Bio


Dr. Ebel is the inaugural Chief of the Section of Hepatology and the Co-Director of the Abdominal Organ Transplant Center of Emphasis.

Dr. Ebel's clinical and research interests lie at the intersection of the heart and the liver, specifically for Fontan associated liver disease and indications for combined heart-liver transplantation. She is the Director of the Alagille Syndrome Program at Stanford and supports a growing international referral program for patients with Alagille syndrome and their complex heart and liver needs.
https://www.stanfordchildrens.org/en/service/alagille-syndrome

Dr. Ebel is the Director of Policy in the Office of Child Health Equity at Stanford and is committed to eliminating health disparities for children with liver disease on both an institutional and national level. She works to galvanize pediatric subspecialists to engage in health policy and health equity work to assure equal care for all children. Her current work centers on equitable access in living donor liver transplantation.
https://tts.org/split-advocacy

Dr. Ebel most recently received early career investigator awards from the American Association for the Study of Liver Diseases (AASLD) and the International Pediatric Transplant Association (IPTA).

She can be found on social media: @NoelleEbelMD (Twitter)

Clinical Focus


  • Pediatric Transplant Hepatology

Academic Appointments


Administrative Appointments


  • Chief of the Section of Hepatology, Division of Pediatric Gastroenterology, Hepatology and Nutrition (2023 - Present)
  • Co-Director of the Transplant Center of Emphasis, Stanford Medicine Children's Health (2023 - Present)
  • Director of Policy, Office of Child Health Equity, Lucile Packard Children's Hospital, Stanford University (2021 - Present)
  • Invited Member, Commission on Justice and Equity, Stanford Medicine (2020 - 2021)
  • Founder, GI Advocacy Group, Division of Pediatric Gastroenterology, Hepatology and Nutrition, Stanford University (2020 - Present)
  • Program Director of the Transplant Hepatology Fellowship Program, Division of Pediatric Gastroenterology, Hepatology and Nutrition, Stanford University (2020 - 2024)
  • Director of the Alagille Syndrome Program, Lucile Packard Children's Hospital, Stanford University (2019 - Present)

Honors & Awards


  • Future Leaders Career Development Award (1 recipient biennially), International Pediatric Transplant Association (IPTA) (3/2022)
  • Leonard B. Seeff Award for Outstanding Research by an Early Career Investigator (1 annual recipient), American Association for the Study of Liver Diseases (AASLD) (11/2021)
  • Young Investigator Travel Award, Society of Pediatric Liver Transplantation (SPLIT) (2019)
  • Advanced/Transplant Hepatology Fellowship Award, American Association for the Study of Liver Diseases (AASLD) (7/2017-7/2018)
  • Travel Award, Society of Pediatric Liver Transplantation (SPLIT) (2017)
  • Council Award to examine Hepatic Artery Thrombosis following Pediatric Liver Transplantation, Society of Pediatric Liver Transplantation (SPLIT) (9/2016-9/2017)
  • T32 Institutional National Research Service Award, NIH (3/2016-3/2017)
  • Young Investigator Travel Award, American Association of the Study of Liver Diseases (AASLD) (2016)
  • Research Grant to support examination of growth differences in children with VEO-IBD, University of California, San Francisco (6/2013-6/2014)
  • Patient Care Fund Award, UCSF, Benioff Children's Hospital (2013)
  • Patient Care Fund Award, San Francisco General Hospital (2013)
  • Teaching and Tomorrow Travel Award, NASPGHAN (2012)
  • Excellence in Pediatrics Award, Boston University School of Medicine (2011)
  • Gold Humanism Honor Society, Boston University School of Medicine (2010)
  • Master's Thesis Honors, Boston University School of Medicine (2007)
  • Arnold Award, Anatomy course, Boston University School of Medicine (2007)

Boards, Advisory Committees, Professional Organizations


  • Hepatology Special Interest Group Member, North American Society of Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) (2019 - Present)
  • Member, Society of Pediatric Liver Transplantation (SPLIT) (2019 - Present)
  • Member, American Association for the Study of Liver Diseases (AASLD) (2015 - Present)
  • Member, North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) (2014 - Present)
  • Member, American Academy of Pediatrics (AAP) (2011 - Present)

Professional Education


  • Board Certification: American Board of Pediatrics, Pediatric Transplant Hepatology (2018)
  • Fellowship: UCSF Pediatric Transplant Hepatology (2018) CA
  • Board Certification: American Board of Pediatrics, Pediatric Gastroenterology (2017)
  • Fellowship: Seattle Children's Hospital Pediatric Gastroenterology Fellowship (2017) WA
  • Residency: UCSF Pediatric Residency (2014) CA
  • Medical Education: Boston University School of Medicine (2011) MA

Current Research and Scholarly Interests


Current projects include:
-indications for combined heart-liver transplantation
-mitigating perioperative bleeding during cardiac surgery in children with Alagille syndrome
-congenital heart disease and liver transplantation
-subspecialty advocacy

Clinical Trials


  • A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS). Recruiting

    This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children <12 months of age with Alagille Syndrome [ALGS] or Progressive Familial Intrahepatic Cholestasis [PFIC].

    View full details

  • Decreasing Hemorrhage Risk in Children With Alagille Syndrome Recruiting

    The goal of this interventional study is to test a hemostasis screening protocol and cardiac peri-procedural and post-operative hemostasis pathway to improving bleeding complications and improve patient survival for children with Alagille syndrome and complex cardiac conditions. The main questions it aims to answer are: - Are children with Alagille syndrome with cardiac anomalies more likely to have acquired von Willebrand syndrome (a condition that causes increased bleeding) - Does implementation of a novel screening protocol to detect pre-operative bleeding conditions decrease intra-operative and/or post-operative bleeding complications and mortality risk? - Does implementation of a novel screening protocol to detect and treat bleeding conditions cause thrombotic complications? Participants will undergo additional hematology and bleeding disorder screening prior to cardiac surgery. They will additionally undergo a detailed family screening for a history of bleeding by a genetic counselor. Researchers will compare these findings with children who have similar complex cardiac conditions requiring surgery, but who do not have Alagille syndrome to see if bleeding conditions and complications are more or less common in children with Alagille syndrome.

    View full details

Graduate and Fellowship Programs


  • Gastroenterology & Hepatology (Fellowship Program)

All Publications


  • Anti-spike antibody durability after SARS-CoV-2 vaccination in adolescent solid organ transplant recipients PEDIATRIC TRANSPLANTATION McAteer, J., Kalluri, D. D., Abedon, R. R., Qin, C. X., Auerbach, S. R., Charnaya, O., Danziger-Isakov, L. A., Ebel, N. H., Feldman, A. G., Hsu, E. K., Mohammad, S., Perito, E. R., Thomas, A. M., Chiang, T. Y., Garonzik-Wang, J. M., Segev, D. L., Werbel, W. A., Mogul, D. B. 2024; 28 (1)

    View details for DOI 10.1111/petr.14671

    View details for Web of Science ID 001145549300001

  • Anti-spike antibody durability after SARS-CoV-2 vaccination in adolescent solid organ transplant recipients. Pediatric transplantation McAteer, J., Kalluri, D. D., Abedon, R. R., Qin, C. X., Auerbach, S. R., Charnaya, O., Danziger-Isakov, L. A., Ebel, N. H., Feldman, A. G., Hsu, E. K., Mohammad, S., Perito, E. R., Thomas, A. M., Chiang, T. P., Garonzik-Wang, J. M., Segev, D. L., Werbel, W. A., Mogul, D. B. 2024; 28 (1): e14671

    Abstract

    Adolescent solid organ transplant recipients (aSOTRs) who received three doses of the COVID-19 mRNA vaccine experience high seroconversion rates and antibody persistence for up to 3 months. Long-term antibody durability beyond this timeframe following three doses of the SARS-CoV-2 mRNA vaccine remains unknown. We describe antibody responses 6 months following the third vaccine dose (D3) of the BNT162b2 mRNA vaccination among aSOTRs.Participants in a multi-center, observational cohort who received the third dose of the vaccine were analyzed for antibodies to the SARS-CoV-2 spike protein receptor-binding domain (Roche Elecsys anti-SARS-CoV-2-S positive: ≥0.8, maximum: >2500 U/mL). Samples were collected at 1-, 3-, and 6-months post-D3. Participants were surveyed at each timepoint and at 12-months post-D3.All 34 participants had positive anti-RBD antibody titers 6 months post-D3. Variations in titers occurred between 3 and 6 months post-D3, with 8/28 (29%) having decreased antibody levels at 6 months compared to 3 months and 2/28 (7%) reporting increased titers at 6 months. The remaining 18/28 (64%) had unchanged antibody titers compared to 3-month post-D3 levels. A total of 4/34 (12%) reported breakthrough infection within 6 months and 3/32 (9%) reported infection after 6-12 months following the third dose of the SARS-CoV-2 mRNA vaccine.The results suggest that antibody durability persists up to 6 months following three doses of the SARS-CoV-2 mRNA in aSOTRs. Demography and transplant characteristics did not differ for those who experienced antibody weaning. Breakthrough infections did occur, reflecting immune-evasive nature of novel variants such as Omicron.

    View details for DOI 10.1111/petr.14671

    View details for PubMedID 38317335

  • Caregivers' and providers' perspectives of social and medical care after pediatric liver transplant: Results from the multi-center SOCIAL-Tx study. Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society Wadhwani, S. I., Alvarado, A., Shifman, H. P., Bautista, B., Yalung, J., Squires, J. E., Campbell, K., Ebel, N. H., Hsu, E., Vittorio, J., Zielsdorf, S., Desai, D. M., Bucuvalas, J. C., Gottlieb, L., Kotagal, U., Lyles, C. R., Ackerman, S. L., Lai, J. C. 2023

    Abstract

    Disparities exist in pediatric liver transplant (LT). We characterized barriers and facilitators to providing transplant and social care within pediatric LT clinics.This was a multi-center qualitative study. We oversampled caregivers reporting household financial strain, material economic hardship, or demonstrating poor health literacy. We also enrolled transplant team members. We conducted semi-structured interviews with participants. Caregiver interviews focused on challenges addressing transplant and household needs. Transplant provider interviews focused on barriers and facilitators to providing social care within transplant teams. Interviews were recorded, transcribed, and coded according to the Capability, Opportunity, Motivation-Behavior model (COM-B).We interviewed 27 caregivers and 27 transplant team members. Fifty-two percent of caregivers reported a household income<$60,000 and 62% reported financial resource strain. Caregivers reported experiencing, (1) high financial burdens after LT, (2) added caregiving labor that compounds the financial burden, (3) dependency on their social network's generosity for financial and logistical support, and (4) additional support being limited to the perioperative period. Transplant providers reported, (1) relying on the pre-transplant psychosocial assessment for identifying social risks, (2) discomfort initiating social risk discussions in the post-transplant period, (3) reliance on social workers to address new social risks, and (4) social workers feeling overburdened by quantity and quality of the social work referrals.We identified barriers to providing effective social care in pediatric LT, primarily a lack of comfort assessing and addressing new social risks in the post-transplant period. Addressing these barriers should enhance social care delivery and improve outcomes for these children.

    View details for DOI 10.1097/LVT.0000000000000327

    View details for PubMedID 38166123

  • Event-free survival of maralixibat-treated patients with Alagille syndrome compared to a real-world cohort from GALA. Hepatology (Baltimore, Md.) Hansen, B. E., Vandriel, S. M., Vig, P., Garner, W., Mogul, D. B., Loomes, K. M., Piccoli, D. A., Rand, E. B., Jankowska, I., Czubkowski, P., Gliwicz-Miedzinska, D., Gonzales, E. M., Jacquemin, E., Bouligand, J., D'Antiga, L., Nicastro, E., Arnell, H., Fischler, B., Sokal, E., Demaret, T., Siew, S., Stormon, M., Karpen, S. J., Romero, R., Ebel, N. H., Feinstein, J. A., Roberts, A. J., Evans, H. M., Sundaram, S. S., Chaidez, A., Hardikar, W., Shankar, S., Fischer, R. T., Lacaille, F., Debray, D., Lin, H. C., Jensen, M. K., Jaramillo, C., Karthikeyan, P., Indolfi, G., Verkade, H. J., Larson-Nath, C., Quiros-Tejeira, R. E., Valentino, P. L., Rogalidou, M., Dezsofi, A., Squires, J. E., Schwarz, K., Calvo, P. L., Bernabeu, J. Q., Zizzo, A. N., Nebbia, G., Bulut, P., Santos-Silva, E., Fawaz, R., Nastasio, S., Karnsakul, W., Tamara, M. L., Busoms, C. M., Kelly, D., Sandahl, T. D., Jimenez-Rivera, C., Banales, J. M., Mujawar, Q., Li, L., She, H., Wang, J., Kim, K. M., Oh, S. H., Sanchez, M. C., Cavalieri, M. L., Lee, W. S., Hajinicolaou, C., Lertudomphonwanit, C., Waisbourd-Zinman, O., Arikan, C., Alam, S., Carvalho, E., Melere, M., Eshun, J., Onal, Z., Desai, D. M., Wiecek, S., Pinto, R. B., Wolters, V. M., Garcia, J., Beretta, M., Kerkar, N., Brecelj, J., Rock, N., Lurz, E., Blondet, N., Shah, U., Thompson, R. J., Kamath, B. M., Global ALagille Alliance (GALA) Study Group 2023

    Abstract

    BACKGROUND AND AIMS: Alagille syndrome (ALGS) is characterized by chronic cholestasis with associated pruritus and extrahepatic anomalies. Maralixibat, an ileal bile acid transporter inhibitor, is the first-approved pharmacologic therapy for cholestatic pruritus in ALGS. Since long-term placebo-controlled studies are not feasible or ethical in children with rare diseases, a novel approach was taken comparing 6-year outcomes from maralixibat trials with an aligned and harmonized natural history cohort from the Global ALagille Alliance (GALA) study.APPROACH AND RESULTS: Maralixibat trials comprise 84 patients with ALGS with≤6 years of treatment. GALA contains retrospective data from 1438 participants. GALA was filtered to align with key maralixibat eligibility criteria, yielding 469 participants. Serum bile acids (sBA) could not be included in the GALA filtering criteria as these are routinely performed in clinical practice. Index time was determined via maximum likelihood estimation in an effort to align the disease severity between the two cohorts with the initiation of maralixibat. Event-free survival (EFS), defined as time to first event of manifestations of portal hypertension (variceal bleeding, ascites requiring therapy), surgical biliary diversion, liver transplant, or death, was analyzed by Cox proportional hazards methods. Sensitivity analyses and adjustments for covariates were applied. Age, total bilirubin (TB), gamma-glutamyl transferase (GGT), and alanine aminotransferase (ALT) were balanced between groups with no statistical differences. EFS in the maralixibat cohort was significantly better than the GALA cohort (hazard ratio 0.305; 95% CI, 0.189-0.491; p<0.0001). Multiple sensitivity and subgroup analyses (including sBA availability) showed similar findings.CONCLUSIONS: This study demonstrates a novel application of a robust statistical method to evaluate outcomes in long-term intervention studies where placebo comparisons are not feasible, providing wide application for rare diseases. This comparison with real-world natural history data suggests that maralixibat improves EFS in patients with ALGS.

    View details for DOI 10.1097/HEP.0000000000000727

    View details for PubMedID 38146932

  • Summary of a Consensus Conference on Heart-Liver Transplantation. American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons Kobashigawa, J., VanWagner, L. B., Hall, S., Emamaullee, J., Entwistle, J. W., Ganger, D., Gebel, H., Jeevanandam, V., Kaldas, F., Kilic, A., Kittleson, M., Kushwaha, S., Kwong, A., Lui, G. K., Motayagheni, N., Patel, J., Patel, N., Pereira, N., Potter, L., Sani, M., Schiano, T. D., Shingina, A. 2023

    Abstract

    Patients with severe heart disease may have co-existing liver disease from various causes. The incidence of combined heart-liver transplant (CHLT) is increasing as more patients with congenital heart disease survive to adulthood and develop advanced heart failure with associated liver disease from chronic right-sided heart or Fontan failure. However, the criteria for CHLT have not been established. To address this unmet need, a virtual consensus conference was organized on June 10, 2022, endorsed by the American Society of Transplantation. The conference represented a collaborative effort by experts in cardiothoracic and liver transplantation from across the United States to assess interdisciplinary criteria for liver transplantation in the CHLT candidate, surgical considerations of CHLT, current allocation system that generally results in the liver following the heart for CHLT, and the optimal post-CHLT management. The conference served as a forum to unify criteria between the different specialties and to forge a pathway for patients who may need dual organ transplantation. Due to the continuing shortage of available donor organs, ethical issues related to multi-organ transplantation were also debated. The findings and consensus statements are presented.

    View details for DOI 10.1016/j.ajt.2023.12.002

    View details for PubMedID 38072122

  • Omicron Infections in Vaccinated Pediatric Solid Organ Transplant Recipients. Journal of the Pediatric Infectious Diseases Society McAteer, J., Kalluri, D. D., Abedon, R. R., Qin, C. X., Auerbach, S. R., Charnaya, O., Danziger-Isakov, L. A., Ebel, N. H., Feldman, A. G., Hsu, E. K., Mohammad, S., Perito, E. R., Thomas, A. M., Chiang, T. P., Garonzik-Wang, J. M., Segev, D. L., Werbel, W. A., Mogul, D. B. 2023

    View details for DOI 10.1093/jpids/piad108

    View details for PubMedID 38035755

  • Caregiver Perceptions of Social Risk Screening in Pediatric Liver Transplantation: From the Multicenter SOCIAL-Tx Study. Transplantation Wadhwani, S. I., Kruse, G., Squires, J., Ebel, N., Gupta, N., Campbell, K., Hsu, E., Zielsdorf, S., Vittorio, J., Desai, D. M., Bucuvalas, J. C., Gottlieb, L. M., Lai, J. C. 2023

    Abstract

    The social determinants of health contribute to adverse post-liver transplant outcomes. Identifying unmet social risks may enable transplant teams to improve long-term outcomes for at-risk children. However, providers may feel uncomfortable asking about household-level social risks in the posttransplant period because they might make their patients/families uncomfortable.We conducted a mixed-methods analysis of caregiver participants (ie, parents/guardians of pediatric liver transplant recipients) in the Social and Contextual Impact on Children Undergoing Liver Transplantation study to assess their perceptions of provider-based social risk screening. Participants (N = 109) completed a 20-min social determinants of health questionnaire that included questions on the acceptability of being asked intimate social risk questions. A subset of participants (N = 37) engaged in an in-depth qualitative interview to share their perceptions of social risk screening.Of 109 participants across 9 US transplant centers, 60% reported financial strain and 30% reported at least 1 material economic hardship (eg, food insecurity, housing instability). Overall, 65% of respondents reported it very or somewhat appropriate and 25% reported being neutral to being screened for social risks in a liver transplant setting. In qualitative analyses, participants reported trust in the providers and a clear understanding of the intention of the screening as prerequisites for liver transplant teams to perform social risk screening.Only a small minority of caregivers found social risk screening unacceptable. Pediatric liver transplant programs should implement routine social risk screening and prioritize the patient and family voices when establishing a screening program to ensure successful implementation.

    View details for DOI 10.1097/TP.0000000000004835

    View details for PubMedID 37831642

  • Safety and Immunogenicity of Live Viral Vaccines in a Multicenter Cohort of Pediatric Transplant Recipients. JAMA network open Feldman, A. G., Beaty, B. L., Ferrolino, J. A., Maron, G., Weidner, H. K., Ali, S. A., Bitterfeld, L., Boulware, M. A., Campbell, K. M., Carr, E., Chapman, S., Chang, Y., Cunningham, R., Dallas, R. H., Dantuluri, K. L., Domenick, B. N., Ebel, N. H., Elisofon, S., Fawaz, R., Foca, M., Gans, H. A., Gopalareddy, V. V., Gu, C., Gupta, N. A., Harmann, K., Hollenbeck, J., Huppler, A. R., Jaramillo, C., Kasi, N., Kerkar, N., Lerret, S., Lobritto, S. J., Lopez, M. J., Marini, E., Mavis, A., Mehra, S., Moats, L., Mohandas, S., Munoz, F. M., Mysore, K. R., Onsan, C., Ovchinsky, N., Perkins, K., Postma, S., Pratscher, L., Rand, E. B., Rowe, R. K., Schultz, D., Sear, K., Sell, M. L., Sharma, T., Stoll, J., Vang, M., Villarin, D., Weaver, C., Wood, P., Woodford-Berry, O., Yanni, G., Danziger-Isakov, L. A. 2023; 6 (10): e2337602

    Abstract

    Importance: Live vaccines (measles-mumps-rubella [MMR] and varicella-zoster virus [VZV]) have not been recommended after solid organ transplant due to concern for inciting vaccine strain infection in an immunocompromised host. However, the rates of measles, mumps, and varicella are rising nationally and internationally, leaving susceptible immunocompromised children at risk for life-threating conditions.Objective: To determine the safety and immunogenicity of live vaccines in pediatric liver and kidney transplant recipients.Design, Setting, and Participants: This cohort study included select pediatric liver and kidney transplant recipients who had not completed their primary MMR and VZV vaccine series and/or who displayed nonprotective serum antibody levels at enrollment between January 1, 2002, and February 28, 2023. Eligibility for live vaccine was determined by individual US pediatric solid organ transplant center protocols.Exposures: Exposure was defined as receipt of a posttransplant live vaccine. Transplant recipients received 1 to 3 doses of MMR vaccine and/or 1 to 3 doses of VZV vaccine.Main Outcome and Measure: Safety data were collected following each vaccination, and antibody levels were obtained at 0 to 3 months and 1 year following vaccination. Comparisons were performed using Mann-Whitney U test, and factors associated with development of postvaccination protective antibodies were explored using univariate analysis.Results: The cohort included 281 children (270 [96%] liver, 9 [3%] kidney, 2 [1%] liver-kidney recipients) from 18 centers. The median time from transplant to enrollment was 6.3 years (IQR, 3.4-11.1 years). The median age at first posttransplant vaccine was 8.9 years (IQR, 4.7-13.8 years). A total of 202 of 275 (73%) children were receiving low-level monotherapy immunosuppression at the time of vaccination. The majority of children developed protective antibodies following vaccination (107 of 149 [72%] varicella, 130 of 152 [86%] measles, 100 of 120 [83%] mumps, and 124 of 125 [99%] rubella). One year post vaccination, the majority of children who initially mounted protective antibodies maintained this protection (34 of 44 [77%] varicella, 45 of 49 [92%] measles, 35 of 42 [83%] mumps, 51 of 54 [94%] rubella). Five children developed clinical varicella, all of which resolved within 1 week. There were no cases of measles or rubella and no episodes of graft rejection within 1 month of vaccination. There was no association between antibody response and immunosuppression level at the time of vaccination.Conclusions and Relevance: The findings suggest that live vaccinations may be safe and immunogenic after solid organ transplant in select pediatric recipients and can offer protection against circulating measles, mumps, and varicella.

    View details for DOI 10.1001/jamanetworkopen.2023.37602

    View details for PubMedID 37824141

  • SERUM BILE ACIDS ARE ASSOCIATED WITH NATIVE LIVER SURVIVAL IN PATIENTS WITH ALAGILLE SYNDROME: RESULTS FROM THE GALA STUDY GROUP Perez, C., Vandriel, S. M., Wang, J., Li, L., She, H., Jankowska, I., Czubkowski, P., Gliwicz-Miedzinska, D., Gonzales, E. M., Jacquemin, E., Bouligand, J., D'Antiga, L., Nicastro, E., Fischler, B., Arnell, H., Siew, S., Stormon, M. O., Loomes, K. M., Piccoli, D. A., Rand, E., Squires, J. E., Karpen, S., Romero, R., Kasahara, M., Onal, Z., Sokal, E. M., Demaret, T., Wiecek, S., Lacaille, F., Debray, D., Hardikar, W., Shankar, S., Valentino, P. L., Sundaram, S. S., Chaidez, A., Ebel, N. H., Feinstein, J. A., Mozer-Glassberg, Y., Lin, H. C., Rock, N., Verkade, H. J., Jensen, M., Jaramillo, C., Kim, K., Oh, S., Brecelj, J., Alam, S., Indolfi, G., Blondet, N., Fawaz, R. L., Nastasio, S., Calvo, P., Nebbia, G., Arikan, C., Larson-Nath, C., Zizzo, A. N., Sandahl, T., Tzivinikos, C., El-Koofy, N. M., Elmonem, M. A., Desai, D. M., Karnsakul, W., Karthikeyan, P., Bulut, P., Kerkar, N., Wolters, V. M., Roberts, A. J., Evans, H. M., Sanchez, M., Cavalieri, M., Kelly, D. A., Lee, W., Hajinicolaou, C., Lertudomphonwanit, C., Fischer, R. T., Bernabeu, J., Quiros-Tejeira, R. E., Melere, M., Carvalho, E., Eshun, J., Zellos, A., Dezsofi, A., Pinto, R., Schwarz, K. B., Rogalidou, M., Garcia, J., Tamara, M., Beretta, M., Mujawar, Q., Santos-Silva, E., Busoms, C., Lurz, E., Goncalves, C., Jimenez-Rivera, C., Banales, J. M., Shah, U., Thompson, R. J., Hansen, B. E., Kamath, B. M., Global ALagille Alliance GALA LIPPINCOTT WILLIAMS & WILKINS. 2023: S121-S124
  • SURGICAL BILIARY DIVERSION IS ASSOCIATED WITH AN INCREASED RISK OF LIVER TRANSPLANTATION OR DEATH IN ALAGILLE SYNDROME Vandriel, S. M., Loomes, K. M., Piccoli, D. A., Rand, E., Li, L., She, H., Wang, J., Fawaz, R. L., Nastasio, S., Verkade, H. J., Jensen, M., Jaramillo, C., Rock, N., Jankowska, I., Czubkowski, P., Gliwicz-Miedzinska, D., Lin, H. C., Kelly, D. A., Larson-Nath, C., Lacaille, F., Debray, D., Karpen, S., Romero, R., Molera Busoms, C., Sokal, E. M., Demaret, T., El-Koofy, N. M., Elmonem, M. A., Sundaram, S. S., Chaidez, A., Karthikeyan, P., Karnsakul, W., Hardikar, W., Shankar, S., Quiros-Tejeira, R. E., Alam, S., Bulut, P., Hajinicolaou, C., Wolters, V. M., Onal, Z., Gonzales, E. M., Jacquemin, E., Bouligand, J., D'Antiga, L., Nicastro, E., Ebel, N. H., Feinstein, J. A., Fischler, B., Arnell, H., Siew, S., Stormon, M. O., Kim, K., Oh, S., Roberts, A. J., Evans, H. M., Camila Sanchez, M., Lorena Cavalieri, M., Lee, W., Lertudomphonwanit, C., Fischer, R. T., Waisbourd-Zinman, O., Squires, J. E., Arikan, C., Quintero Bernabeu, J., Kasahara, M., Carvalho, E., Ferreira, C., Valentino, P. L., Indolfi, G., Eshun, J., Calvo, P., Desai, D. M., Zellos, A., Dezsofi, A., Wiecek, S., Nebbia, G., Pinto, R., Rogalidou, M., Legarda Tamara, M., Zizzo, A. N., Garcia, J., Schwarz, K. B., Blondet, N., Beretta, M., Sandahl, T., Brecelj, J., Goncalves, C., Lurz, E., Santos-Silva, E., Kerkar, N., Mujawar, Q., Tzivinikos, C., Shah, U., Jimenez-Rivera, C., Banales, J. M., Thompson, R. J., Hansen, B. E., Kamath, B. M., Global ALagille Alliance GALA LIPPINCOTT WILLIAMS & WILKINS. 2023: S20-S23
  • Association of State Medicaid Expansion Policies with Pediatric Liver Transplant Outcomes. American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons Shifman, H. P., Huang, C. Y., Beck, A. F., Bucuvalas, J., Perito, E. R., Hsu, E. K., Ebel, N. H., Lai, J. C., Wadhwani, S. I. 2023

    Abstract

    Children from minoritized/socioeconomically deprived backgrounds suffer disproportionately high rates of uninsurance and graft failure/death after liver transplant. Medicaid expansion was developed to expand access to public insurance. Our objective was to characterize the impact of Medicaid expansion policies on long-term graft/patient survival after pediatric liver transplantation. All pediatric patients (<19 years) who received a liver transplant between 1/1/2005-12/31/2020 in the US were identified in the Scientific Registry of Transplant Recipients (N=8489). Medicaid expansion was modeled as a time-varying exposure based on transplant and expansion dates. We used Cox proportional hazards models to evaluate the impact of Medicaid expansion on a composite outcome of graft failure/death over 10 years. As a sensitivity analysis, we conducted an intention-to-treat analysis from time of waitlisting to death (N=11901). In multivariable analysis, Medicaid expansion was associated with a 30% decreased hazard of graft failure/death (HR: 0.70; 95%CI: 0.62,0.79; p<0.001), after adjusting for Black race, public insurance, neighborhood deprivation, and living in a primary care shortage area. In intention-to-treat analyses, Medicaid expansion was associated with a 72% decreased hazard of patient death (HR: 0.28; 95%CI: 0.23-0.35; p<0.001). Policies that enable broader health insurance access may help improve outcomes and reduce disparities for children undergoing liver transplantation.

    View details for DOI 10.1016/j.ajt.2023.09.017

    View details for PubMedID 37776976

  • Serum bile acids are associated with native liver survival in patients with Alagille syndrome: results from the GALA study group Perez, C., Vandriel, S. M., Wang, J., Li Liting, She, H., Jankowska, I., Czubkowski, P., Gliwicz, D., Gonzales, E., Jacquemin, E., Bouligand, J., D'Antiga, L., Nicastro, E., Fischler, B., Arnell, H., Siew, S., Stormon, M., Loomes, K. M., Piccoli, D. A., Rand, E. B., Squires, J. E., Karpen, S. J., Romero, R., Kasahara, M., Onal, Z., Sokal, E., Demaret, T., Wiecek, S., Lacaille, F., Debray, D., Hardikar, W., Shankar, S., Valentino, P., Sundaram, S., Chaidez, A., Ebel, N., Feinstein, J., Mozar-Glazberg, Y., Lin, H., Rock, N., Verkade, H. J., Jensen, M. K., Jaramillo, C., Kim, K., Oh, S., Brecelj, J., Alam, S., Indolfi, G., Blondet, N., Fawaz, R., Nastasio, S., Calvo, P., Nebbia, G., Arikan, C., Larson-Nath, C., Zizzo, A. N., Sandahl, T., Tzivinikos, C., El-Koofy, N., Elmonem, M., Desai, D., Karnsakul, W., Karthikeyan, P., Bulut, P., Kerkar, N., Wolters, V., Roberts, A. J., Evans, H., Sanchez, M., Cavalieri, M., Kelly, D., Lee, W., Hajinicolaou, C., Lertudomphonwanit, C., Fischer, R., Bernabeu, J., Quiros-Tejeira, R. E., Melere, M., Carvalho, E., Eshun, J., Zellos, A., Fi, A., Pinto, R., Schwarz, K., Rogalidou, M., Garcia, J., Tamara, M., Beretta, M., Mujawar, Q., Santos-Silva, E., Busoms, C., Lurz, E., Goncalves, C., Jimenez-Rivera, C., Banales, J. M., Shah, U., Thompson, R., Hansen, B., Kamath, B. M. ELSEVIER. 2023: S971-S973
  • MELD 3.0 for adolescent liver transplant candidates. Hepatology (Baltimore, Md.) Kwong, A. J., Zhang, K. Y., Ebel, N., Mannalithara, A., Kim, W. R. 2023

    Abstract

    Adolescents constitute a unique waitlist cohort that is distinct from younger children. MELD 3.0, which was developed in an adult population of liver transplant candidates, is planned to replace MELD-Na in the current liver allocation system for both adult and adolescents aged 12-17. We evaluated the predictive performance of MELD-Na, MELD 3.0, and PELD, for 90-day waitlist mortality risk among adolescent liver transplant registrants.New waitlist registrations for primary liver transplant among individuals aged 12-17 and aged 18-25 for comparison were identified using OPTN data from Nov 17 2004 to Dec 31 2021. The predictive performance of the current and proposed MELD and PELD scores was assessed using the Harrell's concordance (c) statistic.There were 1,238 eligible listings for adolescents aged 12-17, and 1,740 young adults aged 18-25. In the adolescent group, 90-day survival was 97.8%, compared to 95.9% in those aged 18-25 (log-rank p = 0.005), with no significant differences when stratified by sex or indication. Among adolescents, increasing MELD 3.0 was associated with an increased hazard of mortality (HR 1.27, 95% CI 1.18-1.37), and the c-statistic for 90-day waitlist survival using MELD 3.0 was 0.893, compared with 0.871 using MELD-Na, and 0.852 using PELD.The discriminative ability of MELD 3.0 to rank adolescents according to the risk of death within 90 days was robust. Although MELD 3.0 was initially developed and validated in adults, MELD 3.0 may also improve the prediction of waitlist mortality in adolescents and better represent their urgency for liver transplant.

    View details for DOI 10.1097/HEP.0000000000000352

    View details for PubMedID 36943091

  • Outcomes after liver transplantation in MPV17 deficiency: A rebuttal. Pediatric transplantation Huang, A. C., Ebel, N. H., Romero, D., Enns, G. M., Esquivel, C. O., Bonham, C. 2023: e14472

    View details for DOI 10.1111/petr.14472

    View details for PubMedID 36872458

  • OPTN/SRTR 2021 Annual Data Report: Liver. American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons Kwong, A. J., Ebel, N. H., Kim, W. R., Lake, J. R., Smith, J. M., Schladt, D. P., Schnellinger, E. M., Handarova, D., Weiss, S., Cafarella, M., Snyder, J. J., Israni, A. K., Kasiske, B. L. 2023; 23 (2S1): S178-S263

    Abstract

    In 2021, liver transplant volume continued to grow, with a record 9,234 transplants performed in the United States, 8,665 (93.8%) from deceased donors and 569 (6.2%) from living donors. There were 8,733 (94.6%) adult and 501 (5.4%) pediatric liver transplant recipients. An increase in the number of deceased donor livers corresponded to an increase in the overall transplant rate and shorter waiting times, although still 10.0% of livers that were recovered were not transplanted. Alcohol-associated liver disease was the leading indication for both waitlist registration and liver transplant in adults, outpacing nonalcoholic steatohepatitis, while biliary atresia remained the leading indication for children. Related to allocation policy changes implemented in 2019, the proportion of liver transplants performed for hepatocellular carcinoma has decreased. Among adult candidates listed for liver transplant in 2020, 37.7% received a deceased donor liver transplant within 3 months, 43.8% within 6 months, and 53.3% within 1 year. Pretransplant mortality improved for children following implementation of acuity circle-based distribution. Short-term graft and patient survival outcomes up to 1 year worsened for adult deceased and living donor liver transplant recipients, which is a reversal of previous trends and coincided with the onset of the COVID-19 pandemic in early 2020. Longer-term outcomes among adult deceased donor liver transplant recipients were unaffected, with overall posttransplant mortality rates of 13.3% at 3 years, 18.6% at 5 years, and 35.9% at 10 years. Pretransplant mortality improved for children following implementation of acuity circle-based distribution and prioritization of pediatric donors to pediatric recipients in 2020. Pediatric living donor recipients had superior graft and patient survival outcomes compared with deceased donor recipients at all time points.

    View details for DOI 10.1016/j.ajt.2023.02.006

    View details for PubMedID 37132348

  • BARRIERS TO IDENTIFYING AND INTERVENING ON SOCIAL NEEDS IN PEDIATRIC LIVER TRANSPLANTATION: QUALITATIVE RESULTS FROM THE MULTI-CENTER SOCIAL & CONTEXTUAL IMPACT ON CHILDREN UNDERGOING LIVER TRANSPLANTATION (SOCIAL-TX) STUDY Wadhwani, S., Alvarado, A., Shifman, H., Squires, J. E., Campbell, K. M., Ebel, N., Hsu, E. K., Vittorio, J. M., Zielsdorf, S., Desai, D. M., Bucuvalas, J. C., Gottlieb, L., Kotagal, U., Lyles, C., Lai, J. WILEY. 2022: S1523-S1524
  • MELD 3.0 PREDICTS WAITLIST MORTALITY RISK IN ADOLESCENT LIVER TRANSPLANT REGISTRANTS Kwong, A. J., Zhang, K., Ebel, N., Mannalithara, A., Kim, W. WILEY. 2022: S17-S18
  • Healthcare Resource Utilization by Patients with Alagille Syndrome. The Journal of pediatrics Ebel, N. H., Goldstein, A., Howard, R., Mogul, D. B., Marden, J. R., Anderson, A., Gaburo, K., Kirson, N., Rosenthal, P. 2022

    Abstract

    (s): To assess and characterize healthcare resource utilization (HRU) in children with the rare, genetic, multisystem disorder, Alagille syndrome (ALGS).This retrospective analysis reviewed commercially- and Medicaid-insured claims from October 1, 2015 through December 31, 2019 to assess HRU in patients with ALGS. As there is no specific International Classification of Diseases (ICD)-10 code for ALGS, patients were identified using the following algorithm: ≥1 claim with diagnosis code Q44.7 (other congenital malformations of the liver), <18 years of age, with no history of biliary atresia (ICD-10 code: Q44.2) and ≥6 months of insurance eligibility prior to diagnosis. HRU was summarized per patient per year over all available claims post-diagnosis.171 commercially-insured and 215 Medicaid-insured patients with ALGS were available for analysis. Annually, commercially-insured and Medicaid-insured patients averaged 31 medical visits (range 1.5-237) and 48 medical visits (range 0.7-690), respectively. The most common visits were outpatient with the majority encompassing lab/imaging and primary care visits (commercially-insured: 21 [range 0.0-183]; Medicaid-insured: 26 [range 0.0-609]). Inpatient visits were the highest driver of costs in both the commercial and Medicaid populations.Patients with ALGS have a substantial HRU burden driven largely by numerous outpatient visits and costly inpatient stays. Given the complexity and variability of ALGS presentation, patients may benefit from multidisciplinary and subspecialized care.

    View details for DOI 10.1016/j.jpeds.2022.09.033

    View details for PubMedID 36179890

  • Natural History of Liver Disease in a Large International Cohort of Children with Alagille syndrome: Results from The GALA Study. Hepatology (Baltimore, Md.) Vandriel, S. M., Li, L., She, H., Wang, J., Gilbert, M. A., Jankowska, I., Czubkowski, P., Gliwicz-Miedzinska, D., Gonzales, E. M., Jacquemin, E., Bouligand, J., Spinner, N. B., Loomes, K. M., Piccoli, D. A., D'Antiga, L., Nicastro, E., Sokal, E., Demaret, T., Ebel, N. H., Feinstein, J. A., Fawaz, R., Nastasio, S., Lacaille, F., Debray, D., Arnell, H., Fischler, B., Siew, S., Stormon, M., Karpen, S. J., Romero, R., Kim, K. M., Baek, W. Y., Hardikar, W., Shankar, S., Roberts, A. J., Evans, H. M., Jensen, M. K., Kavan, M., Sundaram, S. S., Chaidez, A., Karthikeyan, P., Sanchez, M. C., Cavalieri, M. L., Verkade, H. J., Lee, W. S., Squires, J. E., Hajinicolaou, C., Lertudomphonwanit, C., Fischer, R. T., Larson-Nath, C., Mozer-Glassberg, Y., Arikan, C., Lin, H. C., Quintero Bernabeu, J., Alam, S., Kelly, D., Carvalho, E., Ferreira, C. T., Indolfi, G., Quiros-Tejeira, R. E., Bulut, P., Calvo, P. L., Onal, Z., Valentino, P. L., Desai, D. M., Eshun, J., Rogalidou, M., Dezsofi, A., Wiecek, S., Nebbia, G., Borges Pinto, R., Wolters, V. M., Tamara, M. L., Zizzo, A. N., Garcia, J., Schwarz, K., Beretta, M., Sandahl, T. D., Jimenez-Rivera, C., Kerkar, N., Brecelj, J., Mujawar, Q., Rock, N., Busoms, C. M., Karnsakul, W., Lurz, E., Santos-Silva, E., Blondet, N., Bujanda, L., Shah, U., Thompson, R. J., Hansen, B. E., Kamath, B. M., Global ALagille Alliance (GALA) Study Group 2022

    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.

    View details for DOI 10.1002/hep.32761

    View details for PubMedID 36036223

  • Antibody Response to Three SARS-CoV-2 mRNA Vaccines in Adolescent Solid Organ Transplant Recipients. American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons Qin, C. X., Auerbach, S. R., Charnaya, O., Danziger-Isakov, L. A., Ebel, N. H., Feldman, A. G., Hsu, E. K., McAteer, J., Mohammad, S., Perito, E. R., Thomas, A. M., Chiang, T. P., Garonzik-Wang, J. M., Segev, D. L., Mogul, D. B. 2022

    View details for DOI 10.1111/ajt.17085

    View details for PubMedID 35510786

  • Addressing Racism in Pediatric Liver Transplantation: A Moral Imperative. The Journal of pediatrics Ebel, N. H., Dike, P. N., Hsu, E. K. 2022

    View details for DOI 10.1016/j.jpeds.2022.04.046

    View details for PubMedID 35504347

  • Outcomes after liver transplantation in MPV17 deficiency (Navajo neurohepatopathy): A single-center case series. Pediatric transplantation Huang, A. C., Ebel, N. H., Romero, D., Martin, B., Jhun, I., Brown, M., Enns, G. M., Esquivel, C., Bonham, C. 2022: e14274

    Abstract

    BACKGROUND: MPV17-related mitochondrial DNA maintenance defect (MPV17 deficiency) is a rare, autosomal recessive mitochondrial DNA depletion syndrome with a high mortality rate in infancy and early childhood due to progression to liver failure. Liver transplantation for children with MPV17 deficiency has been considered controversial due to uncertainty about the potential progression of extrahepatic manifestations following liver transplantation.METHODS: We describe our institution's experience for two infants diagnosed with infantile MPV17 deficiency who presented in acute on chronic liver failure, but with normal development and normal neurological status who successfully underwent liver transplantation.RESULTS: Both patients underwent successful liver transplantation with normal development and neurological status at 3years and 16months post-transplant, respectively.CONCLUSIONS: In this rare disease population, we describe two infants with MPV17 deficiency who underwent liver transplantation for acute on chronic liver failure who continue to have normal development, without progression of neurological disease. MPV17 deficiency should not be considered a contraindication to liver transplantation.

    View details for DOI 10.1111/petr.14274

    View details for PubMedID 35466509

  • Neurologic complications in en bloc pediatric heart-liver transplants Pan, J., Bensen, R., Ebel, N., Mendoza, J., Ma, M., Hollander, S., Gallo, A., Esquivel, C. O., Bonham, A. WILEY. 2022
  • Humoral response to BNT162B2 SARS-COV-2 vaccination in pediatric solid organ transplant recipients Qin, C. X., Auerbach, S. R., Charnaya, O., Danziger-Isakov, L. A., Ebel, N. H., Feldman, A. G., Hsu, E. K., McAteer, J., Mohammad, S., Perito, E. R., Thomas, A. M., Chiang, T. Y., Garonzik-Wang, J. M., Segev, D. L., Mogul, D. B. WILEY. 2022
  • Quality improvement project to safely expedite liver biopsy in pediatric acute liver failure Mendoza, J., Ebel, N. H., Josephs, S., Wolke, O., Depper, J., Bonham, C. A., Damian, M. A., Esquivel, C. O., Gallo, A. WILEY. 2022
  • Underutilized technical variant liver grafts show equal graft survival to whole liver grafts in pediatric liver transplantation Stoltz, D., Bonham, A., Lum, G., Ebel, N., Mendoza, J., Esquivel, C., Gallo, A. WILEY. 2022
  • Towards identifying predictors of pediatric heart only versus combined heart liver transplantation Zhang, K., Chen, S., Syed, A., Gallo, A., Esquivel, C., Bonham, A., Hollander, S. A., Ma, M., Han, J., Ebel, N. H. WILEY. 2022
  • OPTN/SRTR 2020 Annual Data Report: Liver. American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons Kwong, A. J., Ebel, N. H., Kim, W. R., Lake, J. R., Smith, J. M., Schladt, D. P., Skeans, M. A., Foutz, J., Gauntt, K., Cafarella, M., Snyder, J. J., Israni, A. K., Kasiske, B. L. 2022; 22 Suppl 2: 204-309

    Abstract

    This year was marked by the COVID-19 pandemic, which altered transplant program activity and affected waitlist and transplant outcomes. Still, 8906 liver transplants were performed, an all-time high, across 142 centers in the United States, and pretransplant as well as graft and patient survival metrics, continued to improve. Living donation activity decreased after several years of growth. As of June 30, 2020, 98989 liver transplant recipients were alive with a functioning graft, and in the context of increasing liver transplant volume, the size of both the adult and pediatric liver transplant waitlists have decreased. On February 4, 2020, shortly before the pandemic began, a new liver distribution policy based on acuity circles was implemented, replacing donor service area- and region-based boundaries. A policy change to direct pediatric livers to pediatric recipients led to an increase in deceased donor transplant rates and a decrease in pretransplant mortality rate among children, although the absolute number of pediatric transplants did not increase in 2020. Among adults, alcohol-associated liver disease became the predominant indication for liver transplant in 2020. After implementation of the National Liver Review Board and lower waitlist priority for most exception cases in 2019, fewer liver transplants were being performed via exception points, and the transplant rate between those with and without hepatocellular carcinoma has equalized. Women continue to experience higher pretransplant mortality and lower rates of liver transplant than men.

    View details for DOI 10.1111/ajt.16978

    View details for PubMedID 35266621

  • A review of racial, socioeconomic, and geographic disparities in pediatric liver transplantation. Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society Ebel, N. H., Lai, J. C., Bucuvalas, J. C., Wadhwani, S. I. 2022

    Abstract

    Equity is a core principle in both pediatrics and solid organ transplantation. Health inequities, specifically across race, socioeconomic position, or geography, reflect a moral failure. Ethical principles of prudential life span, maximin principle, and fair innings argue for allocation priority to children related to the number of life years gained, equal access to transplant, and equal opportunity for ideal post-transplant outcomes. Iterative policy changes have aimed to narrow these disparities to achieve pediatric transplant equity. These policy changes have focused on modifying pediatric priority for organ allocation to eliminate mortality on the pediatric transplant waitlist. Yet disparities remain in pediatric liver transplantation at all time points: from access to referral for transplantation, likelihood of living donor transplantation, utilization of exception narratives, waitlist mortality, and inequitable post-transplant outcomes. Black children are less likely to be petitioned for exception scores, have higher waitlist mortality, are less likely to be the recipient of living donor transplant, and have worse post-transplant outcomes compared to White children. Children living in the most socioeconomically deprived neighborhoods have worse post-transplant outcomes. Children living further from a transplant center have higher waitlist mortality. Herein, we review the current knowledge of these racial and ethnic, socioeconomic, and geographic disparities for these children. To achieve equity, stakeholder engagement is required at all levels from providers and health delivery systems, learning networks, institutions, and society. Future initiatives must be swift, bold, and effective with the tri-partite mission to inform policy changes, improve healthcare delivery, and optimize resource allocation to provide equitable transplant access, waitlist survival, and post-transplant outcomes for all children.

    View details for DOI 10.1002/lt.26437

    View details for PubMedID 35188708

  • Neurologic complications in en bloc pediatric heart-liver transplants Pan, J., Bensen, R., Ebel, N., Mendoza, J., Ma, M., Hollander, S., Gallo, A., Esquivel, C., Bonham, A. WILEY. 2022: 80
  • 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 Hansen, B. E., Vandriel, S. M., Vig, P., Garner, W., Li, L., She, H., Wang, J., Gilbert, M. A., Jankowska, I., Czubkowski, P., Gliwicz-Miedzinska, D., Gonzales, E. M., Jacquemin, E., Bouligand, J., Spinner, N., Loomes, K. M., Piccoli, D. A., D'Antiga, L., Nicastro, E., Sokal, E., Demaret, T., Ebel, N., Feinstein, J. A., Fawaz, R., Nastasio, S., Lacaille, F., Debray, D., Arnell, H., Fischler, B., Siew, S., Stormon, M., Karpen, S. J., Romero, R., Kim, K., Baek, W., Hardikar, W., Shankar, S., Roberts, A. J., Evans, H. M., Jensen, M., Kavan, M., Sundaram, S. S., Chaidez, A., Karthikeyan, P., Davison, S., Sanchez, M., Cavalieri, M., Verkade, H. J., Lee, W., Squires, J. E., Hajinicolaou, C., Lertudomphonwanit, C., Fischer, R. T., Larson-Nath, C., Mozer-Glassberg, Y., Arikan, C., Lin, H. C., Bernabeu, J., Alam, S., Kelly, D., Carvalho, E., Ferreira, C., Indolfi, G., Quiros-Tejeira, R. E., Bulut, P., Calvo, P., Onal, Z., Valentino, P. L., Desai, D. M., Eshun, J., Rogalidou, M., Dezsofi, A., Wiecek, S., Nebbia, G., Pinto, R., Wolters, V. M., Tamara, M., Zizzo, A. N., Garcia, J., Schwarz, K., Beretta, M., Sandahl, T., Jimenez-Rivera, C., Kerkar, N., Brecelj, J., Mujawar, Q., Rock, N., Molera, C., Karnsakul, W., Lurz, E., Santos-Silva, E., Blondet, N., Bujanda, L., Shah, U., Thompson, R. J., Kamath, B. M. WILEY. 2021: 1387A-1389A
  • The Impact of Severe Acute Respiratory Syndrome Coronavirus Type 2 on Children with Liver Diseases: A Joint European Society for Pediatric Gastroenterology, Hepatology and Nutrition and Society of Pediatric Liver Transplantation Position Paper. Journal of pediatric gastroenterology and nutrition Nicastro, E., Ebel, N. H., Kehar, M., Czubkowski, P., Ng, V. L., Michaels, M. G., Lobritto, S. J., Martinez, M., Indolfi, G. 2021

    Abstract

    ABSTRACT: Children are seldom affected by severe forms of severe acute respiratory syndrome coronavirus type 2 (SARS-CoV2) infection. However, the impact of comorbidities in the clinical presentation and outcome of SARS-CoV2 in children is poorly characterized including that of chronic liver disease (CLD) and those taking immunosuppressive medications for autoimmune liver disease or following liver transplantation (LT). Although not the main target organ, a spectrum of liver involvement has been described in children infected with SARS-CoV2 and those presenting with Multisystem Inflammatory Syndrome in Children (MIS-C). The Hepatology Committee of the European Society for Pediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) and the Society of Pediatric Liver Transplantation (SPLIT) present an evidence-based position paper on liver involvement in children with SARS-CoV2 infection and its impact on those with CLD as well as LT recipients. All children may exhibit acute liver injury from SARS-CoV2 infection, and those with CLD and may experience hepatic decompensation. Preventative and therapeutic measures are discussed.

    View details for DOI 10.1097/MPG.0000000000003339

    View details for PubMedID 34694269

  • Updates from the NASPGHAN/SPLIT SARS-CoV2 International Registry. Journal of pediatric gastroenterology and nutrition Kehar, M., Ebel, N. H., Ng, V., Lobritto, S., Martinez, M. 2021

    View details for DOI 10.1097/MPG.0000000000003326

    View details for PubMedID 34654794

  • THE IMPACT OF SARS-COV2 INFECTION IN CHILDREN WITH LIVER DISEASE: AN INTERNATIONAL OBSERVATIONAL REGISTRY STUDY Kehar, M., Ebel, N. H., Ng, V., Sehgal, A., Slowik, V., Leung, D. H., Shah, A. A., Ovchinsky, N., Kogan-Liberman, D., Arnon, R., Vitola, B., Waheed, N., Lebel, S., Mohammad, S., Squires, J. E., Shteyer, E., Miloh, T. A., Sanchez, M., Hildreth, A., Yerushalmi, B. Y., Chu, C., Kader, H., Book, L., Alrabadi, L., Zheng, M., Namjoshi, S. S., Cagil, Y., Fuchs, Y., Lobritto, S. J., Martinez, M. WILEY. 2021: 1180A-1181A
  • HEALTHCARE RESOURCE UTILIZATION IN PATIENTS WITH ALAGILLE SYNDROME Ebel, N. H., Goldstein, A., Howard, R., Marden, J., Anderson, A., Rosenthal, P. WILEY. 2021: 1162A-1163A
  • THE IMPACT OF SARS-CoV2 INFECTION IN PEDIATRIC LIVER TRANSPLANT RECIPIENTS: AN INTERNATIONAL OBSERVATIONAL REGISTRY STUDY Ebel, N. H., Kehar, M., Ng, V., Sehgal, A., Leung, D. H., Shah, A. A., Gupta, N., Baqueros, J., Botha, J., Slowik, V., Lebel, S., Miloh, T. A., Shteyer, E., Arnon, R., Azzam, R. K., Ovchinsky, N., Mohammad, S., Kogan-Liberman, D., Squires, J. E., Sanchez, M., Hildreth, A., Book, L., Chu, C., Alrabadi, L., Chepuri, B., D'Agostino, D., Elisofon, S., Falik, R., Gallagher, L., Kader, H., Lam, S., Mogul, D., Mujawar, Q., Namjoshi, S. S., Valentino, P. L., Vitola, B., Waheed, N., Zheng, M., Blondet, N., Lobritto, S. J., Martinez, M. WILEY. 2021: 136A-137A
  • Antibody Response to 2-Dose SARS-CoV-2 mRNA Vaccination in Pediatric Solid Organ Transplant Recipients. American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons Qin, C. X., Auerbach, S. R., Charnaya, O., Danziger-Isakov, L. A., Ebel, N. H., Feldman, A. G., Hsu, E. K., McAteer, J., Mohammad, S., Perito, E. R., Thomas, A. M., Chiang, T. P., Garonzik-Wang, J. M., Segev, D. L., Mogul, D. B. 2021

    Abstract

    While many adult solid organ transplant recipients (SOTRs) have impaired antibody response to severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) vaccination, pediatric SOTRs' response has not been assessed.1-2 We report the immunogenicity and safety of BNT162b2 mRNA vaccination in pediatric SOTRs.

    View details for DOI 10.1111/ajt.16841

    View details for PubMedID 34517430

  • Acute Liver Failure in Neonatal Lupus Erythematosus: Novel Treatment With Exchange Transfusion, Intravenous Immunoglobulin, and Steroids. JPGN reports Nguyen, E. L., Huang, A. C., Khalsa, U. K., Saarela, K., Sandborg, C., Ebel, N. H. 2021; 2 (2): e057

    View details for DOI 10.1097/PG9.0000000000000057

    View details for PubMedID 37207059

    View details for PubMedCentralID PMC10191595

  • SARS-CoV2 Infection in Children with Liver Transplant and Native Liver Disease: An International Observational Registry Study. Journal of pediatric gastroenterology and nutrition Kehar, M. n., Ebel, N. H., Ng, V. L., Baquero, J. E., Leung, D. H., Slowik, V. n., Ovchinsky, N. n., Shah, A. A., Arnon, R. n., Miloh, T. n., Gupta, N. n., Mohammad, S. n., Kogan-Liberman, D. n., Squires, J. E., Sanchez, M. C., Hildreth, A. n., Book, L. n., Chu, C. n., Alrabadi, L. n., Azzam, R. n., Chepuri, B. n., Falik, R. n., Gallagher, L. n., Kader, H. n., Mogul, D. n., Mujawar, Q. n., Namjoshi, S. S., Valentino, P. L., Vitola, B. n., Waheed, N. n., Zheng, M. H., Lobritto, S. n., Martinez, M. n. 2021

    Abstract

    Increased mortality risk due to severe acute respiratory syndrome coronavirus-2 (SARS-CoV2) infection in adults with native liver disease (LD) and liver transplant (LT) is associated with advanced age and comorbid conditions. We aim to report outcomes for children with LD and LT enrolled in the NASPGHAN/SPLIT SARS-CoV2 registry.In this multicenter observational cohort study, we collected data from 91 patients <21 years (LD 44, LT 47) with laboratory-confirmed SARS-CoV2 infection between April 21 and September 17, 2020.Patients with LD were more likely to require admission (70% vs 43% LT, p = 0.007) and pediatric intensive care unit (PICU) management (32% vs 4% LT, p = 0.001). Seven LD patients required mechanical ventilation (MV) and 2 patients died; no patients in the LT cohort died or required MV. Four LD patients presented in pediatric acute liver failure (PALF), 2 with concurrent multisystem inflammatory syndrome in children (MIS-C); all recovered without LT. Two LD patients had MIS-C alone and one patient died. Bivariable logistic-regression analysis found that patients with non-alcoholic fatty liver disease (NAFLD) (OR 5.6, p = 0.02) and LD (OR 6.1, p = 0.01, vs LT) had higher odds of severe disease (PICU, vasopressor support, MV, renal replacement therapy or death).Although not directly comparable, LT recipients had lower odds of severe SARS-CoV2 infection (vs LD), despite immunosuppression burden. NAFLD patients reported to the registry had higher odds of severe SARS-CoV2 disease. Future controlled studies are needed to evaluate effective treatments and further stratify LD and LT patients with SARS-CoV2 infection.

    View details for DOI 10.1097/MPG.0000000000003077

    View details for PubMedID 33605666

  • ACUTE LIVER FAILURE IN NEONATAL LUPUS ERYTHEMATOSUS: NOVEL TREATMENT WITH EXCHANGE TRANSFUSION, IVIG, AND STEROIDS Nguyen, E., Huang, A., Khalsa, U., Saarela, K., Sandborg, C., Ebel, N. WILEY. 2020: 131A–132A
  • PHENOTYPIC DIVERGENCE OF JAGGED1 AND NOTCH2-ASSOCIATED ALAGILLE SYNDROME: RESULTS FROM THE INTERNATIONAL MULTICENTER GALA STUDY GROUP Vandriel, S., Li, L., She, H., Wang, J., Gilbert, M. A., Spinner, N. B., Loomes, K. M., Piccoli, D. A., D'Antiga, L., Nicastro, E., Calvo, P., Hardikar, W., Shankar, S., Fawaz, R. L., Nastasio, S., Bulut, P., Jankowska, I., Czubkowski, P., Gliwicz-Miedzinska, D., Sokal, E., Demaret, T., Siew, S. M., Stormon, M., Lacaille, F., Debray, D., Kim, K., Baek, W., Feinstein, J. A., Ebel, N., Karpen, S. J., Romero, R., Karthikeyan, P., Davison, S., Arnell, H., Fischler, B., Squires, J. E., Verkade, H. J., Jensen, M., Kavan, M., Roberts, A. J., Evans, H. M., Lertudomphonwanit, C., Lee, W., Sundaram, S. S., Chaidez, A., Fischer, R. T., Mozer-Glassberg, Y., Larson-Nath, C., Lin, H., Bujanda, L., Kelly, D., Karnsakul, W., Bernabeu, J., Indolfi, G., Mujawar, Q., Valentino, P. L., Nebbia, G., Quiros-Tejeira, R. E., Kerkar, N., Schwarz, K. B., Wolters, V. M., Alam, S., Jimenez-Rivera, C., Santos-Silva, E., Brecelj, J., Sanchez, M., Cavalieri, M., Desai, D. M., Onal, Z., Tamara, M., Molera, C., Arikan, C., Wiecek, S., Gonzales, E. M., Thompson, R. J., Hansen, B. E., The, B., Gala Study Grp WILEY. 2020: 882A–884A
  • PERIOPERATIVE MANAGEMENT AND POST-LIVER TRANSPLANTATION OUTCOMES IN UREA CYCLE DISORDERS, MAPLE SYRUP URINE DISEASE AND ORGANIC ACIDEMIAS: 20 YEARS' EXPERIENCE Ebel, N., Vuong, P., Baker, C., Brubaker, A., Than, P., Enns, G., Esquivel, C. O. WILEY. 2020: 881A–882A
  • PERIOPERATIVE MANAGEMENT AND POST-LIVER TRANSPLANTATION OUTCOMES IN METHYLMALONIC ACIDEMIA, PROPIONIC ACIDEMIA AND UREA CYCLE DISORDERS: OUR 20 YEAR EXPERIENCE Vuong, P., Ebel, N., Baker, C. V., Brubaker, A., Than, P., Enns, G., Esquivel, C. O. LIPPINCOTT WILLIAMS & WILKINS. 2020: S547
  • LIVER TRANSPLANTATION IN INFANTILE NAVAJO NEUROHEPATOPATHY Romero, D., Ebel, N. H., Huang, A., Brown, M., Enns, G. M., Esquivel, C., Bonham, C. A. LIPPINCOTT WILLIAMS & WILKINS. 2020: S494–S495
  • Decreased Incidence of Hepatic Artery Thrombosis in Pediatric Liver Transplantation Utilizing Technical Variant Grafts: Report of the Society of Pediatric Liver Transplantation (SPLIT) Experience. The Journal of pediatrics Ebel, N. H., Hsu, E. K., Dick, A. A., Shaffer, M. L., Carlin, K., Horslen, S. P. 2020

    Abstract

    OBJECTIVE: To evaluate risk factors for hepatic artery thrombosis (HAT) and examine the long-term outcomes of graft and patient survival following HAT in pediatric recipients of liver transplantation.STUDY DESIGN: Utilizing multicenter data from the Society of Pediatric Liver Transplantation (SPLIT), Kaplan-Meier and Cox regression analyses were performed on first-time pediatric (aged <18 years) liver transplant recipients (n=3801) in the United States and Canada between 1995 to 2016.RESULTS: Of children undergoing their first liver transplantation, 7.4% developed HAT within the first 90 days of transplantation and of those who were re-transplanted, 20.7% developed recurrent HAT. Prolonged warm ischemia times increased the odds of developing HAT (OR 1.11, p=0.02). Adolescents aged 11-17 years (OR 0.53, p=0.03) and recipients with split, reduced or living donor grafts had decreased odds of HAT (OR 0.59, P < .001 compared with whole grafts). Fifty percent of children who developed HAT developed graft failure within the first 90 days of transplantation (AHR 11.87, 95% CI 9.02,15.62) and had a significantly higher post-transplant mortality within the first 90 days of transplantation (AHR 6.18, 95% CI 4.01,9.53).CONCLUSIONS: These data from an international registry demonstrate poorer long-term graft and patient survival in pediatric recipients whose post-transplant course is complicated by HAT. Notably, recipients of technical variant grafts had lower odds of HAT compared with whole liver grafts.

    View details for DOI 10.1016/j.jpeds.2020.06.053

    View details for PubMedID 32585237

  • Outcomes in Patients with Alagille Syndrome and Complex Pulmonary Artery Disease. The Journal of pediatrics Luong, R. n., Feinstein, J. A., Ma, M. n., Ebel, N. H., Wise-Faberowski, L. n., Zhang, Y. n., Peng, L. F., Yarlagadda, V. V., Shek, J. n., Hanley, F. L., McElhinney, D. B. 2020

    Abstract

    To assess outcomes in a large cohort of patients with Alagille Syndrome (ALGS) who underwent pulmonary artery reconstruction surgery for complex PA disease.Patients with ALGS who underwent PA reconstruction surgery at Lucile Packard Children's Hospital Stanford were reviewed. Patients were examined as an overall cohort and based on the primary cardiovascular diagnosis: severe isolated branch PA stenosis, tetralogy of Fallot (TOF) without major aortopulmonary collateral arteries (MAPCAs), or TOF with MAPCAs RESULTS: Fifty-one patients with ALGS underwent PA surgery at our center: 22 with severe branch PA stenosis, 9 with TOF without MAPCAs, and 20 with TOF and MAPCAs. Forty-one patients (80%) achieved a complete repair. Five of the patients with TOF with MAPCAs (25%) had a complete repair at the first surgery, compared with 8 (89%) and 19 (86%) with TOF without MAPCAs and isolated branch PA stenosis, respectively. At a median follow-up of 1.7 years after the first surgery, 39 patients (76%) were alive, 36 with a complete repair and a median PA:aortic systolic pressure ratio of 0.38. Nine patients (18%), 8 with isolated branch PA stenosis, underwent liver transplantation.Most patients with ALGS and complex PA disease can undergo complete repair with low postoperative right ventricular pressure. Patients with TOF/MAPCAs had the worst outcome, with higher mortality and more frequent PA interventions compared with patients with TOF without MAPCAs or isolated branch PA stenosis. Complex PA disease is not a contraindication to liver transplantation in patients with ALGS.

    View details for DOI 10.1016/j.jpeds.2020.09.053

    View details for PubMedID 32980376

  • Hepatic Venous Pressure Gradient Measurements in Children: Correlation With Hepatic Histology and Clinical Indicators of Portal Hypertension. Journal of pediatric gastroenterology and nutrition Ebel, N. H., Carlin, K. n., Shaffer, M. L., Shivaram, G. n., Hawkins, M. n., Lane, E. R., Cooper, K. n., Lindquester, W. S., Gadodia, G. n., Murray, K. F. 2019; 68 (6): 788–92

    Abstract

    In adults, elevated hepatic venous pressure gradients (HVPGs) are correlated with the degree of liver fibrosis on histopathology and predict worse outcomes including variceal bleeding and death. We aimed to examine the association between HVPG measurements, histopathologic findings, and clinical indicators of portal hypertension in children.Utilizing retrospective data from 2 pediatric centers between 2006 and 2015, we identified children who underwent simultaneous HVPG measurement and transjugular liver biopsy. Medical charts were reviewed for histopathology, imaging, endoscopic, and clinical data.Forty-one children (median age 11 years) were included in the analysis with diagnoses of acute hepatitis (n = 15), chronic liver disease (n = 12), hepatic noncirrhotic portal hypertension (n = 4), acute liver failure (n = 3), and nonhepatic causes of portal hypertension (n = 7). Elevated mean HVPG measurements were found in children with acute liver failure (10 mmHg, range 4-12) and chronic liver disease (7 mmHg, range 1-12). HVPG measurements did not correlate with the histological severity of fibrosis (ρ = 0.23, P = 0.14) or portal inflammation (ρ = 0.24, P = 0.29), and no difference was found in HVPG when comparing children with and without a history of variceal bleeding (P = 0.43).HVPG measurements do not correlate significantly with the degree of hepatic fibrosis on biopsy. Furthermore, HVPG measurements are not associated with the presence of varices or history of variceal bleeding, suggesting the possibility of intrahepatic shunting in children with advanced liver disease. Therefore, unlike in adults, HVPG measurements may not accurately predict children who are at risk of complications from portal hypertension.

    View details for DOI 10.1097/MPG.0000000000002327

    View details for PubMedID 30921261

  • Editorial: an expert consensus for the management of chronic hepatitis B in Asian Americans. Alimentary pharmacology & therapeutics Ebel, N. H., Rosenthal, P. 2018; 47 (11): 1541-1542

    View details for DOI 10.1111/apt.14620

    View details for PubMedID 29878412

  • Disparities in Waitlist and Posttransplantation Outcomes in Liver Transplant Registrants and Recipients Aged 18 to 24 Years: Analysis of the UNOS Database. Transplantation Ebel, N. H., Hsu, E. K., Berry, K., Horslen, S. P., Ioannou, G. N. 2017; 101 (7): 1616-1627

    Abstract

    We evaluated liver transplantation waitlist and posttransplantation outcomes in those aged 18 to 24 years compared with both younger (0-17 years) and older (25-34 years) registrants and recipients.Using national data from the United Network for Organ Sharing, competing risk, Cox regression and Kaplan-Meier analyses were performed on first-time liver transplant registrants (n = 13 979) and recipients (n = 8718) ages 0 to 34 years between 2002 and 2015.Nonstatus 1A registrants, registrants aged 0 to 17 and 25 to 34 years were less likely to experience dropout from the waiting list compared with those aged 18 to 24 years (adjusted hazard ratio, 0-5 years = 0.36; 6-11 = 0.29; 12-17 = 0.48; 18-24 = 1.00; 25-34 = 0.82). Although there was no difference in risk of graft failure across all age groups, both younger and older age groups had significantly lower risk of posttransplant mortality compared with those aged 18 to 24 years (adjusted hazard ratio, for 0-5 years = 0.53, 6-11 = 0.48, 12-17 = 0.70, 18-24 = 1.00, 25-34 = 0.77). This may be related to lower likelihood of retransplantation after graft failure in those aged 18 to 24 years.This national registry study demonstrates for the first time poorer waitlist and postliver transplant outcomes in young adults ages 18 to 24 years at the time of listing and transplantation compared to older and younger age groups. Given the potential survival benefit in transplanting young adults and the shortage of solid organs for transplant, future studies are critical to identify and target modifiable risk factors to improve waitlist and long-term posttransplant outcomes in 18- to 24-year-old registrants and recipients.

    View details for DOI 10.1097/TP.0000000000001689

    View details for PubMedID 28230640

    View details for PubMedCentralID PMC5481466