>0028-4793</issn><eissn>1533-4406</eissn><coden>NEJMAG</coden><abstract>Congenital osteopetrosis is a group of disorders resulting in decreased osteoclastic function and hence decreased bone resorption. The accumulation of sclerotic bone compromises marrow space and cranial-nerve foramina and predisposes patients to pathologic fractures. Most patients become blind or anemic before six months of age and die because of poor resistance to infection, neurologic deficits, or bone marrow failure during the first decade — often the first two years — of life.
1
–
4
Bone marrow transplantation is curative therapy,
5
but an acceptable donor can be found for only about 40 percent of patients. Overall survival 23 months after transplantation was . . .</abstract><cop>United States</cop><pub>Massachusetts Medical Society</pub><pmid>7753137</pmid><doi>10.1056/NEJM199506153322402</doi><tpages>6</tpages><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0028-4793 |
| ispartof | The New England journal of medicine, 1995-06, Vol.332 (24), p.1594-1599 |
| issn | 0028-4793 1533-4406 |
| language | eng |
| recordid | cdi_proquest_miscellaneous_77276611 |
| source | MEDLINE; 1990; EZB Electronic Journals Library |
| subjects | Adult Biochemical markers Biopsy Body weight Bone imaging Bone marrow Bone Resorption Bone turnover Cancellous bone Child, Preschool Drug dosages Endocrinology Hemoglobin Humans Infections Injections, Subcutaneous Interferon Interferon-gamma - administration & dosage Interferon-gamma - therapeutic use Leukocytes (granulocytic) Macrophages Orthopedics Osteoclasts Osteopetrosis Osteopetrosis - congenital Osteopetrosis - pathology Osteopetrosis - therapy Patients Pediatrics Recombinant Proteins - administration & dosage Recombinant Proteins - therapeutic use Sulfur Superoxide Transplants & implants |
| title | Long-Term Treatment of Osteopetrosis with Recombinant Human Interferon Gamma |
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