9 results on '"DiGuglielmo, Matthew"'
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2. Vedolizumab Therapy in Children With Primary Sclerosing Cholangitis: Data From the Pediatric Primary Sclerosing Cholangitis Consortium
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Laborda, Trevor J., Ricciuto, Amanda, Aumar, Madeleine, Carman, Nicholas, DiGuglielmo, Matthew, Draijer, Laura G., Furuya, Katryn N., Gupta, Nitika, Koot, Bart G.P., Loomes, Kathleen M., Lytvyak, Ellina, Martinez, Mercedes, Miloh, Tamir, Montano-Loza, Aldo J., Perito, Emily R., Sathya, Pushpa, Shah, Uzma, Shteyer, Eyal, Singh, Ruchi, Taylor, Amy, Valentino, Pamela L., Vitola, Bernadette, Zerofsky, Melissa, Zizzo, Andréanne, and Deneau, Mark R.
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- 2020
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3. Assessing the Validity of Adult-derived Prognostic Models for Primary Sclerosing Cholangitis Outcomes in Children
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Deneau, Mark R., Valentino, Pamela L., Mack, Cara, Alqoaer, Khaled, Amin, Mansi, Amir, Achiya Z., Aumar, Madeleine, Auth, Marcus, Broderick, Annemarie, DiGuglielmo, Matthew, Draijer, Laura G., El-Matary, Wael, Ferrari, Federica, Furuya, Katryn N., Gottrand, Frederic, Gupta, Nitika, Homan, Matjaz, Jensen, M.K., Kamath, Binita M., Kim, Kyung Mo, Kolho, Kaija-Leena, Koot, Bart, Iorio, Raffaele, Martinez, Mercedes, Miloh, Tamir, Mohan, Parvathi, Palle, Sirish, Papadopoulou, Alexandra, Ricciuto, Amanda, Saubermann, Lawrence, Sathya, Pushpa, Shteyer, Eyal, Smolka, Vratislav, Tanaka, Atsushi, Varier, Raghu, Venkat, Veena, Vitola, Bernadette, Woynarowski, Marek, and Guthery, Stephen
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- 2020
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4. Oral Vancomycin, Ursodeoxycholic Acid, or No Therapy for Pediatric Primary Sclerosing Cholangitis: A Matched Analysis.
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Deneau, Mark R., Mack, Cara, Mogul, Douglas, Perito, Emily R., Valentino, Pamela L., Amir, Achiya Z., DiGuglielmo, Matthew, Draijer, Laura G., El‐Matary, Wael, Furuya, Katryn N., Gupta, Nitika, Hochberg, Jessica T., Horslen, Simon, Jensen, M. Kyle, Jonas, Maureen M., Kerkar, Nanda, Koot, Bart G.P., Laborda, Trevor J., Lee, Christine K., and Loomes, Kathleen M.
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- 2021
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5. The Sclerosing Cholangitis Outcomes in Pediatrics (SCOPE) Index: A Prognostic Tool for Children.
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Deneau, Mark R., Mack, Cara, Perito, Emily R., Ricciuto, Amanda, Valentino, Pamela L., Amin, Mansi, Amir, Achiya Z., Aumar, Madeleine, Auth, Marcus, Broderick, Annemarie, DiGuglielmo, Matthew, Draijer, Laura G., Tavares Fagundes, Eleonora Druve, El‐Matary, Wael, Ferrari, Federica, Furuya, Katryn N., Gupta, Nitika, Hochberg, Jessica T., Homan, Matjaz, and Horslen, Simon
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- 2021
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6. Sa2020 - Role of Genetic Variants (PNPLA3 and MBOAT7) in Development of NAFLD Amongst Obese Children of Different Ethnicities
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Mansoor, Sana, DiGuglielmo, Matthew, He, Zhaoping, and Holbrook, Jennifer
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- 2018
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7. Gamma Glutamyltransferase Reduction Is Associated With Favorable Outcomes in Pediatric Primary Sclerosing Cholangitis.
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Deneau, Mark R., Mack, Cara, Abdou, Reham, Amin, Mansi, Amir, Achiya, Auth, Marcus, Bazerbachi, Fateh, Marie Broderick, Anne, Chan, Albert, DiGuglielmo, Matthew, El‐Matary, Wael, El‐Youssef, Mounif, Ferrari, Federica, Furuya, Katryn N., Gottrand, Frederic, Gupta, Nitika, Homan, Matjaž, Jensen, M.K., Kamath, Binita M., and Mo Kim, Kyung
- Abstract
Adverse clinical events in primary sclerosing cholangitis (PSC) happen too slowly to capture during clinical trials. Surrogate endpoints are needed, but no such validated endpoints exist for children with PSC. We evaluated the association between gamma glutamyltransferase (GGT) reduction and long‐term outcomes in pediatric PSC patients. We evaluated GGT normalization (< 50 IU/L) at 1 year among a multicenter cohort of children with PSC who did or did not receive treatment with ursodeoxycholic acid (UDCA). We compared rates of event‐free survival (no portal hypertensive or biliary complications, cholangiocarcinoma, liver transplantation, or liver‐related death) at 5 years. Of the 287 children, mean age of 11.4 years old, UDCA was used in 81% at a mean dose of 17 mg/kg/day. Treated and untreated groups had similar GGT at diagnosis (314 versus 300, P= not significant [NS]). The mean GGT was reduced at 1 year in both groups, with lower values seen in treated (versus untreated) patients (99 versus 175, P= 0.002), but 5‐year event‐free survival was similar (74% versus 77%, P= NS). In patients with GGT normalization (versus no normalization) by 1 year, regardless of UDCA treatment status, 5‐year event‐free survival was better (91% versus 67%, P< 0.001). Similarly, larger reduction in GGT over 1 year (> 75% versus < 25% reduction) was also associated with improved outcome (5‐year event‐free survival 88% versus 61%, P= 0.005). Conclusion:A GGT < 50 and/or GGT reduction of > 75% by 1 year after PSC diagnosis predicts favorable 5‐year outcomes in children. GGT has promise as a potential surrogate endpoint in future clinical trials for pediatric PSC. [ABSTRACT FROM AUTHOR]
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- 2018
- Full Text
- View/download PDF
8. The Sclerosing Cholangitis Outcomes in Pediatrics (SCOPE) Index: A Prognostic Tool for Children
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Matjaz Homan, Binita M. Kamath, Bart G. P. Koot, Mansi Amin, Parvathi Mohan, Amanda Ricciuto, Melissa Zerofsky, Madeleine Aumar, Kathleen B. Schwarz, Laura G. Draijer, Annemarie Broderick, Kaija-Leena Kolho, Stephen L. Guthery, Nanda Kerkar, Saeed Mohammad, Nisreen Soufi, Alexandra Papadopoulou, Eyal Shteyer, Raffaele Iorio, Nadia Ovchinsky, M. Kyle Jensen, Simon Horslen, Ruchi Singh, Maureen M. Jonas, Kyung Mo Kim, Alexander Miethke, Girish S. Rao, Federica Ferrari, Achiya Z. Amir, Cara L. Mack, Douglas Mogul, Matthew DiGuglielmo, Vratislav Smolka, Christine K. Lee, Pushpa Sathya, Katryn N. Furuya, Nitika A. Gupta, Mercedes Martinez, Atsushi Tanaka, Tamir Miloh, Kathleen M. Loomes, Bernadette Vitola, Uzma Shah, Pamela L. Valentino, Andréanne Zizzo, Mark Deneau, Jessica T. Hochberg, Wael El-Matary, Stacy Moroz, Marcus Auth, Emily R. Perito, Trevor J. Laborda, Marek Woynarowski, Eleonora Druve Tavares Fagundes, Alexandre Rodrigues Ferreira, Raghu Varier, Sirish Palle, Graduate School, AGEM - Amsterdam Gastroenterology Endocrinology Metabolism, ARD - Amsterdam Reproduction and Development, Paediatric Gastroenterology, R Deneau, Mark, Mack, Cara, R Perito, Emily, Ricciuto, Amanda, L Valentino, Pamela, Amin, Mansi, Z Amir, Achiya, Aumar, Madeleine, Auth, Marcu, Broderick, Annemarie, Diguglielmo, Matthew, G Draijer, Laura, Druve Tavares Fagundes, Eleonora, El-Matary, Wael, Ferrari, Federica, N Furuya, Katryn, Gupta, Nitika, T Hochberg, Jessica, Homan, Matjaz, Horslen, Simon, Iorio, Raffaele, Kyle Jensen, M, M Jonas, Maureen, M Kamath, Binita, Kerkar, Nanda, Mo Kim, Kyung, Kolho, Kaija-Leena, P Koot, Bart G, J Laborda, Trevor, K Lee, Christine, M Loomes, Kathleen, Martinez, Mercede, Miethke, Alexander, Miloh, Tamir, Mogul, Dougla, Mohammad, Saeed, Mohan, Parvathi, Moroz, Stacy, Ovchinsky, Nadia, Palle, Sirish, Papadopoulou, Alexandra, Rao, Girish, Rodrigues Ferreira, Alexandre, Sathya, Pushpa, B Schwarz, Kathleen, Shah, Uzma, Shteyer, Eyal, Singh, Ruchi, Smolka, Vratislav, Soufi, Nisreen, Tanaka, Atsushi, Varier, Raghu, Vitola, Bernadette, Woynarowski, Marek, Zerofsky, Melissa, Zizzo, Andréanne, L Guthery, Stephen, Children's Hospital, and HUS Children and Adolescents
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0301 basic medicine ,Male ,Pediatrics ,medicine.medical_treatment ,Biopsy ,Autoimmune hepatitis ,Liver transplantation ,0302 clinical medicine ,Cholangiography ,Risk Factors ,Retrospective Studie ,Stage (cooking) ,Child ,RISK ,medicine.diagnostic_test ,gamma-Glutamyltransferase ,Prognosis ,3. Good health ,SURVIVAL ,Disease Progression ,030211 gastroenterology & hepatology ,Female ,Human ,medicine.medical_specialty ,Cancer complication ,Adolescent ,Prognosi ,Cholangitis, Sclerosing ,VALIDATION ,Primary sclerosing cholangitis ,CHOLANGIOCARCINOMA ,03 medical and health sciences ,medicine ,Humans ,Serum Albumin ,Retrospective Studies ,Hepatology ,business.industry ,Platelet Count ,Risk Factor ,Retrospective cohort study ,Bilirubin ,NATURAL-HISTORY ,medicine.disease ,Liver Transplantation ,Clinical trial ,MODEL ,030104 developmental biology ,3121 General medicine, internal medicine and other clinical medicine ,AUTOIMMUNE HEPATITIS ,business - Abstract
Background and Aims: Disease progression in children with primary sclerosing cholangitis (PSC) is variable. Prognostic and risk-stratification tools exist for adult-onset PSC, but not for children. We aimed to create a tool that accounts for the biochemical and phenotypic features and early disease stage of pediatric PSC. Approach and Results: We used retrospective data from the Pediatric PSC Consortium. The training cohort contained 1,012 patients from 40 centers. We generated a multivariate risk index (Sclerosing Cholangitis Outcomes in Pediatrics [SCOPE] index) that contained total bilirubin, albumin, platelet count, gamma glutamyltransferase, and cholangiography to predict a primary outcome of liver transplantation or death (TD) and a broader secondary outcome that included portal hypertensive, biliary, and cancer complications termed hepatobiliary complications (HBCs). The model stratified patients as low, medium, or high risk based on progression to TD at rates of
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- 2021
9. Gamma Glutamyltransferase Reduction Is Associated With Favorable Outcomes in Pediatric Primary Sclerosing Cholangitis
- Author
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Amanda Ricciuto, Cara L. Mack, Jason Yap, Lawrence J. Saubermann, Pamela L. Valentino, Pushpa Sathya, Mark Deneau, Binita M. Kamath, Vratislav Smolka, Mercedes Martinez, Atsushi Tanaka, Bart G. P. Koot, Annemarie Broderick, Tamir Miloh, Kaija-Leena Kolho, Reham Abdou, Miriam B. Vos, Federica Ferrari, Eyal Shteyer, Mounif El-Youssef, Katryn N. Furuya, Anastasia Konidari, Frédéric Gottrand, Raghu Varier, Achiya Z. Amir, Albert Chan, Mansi Amin, Sirish Palle, Parvathi Mohan, M.K. Jensen, Nitika A. Gupta, Veena Venkat, Matjaž Homan, Alexandra Papadopoulou, Wael El-Matary, Marcus Auth, Fateh Bazerbachi, Marek Woynarowski, Raffaele Iorio, Bernadette Vitola, Kyung Mo Kim, Matthew DiGuglielmo, Deneau, Mark R, Mack, Cara, Abdou, Reham, Amin, Mansi, Amir, Achiya, Auth, Marcu, Bazerbachi, Fateh, Marie Broderick, Anne, Chan, Albert, Diguglielmo, Matthew, El-Matary, Wael, El-Youssef, Mounif, Ferrari, Federica, Furuya, Katryn N, Gottrand, Frederic, Gupta, Nitika, Homan, Matjaž, Jensen, M K, Kamath, Binita M, Mo Kim, Kyung, Kolho, Kaija-Leena, Konidari, Anastasia, Koot, Bart, Iorio, Raffaele, Martinez, Mercede, Mohan, Parvathi, Palle, Sirish, Papadopoulou, Alexandra, Ricciuto, Amanda, Saubermann, Lawrence, Sathya, Pushpa, Shteyer, Eyal, Smolka, Vratislav, Tanaka, Atsushi, Valentino, Pamela L, Varier, Raghu, Venkat, Veena, Vitola, Bernadette, Vos, Miriam B, Woynarowski, Marek, Yap, Jason, Miloh, Tamir, Children's Hospital, Clinicum, University of Helsinki, HUS Children and Adolescents, AGEM - Re-generation and cancer of the digestive system, AGEM - Digestive immunity, Amsterdam Reproduction & Development, and Paediatric Gastroenterology
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sclerosing cholangitis ,medicine.medical_specialty ,endocrine system diseases ,CHOLESTASIS ,CHILDREN ,DIAGNOSIS ,Gastroenterology ,digestive system ,Article ,Primary sclerosing cholangitis ,03 medical and health sciences ,0302 clinical medicine ,Cholestasis ,Internal medicine ,MANAGEMENT ,medicine ,Gamma-glutamyltransferase ,lcsh:RC799-869 ,GLUTAMYL-TRANSFERASE ,gamma glutamyltransferase ,prognosis ,Hepatology ,biology ,business.industry ,Clinical events ,Surrogate endpoint ,digestive, oral, and skin physiology ,Too slowly ,Original Articles ,DOSE URSODEOXYCHOLIC ACID ,NATURAL-HISTORY ,medicine.disease ,digestive system diseases ,3. Good health ,PROGNOSTIC VALUE ,Clinical trial ,ALKALINE-PHOSPHATASE ,3121 General medicine, internal medicine and other clinical medicine ,030220 oncology & carcinogenesis ,SURVIVAL ,biology.protein ,030211 gastroenterology & hepatology ,lcsh:Diseases of the digestive system. Gastroenterology ,business - Abstract
Adverse clinical events in primary sclerosing cholangitis (PSC) happen too slowly to capture during clinical trials. Surrogate endpoints are needed, but no such validated endpoints exist for children with PSC. We evaluated the association between gamma glutamyltransferase (GGT) reduction and long-term outcomes in pediatric PSC patients. We evaluated GGT normalization (75% versus 75% by 1 year after PSC diagnosis predicts favorable 5-year outcomes in children. GGT has promise as a potential surrogate endpoint in future clinical trials for pediatric PSC.
- Published
- 2018
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