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1. Systemic T-cell activation and IFN-γ activity in indeterminate severe hepatitis are reminiscent of hemophagocytic lymphohistiocytosis: Implications for T-cell– and IFN-γ–directed therapies

Author

Nguyen, Thinh H., Satwani, Prakash, Kumar, Deepak, Kapoor, Urvi, Malik, Sakshi, Prince, Chengyu, Montminy, Taylor, Smiley, Kristi, Martinez, Mercedes, Goldner, Dana, Marsh, Rebecca, Remotti, Helen E., Fazlollahi, Ladan, Rytting, Heather B., Romero, Rene, and Chandrakasan, Shanmuganathan

Published
2024
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4. Case 28-2024: A 75-Year-Old Woman with Edema, Arthritis, and Proteinuria.

Author

Jonas, Beth L., Romero, Rene Balza, Criscione-Schreiber, Lisa G., Cheung, Pui W., and Trivin-Avillach, Claire

Subjects
*BRAIN natriuretic factor, *MEDICAL societies, *LUPUS nephritis, *SJOGREN'S syndrome, *FINGER joint, *JOINTS (Anatomy)
Abstract

This article from the New England Journal of Medicine presents a case study of a 75-year-old woman with multiple medical conditions. The article explores the underlying cause of her symptoms and provides insights into her diagnosis and treatment. The patient's history and laboratory test results suggest the possibility of various conditions, including autoimmune disease, infectious disease, and cancer. The text discusses the importance of understanding the cause of the patient's hypocomplementemia and presents management options for her specific conditions, including AA amyloidosis and lupus nephritis. The challenge lies in finding effective and safe treatment options for her overlapping conditions of rheumatoid arthritis and systemic lupus erythematosus. [Extracted from the article]

Published
2024
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6. Longitudinal Outcomes in Young Patients with Alpha-1-Antitrypsin Deficiency with Native Liver Reveal that Neonatal Cholestasis is a Poor Predictor of Future Portal Hypertension

Author

Alonso, Estella, Bass, Lee, Kelly, Susan, Riordan, Mary, Melin-Aldana, Hector, Bezerra, Jorge, Bove, Kevin, Heubi, James, Miethke, Alexander, Tiao, Greg, Denlinger, Julie, Chapman, Erin, Sokol, Ronald, Feldman, Amy, Mack, Cara, Narkewicz, Michael, Suchy, Frederick, Sundaram, Shikha, Van Hove, Johan, Garcia, Benigno, Kauma, Mikaela, Kocher, Kendra, Steinbeiss, Matthew, Lovell, Mark, Loomes, Kathleen, Piccoli, David, Rand, Elizabeth, Russo, Pierre, Spinner, Nancy, Erlichman, Jessi, Stalford, Samantha, Pakstis, Dina, King, Sakya, Squires, Robert, Sindhi, Rakesh, Venkat, Veena, Bukauskas, Kathy, McKiernan, Patrick, Haberstroh, Lori, Squires, James, Rosenthal, Philip, Bull, Laura, Curry, Joanna, Langlois, Camille, Kim, Grace, Teckman, Jeffery, Kociela, Vikki, Nagy, Rosemary, Patel, Shraddha, Cerkoski, Jacqueline, Molleston, Jean P., Bozic, Molly, Subbarao, Girish, Klipsch, Ann, Sawyers, Cindy, Cummings, Oscar, Horslen, Simon, Murray, Karen, Hsu, Evelyn, Cooper, Kara, Young, Melissa, Finn, Laura, Kamath, Binita, Ng, Vicky, Quammie, Claudia, Putra, Juan, Sharma, Deepika, Parmar, Aishwarya, Guthery, Stephen, Jensen, Kyle, Rutherford, Ann, Lowichik, Amy, Book, Linda, Meyers, Rebecka, Hall, Tyler, Wang, Kasper, Michail, Sonia, Thomas, Danny, Goodhue, Catherine, Kohli, Rohit, Wang, Larry, Soufi, Nisreen, Thomas, Daniel, Karpen, Saul, Gupta, Nitika, Romero, Rene, Jr., Vos, Miriam B., Tory, Rita, Berauer, John-Paul, Abramowsky, Carlos, McFall, Jeanette, Shneider, Benjamin, Harpavat, Sanjiv, Hertel, Paula, Leung, Daniel, Tessier, Mary, Schady, Deborah, Cavallo, Laurel, Olvera, Diego, Banks, Christina, Tsai, Cynthia, Thompson, Richard, Doo, Edward, Hoofnagle, Jay, Sherker, Averell, Torrance, Rebecca, Hall, Sherry, Magee, John, Merion, Robert, Spino, Cathie, Ye, Wen, Teckman, Jeffrey, Hawthorne, Kieran, Bass, Lee M., Murray, Karen F., Kerkar, Nanda, Magee, John C., Heubi, James E., Squires, Robert H., Kamath, Binita M., Guthery, Stephen L., Loomes, Kathleen M., Sherker, Averell H., and Sokol, Ronald J.

Published
2020
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7. Changes in energy expenditure and physical activity over 15 years of environmental changes: The Maycoba project.

Author

Niclou, Alexandra, Esparza‐Romero, Julián, Urquidez‐Romero, Rene, Lam, Yan Y., Rood, Jennifer, Schulz, Leslie O., Bennett, Peter H., Valencia, Mauro E., and Ravussin, Eric

Subjects
PHYSICAL activity, BODY composition, ADIPOSE tissues, WEIGHT gain, BODY weight
Abstract

Objective: This work aimed to parse out the role of changing environments on body composition, total energy expenditure, and physical activity in the Mexican Pima, a population experiencing rapid industrialization. Methods: Using doubly labeled water, we compared energy expenditure and physical activity in a longitudinal cohort of Mexican Pima (n = 26; female: 12) in 1995 and 2010. Body mass and composition were assessed by bioimpedance analysis. To determine the effects of environmental factors on body weight independent of age, we compared the 1995 longitudinal cohort with an age‐ and sex‐matched cross‐sectional cohort (n = 26) in 2010. Results: Body mass, fat mass, and fat‐free mass all significantly increased between 1995 and 2010. Despite a 13% average increase in body weight, weight‐adjusted total daily energy expenditure decreased significantly. Measured physical activity levels also decreased between 1995 and 2010, after we adjusted for weight. Conclusions: Our results suggest that the recent industrialization of the Maycoba region in Sonora, Mexico, has contributed to a decrease in physical activity, in turn contributing to weight gain and metabolic disease among the Mexican Pima. [ABSTRACT FROM AUTHOR]

Published
2024
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8. Sofosbuvir–velpatasvir in children 3–17 years old with hepatitis C virus infection.

Author

Jonas, Maureen M., Romero, Rene, Rosenthal, Philip, Lin, Chuan‐Hao, Verucchi, Gabriella, Wen, Jessica, Balistreri, William F., Whitworth, Suzanne, Bansal, Sanjay, Leung, Daniel H., Narkewicz, Michael R., Gonzalez‐Peralta, Regino P., Mangia, Alessandra, Karnsakul, Wikrom, Rao, Girish S., Shao, Jiang, de Jong, Jan, Parhy, Bandita, Osinusi, Anu, and Kersey, Kathryn

Published
2024
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9. Quality of Life and Its Determinants in a Multicenter Cohort of Children with Alagille Syndrome

Author

Kamath, Binita M, Chen, Zhen, Romero, Rene, Fredericks, Emily M, Alonso, Estella M, Arnon, Ronen, Heubi, James, Hertel, Paula M, Karpen, Saul J, Loomes, Kathleen M, Murray, Karen F, Rosenthal, Philip, Schwarz, Kathleen B, Subbarao, Girish, Teckman, Jeffrey H, Turmelle, Yumirle P, Wang, Kasper S, Sherker, Averell H, Sokol, Ronald J, Magee, John C, and Network, Childhood Liver Disease Research

Subjects
Clinical Research, Liver Disease, Pediatric, Digestive Diseases, Chronic Liver Disease and Cirrhosis, Adolescent, Alagille Syndrome, Case-Control Studies, Child, Child, Preschool, Cohort Studies, Emotions, Female, Health Status, Humans, Male, Quality of Life, Social Behavior, Surveys and Questionnaires, alpha 1-Antitrypsin Deficiency, Childhood Liver Disease Research Network, Human Movement and Sports Sciences, Paediatrics and Reproductive Medicine, Pediatrics
Abstract

ObjectivesTo assess health-related quality of life (HRQOL) in children with Alagille syndrome (ALGS) in comparison with healthy and other liver disease cohorts, and to identify determinants of HRQOL in patients with ALGS.Study designWithin the Childhood Liver Disease Research Network prospective study of cholestasis, Pediatric Quality of Life Inventory (PedsQL) questionnaires were administered to 70 children with ALGS, 95 children with alpha-1-antitrypsin deficiency (A1ATD), and 49 children with other causes of chronic intrahepatic cholestasis (IHC) aged 5-18 years. Parent proxy PedsQL scores were recorded for children aged 2-18 years (98 ALGS, 123 A1ATD, and 68 IHC).ResultsMean ages and total bilirubin (mg/dL) were ALGS 9.4 years; 4.4, A1ATD 9.5 years; 0.7, and IHC 10.3 years; 2.9. ALGS child PedsQL scores were lower than in healthy children and children with A1ATD (mean 73 vs 83; P = .001). Children with ALGS and IHC were similar, except in physical scores (73 vs 79; P = .05). Parents of children with ALGS perceived their children to have worse HRQOL than A1ATD (P ≤ .001) and marginally lower compared with IHC. Univariate analysis revealed ALGS child-reported scores were positively associated with better growth and inversely with total bilirubin. Growth failure, elevated international normalized ratio, and an intracardiac defect were predictive of poor parental scores (P ≤ .05). In multivariate analysis, only weight z-score remained significant for child- and parent-reported scores.ConclusionsHRQOL is impaired in children with ALGS compared with healthy and children with A1ATD, similar to children with IHC and is associated with growth failure, which is a potentially treatable cause of impaired HRQOL.

Published
2015

10. Baseline Analysis of a Young α‐1‐Antitrypsin Deficiency Liver Disease Cohort Reveals Frequent Portal Hypertension

Author

Teckman, Jeffrey H, Rosenthal, Philip, Abel, Robert, Bass, Lee M, Michail, Sonia, Murray, Karen F, Rudnick, David A, Thomas, Daniel W, Spino, Cathie, Arnon, Ronen, Hertel, Paula M, Heubi, James, Kamath, Binita M, Karnsakul, Wikrom, Loomes, Kathleen M, Magee, John C, Molleston, Jean P, Romero, Rene, Shneider, Benjamin L, Sherker, Averell H, Sokol, Ronald J, and Network, Childhood Liver Disease Research

Subjects
Biomedical and Clinical Sciences, Clinical Sciences, Clinical Research, Digestive Diseases, Liver Disease, Rare Diseases, Chronic Liver Disease and Cirrhosis, Pediatric, Aetiology, 2.1 Biological and endogenous factors, Oral and gastrointestinal, Adolescent, Adult, Child, Child, Preschool, Cohort Studies, Female, Humans, Hypertension, Portal, Infant, Infant, Newborn, Jaundice, Liver, Longitudinal Studies, Male, Prospective Studies, Young Adult, alpha 1-Antitrypsin, alpha 1-Antitrypsin Deficiency, cirrhosis, jaundice, liver enzymes, liver transplant, metabolic liver disease, Childhood Liver Disease Research Network, Medical and Health Sciences, Gastroenterology & Hepatology, Clinical sciences, Nutrition and dietetics, Paediatrics
Abstract

Objectivesα-1-Antitrypsin (A1AT) deficiency is a common genetic disease with an unpredictable and highly variable course. The Childhood Liver Disease Research and Education Network is a National Institutes of Health, multicenter, longitudinal consortium studying pediatric liver diseases, with the objective of prospectively defining natural history and identifying disease modifiers.MethodsLongitudinal, cohort study of A1AT patients' birth through 25 years diagnosed as having liver disease, type PIZZ or PISZ. Medical history, physical examination, laboratory, imaging, and standardized survey tool data were collected during the provision of standard of care.ResultsIn the present report of the cohort at baseline, 269 subjects were enrolled between November 2008 and October 2012 (208 with their native livers and 61 postliver transplant). Subjects with mild disease (native livers and no portal hypertension [PHT]) compared to severe disease (with PHT or postliver transplant) were not different in age at presentation. A total of 57% of subjects with mild disease and 76% with severe disease were jaundiced at presentation (P = 0.0024). A total of 29% of subjects with native livers had PHT, but age at diagnosis and growth were not different between the no-PHT and PHT groups (P > 0.05). Subjects with native livers and PHT were more likely to have elevated bilirubin, ALT, AST, INR, and GGTP than the no-PHT group (P <

Published
2015

12. Medical Status of 219 Children with Biliary Atresia Surviving Long-Term with Their Native Livers: Results from a North American Multicenter Consortium

Author

Ng, Vicky Lee, Haber, Barbara H, Magee, John C, Miethke, Alexander, Murray, Karen F, Michail, Sonia, Karpen, Saul J, Kerkar, Nanda, Molleston, Jean P, Romero, Rene, Rosenthal, Philip, Schwarz, Kathleen B, Shneider, Benjamin L, Turmelle, Yumirle P, Alonso, Estella M, Sherker, Averell H, Sokol, Ronald J, and Network, Childhood Liver Disease Research and Education

Subjects
Biomedical and Clinical Sciences, Clinical Sciences, Rare Diseases, Clinical Research, Liver Disease, Pediatric, Digestive Diseases, Chronic Liver Disease and Cirrhosis, Perinatal Period - Conditions Originating in Perinatal Period, Oral and gastrointestinal, Good Health and Well Being, Biliary Atresia, Canada, Child, Enterostomy, Female, Health Status, Humans, Liver, Male, Quality of Life, Survivors, Time Factors, United States, Childhood Liver Disease Research and Education Network, Human Movement and Sports Sciences, Paediatrics and Reproductive Medicine, Pediatrics, Paediatrics
Abstract

ObjectivesTo examine the medical status of children with biliary atresia (BA) with their native livers after hepato- portoenterostomy (HPE) surgery.Study designThe Childhood Liver Disease Research and Education Network database was utilized to examine subjects with BA living with their native livers 5 or more years after HPE and to describe the prevalence of subjects with BA with an "ideal" outcome, defined as no clinical evidence of chronic liver disease, normal liver biochemical indices (aspartate aminotransferase, alanine aminotransferase, γ-glutamyl transpeptidase, platelet count, total bilirubin, international normalized ratio, and albumin), and normal health-related quality of life 5 or more years after HPE.ResultsChildren with BA (n = 219; 43% male) with median age 9.7 years were studied. Median age at HPE was 56 (range 7-125) days. Median age- and sex-adjusted height and weight z-scores at 5-year follow-up were 0.487 (IQR -0.27 to 1.02) and 0.00 (IQR -0.74 to 0.70), respectively. During the 12 preceding months, cholangitis and bone fractures occurred in 17% and 5.5%, respectively. Health-related quality of life was reported normal by 53% of patients. However, only 1.8% met the study definition of "ideal" outcome. Individual tests of liver synthetic function (total bilirubin, albumin, and international normalized ratio) were normal in 75%, 85%, and 73% of the study cohort.ConclusionCholangitis and fractures in long-term survivors underscore the importance of ongoing medical surveillance. Over 98% of this North American cohort of subjects with BA living with native livers 5 or more years after HPE have clinical or biochemical evidence of chronic liver disease.

Published
2014

13. Use of Corticosteroids After Hepatoportoenterostomy for Bile Drainage in Infants With Biliary Atresia: The START Randomized Clinical Trial

Author

Bezerra, Jorge A, Spino, Cathie, Magee, John C, Shneider, Benjamin L, Rosenthal, Philip, Wang, Kasper S, Erlichman, Jessi, Haber, Barbara, Hertel, Paula M, Karpen, Saul J, Kerkar, Nanda, Loomes, Kathleen M, Molleston, Jean P, Murray, Karen F, Romero, Rene, Schwarz, Kathleen B, Shepherd, Ross, Suchy, Frederick J, Turmelle, Yumirle P, Whitington, Peter F, Moore, Jeffrey, Sherker, Averell H, Robuck, Patricia R, and Sokol, Ronald J

Subjects
Biomedical and Clinical Sciences, Clinical Sciences, Clinical Trials and Supportive Activities, Digestive Diseases, Rare Diseases, Liver Disease, Patient Safety, Clinical Research, Pediatric, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, Oral and gastrointestinal, Administration, Oral, Adrenal Cortex Hormones, Biliary Atresia, Bilirubin, Double-Blind Method, Drainage, Female, Humans, Infant, Infusions, Intravenous, Male, Methylprednisolone, Portoenterostomy, Hepatic, Prednisolone, Survival Analysis, Treatment Outcome, Childhood Liver Disease Research and Education Network, Medical and Health Sciences, General & Internal Medicine, Biomedical and clinical sciences, Health sciences
Abstract

ImportanceBiliary atresia is the most common cause of end-stage liver disease in children. Controversy exists as to whether use of steroids after hepatoportoenterostomy improves clinical outcome.ObjectiveTo determine whether the addition of high-dose corticosteroids after hepatoportoenterostomy is superior to surgery alone in improving biliary drainage and survival with the native liver.Design, setting, and patientsThe multicenter, double-blind Steroids in Biliary Atresia Randomized Trial (START) was conducted in 140 infants (mean age, 2.3 months) between September 2005 and February 2011 in the United States; follow-up ended in January 2013.InterventionsParticipants were randomized to receive intravenous methylprednisolone (4 mg/kg/d for 2 weeks) and oral prednisolone (2 mg/kg/d for 2 weeks) followed by a tapering protocol for 9 weeks (n = 70) or placebo (n = 70) initiated within 72 hours of hepatoportoenterostomy.Main outcomes and measuresThe primary end point (powered to detect a 25% absolute treatment difference) was the percentage of participants with a serum total bilirubin level of less than 1.5 mg/dL with his/her native liver at 6 months posthepatoportoenterostomy. Secondary outcomes included survival with native liver at 24 months of age and serious adverse events.ResultsThe proportion of participants with improved bile drainage was not statistically significantly improved by steroids at 6 months posthepatoportoenterostomy (58.6% [41/70] of steroids group vs 48.6% [34/70] of placebo group; adjusted relative risk, 1.14 [95% CI, 0.83 to 1.57]; P = .43). The adjusted absolute risk difference was 8.7% (95% CI, -10.4% to 27.7%). Transplant-free survival was 58.7% in the steroids group vs 59.4% in the placebo group (adjusted hazard ratio, 1.0 [95% CI, 0.6 to 1.8]; P = .99) at 24 months of age. The percentage of participants with serious adverse events was 81.4% [57/70] of the steroids group and 80.0% [56/70] of the placebo group (P > .99); however, participants receiving steroids had an earlier time of onset of their first serious adverse event by 30 days posthepatoportoenterostomy (37.2% [95% CI, 26.9% to 50.0%] of steroids group vs 19.0% [95% CI, 11.5% to 30.4%] of placebo group; P = .008).Conclusions and relevanceAmong infants with biliary atresia who have undergone hepatoportoenterostomy, high-dose steroid therapy following surgery did not result in statistically significant treatment differences in bile drainage at 6 months, although a small clinical benefit could not be excluded. Steroid treatment was associated with earlier onset of serious adverse events in children with biliary atresia.Trial registrationclinicaltrials.gov Identifier: NCT00294684.

Published
2014

15. A Learning Collaborative Approach Increases Specificity of Diagnosis of Acute Liver Failure in Pediatric Patients

Author

Squires, Robert H., Bukauskas, Kathryn, Schulte, Madeline, Narkewicz, Michael R., Hite, Michelle, Loomes, Kathleen M., Rand, Elizabeth B., Piccoli, David, Kawchak, Deborah, Seidman, Christa, Romero, Rene, Karpen, Saul, de la Cruz-Tracy, Liezl, Ng, Vicky, Hunt, Kelsey, Subbarao, Girish C., Klipsch, Ann, Munson, Sarah, Alonso, Estella M., Sorenson, Lisa, Kelly, Susan, Neighbors, Katie, Rosenthal, Philip, Fleck, Shannon, Leonis, Mike A., Bucuvalas, John, Horning, Tracie, Rodriguez Baez, Norberto, Montanye, Shirley, Cowie, Margaret, Horslen, Simon P., Murray, Karen, Young, Melissa, Nielson, Heather, Klein, Jani, Rudnick, David A., Shepherd, Ross W., Harris, Kathy, Karpen, Saul J., De La Torre, Alejandro, Dell Olio, Dominic, Kelly, Deirdre, Lloyd, Carla, Lobritto, Steven J., Bakhsh, Sumerah, Jonas, Maureen, Elifoson, Scott A., Raza, Roshan, Schwarz, Kathleen B., Karnsakul, Wikrom W., Alford, Mary Kay, Dhawan, Anil, Fitzpatrick, Emer, Shneider, Benjamin L., Kerkar, Nanda N., Haydel, Brandy, Narayanappa, Sreevidya, Lopez, M. James, Shieck, Victoria, Doo, Edward, Sherker, Averell H., Belle, Steven H., Horslen, Simon, Hardison, Regina M., Rodriguez-Baez, Norberto, Ng, Vicky L., and Li, Ruosha

Published
2018
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16. Impact of Steroid Therapy on Early Growth in Infants with Biliary Atresia: The Multicenter Steroids in Biliary Atresia Randomized Trial

Author

Alonso, Estella M., Ye, Wen, Hawthorne, Kieran, Venkat, Veena, Loomes, Kathleen M., Mack, Cara L., Hertel, Paula M., Karpen, Saul J., Kerkar, Nanda, Molleston, Jean P., Murray, Karen F., Romero, Rene, Rosenthal, Philip, Schwarz, Kathleen B., Shneider, Benjamin L., Suchy, Frederick J., Turmelle, Yumirle P., Wang, Kasper S., Sherker, Averell H., Sokol, Ronald J., Bezerra, Jorge A., and Magee, John C.

Published
2018
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18. Health Related Quality of Life and Neurocognitive Outcomes in the First Year after Pediatric Acute Liver Failure

Author

Bukauskas, Kathryn, Schulte, Madeline, Narkewicz, Michael R., Hite, Michelle, Rand, Elizabeth B., Piccoli, David, Kawchak, Deborah, Seidman, Christa, Romero, Rene, Karpen, Saul, de la Cruz-Tracy, Liezl, Hunt, Kelsey, Subbarao, Girish C., Klipsch, Ann, Munson, Sarah, Kelly, Susan, Rosenthal, Philip J., Fleck, Shannon, Leonis, Mike A., Bucuvalas, John, Horning, Tracie, Baez, Norberto Rodriguez, Montanye, Shirley, Cowie, Margaret, Horslen, Simon P., Murray, Karen, Young, Melissa, Nielson, Heather, Klein, Jani, Rudnick, David A., Shepherd, Ross W., Harris, Kathy, Karpen, Saul J., De La Torre, Alejandro, Dell Olio, Dominic, Kelly, Deirdre, Lloyd, Carla, Lobritto, Steven J., Bakhsh, Sumerah, Jonas, Maureen, Elifoson, Scott A., Raza, Roshan, Schwarz, Kathleen B., Karnsakul, Wikrom W., Alford, Mary Kay, Dhawan, Anil, Fitzpatrick, Emer, Kerkar, Nanda N., Haydel, Brandy, Narayanappa, Sreevidya, Lopez, M. James, Shieck, Victoria, Sorensen, Lisa G., Neighbors, Katie, Hardison, Regina M., Loomes, Kathleen M., Varni, James W., Ng, Vicky L., Squires, Robert H., and Alonso, Estella M.

Published
2018
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19. Neurodevelopmental Outcome of Young Children with Biliary Atresia and Native Liver: Results from the ChiLDReN Study

Author

Hertel, Paula M., Harpavat, Sanjiv, Brandt, Mary L., Leung, Daniel H., Karnsakul, Wikrom, Torrance, Rebecca, Hall, Sherry, Doo, Edward, Hoofnagle, Jay H., Whitington, Peter, Bass, Lee, Miethke, Alexander G., Heubi, James E., Setchell, Kenneth, Bove, Kevin E., Tiao, Greg, Mack, Cara L., Narkewicz, Michael R., Feldman, Amy G., Sundaram, Shikha S., Suchy, Frederick J., Karrer, Frederick M., Lovell, Mark, Van Hove, Johan L., Rand, Elizabeth B., Squires, James E., Venkat, Veena L., Sindhi, Rakesh, Ranganathan, Sarangarajan, Bull, Laura, Teckman, Jeffrey, Bozic, Molly, Subbarao, Girish, Horslen, Simon, Hsu, Evelyn, Finn, Laura, Healey, Patrick, Kohli, Rohit, Thomas, Danny, Soufi, Nisreen, Michail, Sonia, Clifton, Matt, Gupta, Nitika, Romero, Rene, Vos, Miriam, Caltharp, Shelley, Kamath, Binita M., Ling, Simon C., Gold, Anna, Fecteau, Annie, Guthery, Stephen L., Jensen, Kyle, Meyers, Rebecka, Lowichik, Amy, Book, Linda, Merion, Robert M., Spino, Cathie, Jones, Karen, Ng, Vicky L., Sorensen, Lisa G., Alonso, Estella M., Fredericks, Emily M., Ye, Wen, Moore, Jeff, Karpen, Saul J., Shneider, Benjamin L., Molleston, Jean P., Bezerra, Jorge A., Murray, Karen F., Loomes, Kathleen M., Rosenthal, Philip, Squires, Robert H., Wang, Kasper, Arnon, Ronen, Schwarz, Kathleen B., Turmelle, Yumirle P., Haber, Barbara H., Sherker, Averell H., Magee, John C., and Sokol, Ronald J.

Published
2018
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20. Intravenous N‐acetylcysteine in pediatric patients with nonacetaminophen acute liver failure: A placebo‐controlled clinical trial

Author

Squires, Robert H, Dhawan, Anil, Alonso, Estella, Narkewicz, Michael R, Shneider, Benjamin L, Rodriguez‐Baez, Norberto, Olio, Dominic Dell, Karpen, Saul, Bucuvalas, John, Lobritto, Steven, Rand, Elizabeth, Rosenthal, Philip, Horslen, Simon, Ng, Vicky, Subbarao, Girish, Kerkar, Nanda, Rudnick, David, Lopez, M James, Schwarz, Kathleen, Romero, Rene, Elisofon, Scott, Doo, Edward, Robuck, Patricia R, Lawlor, Sharon, Belle, Steven H, and Group, for the Pediatric Acute Liver Failure Study

Subjects
Biomedical and Clinical Sciences, Clinical Sciences, Liver Disease, Digestive Diseases, Clinical Research, Pediatric, Clinical Trials and Supportive Activities, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, Oral and gastrointestinal, Acetylcysteine, Adolescent, Child, Child, Preschool, Double-Blind Method, Female, Free Radical Scavengers, Hepatic Encephalopathy, Humans, Infant, Infant, Newborn, Infusions, Intravenous, Liver Failure, Acute, Liver Transplantation, Male, Severity of Illness Index, Survival Rate, Treatment Outcome, Pediatric Acute Liver Failure Study Group, Medical Biochemistry and Metabolomics, Immunology, Gastroenterology & Hepatology, Clinical sciences
Abstract

UnlabelledN-acetylcysteine (NAC) was found to improve transplantation-free survival in only those adults with nonacetaminophen (non-APAP) acute liver failure (ALF) and grade 1-2 hepatic encephalopathy (HE). Because non-APAP ALF differs significantly between children and adults, the Pediatric Acute Liver Failure (PALF) Study Group evaluated NAC in non-APAP PALF. Children from birth through age 17 years with non-APAP ALF enrolled in the PALF registry were eligible to enter an adaptively allocated, doubly masked, placebo-controlled trial using a continuous intravenous infusion of NAC (150 mg/kg/day in 5% dextrose in water [D5W]) or placebo (D5W) for up to 7 days. The primary outcome was 1-year survival. Secondary outcomes included liver transplantation-free survival, liver transplantation (LTx), length of intensive care unit (ICU) and hospital stays, organ system failure, and maximum HE score. A total of 184 participants were enrolled in the trial with 92 in each arm. The 1-year survival did not differ significantly (P = 0.19) between the NAC (73%) and placebo (82%) treatment groups. The 1-year LTx-free survival was significantly lower (P = 0.03) in those who received NAC (35%) than those who received placebo (53%), particularly, but not significantly so, among those less than 2 years old with HE grade 0-1 (NAC 25%; placebo 60%; P = 0.0493). There were no significant differences between treatment arms for hospital or ICU length of stay, organ systems failing, or highest recorded grade of HE.ConclusionNAC did not improve 1-year survival in non-APAP PALF. One-year LTx-free survival was significantly lower with NAC, particularly among those

Published
2013

21. Portal Hypertension in Children and Young Adults With Biliary Atresia

Author

Shneider, Benjamin L, Abel, Bob, Haber, Barbara, Karpen, Saul J, Magee, John C, Romero, Rene, Schwarz, Kathleen, Bass, Lee M, Kerkar, Nanda, Miethke, Alexander G, Rosenthal, Philip, Turmelle, Yumirle, Robuck, Patricia R, and Sokol, Ronald J

Subjects
Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Clinical Research, Liver Disease, Chronic Liver Disease and Cirrhosis, Perinatal Period - Conditions Originating in Perinatal Period, Pediatric, Digestive Diseases, 2.1 Biological and endogenous factors, Aetiology, Adolescent, Alanine Transaminase, Aspartate Aminotransferases, Bile Ducts, Biliary Atresia, Bilirubin, Child, Child, Preschool, Cross-Sectional Studies, Humans, Hypertension, Portal, Leukocyte Count, Liver, North America, Platelet Count, Prevalence, Prothrombin Time, Serum Albumin, Splenomegaly, Thrombocytopenia, ascites, hepatopulmonary syndrome, hypersplenism, pediatric, varices, Childhood Liver Disease Research and Education Network, Medical and Health Sciences, Gastroenterology & Hepatology, Clinical sciences, Nutrition and dietetics, Paediatrics
Abstract

ObjectiveBiliary atresia (BA) frequently results in portal hypertension (PHT), complications of which lead to significant morbidity and mortality. The Childhood Liver Disease Research and Education Network was used to perform a cross-sectional multicentered analysis of PHT in children with BA.MethodsSubjects with BA receiving medical management at a Childhood Liver Disease Research and Education Network site were enrolled. A priori, clinically evident PHT was defined as "definite" when there was either history of a complication of PHT or clinical findings consistent with PHT (both splenomegaly and thrombocytopenia). PHT was denoted as "possible" if one of the findings was present in the absence of a complication, whereas PHT was "absent" if none of the criteria were met.ResultsA total of 163 subjects were enrolled between May 2006 and December 2009. At baseline, definite PHT was present in 49%, possible in 17%, and absent in 34% of subjects. Demographics, growth, and anthropometrics were similar amongst the 3 PHT categories. Alanine aminotransferase, γ-glutamyl transpeptidase, and sodium levels were similar, whereas there were significant differences in aspartate aminotransferase (AST), AST/alanine aminotransferase, albumin, total bilirubin, prothrombin time, white blood cell count, platelet count, and AST/platelet count between definite and absent PHT. Thirty-four percent of those with definite PHT had either prothrombin time >15 seconds or albumin

Published
2012

24. Serum bile acids are associated with native liver survival in patients with Alagille syndrome: results from the GALA study group

Author

Murillo Perez, Carla Fiorella, primary, Vandriel, Shannon M., additional, Wang, Jian-She, additional, Liting, Li, additional, She, Huiyu, additional, Jankowska, Irena, additional, Czubkowski, Piotr, additional, Gliwicz, Dorota, additional, Gonzalès, Emmanuel, additional, Jacquemin, Emmanuel, additional, Bouligand, Jérôme, additional, D’Antiga, Lorenzo, additional, Nicastro, Emanuele, additional, Fischler, Björn, additional, Arnell, Henrik, additional, Siew, Susan, additional, Stormon, Michael, additional, Loomes, Kathleen M., additional, Piccoli, David A., additional, Rand, Elizabeth B., additional, Squires, James E., additional, Karpen, Saul J., additional, Romero, Rene, additional, Kasahara, Mureo, additional, Önal, Zerrin, additional, Sokal, Etienne, additional, Demaret, Tanguy, additional, Wiecek, Sabina, additional, Lacaille, Florence, additional, Debray, Dominique, additional, Hardikar, Winita, additional, Shankar, Sahana, additional, Valentino, Pamela, additional, Sundaram, Shikha, additional, Chaidez, Alexander, additional, Ebel, Noelle, additional, Feinstein, Jeffrey, additional, Mozar-Glazberg, Yael, additional, Lin, Henry, additional, Rock, Nathalie, additional, Verkade, Henkjan J., additional, Jensen, M.K., additional, Jaramillo, Catalina, additional, Kim, Kyungmo, additional, Oh, Seak Hee, additional, Brecelj, Jernej, additional, Alam, Seema, additional, Indolfi, Giuseppe, additional, Blondet, Niviann, additional, Fawaz, Rima, additional, Nastasio, Silvia, additional, Calvo, Pier Luigi, additional, Nebbia, Gabriella, additional, Arıkan, Cigdem, additional, Larson-Nath, Catherine, additional, Zizzo, Andréanne N., additional, Sandahl, Thomas Damgaard, additional, Tzivinikos, Christos, additional, El-Koofy, Nehal, additional, Elmonem, Mohamed, additional, Desai, Dev, additional, Karnsakul, Wikrom, additional, Karthikeyan, Palaniswamy, additional, Bulut, Pinar, additional, Kerkar, Nanda, additional, Wolters, Victorien, additional, Roberts, Amin J, additional, Evans, Helen, additional, Sanchez, Maria Camila, additional, Cavalieri, Maria Lorena, additional, Kelly, Deirdre, additional, Lee, Way Seah, additional, Hajinicolaou, Christina, additional, Lertudomphonwanit, Chatmanee, additional, Fischer, Ryan, additional, Bernabeu, Jesús Quintero, additional, Quiros-Tejeira, Ruben E., additional, Melere, Melina, additional, Carvalho, Elisa, additional, Eshun, John, additional, Zellos, Aglaia, additional, Dezsőfi, Antal, additional, Pinto, Raquel Borges, additional, Schwarz, Kathleen, additional, Rogalidou, Maria, additional, Garcia, Jennifer, additional, Tamara, María Legarda, additional, Beretta, Marisa, additional, Mujawar, Quais, additional, Santos-Silva, Ermelinda, additional, Busoms, Cristina Molera, additional, Lurz, Eberhard, additional, Gonçalves, Cristina, additional, Jimenez-Rivera, Carolina, additional, Bañales, Jesús M., additional, Shah, Uzma, additional, Thompson, Richard, additional, Hansen, Bettina, additional, and Kamath, Binita M., additional

Published
2023
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25. Protein biomarkers GDF15 and FGF21 to differentiate mitochondrial hepatopathies from other pediatric liver diseases.

Author

Van Hove, Johan L. K., Friederich, Marisa W., Strode, Dana K., Van Hove, Roxanne A., Miller, Kristen R., Sharma, Rohit, Shah, Hardik, Estrella, Jane, Gabel, Linda, Horslen, Simon, Kohli, Rohit, Lovell, Mark A., Miethke, Alexander G., Molleston, Jean P., Romero, Rene, Squires, James E., Alonso, Estella M., Guthery, Stephen L., Kamath, Binita M., and Loomes, Kathleen M.

Published
2024
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27. Factors associated with thrombotic and hemorrhagic complications in pediatric liver transplant: A multi‐center analysis from the Starzl Network for Excellence in Pediatric Transplantation

Author

Soltys, Kyle, primary, Lemoine, Caroline, additional, Zhang, Xingyu, additional, Bhat, Rukhmi, additional, Bucuvalas, John, additional, Rasmussen, Sara, additional, Romero, Rene, additional, Batsis, Irini, additional, Sayed, Blayne, additional, Tunno, John, additional, Confair, Cassandra, additional, Vargas, Sarah, additional, Superina, Riccardo, additional, and Mazariegos, George, additional

Published
2023
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29. Natural History of Liver Disease in a Large International Cohort of Children with Alagille syndrome: Results from The <scp>GALA</scp> Study

Author

UCL - SSS/IREC/PEDI - Pôle de Pédiatrie, UCL - (SLuc) Service de gastro-entérologie et hépatologie pédiatrique, Vandriel, Shannon M., Li, Li‐Ting, She, Huiyu, Wang, Jian‐She, Gilbert, Melissa A., Jankowska, Irena, Czubkowski, Piotr, Gliwicz‐Miedzińska, Dorota, Gonzales, Emmanuel M., Jacquemin, Emmanuel, Bouligand, Jérôme, Spinner, Nancy B., Loomes, Kathleen M., Piccoli, David A., D'Antiga, Lorenzo, Nicastro, Emanuele, Sokal, Etienne, Demaret,Tanguy, Ebel, Noelle H., Feinstein, Jeffrey A., Fawaz, Rima, Nastasio, Silvia, Lacaille, Florence, Debray, Dominique, Arnell, Henrik, Fischler, Björn, Siew, Susan, Stormon, Michael, Karpen, Saul J., Romero, Rene, Kim, Kyung Mo, Baek, Woo Yim, Hardikar, Winita, Shankar, Sahana, Roberts, Amin J., Evans, Helen M., Jensen, M. Kyle, Kavan, Marianne, Sundaram, Shikha S., Chaidez, Alexander, Karthikeyan, Palaniswamy, Sanchez, Maria Camila, Cavalieri, Maria Lorena, Verkade, Henkjan J., Lee, Way Seah, Squires, James E., Hajinicolaou, Christina, Lertudomphonwanit, Chatmanee, Fischer, Ryan T., Larson‐Nath, Catherine, Mozer‐Glassberg, Yael, Arikan, Cigdem, Lin, Henry C., Quintero Bernabeu, Jesus, Alam, Seema, Kelly, Deirdre, Carvalho, Elisa, Ferreira, Cristina Targa, Indolfi, Giuseppe, Quiros‐Tejeira, Ruben E., Bulut, Pinar, Calvo, Pier Luigi, Önal, Zerrin, Valentino, Pamela L., Desai, Dev M., Eshun, John, Rogalidou, Maria, Dezsőfi, Antal, Wiecek, Sabina, Nebbia, Gabriella, Borges Pinto, Raquel, Wolters, Victorien M., Tamara, María Legarda, Zizzo, Andréanne N., Garcia, Jennifer, Schwarz, Kathleen, Beretta, Marisa, Sandahl, Thomas Damgaard, Jimenez‐Rivera, Carolina, Kerkar, Nanda, Brecelj, Jernej, Mujawar, Quais, Rock, Nathalie, Busoms, Cristina Molera, Karnsakul, Wikrom, Lurz, Eberhard, Santos‐Silva, Ermelinda, Blondet, Niviann, Bujanda, Luis, Shah, Uzma, Thompson, Richard J., Hansen, Bettina E., Kamath, Binita M., UCL - SSS/IREC/PEDI - Pôle de Pédiatrie, UCL - (SLuc) Service de gastro-entérologie et hépatologie pédiatrique, Vandriel, Shannon M., Li, Li‐Ting, She, Huiyu, Wang, Jian‐She, Gilbert, Melissa A., Jankowska, Irena, Czubkowski, Piotr, Gliwicz‐Miedzińska, Dorota, Gonzales, Emmanuel M., Jacquemin, Emmanuel, Bouligand, Jérôme, Spinner, Nancy B., Loomes, Kathleen M., Piccoli, David A., D'Antiga, Lorenzo, Nicastro, Emanuele, Sokal, Etienne, Demaret,Tanguy, Ebel, Noelle H., Feinstein, Jeffrey A., Fawaz, Rima, Nastasio, Silvia, Lacaille, Florence, Debray, Dominique, Arnell, Henrik, Fischler, Björn, Siew, Susan, Stormon, Michael, Karpen, Saul J., Romero, Rene, Kim, Kyung Mo, Baek, Woo Yim, Hardikar, Winita, Shankar, Sahana, Roberts, Amin J., Evans, Helen M., Jensen, M. Kyle, Kavan, Marianne, Sundaram, Shikha S., Chaidez, Alexander, Karthikeyan, Palaniswamy, Sanchez, Maria Camila, Cavalieri, Maria Lorena, Verkade, Henkjan J., Lee, Way Seah, Squires, James E., Hajinicolaou, Christina, Lertudomphonwanit, Chatmanee, Fischer, Ryan T., Larson‐Nath, Catherine, Mozer‐Glassberg, Yael, Arikan, Cigdem, Lin, Henry C., Quintero Bernabeu, Jesus, Alam, Seema, Kelly, Deirdre, Carvalho, Elisa, Ferreira, Cristina Targa, Indolfi, Giuseppe, Quiros‐Tejeira, Ruben E., Bulut, Pinar, Calvo, Pier Luigi, Önal, Zerrin, Valentino, Pamela L., Desai, Dev M., Eshun, John, Rogalidou, Maria, Dezsőfi, Antal, Wiecek, Sabina, Nebbia, Gabriella, Borges Pinto, Raquel, Wolters, Victorien M., Tamara, María Legarda, Zizzo, Andréanne N., Garcia, Jennifer, Schwarz, Kathleen, Beretta, Marisa, Sandahl, Thomas Damgaard, Jimenez‐Rivera, Carolina, Kerkar, Nanda, Brecelj, Jernej, Mujawar, Quais, Rock, Nathalie, Busoms, Cristina Molera, Karnsakul, Wikrom, Lurz, Eberhard, Santos‐Silva, Ermelinda, Blondet, Niviann, Bujanda, Luis, Shah, Uzma, Thompson, Richard J., Hansen, Bettina E., and Kamath, Binita M.

Abstract

Background: Alagille syndrome (ALGS) is a multisystem disorder, characterized by cholestasis. Existing outcome data are largely derived from tertiary centers and real-world data are lacking. This study aimed to elucidate the natural history of liver disease in a contemporary, international, cohort of children with ALGS. Methods: Multicenter retrospective study of children with a clinically and/or genetically confirmed ALGS diagnosis, born Jan-1997 - Aug-2019. Native liver survival (NLS) and event-free survival rates were assessed. Cox models were constructed to identify early biochemical predictors of clinically evident portal hypertension (CEPH) and NLS. Results: 1433 children (57% male) from 67 centers in 29 countries were included. 10 and 18-years NLS rates were 54.4% and 40.3%. By 10 and 18-years, 51.5% and 66.0% of ALGS children experienced ≥1 adverse liver-related event (CEPH, transplant or death). Children (>6 and ≤12 months) with median total bilirubin (TB) levels between ≥5.0 and <10.0 mg/dL had a 4.1-fold (95% CI 1.6 - 10.8) and those ≥10.0 mg/dL had an 8.0-fold (95% CI 3.4 - 18.4) increased risk of developing CEPH compared with those <5.0 mg/dL. Median TB levels between ≥5.0 and <10.0 mg/dL and >10.0 mg/dL were associated with a 4.8 (95% CI 2.4 - 9.7) and 15.6 (95% CI 8.7 - 28.2) increased risk of transplantation relative to <5.0 mg/dL. Median TB <5.0 mg/dL were associated with higher NLS rates relative to ≥5.0 mg/dL, with 79% reaching adulthood with native liver (p<0.001). Conclusions: In this large international cohort of ALGS, only 40.3% of children reach adulthood with their native liver. A TB <5.0 mg/dL between 6-and-12-months of age is associated with better hepatic outcomes. These thresholds provide clinicians with an objective tool to assist with clinical decision-making and in the evaluation of novel therapies.

Published
2023

30. Clinical Course among Cases of Acute Liver Failure of Indeterminate Diagnosis

Author

Bukauskas, Kathryn, Narkewicz, Michael R., Hite, Michelle, Loomes, Kathleen M., Rand, Elizabeth B., Piccoli, David, Kawchak, Deborah, Romero, Rene, Karpen, Saul, de la Cruz-Tracy, Liezl, Ng, Vicky, Hunt, Kelsey, Subbarao, Girish C., Klipsch, Ann, Alonso, Estella M., Sorenson, Lisa, Kelly, Susan, Delute, Dhey, Neighbors, Katie, Rosenthal, Philip J., Fleck, Shannon, Leonis, Mike A., Bucuvalas, John, Horning, Tracie, Baez, Norberto Rodriguez, Montanye, Shirley, Cowie, Margaret, Murray, Karen, Young, Melissa, Vendettuoli, Heather, Rudnick, David A., Shepherd, Ross W., Harris, Kathy, Karpen, Saul J., De La Torre, Alejandro, Dell Olio, Dominic, Kelly, Deirdre, Lloyd, Carla, Lobritto, Steven J., Bakhsh, Sumerah, Jonas, Maureen, Elifoson, Scott A., Raza, Roshan, Schwarz, Kathleen B., Karnsakul, Wikrom W., Alford, Mary Kay, Dhawan, Anil, Fitzpatrick, Emer, Kerkar, Nanda N., Haydel, Brandy, Narayanappa, Sreevidya, Lopez, M. James, Shieck, Victoria, Li, Ruosha, Belle, Steven H., Horslen, Simon, Chen, Ling-wan, Zhang, Song, and Squires, Robert H.

Published
2016
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31. Total Serum Bilirubin within 3 Months of Hepatoportoenterostomy Predicts Short-Term Outcomes in Biliary Atresia

Author

Alonso, Estella, Kaurs, Elizabeth, Kelly, Sue, Bove, Kevin, Heubi, James, Miethke, Alexander, Tiao, Greg, Denlinger, Julie, Ferris, Andrea, Feldman, Amy, Mack, Cara, Suchy, Frederick, Sundaram, Shikha, Van Hove, Johan, Hite, Michelle, Kantor, Susanna, Miller, Todd, Smith, Julia, VanWinkle, Becky, Loomes, Kathleen, Lin, Henry, Piccoli, David, Russo, Pierre, Spinner, Nancy, Brown, Lindsay, Elgert, Emily, Erlichman, Jessi, Alissa, Feras, Lindblad, Douglas, Mazariegos, George, Ortiz-Aguayo, Roberto, Perlmutter, David, Sindhi, Rakesh, Venkat, Veena, Vockley, Jerry, Bukauskas, Kathy, Kufen, Adam, Schulte, Madeline, Bull, Laura, Fleck, Shannon, Langlois, Camille, Teckman, Jeffery, Kociela, Vikki, Postma, Stacy, Harris, Kathleen, Bozic, Molly, Subbarao, Girish, Byam, Beth, Klipsch, Ann, Sawyers, Cindy, Horslen, Simon, Hsu, Evelyn, Cooper, Kara, Young, Melissa, Kamath, Binita, DeAngelis, Maria, O'Connor, Constance, VanRoestel, Krista, Parmar, Arpita, Quammie, Claudia, Hung, Kelsey, Guthery, Stephen, Jensen, Kyle, Rutherford, Ann, Kerker, Nanda, Michail, Sonia, Thomas, Danny, Goodhue, Catherine, Gupta, Nikita, Vos, Mariam, de la Cruz-Tracey, Liezl, Hankerson-Dyson, Dana, Tory, Rita, Turner-Green, Taieshia, Wellons, Allison, Brandt, Mary, Finegold, Milton, Harpavat, Sanjiv, Hertel, Paula, Leung, Daniel, Liwanag, Loriel, Thompson, Richard, Brown, Sherry, Doo, Edward, Hoofnagle, Jay, Hall, Sherry, Torrance, Rebecca, Brown, Jameisha, Kafka, Kimberly, Merion, Robert, Spino, Cathie, Shneider, Benjamin L., Magee, John C., Karpen, Saul J., Rand, Elizabeth B., Narkewicz, Michael R., Bass, Lee M., Schwarz, Kathleen, Whitington, Peter F., Bezerra, Jorge A., Kerkar, Nanda, Haber, Barbara, Rosenthal, Philip, Turmelle, Yumirle P., Molleston, Jean P., Murray, Karen F., Ng, Vicky L., Wang, Kasper S., Romero, Rene, Squires, Robert H., Arnon, Ronen, Sherker, Averell H., Moore, Jeffrey, Ye, Wen, and Sokol, Ronald J.

Published
2016
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33. Elbasvir/grazoprevir in children aged 3–18 years with chronic HCV genotype 1 or 4 infection: a pharmacokinetic modeling study

Author

Gonzalez-Peralta, Regino P., primary, Wirth, Stefan, additional, Squires, Robert H., additional, Mutschler, Frauke, additional, Lang, Thomas, additional, Pawlowska, Malgorzata, additional, Sluzewski, Wojciech, additional, Majda-Stanislawska, Ewa, additional, Fischler, Bjorn, additional, Balistreri, William F., additional, Jonas, Maureen M., additional, Blondet, Niviann, additional, Rosenthal, Philip, additional, Alkhouri, Naim, additional, Romero, Rene, additional, Grandhi, Anjana, additional, Castronuovo, Patricia, additional, Caro, Luzelena, additional, Du, Lihong, additional, Rosenbloom, Daniel I.S., additional, and Haber, Barbara A., additional

Published
2023
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34. Effectiveness of a Lifestyle Change Program on Insulin Resistance in Yaquis Indigenous Populations in Sonora, Mexico: PREVISY

Author

Castro-Juarez, Alejandro Arturo, primary, Serna-Gutiérrez, Araceli, additional, Alemán-Mateo, Heliodoro, additional, Gallegos-Aguilar, Ana Cristina, additional, Dórame-López, Norma Alicia, additional, Valenzuela-Sánchez, Abraham, additional, Valenzuela-Guzmán, Diana Marcela, additional, Díaz-Zavala, Rolando Giovanni, additional, Urquidez-Romero, Rene, additional, and Esparza-Romero, Julián, additional

Published
2023
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35. Baseline Ultrasound and Clinical Correlates in Children with Cystic Fibrosis

Author

Alonso, Estella M., Nicholas, Jennifer L., Kaurs, Elizabeth, Narkewicz, Michael R., Sokol, Ronald J., Harned, Roger, Burr, Susanna, Romero, Rene, Freeman, Jay, Alazraki, Adina, Patrick, Ellen, Hunter, Eric, Ling, Simon C., Navarro, Oscar, Ling, Julie P., Palermo, Joe J., Towbin, Alex, Ferris, Andrea, Denlinger, Julie, Molleston, Jean P., Bozic, Molly A., Subbarao, Girish, Karmazyn, Boaz, Klipsch, Ann, Paranjape, Shruti M., Karnsakul, Wikrom, Benson, Jane E., Callahan, Karen A., Kafka, Kim, Murray, Karen F., Gibson, Ron, Otto, Randolph, Genatossio, Alan, Young, Melissa, Weymann, Alexander, Siegel, Marilyn J., Harris, Kathy, Leung, Daniel H., Krishnamurthy, Rajesh, Brown, Jameisha, Schwarzenberg, Sara Jane, Stacklie, Denise, Seidel, F. Glenn, Doo, Edward, Sherker, Averell H., Hall, Sherry R., Torrance, Rebecca, Ye, Wen, Ling, Simon, Palermo, Joseph, Marshall, Bruce C., and Magee, John C.

Published
2015
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37. THU-291 - Serum bile acids are associated with native liver survival in patients with Alagille syndrome: results from the GALA study group

Author

Murillo Perez, Carla Fiorella, Vandriel, Shannon M., Wang, Jian-She, Liting, Li, She, Huiyu, Jankowska, Irena, Czubkowski, Piotr, Gliwicz, Dorota, Gonzalès, Emmanuel, Jacquemin, Emmanuel, Bouligand, Jérôme, D’Antiga, Lorenzo, Nicastro, Emanuele, Fischler, Björn, Arnell, Henrik, Siew, Susan, Stormon, Michael, Loomes, Kathleen M., Piccoli, David A., Rand, Elizabeth B., Squires, James E., Karpen, Saul J., Romero, Rene, Kasahara, Mureo, Önal, Zerrin, Sokal, Etienne, Demaret, Tanguy, Wiecek, Sabina, Lacaille, Florence, Debray, Dominique, Hardikar, Winita, Shankar, Sahana, Valentino, Pamela, Sundaram, Shikha, Chaidez, Alexander, Ebel, Noelle, Feinstein, Jeffrey, Mozar-Glazberg, Yael, Lin, Henry, Rock, Nathalie, Verkade, Henkjan J., Jensen, M.K., Jaramillo, Catalina, Kim, Kyungmo, Oh, Seak Hee, Brecelj, Jernej, Alam, Seema, Indolfi, Giuseppe, Blondet, Niviann, Fawaz, Rima, Nastasio, Silvia, Calvo, Pier Luigi, Nebbia, Gabriella, Arıkan, Cigdem, Larson-Nath, Catherine, Zizzo, Andréanne N., Sandahl, Thomas Damgaard, Tzivinikos, Christos, El-Koofy, Nehal, Elmonem, Mohamed, Desai, Dev, Karnsakul, Wikrom, Karthikeyan, Palaniswamy, Bulut, Pinar, Kerkar, Nanda, Wolters, Victorien, Roberts, Amin J, Evans, Helen, Sanchez, Maria Camila, Cavalieri, Maria Lorena, Kelly, Deirdre, Lee, Way Seah, Hajinicolaou, Christina, Lertudomphonwanit, Chatmanee, Fischer, Ryan, Bernabeu, Jesús Quintero, Quiros-Tejeira, Ruben E., Melere, Melina, Carvalho, Elisa, Eshun, John, Zellos, Aglaia, Dezsőfi, Antal, Pinto, Raquel Borges, Schwarz, Kathleen, Rogalidou, Maria, Garcia, Jennifer, Tamara, María Legarda, Beretta, Marisa, Mujawar, Quais, Santos-Silva, Ermelinda, Busoms, Cristina Molera, Lurz, Eberhard, Gonçalves, Cristina, Jimenez-Rivera, Carolina, Bañales, Jesús M., Shah, Uzma, Thompson, Richard, Hansen, Bettina, and Kamath, Binita M.

Published
2023
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38. Clinical spectrum and genetic causes of mitochondrial hepatopathy phenotype in children.

Author

Squires, James E., Miethke, Alexander G., Valencia, C. Alexander, Hawthorne, Kieran, Henn, Lisa, Van Hove, Johan L. K., Squires, Robert H., Bove, Kevin, Horslen, Simon, Kohli, Rohit, Molleston, Jean P., Romero, Rene, Alonso, Estella M., Bezerra, Jorge A., Guthery, Stephen L., Hsu, Evelyn, Karpen, Saul J., Loomes, Kathleen M., Ng, Vicky L., and Rosenthal, Philip

Published
2023
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39. Finding Spanish Syllabification Rules with Decision Trees

Author

Goddard, John, MacKinney-Romero, René, Hutchison, David, editor, Kanade, Takeo, editor, Kittler, Josef, editor, Kleinberg, Jon M., editor, Mattern, Friedemann, editor, Mitchell, John C., editor, Naor, Moni, editor, Nierstrasz, Oscar, editor, Pandu Rangan, C., editor, Steffen, Bernhard, editor, Sudan, Madhu, editor, Terzopoulos, Demetri, editor, Tygar, Dough, editor, Vardi, Moshe Y., editor, Weikum, Gerhard, editor, Carbonell, Jaime G., editor, Siekmann, Jörg, editor, Salakoski, Tapio, editor, Ginter, Filip, editor, Pyysalo, Sampo, editor, and Pahikkala, Tapio, editor

Published
2006
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40. Inferring Rules for Finding Syllables in Spanish

Author

MacKinney-Romero, René, Goddard, John, Hutchison, David, editor, Kanade, Takeo, editor, Kittler, Josef, editor, Kleinberg, Jon M., editor, Mattern, Friedemann, editor, Mitchell, John C., editor, Naor, Moni, editor, Nierstrasz, Oscar, editor, Pandu Rangan, C., editor, Steffen, Bernhard, editor, Sudan, Madhu, editor, Terzopoulos, Demetri, editor, Tygar, Dough, editor, Vardi, Moshe Y., editor, Weikum, Gerhard, editor, Carbonell, Jaime G., editor, Siekmann, Jörg, editor, Gelbukh, Alexander, editor, de Albornoz, Álvaro, editor, and Terashima-Marín, Hugo, editor

Published
2005
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41. Predictive equations for fat mass in older Hispanic adults with excess adiposity using the 4‐compartment model as a reference method

Author

González-Arellanes, Rogelio, primary, Urquidez-Romero, Rene, additional, Rodríguez-Tadeo, Alejandra, additional, Esparza-Romero, Julián, additional, Méndez-Estrada, Rosa Olivia, additional, Ramírez-López, Erik, additional, Robles-Sardin, Alma-Elizabeth, additional, Pacheco-Moreno, Bertha-Isabel, additional, and Alemán-Mateo, Heliodoro, additional

Published
2022
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49. Application of real-world evidence analytics: A 6-year event-free survival analysis in alagille syndrome of the gala clinical research database and maralixibat treated patients

Author

Hansen, Bettina E., Vandriel, Shannon M., Vig, Pamela, Garner, Will, Li, Li-Ting, She, Huiyu, Wang, Jian-She, Gilbert, Melissa A., Jankowska, Irena, Czubkowski, Piotr, Gliwicz-Miedzinska, Dorota, Gonzales, Emmanuel M., Jacquemin, Emmanuel, Bouligand, Jerome, Spinner, Nancy, Loomes, Kathleen M., Piccoli, David A., D'Antiga, Lorenzo, Nicastro, Emanuele, Sokal, Etienne, Demaret, Tanguy, Ebel, Noelle, Feinstein, Jeffrey A., Fawaz, Rima, Nastasio, Silvia, Lacaille, Florence, Debray, Dominique, Arnell, Henrik, Fischler, Bjorn, Siew, Susan, Stormon, Michael, Karpen, Saul J., Romero, Rene, Kim, Kyung Mo, Baek, Woo Yim, Hardikar, Winita, Shankar, Sahana, Roberts, Amin J., Evans, Helen M., Jensen, M. Kyle, Kavan, Marianne, Sundaram, Shikha S., Chaidez, Alexander, Karthikeyan, Palaniswamy, Davison, Suzanne, Sanchez, Maria Camila, Cavalieri, Maria Lorena, Verkade, Henkjan J., Lee, Way Seah, Squires, James E., and Center for Liver, Digestive and Metabolic Diseases (CLDM)

Published
2021

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