{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T12:14:08Z","timestamp":1777637648408,"version":"3.51.4"},"reference-count":16,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2008,11,1]],"date-time":"2008-11-01T00:00:00Z","timestamp":1225497600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. pediatr. gastroenterol. nutr."],"published-print":{"date-parts":[[2008,11]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Objectives:<\/jats:title><jats:p>To investigate the effect of ursodeoxycholic acid (UDCA) in children with liver disease associated with ZZ \u03b1<jats:sub>1<\/jats:sub>\u2010antitrypsin (AAT) deficiency.<\/jats:p><\/jats:sec><jats:sec><jats:title>Patients and Methods:<\/jats:title><jats:p>A total of 42 affected children received UDCA (30 mg \u00b7 kg<jats:sup>\u22121<\/jats:sup> \u00b7 day<jats:sup>\u22121<\/jats:sup>) and underwent clinical and biochemical follow\u2010up at least yearly.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results:<\/jats:title><jats:p>In group 1, 22 children whose mean initial \u03b3\u2010glutamyl\u2010transpeptidase (GGT) was 7.4 \u00d7 N normalized serum liver test results after a mean treatment of 2.6 years. In 16 of these children, UDCA was discontinued. Relapse was observed in 11 children, and liver test results returned to normal after UDCA resumption. In the other 5 children, liver test results remained normal during a mean period of 2.5 years. In group 2, 11 children (mean initial GGT 12.8 \u00d7 N) had improved liver test results after a mean treatment of 2.3 years. In group 3, 9 children (mean initial GGT 33.8 \u00d7 N) had no liver test result improvement and evolution toward cirrhosis, requiring liver transplantation in 7. Most of the children in group 1 had normal results of clinical examination after UDCA treatment, versus none in group 3 (<jats:italic>P<\/jats:italic> \u2264 0.00001). Initial GGT (<jats:italic>P<\/jats:italic> \u2264 0.002) and total bilirubin (<jats:italic>P<\/jats:italic> \u2264 0.05) levels were significantly lower in group 1 than in group 3. Combined initial values of GGT \u22645.5 \u00d7 N and total bilirubin \u226466 \u03bcmol\/L were associated with normalization of liver test results in 90% of children.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions:<\/jats:title><jats:p>UDCA may significantly improve clinical status and liver test results in some children with liver disease associated with ZZ AAT deficiency. No beneficial effect of UDCA was shown in children with the most severe liver involvement. Initial levels of GGT and total bilirubin may be of prognostic value for therapy effectiveness.<\/jats:p><\/jats:sec>","DOI":"10.1097\/mpg.0b013e31817b6dfb","type":"journal-article","created":{"date-parts":[[2010,2,25]],"date-time":"2010-02-25T01:42:44Z","timestamp":1267062164000},"page":"623-629","source":"Crossref","is-referenced-by-count":35,"title":["Liver Disease Associated with ZZ \u03b11\u2010Antitrypsin Deficiency and Ursodeoxycholic Acid Therapy in Children"],"prefix":"10.1002","volume":"47","author":[{"given":"Panayotis","family":"Lykavieris","sequence":"first","affiliation":[{"name":"Pediatric Hepatology and National Reference Centre for Biliary Atresia France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"B\u00e9atrice","family":"Ducot","sequence":"additional","affiliation":[{"name":"INSERM U 822 \u00abEpidemiology Demography and Social Sciences\u00bb H\u00f4pital Bic\u00eatre Universit\u00e9 Paris\u2010Sud 11 Facult\u00e9 de M\u00e9decine Paris\u2010Sud Assistance Publique\u2010H\u00f4pitaux de Paris Le Kremlin Bic\u00eatre France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alain","family":"Lachaux","sequence":"additional","affiliation":[{"name":"Pediatric Gastroenterology H\u00f4pital Edouard Herriot Lyon France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alain","family":"Dabadie","sequence":"additional","affiliation":[{"name":"Pediatric Department H\u00f4pital Sud Rennes France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pierre","family":"Brou\u00e9","sequence":"additional","affiliation":[{"name":"Pediatric Department H\u00f4pital des Enfants Toulouse France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jacques","family":"Sarles","sequence":"additional","affiliation":[{"name":"Pediatric Department H\u00f4pital des Enfants de la Timone Marseille France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Olivier","family":"Bernard","sequence":"additional","affiliation":[{"name":"Pediatric Hepatology and National Reference Centre for Biliary Atresia France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Emmanuel","family":"Jacquemin","sequence":"additional","affiliation":[{"name":"Pediatric Hepatology and National Reference Centre for Biliary Atresia France"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2008,11]]},"reference":[{"key":"e_1_2_5_2_1","doi-asserted-by":"publisher","DOI":"10.1136\/adc.85.1.2"},{"key":"e_1_2_5_3_1","doi-asserted-by":"publisher","DOI":"10.1055\/s-2007-1007158"},{"key":"e_1_2_5_4_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra010772"},{"key":"e_1_2_5_5_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM197606102942404"},{"key":"e_1_2_5_6_1","doi-asserted-by":"publisher","DOI":"10.1002\/hep.1840220221"},{"key":"e_1_2_5_7_1","doi-asserted-by":"publisher","DOI":"10.1378\/chest.128.4.2076"},{"key":"e_1_2_5_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0168-8278(05)80469-6"},{"key":"e_1_2_5_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0168-8278(00)80103-8"},{"key":"e_1_2_5_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0022-3476(83)80230-3"},{"key":"e_1_2_5_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/hep.21628"},{"key":"e_1_2_5_12_1","doi-asserted-by":"publisher","DOI":"10.1097\/00005176-199705000-00016"},{"key":"e_1_2_5_13_1","doi-asserted-by":"publisher","DOI":"10.1002\/hep.510250303"},{"key":"e_1_2_5_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1089-3261(03)00135-1"},{"key":"e_1_2_5_15_1","doi-asserted-by":"publisher","DOI":"10.1136\/adc.58.11.882"},{"key":"e_1_2_5_16_1","doi-asserted-by":"publisher","DOI":"10.1053\/gast.2001.23984"},{"key":"e_1_2_5_17_1","doi-asserted-by":"publisher","DOI":"10.1002\/hep.21689"}],"container-title":["Journal of Pediatric Gastroenterology and Nutrition"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1097\/MPG.0b013e31817b6dfb","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,1,18]],"date-time":"2024-01-18T08:30:28Z","timestamp":1705566628000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1097\/MPG.0b013e31817b6dfb"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2008,11]]},"references-count":16,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2008,11]]}},"alternative-id":["10.1097\/MPG.0b013e31817b6dfb"],"URL":"https:\/\/doi.org\/10.1097\/mpg.0b013e31817b6dfb","archive":["Portico"],"relation":{},"ISSN":["0277-2116","1536-4801"],"issn-type":[{"value":"0277-2116","type":"print"},{"value":"1536-4801","type":"electronic"}],"subject":[],"published":{"date-parts":[[2008,11]]}}}