Hepatocyte Transplantation Followed by Auxiliary Liver Transplantation—a Novel Treatment for Ornithine Transcarbamylase Deficiency
We report the first successful use of hepatocyte transplantation as a bridge to subsequent auxiliary partial orthotopic liver transplantation (APOLT) in a child antenatally diagnosed with severe ornithine transcarbamylase (OTC) deficiency. A total of 1.74 × 109 fresh and cryopreserved hepatocytes we...
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| Veröffentlicht in: | American journal of transplantation 2008-02, Vol.8 (2), p.452-457 |
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| container_title | American journal of transplantation |
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| creator | Puppi, J. Tan, N. Mitry, R. R. Hughes, R. D. Lehec, S. Mieli‐Vergani, G. Karani, J. Champion, M. P. Heaton, N. Mohamed, R. Dhawan, A. |
| description | We report the first successful use of hepatocyte transplantation as a bridge to subsequent auxiliary partial orthotopic liver transplantation (APOLT) in a child antenatally diagnosed with severe ornithine transcarbamylase (OTC) deficiency. A total of 1.74 × 109 fresh and cryopreserved hepatocytes were administered intraportally into the liver over a period of 6 months. Immunosuppression was with tacrolimus and prednisolone. A sustained decrease in ammonia levels and a gradual increase in serum urea were observed except during episodes of sepsis in the first 6 months of life. The patient was able to tolerate a normal protein intake and presented a normal growth and neurological development. APOLT was successfully performed at 7 months of age. We conclude that hepatocyte transplantation can be used in conjunction with APOLT as an effective treatment for severe OTC‐deficient patients, improving neurodevelopmental outcomes.
This child with antenatally diagnosed severe ornithine transcarbamylase deficiency was successfully bridged to auxiliary partial orthotopic liver transplantation by hepatocyte transplantation. |
| doi_str_mv | 10.1111/j.1600-6143.2007.02058.x |
| format | Article |
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This child with antenatally diagnosed severe ornithine transcarbamylase deficiency was successfully bridged to auxiliary partial orthotopic liver transplantation by hepatocyte transplantation.</description><identifier>ISSN: 1600-6135</identifier><identifier>EISSN: 1600-6143</identifier><identifier>DOI: 10.1111/j.1600-6143.2007.02058.x</identifier><identifier>PMID: 18211511</identifier><language>eng</language><publisher>Malden, USA: Blackwell Publishing Inc</publisher><subject>Adolescent ; APOLT ; auxiliary liver transplantation ; Biological and medical sciences ; Female ; hepatocyte transplantation ; Hepatocytes - transplantation ; Humans ; Infant, Newborn ; Liver Transplantation ; Liver, biliary tract, pancreas, portal circulation, spleen ; Male ; Medical sciences ; metabolic disorders ; Ornithine Carbamoyltransferase Deficiency Disease - surgery ; Ornithine Carbamoyltransferase Deficiency Disease - therapy ; Pregnancy ; Prenatal Diagnosis ; Surgery (general aspects). Transplantations, organ and tissue grafts. Graft diseases ; Surgery of the digestive system ; Treatment Outcome ; urea cycle</subject><ispartof>American journal of transplantation, 2008-02, Vol.8 (2), p.452-457</ispartof><rights>2008 INIST-CNRS</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c5468-4c1746254591d92fb931b166cbdd73f16b7f48b03db2a69c3faf0ee373f4ab443</citedby><cites>FETCH-LOGICAL-c5468-4c1746254591d92fb931b166cbdd73f16b7f48b03db2a69c3faf0ee373f4ab443</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1111%2Fj.1600-6143.2007.02058.x$$EPDF$$P50$$Gwiley$$H</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1111%2Fj.1600-6143.2007.02058.x$$EHTML$$P50$$Gwiley$$H</linktohtml><link.rule.ids>314,776,780,1411,27901,27902,45550,45551</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=20106923$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/18211511$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Puppi, J.</creatorcontrib><creatorcontrib>Tan, N.</creatorcontrib><creatorcontrib>Mitry, R. R.</creatorcontrib><creatorcontrib>Hughes, R. D.</creatorcontrib><creatorcontrib>Lehec, S.</creatorcontrib><creatorcontrib>Mieli‐Vergani, G.</creatorcontrib><creatorcontrib>Karani, J.</creatorcontrib><creatorcontrib>Champion, M. P.</creatorcontrib><creatorcontrib>Heaton, N.</creatorcontrib><creatorcontrib>Mohamed, R.</creatorcontrib><creatorcontrib>Dhawan, A.</creatorcontrib><title>Hepatocyte Transplantation Followed by Auxiliary Liver Transplantation—a Novel Treatment for Ornithine Transcarbamylase Deficiency</title><title>American journal of transplantation</title><addtitle>Am J Transplant</addtitle><description>We report the first successful use of hepatocyte transplantation as a bridge to subsequent auxiliary partial orthotopic liver transplantation (APOLT) in a child antenatally diagnosed with severe ornithine transcarbamylase (OTC) deficiency. A total of 1.74 × 109 fresh and cryopreserved hepatocytes were administered intraportally into the liver over a period of 6 months. Immunosuppression was with tacrolimus and prednisolone. A sustained decrease in ammonia levels and a gradual increase in serum urea were observed except during episodes of sepsis in the first 6 months of life. The patient was able to tolerate a normal protein intake and presented a normal growth and neurological development. APOLT was successfully performed at 7 months of age. We conclude that hepatocyte transplantation can be used in conjunction with APOLT as an effective treatment for severe OTC‐deficient patients, improving neurodevelopmental outcomes.
This child with antenatally diagnosed severe ornithine transcarbamylase deficiency was successfully bridged to auxiliary partial orthotopic liver transplantation by hepatocyte transplantation.</description><subject>Adolescent</subject><subject>APOLT</subject><subject>auxiliary liver transplantation</subject><subject>Biological and medical sciences</subject><subject>Female</subject><subject>hepatocyte transplantation</subject><subject>Hepatocytes - transplantation</subject><subject>Humans</subject><subject>Infant, Newborn</subject><subject>Liver Transplantation</subject><subject>Liver, biliary tract, pancreas, portal circulation, spleen</subject><subject>Male</subject><subject>Medical sciences</subject><subject>metabolic disorders</subject><subject>Ornithine Carbamoyltransferase Deficiency Disease - surgery</subject><subject>Ornithine Carbamoyltransferase Deficiency Disease - therapy</subject><subject>Pregnancy</subject><subject>Prenatal Diagnosis</subject><subject>Surgery (general aspects). Transplantations, organ and tissue grafts. 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R.</creator><creator>Hughes, R. D.</creator><creator>Lehec, S.</creator><creator>Mieli‐Vergani, G.</creator><creator>Karani, J.</creator><creator>Champion, M. P.</creator><creator>Heaton, N.</creator><creator>Mohamed, R.</creator><creator>Dhawan, A.</creator><general>Blackwell Publishing Inc</general><general>Blackwell</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>7T5</scope><scope>H94</scope><scope>7X8</scope></search><sort><creationdate>200802</creationdate><title>Hepatocyte Transplantation Followed by Auxiliary Liver Transplantation—a Novel Treatment for Ornithine Transcarbamylase Deficiency</title><author>Puppi, J. ; Tan, N. ; Mitry, R. R. ; Hughes, R. D. ; Lehec, S. ; Mieli‐Vergani, G. ; Karani, J. ; Champion, M. 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P.</au><au>Heaton, N.</au><au>Mohamed, R.</au><au>Dhawan, A.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Hepatocyte Transplantation Followed by Auxiliary Liver Transplantation—a Novel Treatment for Ornithine Transcarbamylase Deficiency</atitle><jtitle>American journal of transplantation</jtitle><addtitle>Am J Transplant</addtitle><date>2008-02</date><risdate>2008</risdate><volume>8</volume><issue>2</issue><spage>452</spage><epage>457</epage><pages>452-457</pages><issn>1600-6135</issn><eissn>1600-6143</eissn><abstract>We report the first successful use of hepatocyte transplantation as a bridge to subsequent auxiliary partial orthotopic liver transplantation (APOLT) in a child antenatally diagnosed with severe ornithine transcarbamylase (OTC) deficiency. A total of 1.74 × 109 fresh and cryopreserved hepatocytes were administered intraportally into the liver over a period of 6 months. Immunosuppression was with tacrolimus and prednisolone. A sustained decrease in ammonia levels and a gradual increase in serum urea were observed except during episodes of sepsis in the first 6 months of life. The patient was able to tolerate a normal protein intake and presented a normal growth and neurological development. APOLT was successfully performed at 7 months of age. We conclude that hepatocyte transplantation can be used in conjunction with APOLT as an effective treatment for severe OTC‐deficient patients, improving neurodevelopmental outcomes.
This child with antenatally diagnosed severe ornithine transcarbamylase deficiency was successfully bridged to auxiliary partial orthotopic liver transplantation by hepatocyte transplantation.</abstract><cop>Malden, USA</cop><pub>Blackwell Publishing Inc</pub><pmid>18211511</pmid><doi>10.1111/j.1600-6143.2007.02058.x</doi><tpages>6</tpages><oa>free_for_read</oa></addata></record> |
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| ispartof | American journal of transplantation, 2008-02, Vol.8 (2), p.452-457 |
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| source | MEDLINE; Wiley Online Library Journals Frontfile Complete; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals; Alma/SFX Local Collection |
| subjects | Adolescent APOLT auxiliary liver transplantation Biological and medical sciences Female hepatocyte transplantation Hepatocytes - transplantation Humans Infant, Newborn Liver Transplantation Liver, biliary tract, pancreas, portal circulation, spleen Male Medical sciences metabolic disorders Ornithine Carbamoyltransferase Deficiency Disease - surgery Ornithine Carbamoyltransferase Deficiency Disease - therapy Pregnancy Prenatal Diagnosis Surgery (general aspects). Transplantations, organ and tissue grafts. Graft diseases Surgery of the digestive system Treatment Outcome urea cycle |
| title | Hepatocyte Transplantation Followed by Auxiliary Liver Transplantation—a Novel Treatment for Ornithine Transcarbamylase Deficiency |
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