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3. International Liver Transplantation Society Global Census: First Look at Pediatric Liver Transplantation Activity Around the World

Author

Rodriguez-Davalos, Manuel I., Lopez-Verdugo, Fidel, Kasahara, Mureo, Muiesan, Paolo, Reddy, Mettu S., Flores-Huidobro Martinez, Angel, Xia, Qiang, Hong, Johnny C., Niemann, Claus U., Seda-Neto, Joao, Miloh, Tamir A., Yi, Nam-Joon, Mazariegos, George V., Ng, Vicky L., Esquivel, Carlos O., Lerut, Jan, Rela, Mohamed, Botha, Jean, Mehta, Naimish N., Bakar Hafeez Bhatti, Abu, Zamora-Valdes, Daniel, Anand, Superina, Riccardo, de Kleine, Ruben, Matamoros, Maria A., Bedregal-Mendoza, Tayana, Steve Bynon, J., Rodriguez-Sancho, Luis C., Langnas, Alan, Dhawan, Anil, Ogura, Yasuhiro, Vianna, Rodrigo, Ilyassova, Bibigul, Tantemsapya, Niramol, Bellemare, Sarah, Ovchinsky, Nadia, Bhalerao, Suryabhan, Balci, Deniz, Dip, Marcelo, Imventarza, Oscar, Mangus, Richard S., Fawcett, Jonathan, Antonini, Teresa M., Shrimal, Anurag, Zhu, Zhi-Jun, Rivera, Jairo, Dayangac, Murat, McCall, John, Utz Melere, Melina, Stormon, Michael, Mizuta, Koichi, Gao, Wei, Hening Rahayatri, Tri, Costaguta, Alejandro, Gautier, Sergei, Kazemi, Kourosh, Martinez-Castillo, Jorge, Apinyachon, Worapot, Rastogi, Mukul, Restrepo, Juan C., Takahito, Yagi, Ravichandra, Srinivasa, Vanin, Anabel, Vieira, Sandra, Luque, Carlos, Gondolesi, Gabriel, Hashimoto, Koji, Yazigi, Nada, Lebel, Sylvie, Mavis, Alisha, Arnon, Ronen, Chavez-Vargas, Ricardo, Elisofon, Scott, Desai, Dev M., Okajima, Hideaki, Chinnakotla, Srinath, Hsu, Evelyn, Reyes, Jorge, Orgoi, Sergelen, Raut, Vikram, Hee Oh, Seak, Valentino, Pamela, Emre, Sukru, Spada, Marco, Leung, Dan, Charco, Ramon, Ericzon, Bo-Goran, Fischer, Ryan, Del Rio-Martin, Juan V., Cillo, Umberto, Soliman, Hossam, Varela-Fascinetto, Gustavo, de Villa, Vanessa, Haberal, Mehmet, Covarrubias, Joshue, Colledan, Michele, de Ville de Goyet, Jean, Desai, Chirag, Sundaram, Shikha, Fronek, Jiri, Nadig, Satish N., Mali, Vidyadhar P., Tandoi, Francesco, Kohli, Rohit, Mohan, Neelam, Goyal, Neerav, Santhosh, Drisya, Debray, Dominique, Durmaz, Ozlem, De Bruyne, Ruth, Kaliciński, Piotr, Hierro, Loreto, Venick, Robert, Perito, Emily, Mozer-Glassberg, Yael, Chen, Chao-Long, Rand, Elizabeth, Nikeghbalian, Saman, Kelly, Deirdre, Boillot, Olivier, and Book, Linda S.

Published
2023
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4. Predictors of survival following liver transplantation for pediatric hepatoblastoma and hepatocellular carcinoma: Experience from the Society of Pediatric Liver Transplantation (SPLIT)

Author

Boster, Julia M., Superina, Riccardo, Mazariegos, George V., Tiao, Gregory M., Roach, Jonathan P., Lovell, Mark A., Greffe, Brian S., Yanni, George, Leung, Daniel H., Elisofon, Scott A., McDiarmid, Suzanne V., Gupta, Nitika A., Lobritto, Steven J., Lemoine, Caroline, Stoll, Janis M., Vitola, Bernadette E., Daniel, James F., Sayed, Blayne A., Desai, Dev M., Martin, Abigail E., Amin, Arpit, Anand, Ravinder, Anderson, Sarah G., and Sundaram, Shikha S.

Published
2022
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5. Early Graft Loss With Suspected Seventh‐Day Syndrome Following Pediatric Liver Transplantation.

Author

Hartjes, Kayla, Koo, Donna, Al‐Ibraheemi, Alyaa, Sweeny, Katherine F., Wehrman, Andrew, Elisofon, Scott, Lee, Christine K., Cuenca, Alex G., Kim, Heung Bae, and Lee, Eliza J.

Subjects
LIVER transplantation, SYNDROMES in children, LITERATURE reviews, CHILDREN'S literature, CLINICAL deterioration
Abstract

Introduction: Allograft dysfunction within the first week posttransplant is an uncommon but known complication following liver transplantation. Seventh‐Day Syndrome (7DS) is a rare complication of allograft dysfunction following liver transplantation characterized by the rapid clinical deterioration of a formerly well‐functioning allograft within the first week posttransplant. The etiology of 7DS is unknown, and treatment options remain limited. While cases of graft survival have been reported, the risk of mortality remains exceedingly high without urgent retransplantation. Methods: Patient data was retrospectively analyzed and a literature review performed. Results: We present a unique case of split liver transplantation into two pediatric recipients in which one recipient developed rapidly progressive graft failure approximately 1 week postoperatively requiring urgent retransplantation while the other recipient had an unremarkable postoperative course. Upon clinical manifestation of progressive graft failure, the patient was treated with thymoglobulin, rituximab, intravenous immunoglobulin, and plasmapheresis. Despite this, the patient's clinical status continued to decline and she underwent retransplantation 11 days following her initial liver transplant. Conclusion: Seventh‐Day Syndrome is a rare complication following liver transplantation that is associated with a high risk of morbidity and mortality. Our case adds to the limited literature on 7DS in children and is the first to report a comparative posttransplant clinical course in two recipients who received split grafts from the same donor. [ABSTRACT FROM AUTHOR]

Published
2024
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6. Safety and Immunogenicity of Live Viral Vaccines in a Multicenter Cohort of Pediatric Transplant Recipients

Author

Feldman, Amy G., primary, Beaty, Brenda L., additional, Ferrolino, Jose A., additional, Maron, Gabriela, additional, Weidner, Hillary K., additional, Ali, Saira A., additional, Bitterfeld, Leandra, additional, Boulware, Mary Alice, additional, Campbell, Kathleen M., additional, Carr, Emily, additional, Chapman, Shelley, additional, Chang, Yeh-Chung, additional, Cunningham, Ryan, additional, Dallas, Ronald H., additional, Dantuluri, Keerti L., additional, Domenick, Bryanna N., additional, Ebel, Noelle H., additional, Elisofon, Scott, additional, Fawaz, Rima, additional, Foca, Marc, additional, Gans, Hayley A., additional, Gopalareddy, Vani V., additional, Gu, Cindy, additional, Gupta, Nitika A., additional, Harmann, Katherine, additional, Hollenbeck, Jessica, additional, Huppler, Anna R., additional, Jaramillo, Catalina, additional, Kasi, Nagraj, additional, Kerkar, Nanda, additional, Lerret, Stacee, additional, Lobritto, Steven J., additional, Lopez, Maclovio J., additional, Marini, Elizabeth, additional, Mavis, Alisha, additional, Mehra, Sonia, additional, Moats, Lynnette, additional, Mohandas, Sindhu, additional, Munoz, Flor M., additional, Mysore, Krupa R., additional, Onsan, Ceren, additional, Ovchinsky, Nadia, additional, Perkins, Kerrigan, additional, Postma, Stacy, additional, Pratscher, Lauren, additional, Rand, Elizabeth B., additional, Rowe, Regina K., additional, Schultz, Danielle, additional, Sear, Katherine, additional, Sell, Megan L., additional, Sharma, Tanvi, additional, Stoll, Janis, additional, Vang, Mychoua, additional, Villarin, Dominique, additional, Weaver, Carly, additional, Wood, Phoebe, additional, Woodford-Berry, Olivia, additional, Yanni, George, additional, and Danziger-Isakov, Lara A., additional

Published
2023
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7. 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

9. Bilateral native nephrectomy to reduce oxalate stores in children at the time of combined liver-kidney transplantation for primary hyperoxaluria type 1

Author

Lee, Eliza, Ramos-Gonzalez, Gabriel, Rodig, Nancy, Elisofon, Scott, Vakili, Khashayar, and Kim, Heung Bae

Subjects
Hyperoxaluria -- Care and treatment -- Complications and side effects -- Patient outcomes, Pediatric diseases -- Care and treatment -- Complications and side effects -- Patient outcomes, Kidney transplantation -- Patient outcomes, Liver transplantation -- Patient outcomes, Nephrectomy -- Methods -- Patient outcomes, Oxalates -- Physiological aspects -- Health aspects, Health
Abstract

Objective Primary hyperoxaluria type-1 (PH-1) is a rare genetic disorder in which normal hepatic metabolism of glyoxylate is disrupted resulting in diffuse oxalate deposition and end-stage renal disease (ESRD). While most centers agree that combined liver-kidney transplant (CLKT) is the appropriate treatment for PH-1, perioperative strategies for minimizing recurrent oxalate-related injury to the transplanted kidney remain unclear. We present our management of children with PH-1 and ESRD on hemodialysis (HD) who underwent CLKT at our institution from 2005 to 2015. Methods On chart review, three patients (2 girls, 1 boy) met study criteria. Two patients received deceased-donor split-liver grafts, while one patient received a whole liver graft. All patients underwent bilateral native nephrectomy at transplant to minimize the total body oxalate load. Median preoperative serum oxalate was 72 [mu]mol/L (range 17.8-100). All patients received HD postoperatively until predialysis serum oxalate levels fell Conclusions While CLKT remains the definitive treatment for PH-1, bilateral native nephrectomy at the time of transplant reduces postoperative oxalate stores and may mitigate damage to the renal allograft., Author(s): Eliza Lee [sup.1] , Gabriel Ramos-Gonzalez [sup.2] , Nancy Rodig [sup.3] , Scott Elisofon [sup.4] , Khashayar Vakili [sup.2] , Heung Bae Kim [sup.2] Author Affiliations: (Aff1) 0000 0000 [...]

Published
2018
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11. Hematopoietic stem cell transplantation outcomes for 11 patients with dedicator of cytokinesis 8 deficiency

Author

Al-Herz, Waleed, Chu, Julia I., van der Spek, Jet, Raghupathy, Raj, Massaad, Michel J., Keles, Sevgi, Biggs, Catherine M., Cockerton, Lucinda, Chou, Janet, Dbaibo, Ghassan, Elisofon, Scott A., Hanna-Wakim, Rima, Kim, Heung Bae, Lehmann, Leslie E., McDonald, Douglas R., Notarangelo, Luigi D., Veys, Paul, Chatila, Talal A., Geha, Raif S., Gaspar, H. Bobby, and Pai, Sung-Yun

Published
2016
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14. Severe Acute Respiratory Syndrome Coronavirus-2 Infection in Children With Liver Transplant and Native Liver Disease: An International Observational Registry Study

Author

Kehar, Mohit, primary, Ebel, Noelle H., additional, Ng, Vicky L., additional, Baquero, Jairo Eduardo Rivera, additional, Leung, Daniel H., additional, Slowik, Voytek, additional, Ovchinsky, Nadia, additional, Shah, Amit A., additional, Arnon, Ronen, additional, Miloh, Tamir, additional, Gupta, Nitika, additional, Mohammad, Saeed, additional, Kogan-Liberman, Debora, additional, Squires, James E., additional, Sanchez, Maria Camila, additional, Hildreth, Amber, additional, Book, Linda, additional, Chu, Christopher, additional, Alrabadi, Leina, additional, Azzam, Ruba, additional, Chepuri, Bhavika, additional, Elisofon, Scott, additional, Falik, Rachel, additional, Gallagher, Lisa, additional, Kader, Howard, additional, Mogul, Douglas, additional, Mujawar, Quais, additional, Namjoshi, Shweta S., additional, Valentino, Pamela L., additional, Vitola, Bernadette, additional, Waheed, Nadia, additional, Zheng, Ming-Hua, additional, Lobritto, Steven, additional, and Martinez, Mercedes, additional

Published
2021
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15. Contributors

Author

Adams, Paul C., primary, Albrecht, Helmut, additional, Bajaj, Jasmohan, additional, Baik, Soon Koo, additional, Beuers, Ulrich, additional, Biggins, Scott W., additional, Bismuth, Henri, additional, Boberg, Kirsten Muri, additional, Bonkovsky, Herbert L., additional, Bosch, Jaime, additional, Boyer, Thomas D., additional, Brunt, Elizabeth M., additional, Caselitz, Martin, additional, Caselmann, Wolfgang H., additional, Cave, Matt, additional, Chan, Henry Lik-Yuen, additional, Chang, Kyong-Mi, additional, Ciancio, Alessia, additional, Colombo, Massimo, additional, Conjeevaram, Hari S., additional, Cox, Diane W., additional, Day, Chris P., additional, Doctor, R. Brian, additional, Oude Elferink, Ronald P.J., additional, Elisofon, Scott A., additional, El-Serag, Hashem B., additional, Esmat, Gamal, additional, Everson, Gregory T., additional, Falkner, Keith Cameron, additional, Fallon, Michael B., additional, Feng, Sandy, additional, Fischer, Hans-Peter, additional, Fortune, Brett E., additional, Friedman, Scott L., additional, García-Pagán, J.C., additional, Garcia-Tsao, Guadalupe, additional, Ghishan, Fayez K., additional, Gores, Gregory J., additional, Gruessner, Rainer W., additional, Gupta, Sanjeev, additional, Hammoud, Ghassan M., additional, Heathcote, E.J., additional, Helmke, Steve M., additional, Hirschfield, G.M., additional, Hunt, Douglas, additional, Iavarone, Massimo, additional, Ibdah, Jamal A., additional, Imbert-Bismut, Françoise, additional, Jansen, Peter L.M., additional, Johnson, Benjamin F., additional, F. Jonas, Maureen M., additional, Jones, Dean P., additional, Kamath, Patrick S., additional, Karlsen, Tom H., additional, Kelly, Deirdre, additional, Kettelle, J., additional, Khan, Khalid M., additional, Knolle, Percy, additional, Kramer, David J., additional, Kumar, Manoj, additional, Kumar, Navaneeth C., additional, Lai, Jennifer C., additional, Lapointe, Réal, additional, Lazaridis, Konstantinos N., additional, Lechel, André, additional, Lee, Samuel S., additional, Lencioni, Riccardo, additional, Levy, Cynthia, additional, Lindor, Keith D., additional, Locarnini, Stephen, additional, Lok, Anna S.F., additional, Malhi, Harmeet, additional, Manns, Michael P., additional, Marrero, Jorge A., additional, McClain, Craig, additional, McCuskey, Robert, additional, McGovern, Barbara H., additional, McHutchison, John G., additional, Moradpour, Darius, additional, Navarro, Victor J., additional, Neuberger, James, additional, Nevah R., Moises I., additional, Nevzorova, Yulia A., additional, Patton, Heather M., additional, Penin, François, additional, Perlmutter, David, additional, Plessier, Aurélie, additional, Poynard, Thierry, additional, Puri, Puneet, additional, Rice, Charles, additional, Rizzetto, Mario, additional, Roberts, Eve A., additional, Rockey, Don C., additional, Roy-Chowdhury, Jayanta, additional, Roy-Chowdhury, Namita, additional, Rudolph, K. Lenhard, additional, Russo, Mark W., additional, Saab, Sammy, additional, Sanyal, Arun J., additional, Sarin, S.K., additional, Schrumpf, Erik, additional, Seeff, Leonard B., additional, Seijo, S., additional, Shah, Vijay H., additional, Shedlofsky, Steven I., additional, Shouval, Daniel, additional, Sirlin, Claude B., additional, Smith, Maxwell L., additional, Smorodinsky, Emmanuil, additional, Spengler, Ulrich, additional, Sterling, Richard K., additional, Stewart, Stephen F., additional, Strader, Doris B., additional, Strassburg, Christian P., additional, Stravitz, R. Todd, additional, Sud, Priti, additional, Sulkowski, Mark S., additional, Talwalkar, Jayant A., additional, Terrault, Norah A., additional, Tillmann, Hans L., additional, Trautwein, Christian, additional, Vagefi, Parsia A., additional, Valla, Dominique, additional, Wagner, Siegfried, additional, Warner, Nadia, additional, Wong, Vincent Wai-Sun, additional, and Zayed, Naglaa, additional

Published
2012
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20. The re‐occurrence of cardiomyopathy in propionic acidemia after liver transplantation

Author

Berry, Gerard T., primary, Blume, Elizabeth D., additional, Wessel, Ann, additional, Singh, Tajinder, additional, Hecht, Leah, additional, Marsden, Deborah, additional, Sahai, Inderneel, additional, Elisofon, Scott, additional, Ferguson, Michael, additional, Kim, Heung Bae, additional, Harris, David J., additional, Demirbas, Didem, additional, Almuqbil, Mohammed, additional, and Nyhan, William L., additional

Published
2020
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22. Contributors

Author

Adams, David, primary, Adams, Sherri L., additional, Agarwal, Chhavi, additional, Alpern, Elizabeth R., additional, Matheny Antommaria, Armand H., additional, Bair-Merritt, Megan H., additional, Baptista-Neto, Lourival, additional, Baren, Jill, additional, Baum, Carl R., additional, Baum, Eric D., additional, Beasley, Pamela J., additional, Beno, Suzanne, additional, Bernard, Laurie A., additional, Bernstein, Stacey E., additional, Brands, Chad K., additional, Brennan, Laura K., additional, Brett-Fleegler, Marisa B., additional, Butte, Manish J., additional, Byerley, Julie Story, additional, Calello, Diane P., additional, Caplin, Deirdre, additional, Carlisle, Rebecca G., additional, Carlson, Douglas W., additional, Carroll, Jean Marie, additional, Chang, Mary Wu, additional, Cheng, Grace M., additional, Chidekel, Aaron S., additional, Chitkara, Denesh K., additional, Chiu, Bill, additional, Cho, Christine S., additional, Chow, Jeanne S., additional, Cilento, Bartley G., additional, Coffin, Susan E., additional, Cohen, Bernard A., additional, Cole, Kristina A., additional, Conway, Patrick H., additional, Cooper, Maura, additional, Cornell, Timothy, additional, Cronan, Kate M., additional, Cross, Catherine, additional, Cunningham, Bari B., additional, Cunningham, Melody J., additional, Daru, Jennifer A., additional, Davis, Ian J., additional, Deardorff, Matthew A., additional, Degar, Barbara, additional, DelVecchio, Michael, additional, DeMaso, David Ray, additional, Ungria, Marissa de, additional, Dewar, Stephanie B., additional, DeWolfe, Craig C., additional, Dimmers, Martha, additional, Dinulos, James G.H., additional, Donovan, Ed, additional, Dooley, Kenneth J., additional, Doyne, Emmanuel, additional, Duncan, Christine N., additional, Egan, Marie, additional, Eichenfield, Lawrence F., additional, El-hallak, Moussa, additional, Elisofon, Scott A., additional, Eppes, Stephen C., additional, Ewald, Michele Burns, additional, Farah, Mirna M., additional, Feudtner, Chris, additional, Fine, Andrew M., additional, Frangiskakis, Susan Hetzel, additional, Frank, Gary, additional, Frehm, Eric, additional, Frei, Nicole R., additional, Frieden, Ilona J., additional, Friedlaender, Eron Y., additional, Friedman, Jeremy, additional, Fryer, Robert Hugh, additional, Fulton, David R., additional, Galardy, Paul J., additional, Galashan, Mirabai, additional, Gallagher, Mary Pat, additional, Gamulka, Beth D., additional, Gandhi, Rupali, additional, Garza, Mary B., additional, Garzon, Maria C., additional, Geggel, Robert L., additional, Gewitz, Michael H., additional, Gibson, Timothy, additional, Gilliam, Amy E., additional, Ginnis, Katherine B., additional, Goldberg, Amy, additional, Golja, Anna M., additional, Gregory, Melissa J., additional, Harper, April A., additional, Harris, Mary Catherine, additional, Hayes, Natalie, additional, Heeney, Matthew M., additional, Heinzman, Diana M., additional, Heltzer, Meredith Lee, additional, Herzog, Keith D., additional, Hill, Malinda Ann, additional, Hills, Jessica L., additional, Hoberman, Alejandro, additional, Hoehn, K. Sarah, additional, Hoffman, Amber M., additional, Hoffman, Robert J., additional, Holst, Amy P., additional, Homer, Charles J., additional, Honig, Paul J., additional, Hopkins, Patricia M., additional, Hormann, Mark D., additional, Horn, B. David, additional, Isakoff, Michael S., additional, Janeway, Katherine A., additional, Jin, Katherine Ahn, additional, Jonas, Maureen M., additional, Kang, Tammy, additional, Keilty, Krista, additional, Keren, Ron, additional, Kharbanda, Anupam, additional, Kiesau, Marin, additional, Kim, Caroline C., additional, Kim, Jason Y., additional, Kim, Juliann Lipps, additional, Klein, Nicola, additional, Kleinman, Paul K., additional, Korin, Joel B., additional, Kotagal, Uma, additional, Kresnicka, Lisa K., additional, Kronfol, Rana N., additional, Kuelbs, Cynthia L., additional, Kugathasan, Subra, additional, Kurbegov, Amethyst C., additional, Landrigan, Christopher P., additional, Laufer, Miriam, additional, Lauren, Christine, additional, Lebovitz, Daniel J., additional, Leibel, Natasha, additional, Leung, Lucinda P., additional, Levine, Leonard J., additional, Levy, Jason A., additional, Lewis, Phyllis A., additional, Liang, Marilyn G., additional, Licht, Daniel J., additional, Long, Carolyn M., additional, Louie, Jeffrey P., additional, Love, Barry A., additional, Lowery, Patricia V., additional, MacLusky, Ian B., additional, Madejczyk, Katarzyna, additional, Madonna, Mary Beth, additional, Mahant, Sanjay, additional, Manicone, Paul E., additional, Maniscalco, Jennifer, additional, Mann, Keith, additional, Mannix, Rebekah, additional, Mansbach, Jonathan M., additional, Mattei, Peter, additional, Mayer, Oscar H., additional, McBride, Sarah C., additional, McBryde, Kevin D., additional, McKee, Michele R., additional, McNett, William, additional, Melzer, Sanford M., additional, Metjian, Talene A., additional, Metry, Denise W., additional, Muething, Stephen E., additional, Milliken, Emily E., additional, Mirkinson, Laura J., additional, Mittal, Manoj K., additional, Mix, Angela C., additional, Monzack, Debra, additional, Morel, Kimberly D., additional, Moses, Douglas E., additional, Mowad, Eugene M., additional, Mullen, Elizabeth A., additional, Mulliken, John B., additional, Muret-Wagstaff, Sharon, additional, Murphy, Nancy, additional, Nadel, Frances M., additional, Nagler, Joshua, additional, Nard, James A., additional, Neuman, Mark I., additional, Newland, Jason G., additional, Newton, Alice W., additional, Nichol, Peter F., additional, Nigrovic, Lise E., additional, Noel, Richard J., additional, Oberfield, Sharon E., additional, O'Brien, Maureen M., additional, O'Connell, Karen J., additional, Osterhoudt, Kevin C., additional, Ottolini, Mary, additional, Padman, Raj, additional, Padua, Horacio M., additional, Patel, Alka, additional, Pati, Susmita, additional, Percelay, Jack M., additional, Perez-Rossello, Jeannette M., additional, Phelan, Kieran J., additional, Poduri, Annapurna, additional, Poley, J. Rainer, additional, Posner, Jill C., additional, Prahalad, Sampath, additional, Pride, Howard B., additional, Rauch, Daniel, additional, Rawat, David J., additional, Reeves, Scott, additional, Reirden, Daniel H., additional, Roberts, Brandie J., additional, Rodgers, Jack, additional, Romero, José R., additional, Rosen, Paul, additional, Rubin, David M., additional, Sampayo, Esther Maria, additional, Samson-Fang, Lisa, additional, Santucci, Gina, additional, Schaffer, Julie V., additional, Schetzina, Karen E., additional, Schwab, Sandra, additional, Schwarz, Donald F., additional, Scott, Jordan, additional, Selbst, Steven M., additional, Shah, Kara N., additional, Shah, Samir S., additional, Shaikh, Nader, additional, Shannon, Michael W., additional, Sharma, Adhi N., additional, Siberry, George K., additional, Smith, Karen, additional, Smith, Michael J., additional, Somers, Michael J.G., additional, Sondheimer, Neal, additional, Spalding, Steven J., additional, Spandorfer, Philip R., additional, Spergel, Jonathan M., additional, Sperring, Jeffrey L., additional, Spiegel, David A., additional, Srivastava, Rajendu, additional, St. John, Keith H., additional, Stephens, Michael C., additional, Stewart, Christopher C., additional, Stone, Bryan L., additional, Stucky, Erin R., additional, Sundel, Eric R., additional, Sundel, Robert, additional, Swanson, Suzanne, additional, Taylor, Lesli, additional, Thompson, E. Douglas, additional, Traum, Avram Z., additional, Trivedi, Harsh K., additional, Upham, Bryan D., additional, Vandeven, Andrea M., additional, Vaughan, Brigid L., additional, Venditti, Charles P., additional, Villalva, Venus M., additional, Vincent, Robert N., additional, Volchenboum, Samuel, additional, Vossmeyer, Michael T., additional, Wachter, Robert M., additional, Weiner, Daniel J., additional, Weinstein, Michael, additional, Wharff, Elizabeth A., additional, Wilson, Stephen D., additional, Winkelstein, Jerry A., additional, Wolf, Heidi, additional, Woodward, George A., additional, Yan, Albert C., additional, Zackai, Elaine H., additional, Zaenglein, Andrea L., additional, Zaoutis, Theoklis E., additional, and Zipes, David, additional

Published
2007
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31. Hematopoietic Stem Cell Transplantation Outcomes for 11 Patients with Dedicator of Cytokinesis 8 (DOCK8) Deficiency

Author

Al-Herz, Waleed, Chu, Julia I., van der Spek, Jet, Raghupathy, Raj, Massaad, Michel J., Keles, Sevgi, Biggs, Catherine M., Cockerton, Lucinda, Chou, Janet, Dbaibo, Ghassan, Elisofon, Scott A., Hanna-Wakim, Rima, Kim, Heung Bae, Lehmann, Leslie E., McDonald, Douglas R., Notarangelo, Luigi D., Veys, Paul, Chatila, Talal, Geha, Raif S., Gaspar, H. Bobby, and Pai, Sung-Yun

Subjects
Male, B-Lymphocytes, T-Lymphocytes, Hematopoietic Stem Cell Transplantation, Immunologic Deficiency Syndromes, Article, Treatment Outcome, Child, Preschool, Leukocytes, Mononuclear, Cytokines, Guanine Nucleotide Exchange Factors, Humans, Female, Child
Abstract

Dedicator of cytokinesis 8 (DOCK8) deficiency can be cured by allogeneic hematopoietic stem cell transplantation (HSCT). Reports of outcomes are still limited.We sought to analyze the results of HSCT in patients with DOCK8 deficiency and report whether approaches resulting in mixed chimerism result in clinically relevant immune reconstitution.We performed a retrospective chart review of 11 patients with DOCK8 deficiency and measured DOCK8 expression and cytokine production.Of 11 patients, 7 received HSCT from related and 4 from unrelated donors; 9 patients received busulfan-based conditioning regimens. Survival was excellent (10 [91%] of 11 patients alive), including a patient who had undergone liver transplantation. Patients showed significant improvements in the frequency and severity of infections. Although eczema resolved in all, food allergies and high IgE levels persisted in some patients. Lymphopenia, eosinophilia, low numbers of naive CD8(+) T cells and switched memory B cells, and TH1/TH2 cytokine imbalance improved in most patients. Although the 8 matched related or unrelated donor recipients had full donor chimerism, all 3 recipients of mismatched unrelated donor HSCT had high levels of donor T-cell chimerism and low B-cell and myeloid cell chimerism (0% to 46%). Almost all switched memory B cells were of donor origin. All patients, including those with mixed chimerism, mounted robust antibody responses to vaccination.Allogeneic HSCT ameliorated the infectious and atopic symptoms of patients with DOCK8 deficiency. In patients with mixed chimerism, selective advantage for donor-derived T cells and switched memory B cells promoted restoration of cellular and humoral immunity and protection against opportunistic infection.

Published
2016

32. Treatment and Outcomes of Immune Cytopenias Following Solid Organ Transplant in Children

Author

Schoettler, Michelle, Elisofon, Scott A, Kim, Heung Bae, Blume, Elizabeth D., Rodig, Nancy, Boyer, Debra, Neufeld, Ellis J, and Grace, Rachael F

Subjects
Male, Neutropenia, Infant, Organ Transplantation, Thrombocytopenia, Article, Tacrolimus, Treatment Outcome, Child, Preschool, Humans, Immunologic Factors, Female, Anemia, Hemolytic, Autoimmune, Child, Rituximab, Immunosuppressive Agents, Retrospective Studies
Abstract

Immune cytopenias are a recognized life-threatening complication following pediatric solid organ transplants (SOT), but treatment responses and overall outcome are not well described. The aim of this study was to evaluate the demographic characteristics, response to treatments, and outcomes of a cohort of patients who developed immune cytopenias following SOT.In this single center retrospective review, patients with immune cytopenias after SOT were identified by electronic medical record (EMR) search and transplant databases from 1995-2012.Of 764 SOT patients, 19 (2.4%) developed immune cytopenias. Incidence varied widely by transplant type from 1.2% (renal) to 23.5% (multivisceral). Autoimmune hemolytic anemia (AIHA) was the most common immune cytopenia. Overall median time from transplant to immune cytopenia was 8 m and varied by transplant type from 3 m (liver) to 74 m (heart). Standard therapies for immune cytopenias were often used and ineffective. The most effective therapy for the immune cytopenia was changing immunosuppression from tacrolimus to another agent. Three of 19 patients died; none directly attributed to the immune cytopenia.Immune cytopenias are not rare after SOT, and patients usually do not respond well to traditional first line therapies. Provided that the risk of organ rejection is otherwise manageable, temporary cessation of tacrolimus could be more widely explored in this challenging clinical context. Pediatr Blood Cancer 2015;62:214-218. © 2014 Wiley Periodicals, Inc.

Published
2014

36. A Prospective Clinical Trial Shows That Intravenous N-Acetylcysteine (NAC) Does Not Improve Survival in Pediatric Patients With Non-Acetaminophen Acute Liver Failure

Author

Squires, Robert, primary, Dhawan, Anil, additional, Alonso, Estella M., additional, Narkewicz, Michael R., additional, Rodriguez-Baez, Norberto, additional, Kelly, Deirdre A., additional, Bucuvalas, John, additional, Karpen, Saul J., additional, Shneider, Benjamin L., additional, Rand, Elizabeth B., additional, Lobritto, Steven J., additional, Rosenthal, Philip, additional, Murray, Karen F., additional, Kerkar, Nanda, additional, Ng, Vicky L., additional, Subbarao, Girish, additional, Rudnick, David A., additional, Lopez, M. James, additional, Schwarz, Kathleen B., additional, Romero, Rene, additional, Elisofon, Scott, additional, Robuck, Patricia R., additional, Lawlor, Sharon M., additional, and Belle, Steven H., additional

Published
2011
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45. Society of pediatric liver transplantation: Current registry status 2011‐2018.

Author

Elisofon, Scott A., Magee, John C., Ng, Vicky L., Horslen, Simon P., Fioravanti, Vicki, Economides, Julie, Erinjeri, Jinson, Anand, Ravinder, Mazariegos, George V., Dunn, S., Martin, A., Mannino, D., Flynn, L., Mohammad, S., Alonso, E., Superina, R., Brandt, K., Riordan, M., Lokar, J., and Ito, J.

Subjects
*LIVER transplantation, *PANCREAS transplantation, *SURGICAL complications, *BILIARY tract, *BILIARY atresia, *HEPATIC artery
Abstract

Background: SPLIT was founded in 1995 in order to collect comprehensive prospective data on pediatric liver transplantation, including waiting list data, transplant, and early and late outcomes. Since 2011, data collection of the current registry has been refined to focus on prospective data and outcomes only after transplant to serve as a foundation for the future development of targeted clinical studies. Objective: To report the outcomes of the SPLIT registry from 2011 to 2018. Methods: This is a multicenter, cross‐sectional analysis characterizing patients transplanted and enrolled in the SPLIT registry between 2011 and 2018. All patients, <18 years of age, received a first liver‐only, a combined liver‐kidney, or a combined liver‐pancreas transplant during this study period. Results: A total of 1911 recipients from 39 participating centers in North America were registered. Indications included biliary atresia (38.5%), metabolic disease (19.1%), tumors (11.7%), and fulminant liver failure (11.5%). Greater than 50% of recipients were transplanted as either Status 1A/1B or with a MELD/PELD exception score. Incompatible transplants were performed in 4.1%. Kaplan‐Meier estimates of 1‐year patient and graft survival were 97.3% and 96.6%. First 30 days of surgical complications included reoperation (31.7%), hepatic artery thrombosis (6.3%), and portal vein thrombosis (3.2%). In the first 90 days, biliary tract complications were reported in 13.6%. Acute cellular rejection during first year was 34.7%. At 1 and 2 years of follow‐up, 39.2% and 50.6% had normal liver tests on monotherapy (tacrolimus or sirolimus). Further surgical, survival, allograft function, and complications are detailed. [ABSTRACT FROM AUTHOR]

Published
2020
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47. Contributors

Author

Adams, Paul C., Albrecht, Helmut, Bajaj, Jasmohan, Baik, Soon Koo, Beuers, Ulrich, Biggins, Scott W., Bismuth, Henri, Boberg, Kirsten Muri, Bonkovsky, Herbert L., Bosch, Jaime, Boyer, Thomas D., Brunt, Elizabeth M., Caselitz, Martin, Caselmann, Wolfgang H., Cave, Matt, Chan, Henry Lik-Yuen, Chang, Kyong-Mi, Ciancio, Alessia, Colombo, Massimo, Conjeevaram, Hari S., Cox, Diane W., Day, Chris P., Doctor, R. Brian, Oude Elferink, Ronald P.J., Elisofon, Scott A., El-Serag, Hashem B., Esmat, Gamal, Everson, Gregory T., Falkner, Keith Cameron, Fallon, Michael B., Feng, Sandy, Fischer, Hans-Peter, Fortune, Brett E., Friedman, Scott L., García-Pagán, J.C., Garcia-Tsao, Guadalupe, Ghishan, Fayez K., Gores, Gregory J., Gruessner, Rainer W., Gupta, Sanjeev, Hammoud, Ghassan M., Heathcote, E.J., Helmke, Steve M., Hirschfield, G.M., Hunt, Douglas, Iavarone, Massimo, Ibdah, Jamal A., Imbert-Bismut, Françoise, Jansen, Peter L.M., Johnson, Benjamin F., F. Jonas, Maureen M., Jones, Dean P., Kamath, Patrick S., Karlsen, Tom H., Kelly, Deirdre, Kettelle, J., Khan, Khalid M., Knolle, Percy, Kramer, David J., Kumar, Manoj, Kumar, Navaneeth C., Lai, Jennifer C., Lapointe, Réal, Lazaridis, Konstantinos N., Lechel, André, Lee, Samuel S., Lencioni, Riccardo, Levy, Cynthia, Lindor, Keith D., Locarnini, Stephen, Lok, Anna S.F., Malhi, Harmeet, Manns, Michael P., Marrero, Jorge A., McClain, Craig, McCuskey, Robert, McGovern, Barbara H., McHutchison, John G., Moradpour, Darius, Navarro, Victor J., Neuberger, James, Nevah R., Moises I., Nevzorova, Yulia A., Patton, Heather M., Penin, François, Perlmutter, David, Plessier, Aurélie, Poynard, Thierry, Puri, Puneet, Rice, Charles, Rizzetto, Mario, Roberts, Eve A., Rockey, Don C., Roy-Chowdhury, Jayanta, Roy-Chowdhury, Namita, Rudolph, K. Lenhard, Russo, Mark W., Saab, Sammy, Sanyal, Arun J., Sarin, S.K., Schrumpf, Erik, Seeff, Leonard B., Seijo, S., Shah, Vijay H., Shedlofsky, Steven I., Shouval, Daniel, Sirlin, Claude B., Smith, Maxwell L., Smorodinsky, Emmanuil, Spengler, Ulrich, Sterling, Richard K., Stewart, Stephen F., Strader, Doris B., Strassburg, Christian P., Stravitz, R. Todd, Sud, Priti, Sulkowski, Mark S., Talwalkar, Jayant A., Terrault, Norah A., Tillmann, Hans L., Trautwein, Christian, Vagefi, Parsia A., Valla, Dominique, Wagner, Siegfried, Warner, Nadia, Wong, Vincent Wai-Sun, and Zayed, Naglaa

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