About

Elizabeth B. Rand, M.D., is an Emeritus Professor and the Chief of Pediatrics at the Children's Hospital of Philadelphia (CHOP). She serves as the Director of the Hepatology Fellowship Training Program at CHOP and is a physician in the Department of Pediatrics. Her educational background includes an A.B. in Chemistry from the University of Chicago, earned in 1983, and an M.D. from the same institution, completed in 1987. Dr. Rand's professional focus is on pediatric hepatology, with particular expertise in liver transplant and liver disease. She has contributed to research on Fontan-associated liver disease, pediatric immunosuppression minimization, and the phenotypic spectrum of Hardikar syndrome. Her work involves advancing understanding of pediatric liver conditions and improving clinical outcomes for affected children.

Research topics

• Surgery
• Medicine
• Gastroenterology
• Internal medicine

Selected publications

• The Landscape of Status <scp>1B</scp> by Exception for Pediatric Acute on Chronic Liver Failure—High Risk, High Mortality, and Low Transplant Rate

  Pediatric Transplantation · 2025-12-22

  article

  BACKGROUND: Acute on chronic liver failure (ACLF) leads to increased mortality from multiorgan failure. METHODS: Our objective was to evaluate differences in characteristics and outcomes of children with ACLF listed as Status 1B for liver transplantation (LT) by (1) explicit criteria or (2) exception. We identified 367 children with ACLF who were listed Status 1B for LT in the United Network for Organ Sharing (UNOS) database between January 2007 and March 2019. Children with metabolic diseases and malignancies were excluded. RESULTS: Of 367 children with ACLF listed as Status 1B, 110 (30%) were listed by exception. There were no differences between groups in age, sex, race/ethnicity, or diagnosis. Incidence of cumulative organ failures and MELD/PELD scores were lower in the exception group at waitlisting and at waitlist removal (all p < 0.05). There were regional differences in exception utilization with regions 2 and 10 having a higher proportion of exception listing (43% and 47% vs. overall 30%, p = 0.03). There was no difference in transplantation rate, recovery without transplant, or pre-/post-LT mortality between groups. Narrative summaries in the exception group cited the critical or refractory nature of the patient's illness, with increased risk of waitlist mortality as reasons for urgent LT. CONCLUSION: Our study showed that one third of children with ACLF listed as Status 1B receive this designation using exception criteria. Children listed by exception had similar pre-/post-LT mortality compared to the explicit group. Further study is needed to understand why these children are at higher risk than expected.

  PublisherDOI

• Clinical Characteristics Associated With Variability in Biopsy Grade Severity of Liver Fibrosis in Fontan Circulation

  JACC Advances · 2025-12-15 · 1 citations

  articleOpen access

  BACKGROUND: Liver fibrosis in Fontan circulation (FC) is universal with variable severity. We explore associations between patient-specific characteristics and fibrosis magnitude measured by liver biopsy. OBJECTIVES: The purpose of this study was to identify clinical and hemodynamic factors associated with fibrosis severity in FC, quantified by Sirius red staining of liver biopsy specimens, and to evaluate relationships with patient characteristics, Fontan hemodynamics, and noninvasive liver assessments. METHODS: Subjects with FC underwent a standardized evaluation including cardiac catheterization, percutaneous liver biopsy, liver elastography, and blood laboratories. Sirius red % staining was used to quantitatively assess collagen deposition per liver tissue to measure fibrotic severity and associations with clinical characteristics explored. RESULTS: Among 173 subjects, median age at Fontan operation (FO) was 2.2 years (range: 0.5-15), and median age at biopsy was 17.5 years (range: 4.7-39.4). Sixty-seven percent had dominant right ventricular morphology. Median systemic venous pressure at catheterization was 13 mm Hg (IQR: 11.0,15.0). Median Sirius red % staining was 21.6% (IQR: 17.3,30.2). Sirius red % staining was not associated with ventricular morphology, catheter hemodynamics, or liver elastography. Sirius red % staining correlated with aspartate aminotransferase/platelet ratio (P = 0.026). Years of FC exposure correlated positively with Sirius red % staining, hemoglobin, gamma glutamyl transferase, and liver elastography. Age at FO was inversely correlated with Sirius red % staining. CONCLUSIONS: Liver fibrosis in FC progresses with duration of exposure, independent of venous pressure, suggesting ongoing fibrogenesis even at acceptable hemodynamics. Younger age at FO is associated with greater fibrosis severity and may represent a modifiable factor influencing liver health.

  PublisherDOI

• Treatment of intractable pruritus with maralixibat in patients with Alagille syndrome before and after reversal of biliary diversion

  JPGN Reports · 2025-09-30

  articleOpen access

  Alagille syndrome (ALGS) is a rare, cholestatic, multisystemic disorder characterized by bile duct paucity. Cholestatic pruritus is a common, and often severe, symptom of ALGS and is the leading cause of liver transplantation. The treatment of cholestatic pruritus is challenging and involves medical and surgical options, such as surgical biliary diversion (SBD) for refractory cases. However, SBD is associated with medical/lifestyle challenges. Maralixibat, an ileal bile acid transporter inhibitor, is a recently approved treatment for cholestatic pruritus in patients with ALGS and is used as part of standard of care. We present cases of two patients with ALGS who initiated treatment with maralixibat: one before, with continuation after, reversal of SBD, and one after SBD reversal. In both cases, treatment with maralixibat was well-tolerated and demonstrated marked improvements in cholestatic pruritus. This suggests that maralixibat is a pharmacological alternative for patients who would like to pursue reversal of SBD.

  PublisherOA PDFDOI

• Condition-Specific Growth Charts for Children With Alagille Syndrome

  JAMA Network Open · 2025-11-24 · 1 citations

  articleOpen access

  Importance: Different degrees of growth delay have been reported in children with Alagille syndrome (ALGS), yet these patients are routinely evaluated using standard growth charts. Objective: To develop condition-specific growth charts for ALGS using modern statistical approaches. Design, Setting, and Participants: This case series used data from the international, multicenter Global Alagille Alliance (GALA) study accrued between May 14, 2018, and March 20, 2023. Children born at full term between January 1, 1997, and August 31, 2019, with a clinically and/or genetically confirmed ALGS diagnosis and their native liver were included. Data from children with a known history of prematurity were excluded for the development of the growth charts. Data were analyzed from March 25, 2023, to December 30, 2024. Exposure: Growth of children with Alagille syndrome. Main Outcomes and Measures: Generalized additive models for location scale and shape were fitted to generate percentile plots for weight and height relative to age and superimposed on US Centers for Disease Control and Prevention (CDC) growth charts to illustrate differences in growth patterns compared with children with typical development. Results: Data from 1204 children with ALGS in overlapping cohorts (median [IQR] gestational age, 38 [37-39] weeks) were analyzed (1204 in the weight cohort; 695 boys [57.7%]; 9855 weight observations; 995 with neonatal cholestasis [82.6%]; 306 receiving a liver transplant [25.4%]; 98 deaths [8.1%] and 1106 in the height cohort, 635 boys [57.4%]; 8464 height observations; 906 with neonatal cholestasis [81.9%]; 287 receiving a liver transplant [25.9%]; 86 deaths [7.8%]) were included for the modeling of the weight-for-age and height-for-age charts, respectively. The median birth weight was 2.8 kg (IQR, 2.5-3.0 kg) for boys and 2.6 kg (IQR, 2.4-2.9 kg) for girls. The median birth length was 48.0 cm (IQR, 46.0-50.0 cm) for boys and 47.0 cm (IQR, 45.0-49.0 cm) for girls. The weight-for-age and height-for-age growth charts for boys and girls with AGLS differed significantly from CDC growth charts. The estimated height at age 18 years corresponded to the 50th percentile was 171.5 cm for boys and 156.5 cm for girls on the condition-specific charts vs 176 cm and 163 cm, respectively, on the CDC growth charts. Conclusions and Relevance: These findings suggest that condition-specific growth charts for ALGS may provide a crucial tool for clinicians to evaluate growth and aid in decision-making around listing children for liver transplant.

  PublisherDOI

• Postvaccination Immunogenicity Among Pediatric Solid Organ Transplant Recipients

  JAMA Pediatrics · 2025-02-24 · 6 citations

  articleOpen access

  This cohort study assesses the protection provided by live attenuated viral vaccines among a pediatric population of solid organ transplant recipients 1 year after vaccination.

  PublisherOA PDFDOI

• Liver Transplant From a Deceased Donor With Cystinosis: A Case Report

  JIMD Reports · 2025-01-01

  articleOpen access

  Many inherited metabolic disorders (IMD) are associated with end-organ damage necessitating organ transplantation. Although utilization of deceased donors with history of IMD warrants caution, there may be circumstances under which such donors could be considered as suitable organ donor candidates. We present the first known report of liver transplantation from a deceased donor with cystinosis. The donor was a 20-year-old male with infantile cystinosis who had previously undergone two deceased donor kidney transplants. Unfortunately, he incurred cranial trauma, and after careful consideration of the metabolic consequences, his liver was deemed suitable for transplantation. The liver was successfully transplanted into a 65-year-old female recipient with hepatitis C (HCV) cirrhosis. The recipient is currently 12 months post-transplant and experiencing good graft function without evidence of cystine crystals on liver biopsy. This case highlights that liver transplantation from donors with rare IMD can result in favorable outcomes. However, it is crucial to approach the use of such livers with caution. These transplants should be considered after a thorough assessment, ensuring that a comprehensive decision-making process is in place to mitigate potential risks.

  PublisherOA PDFDOI

• Sound it out: Ultrasound findings in infantile cholestasis

  Journal of Pediatric Gastroenterology and Nutrition · 2025-06-02 · 1 citations

  article1st authorCorresponding

  The author declares no conflict of interest.

  PublisherDOI

• Condition-Specific Growth Charts for Children With Alagille Syndrome

  Utrecht University Repository (Utrecht University) · 2025-11-03

  articleOpen access

  IMPORTANCE: Different degrees of growth delay have been reported in children with Alagille syndrome (ALGS), yet these patients are routinely evaluated using standard growth charts. OBJECTIVE: To develop condition-specific growth charts for ALGS using modern statistical approaches. DESIGN, SETTING, AND PARTICIPANTS: This case series used data from the international, multicenter Global Alagille Alliance (GALA) study accrued between May 14, 2018, and March 20, 2023. Children born at full term between January 1, 1997, and August 31, 2019, with a clinically and/or genetically confirmed ALGS diagnosis and their native liver were included. Data from children with a known history of prematurity were excluded for the development of the growth charts. Data were analyzed from March 25, 2023, to December 30, 2024. EXPOSURE: Growth of children with Alagille syndrome. MAIN OUTCOMES AND MEASURES: Generalized additive models for location scale and shape were fitted to generate percentile plots for weight and height relative to age and superimposed on US Centers for Disease Control and Prevention (CDC) growth charts to illustrate differences in growth patterns compared with children with typical development. RESULTS: Data from 1204 children with ALGS in overlapping cohorts (median [IQR] gestational age, 38 [37-39] weeks) were analyzed (1204 in the weight cohort; 695 boys [57.7%]; 9855 weight observations; 995 with neonatal cholestasis [82.6%]; 306 receiving a liver transplant [25.4%]; 98 deaths [8.1%] and 1106 in the height cohort, 635 boys [57.4%]; 8464 height observations; 906 with neonatal cholestasis [81.9%]; 287 receiving a liver transplant [25.9%]; 86 deaths [7.8%]) were included for the modeling of the weight-for-age and height-for-age charts, respectively. The median birth weight was 2.8 kg (IQR, 2.5-3.0 kg) for boys and 2.6 kg (IQR, 2.4-2.9 kg) for girls. The median birth length was 48.0 cm (IQR, 46.0-50.0 cm) for boys and 47.0 cm (IQR, 45.0-49.0 cm) for girls. The weight-for-age and height-for-age growth charts for boys and girls with AGLS differed significantly from CDC growth charts. The estimated height at age 18 years corresponded to the 50th percentile was 171.5 cm for boys and 156.5 cm for girls on the condition-specific charts vs 176 cm and 163 cm, respectively, on the CDC growth charts. CONCLUSIONS AND RELEVANCE: These findings suggest that condition-specific growth charts for ALGS may provide a crucial tool for clinicians to evaluate growth and aid in decision-making around listing children for liver transplant.

  Publisher

• Characteristics and Survival Outcomes of Hepatocellular Carcinoma After the Fontan Operation

  JACC Advances · 2025-03-12 · 10 citations

  articleOpen access

  BACKGROUND: The Fontan operation is a surgical procedure to palliate single ventricle congenital heart disease. Hepatocellular carcinoma (HCC) is a rare complication of Fontan-associated liver disease (FALD). OBJECTIVES: The authors aim to examine characteristics of individuals with Fontan circulation diagnosed with HCC and to describe tumor characteristics, treatment, and survival outcomes of these patients. METHODS: This was a multicenter retrospective case-control study of adults with Fontan circulation between 2005 and 2021. HCC cases were included based on histology or imaging-based diagnosis. Controls were randomly selected in a 3:1 ratio from the center in which the case was derived. Descriptive statistics were used to compare groups and Kaplan-Meier survival analysis was performed. RESULTS: There were 58 cases of HCC diagnosed at a median age of 31 (IQR: 26-38) years. Diagnosis was made at very early or early stage disease in 68%. Compared to controls, cases had higher prevalence of advanced FALD including varices, ascites, splenomegaly, and decreased platelets. Treatment with curative intent (combined heart-liver transplantation, resection, or ablation) was performed in 41%. Survival at 1 year was 78.9% and highest among those diagnosed at very early or early stage. Over half were undergoing active surveillance at diagnosis, which showed a nonsignificant trend toward higher survival (P = 0.088). CONCLUSIONS: We describe the clinical characteristics, treatment, and survival in patients with FALD-HCC. Results suggest that adults with FALD-HCC diagnosed with early stage disease may have survival benefit. Our findings underscore the importance of HCC screening for early detection in individuals after the Fontan operation.

  PublisherDOI

• Elevated Serum Bile Acids Predict Poor Liver Outcomes in Children With Alagille Syndrome: Results From the <scp>GALA</scp> Study Group

  Liver International · 2025-11-18 · 1 citations

  articleOpen access

  BACKGROUND AND AIM: Alagille syndrome (ALGS) is a rare disorder characterised by cholestasis and extrahepatic manifestations. Given the current era of ileal bile acid transporter (IBAT) inhibitor therapies that reduce serum bile acid (SBA) levels, we evaluated whether SBA predicts liver disease outcomes in ALGS. METHODS: Patients were ascertained from the Global ALagille Alliance (GALA) cohort. A prognostic threshold of SBA 102 μmol/L was assessed as a time-dependent covariate in Cox regression analyses for native liver survival (NLS) and event-free survival (EFS), while adjusting for total bilirubin (TB) levels. RESULTS: 570 GALA patients were included (348 [61%] male). There was a moderate positive correlation between SBA and TB (Pearson correlation = 0.47, p < 0.001). SBA below 102 μmol/L was a significant predictor of outcomes (NLS: HR = 3.78, 95% CI 2.39-5.99, p < 0.001; EFS: HR = 3.44, 95% CI 2.35-5.04, p < 0.001). SBA remained a significant predictor for improved EFS after adjusting for TB clearance at 1 year (TB < 2 mg/dL; HR = 2.00, 95% CI 1.10-3.65, p = 0.02). Median SBA in the first year of life above 102 μmol/L, predicted lower NLS (67.2% vs. 83.5% at 7 years p = 0.05) and EFS (63.4% vs. 80.9% at 7 years, p = 0.02). CONCLUSION: Lower SBA in children with ALGS liver disease predicts improved NLS and EFS. SBA is also associated with NLS in children with ALGS who clear their bilirubin, that is, those with anicteric cholestasis. Although the patients studied here did not receive IBAT inhibition, these data suggest that lowering SBA may improve important clinical outcomes.

  PublisherOA PDFDOI

Recent grants

• NIH Grant K08DK002338

  NIH · $522k · 2001

Frequent coauthors

• Rebecca Taub

  Madrigal Pharmaceuticals (United States)

  41 shared

• Nancy B. Spinner

  Children's Hospital of Philadelphia

  37 shared

• Jack Rychik

  University of Pennsylvania

  36 shared

• Binita M. Kamath

  SickKids Foundation

  35 shared

• David A. Piccoli

  34 shared

• David Goldberg

  Children's Hospital of Philadelphia

  34 shared

• Pierre Russo

  31 shared

• Kathleen M. Loomes

  Children's Hospital of Philadelphia

  30 shared

Labs

• Elizabeth B. Rand LabPI

Education

• MD, School of Medicine

  The University of Chicago

  1987