Long-term efficacy and safety of sofosbuvir-based direct-acting antiviral regimens in paediatric patients with hepatitis C virus infection: an international registry study

Authors: Gonzalez-Peralta, Regino P ^{a, *}, Wen, Jessica W ^b, Hardikar, Winita ^c, Karnsakul, Wikrom W ^d, Whitworth, Suzanne ^e, Lin, Chuan-Hao ^f, Indolfi, Giuseppe ^g, Rosenthal, Philip ^h, Balistreri, William ⁱ, Schwarz, Kathleen B ^d, Honegger, Jonathan R ^j, Zhang, Xu ^k, Svarovskaia, Evguenia C ^k, Suri, Vithika ^k, Kersey, Kathryn ^k, Leung, Daniel H ^l

Source: In The Lancet Child & Adolescent Health April 2025 9(4):248-254

Maralixibat in progressive familial intrahepatic cholestasis (MARCH-PFIC): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Authors: Miethke, Alexander G ^*, Moukarzel, Adib, Porta, Gilda, Covarrubias Esquer, Joshue, Czubkowski, Piotr, Ordonez, Felipe, Mosca, Antonella, Aqul, Amal A, Squires, Robert H, Sokal, Etienne, D'Agostino, Daniel, Baumann, Ulrich, D'Antiga, Lorenzo, Kasi, Nagraj, Laborde, Nolwenn, Arikan, Cigdem, Lin, Chuan-Hao, Gilmour, Susan, Mittal, Naveen, Chiou, Fang Kuan, Horslen, Simon P, Huber, Wolf-Dietrich, Jaecklin, Thomas, Nunes, Tiago, Lascau, Anamaria, Longpre, Lara, Mogul, Douglas B, Garner, Will, Vig, Pamela, Hupertz, Vera F, Gonzalez-Peralta, Regino P, Ekong, Udeme, Hartley, Jane, Laverdure, Noemie, Ovchinsky, Nadia, Thompson, Richard J

Source: In The Lancet Gastroenterology & Hepatology July 2024 9(7):620-631

Major Depressive Disorder in an International Multisite Wilson Disease Registry

Authors: Camarata, Michelle A., Ala, Aftab, Coskun, Ayse K., Deng, Yanhong, Embel, Veysel K., Gonzalez-Peralta, Regino, Maciejewski, Kaitlin R., Patel, Amar, Rubman, Susan, To, Uyen, Tomlin, Ricarda, Schilsky, Michael L., Zimbrean, Paula C.

Source: In Journal of the Academy of Consultation-Liaison Psychiatry March-April 2023 64(2):106-117

Procedure Costs Associated With Management of Biliary Strictures in Pediatric Liver Transplant Recipients in the Society of Pediatric Liver Transplantation (SPLIT) Registry.

Authors: Valentino, Pamela L., Perkins, James D., Taylor, Sarah A., Feldman, Amy G., Banc‐Husu, Anna M., Fishman, Douglas S., Bucuvalas, John C., Gonzalez‐Peralta, Regino P., Mazariegos, George, Mullapudi, Bhargava, Ng, Vicky L., Sundaram, Shikha S., Yazigi, Nada A., Kennedy, Jennie, Soltys, Kyle, McDiarmid, S., Feist, S., Hawkins, A., Hite, M., Azzam, R.

Source: Pediatric Transplantation; May2025, Vol. 29 Issue 3, p1-10, 10p

The Effect of Mental Health, Neurological Disease, and Liver Disease on Quality of Life in Patients With Wilson Disease

Authors: Camarata, Michelle A., Ala, Aftab, Coskun, Ayse K., Deng, Yanhong, Gonzalez-Peralta, Regino P., Maciejewski, Kaitlin R., Patel, Amar, Rubman, Susan, To, Uyen, Tomlin, Ricarda, Schilsky, Michael L., Zimbrean, Paula C.

Source: In Journal of the Academy of Consultation-Liaison Psychiatry September-October 2021 62(5):528-537

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

Authors: 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, Kersey, Kathryn

Source: Journal of Pediatric Gastroenterology & Nutrition; Jun2024, Vol. 78 Issue 6, p1342-1354, 13p

ESPGHAN recommendations on treatment of chronic hepatitis C virus infection in adolescents and children including those living in resource‐limited settings.

Authors: Indolfi, Giuseppe, Gonzalez‐Peralta, Regino P., Jonas, Maureen M., Sayed, Manal Hamdy‐El, Fischler, Björn, Sokal, Etienne, Wirth, Stefan, Nicastro, Emanuele, Kohlmaier, Benno, Fitzpatrick, Emer, Gonzales, Emmanuel, Junge, Norman, Mancell, Sarah, Mozer‐Glassberg, Yael, Pop, Tudor, Samyn, Marianne, Stephenne, Xavier, Zellos, Aglaia

Source: Journal of Pediatric Gastroenterology & Nutrition; Apr2024, Vol. 78 Issue 4, p957-972, 16p

Real‐world experience of maralixibat in Alagille syndrome: Novel findings outside of clinical trials.

Authors: Himes, Ryan, Rosenthal, Philip, Dilwali, Natasha, Smith, Kathryn, Venick, Robert, Gonzalez‐Peralta, Regino P.

Source: Journal of Pediatric Gastroenterology & Nutrition; Mar2024, Vol. 78 Issue 3, p506-513, 8p

Impact of early immunosuppression on pediatric liver transplant outcomes within 1 year.

Authors: Raghu, Vikram K., Zhang, Xingyu, Squires, James E., Eisenberg, Elizabeth, Feldman, Amy G., Halma, Jennifer, Peters, Anna L., Gonzalez‐Peralta, Regino P., Ng, Vicky L., Horslen, Simon P., Lobritto, Steven J., Bucuvalas, John, Mazariegos, George V., Perito, Emily R.

Source: Journal of Pediatric Gastroenterology & Nutrition; Feb2024, Vol. 78 Issue 2, p328-338, 11p

Expected and Actual Case Ascertainment and Treatment Rates for Children Infected with Hepatitis C in Florida and the United States: Epidemiologic Evidence from Statewide and Nationwide Surveys

Authors: Delgado-Borrego, Aymin, Smith, Lesley, Jonas, Maureen M., Hall, Cyndena A., Negre, Betania, Jordan, Sergio H., Ogrodowicz, Matthew, Raza, Roshan, Ludwig, David A., Miller, Tracie, Lipshultz, Steven E., Gonzalez-Peralta, Regino, Chung, Raymond T.

Source: In The Journal of Pediatrics November 2012 161(5):915-921

Herpes Simplex Virus Hepatitis in Infants: Clinical Outcomes and Correlates of Disease Severity

Authors: McGoogan, Katherine E., Haafiz, Allah B., González Peralta, Regino P.

Source: In The Journal of Pediatrics 2011 159(4):608-611

The Combination of Ribavirin and Peginterferon Is Superior to Peginterferon and Placebo for Children and Adolescents With Chronic Hepatitis C

Authors: Schwarz, Kathleen B., Gonzalez–Peralta, Regino P., Murray, Karen F., Molleston, Jean P., Haber, Barbara A., Jonas, Maureen M., Rosenthal, Philip, Mohan, Parvathi, Balistreri, William F., Narkewicz, Michael R., Smith, Lesley, Lobritto, Steven J., Rossi, Stephen, Valsamakis, Alexandra, Goodman, Zachary, Robuck, Patricia R., Barton, Bruce A.

Source: In Gastroenterology 2011 140(2):450-458

Wilson disease: a summary of the updated AASLD Practice Guidance.

Authors: Alkhouri, Naim, Gonzalez-Peralta, Regino P., Medici, Valentina

Source: Hepatology Communications; Jun2023, Vol. 7 Issue 6, p1-13, 13p

WED-164 Interim safety results of the ongoing international phase I/II GATEWAY gene therapy trial with VTX-801 conducted in adult patients with Wilson disease

Authors: Sandahl, Thomas Damgaard, Lee, William M., Ala, Aftab, Gonzalez-Peralta, Regino P., Medici, Valentina, Askari, Fred, Rudnick, Sean, Lauer, Ulrich, Schmidt, Hartmut, Valero, Sonia, Gonzalez, Gloria, Combal, Jean Philippe, D'Antiga, Lorenzo, Benichou, Bernard, Schilsky, Michael

Source: In Journal of Hepatology June 2024 80 Supplement 1:S714-S715

THU-129 Maralixibat improves growth in patients with progressive familial intrahepatic cholestasis: data from the MARCH/MARCH-ON trials

Authors: Gonzalez-Peralta, Regino P., Miethke, Alexander, Aqul, Amal A., Mogul, Douglas B., Nunes, Tiago, Garner, Will, Vig, Pamela, Thompson, Richard J.

Source: In Journal of Hepatology June 2024 80 Supplement 1:S323-S324

P-6 SOFOSBUVIR CONTAINING REGIMENS ARE SAFE AND EFFECTIVE IN ADOLESCENTS WITH CHRONIC HEPATITIS C INFECTION

Authors: Wirth, Stefan, Gonzalez-Peralta, Regino, Rosenthal, Philip, Hardikar, Winita, Wen, Jessica, Jonas, Maureen M., Mittal, Naveen, Whitworth, Mary, Arnon, Ronen, Lin, Chuan-Hao, Lobzin, Yury, Romero, Rene, Chulanov, Vladimir, Subbarao, Girish, Teckman, Jeffrey, Morozov, Vyacheslav, Bassetti, Eric, Kersey, Kathryn, Massetto, Benedetta, Zhu, Yanni, German, Polina, Brainard, Diana M., Bansal, Sanjay, Murray, Karen F., Schwarz, Kathleen, Balistreri, William

Source: In Annals of Hepatology September 2021 24 Supplement 1

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

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

Source: Hepatology Communications; Mar2023, Vol. 7 Issue 3, p1-11, 11p

Subject Terms: PHARMACOKINETICS, ADVERSE health care events, GENOTYPES, FATIGUE (Physiology)

Feasibility of using a patient‐reported outcome measure into clinical practice following pediatric liver transplantation: The Starzl Network experience.

Authors: Ng, Vicky Lee, Dunphy, Claire, Shemesh, Eyal, Lobritto, Steven, Eisenberg, Elizabeth, Pomponi, Claudia, Szolna, Jonathan, Wilkerson, Dawn, Gupta, Nitika, Romero, Rene, Perito, Emily R., DiPaola, Frank, Gonzalez‐Peralta, Regino P., Hsu, Evelyn, Saarela, Katelyn, Mohammad, Saeed, Superina, Riccardo, Logan, Sherrie, Miller, Daniel W., Krise‐Confair, Cassandra

Source: Pediatric Transplantation; Feb2023, Vol. 27 Issue 1, p1-13, 13p

Subject Terms: PATIENT reported outcome measures, LIVER transplantation, MOBILE app development, QUALITY of life, CLINICAL medicine

WED-282 - Analysis of safety in maralixibat-treated participants with progressive familial intrahepatic cholestasis: data from MARCH-PFIC

Authors: Miethke, Alexander, Moukarzel, Adib, Porta, Gilda, Esquer, Joshue Covarrubias, Czubkowski, Piotr, Ordoñez, Felipe, Candusso, Manila, Aqul, Amal A., Squires, Robert H., Sokal, Etienne, D’Agostino, Daniel, Baumann, Ulrich, D’Antiga, Lorenzo, Kasi, Nagraj, Laborde, Nolwenn, Arikan, Cigdem, Lin, Chuan-Hao, Gilmour, Susan, Mittal, Naveen, Chiou, Fang Kuan, Horslen, Simon P., Huber, Wolf-Dietrich, David-Feliciano, Susan, Chien, Elaine, Mogul, Douglas, Garner, Will, Nunes, Tiago, Lascau, Anamaria, Vig, Pamela, Hupertz, Vera, Gonzalez-Peralta, Regino, Ekong, Udeme, Hartley, Jane, Laverdure, Noemie, Ovchinsky, Nadia, Thompson, Richard

Source: In Journal of Hepatology June 2023 78 Supplement 1:S387-S388

LBP-35 - Long-term maintenance of response and improved liver health with maralixibat in patients with progressive familial intrahepatic cholestasis (PFIC): data from the MARCH-ON study

Authors: Miethke, Alexander, Moukarzel, Adib, Porta, Gilda, Esquer, Joshue Covarrubias, Czubkowski, Piotr, Ordonez, Felipe, Candusso, Manila, Aqul, Amal A., Squires, Robert H., Sokal, Etienne, D’Agostino, Daniel, Baumann, Ulrich, D’Antiga, Lorenzo, Kasi, Nagraj, Laborde, Nolwenn, Arıkan, Cigdem, Lin, Chuan-Hao, Gilmour, Susan, Mittal, Naveen, Chiou, Fang Kuan, Horslen, Simon P., Huber, Wolf-Dietrich, Nunes, Tiago, Lascau, Anamaria, Longpre, Lara, Mogul, Douglas, Aguilar, Raul, Vig, Pamela, Hupertz, Vera, Gonzalez-Peralta, Regino, Ekong, Udeme, Hartley, Jane, Laverdure, Noemie, Ovchinsky, Nadia, Thompson, Richard

Source: In Journal of Hepatology June 2023 78 Supplement 1:S123-S123