Evidence map›Paper›PMID 42288684›Full record

ArticleWorld journal of pediatrics : WJP2026

Pediatric liver transplantation for metabolic diseases: a single-center experience.

Gabriel E Gondolesi, Erica Chen, William Zhu, Chau Nguyen, Megan Song, Chelsea Sooy, Khalid Khan, Alexander Kroemer, Udeme Ekong, Brian Nguyen and 21 more

Abstract readComparative Study
PubMed Publisher
In one paragraph

Article in World journal of pediatrics : WJP, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

31 authors.

Gabriel E GondolesiMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA. gabriel.e.gondolesi@medstar.net.ORCID http://orcid.org/0000-0002-3869-6213
Erica ChenGeorgetown University School of Medicine, Washington, DC, USA.
William ZhuGeorgetown University School of Medicine, Washington, DC, USA.
Chau NguyenGeorgetown University School of Medicine, Washington, DC, USA.
Megan SongMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Chelsea SooyMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Khalid KhanMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Alexander KroemerMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Udeme EkongMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Brian NguyenMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Carolina RumboMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Megan JohnsonMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Kathryn LaRussoMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Lindsey BledsoeMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
David WallsMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Tammy HanelMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Michelle EatonMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Julia ClaytonMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Erin MacLeodChildren's National Rare Disease Institute, Washington, DC, USA.
Kelly WeaverMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Megha FitzpatrickMedStar Georgetown Pediatric Department, MedStar Georgetown University Hospital, Washington, DC, USA.
Deborah RegierChildren's National Rare Disease Institute, Washington, DC, USA.
Nicholas Ah MewChildren's National Rare Disease Institute, Washington, DC, USA.
Gabriel HauserMedStar Georgetown Pediatric Department, MedStar Georgetown University Hospital, Washington, DC, USA.
Sumeet GopwaniMedStar Georgetown Pediatric Department, MedStar Georgetown University Hospital, Washington, DC, USA.
Cambria HeustonChildren's National Rare Disease Institute, Washington, DC, USA.
Cal MatsumotoMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Kimberly ChapmanChildren's National Rare Disease Institute, Washington, DC, USA.
Stuart S KaufmanMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Nada YazigiMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.
Thomas FishbeinMedStar Georgetown Transplantation Institute, MedStar Georgetown University Hospital, 3800 Reservoir Road Northwest, Suite 2, Washington, DC, 20007, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundInborn errors of metabolism (IEM) are rare enzymatic disorders that often result in severe multiorgan dysfunction and death in pediatric patients. While advances in medical therapy of IEM have improved considerably, pediatric liver transplantation (pLT) has become an established treatment for many metabolic conditions. This study aims to compare the prognosis of patients with and without IEM indications after pLT.

methodsA mixed retrospective and prospective cohort study of 330 pediatric patients who underwent pLT from 2003 to 2025 was performed. Patients were categorized into IEM and non-IEM cohorts, with the IEM group further stratified by indication into two subgroups: pLT indicated for primary progressive liver disease (IEM-LD) or for neuroprotection (IEM-NP). Clinical characteristics, perioperative variables, complications, and long-term outcomes were compared between groups, with additional analyses by cholestatic phenotype. Survival was assessed using Kaplan-Meier (KM) methods and compared using the log-rank test, and longitudinal outcomes were evaluated using generalized estimating equations.

resultsOf 330 pLT recipients, 117 (35%) had IEM and 213 (65%) had non-IEM indications. IEM recipients had significantly fewer post-transplant vascular complications than non-IEM recipients and experienced shorter hospital stays (P = 0.016) and improved long-term patient (P = 0.040) and graft survival (P = 0.030). There were no differences in biliary complications, rejection episodes, cytomegalovirus (CMV) or Epstein Barr virus (EBV) disease, or post-transplant lymphoproliferative diseases (PTLD) between groups. Reduced-size grafts were used more frequently in IEM recipients (P < 0.002), whereas living-donor grafts were more common in non-IEM recipients (P < 0.003). Long-term metabolic and renal outcomes, including BMI, obesity, insulin use, and renal function, were similar between groups. Within the IEM cohort, IEM-NP recipients had improved patient survival (P = 0.020) compared with IEM-LD, with no difference in graft survival. IEM-NP recipients demonstrated reduced dependence on restrictive diets and disease-specific medications.

conclusionsLiver transplantation has become an effective intervention strategy for many IEMs that are refractory to conventional non-surgical management. This progress has been facilitated by advances in surgical techniques, immunosuppression, close collaboration between transplant and metabolic disease teams, and innovative organ sourcing. Our findings further support pLT as a cornerstone therapy for IEM patients. Ongoing assessment of optimal timing, technical strategies, and follow-up after pLT may further improve patients' prognoses and quality of life.

Indexed as

Liver TransplantationMetabolic DiseasesMetabolism, Inborn ErrorsAdolescentChildChild, PreschoolCohort StudiesFemaleGraft SurvivalHumansInfantMalePrognosisProspective StudiesRetrospective StudiesTreatment OutcomeInborn errors of metabolismLiver transplantationMetabolic diseasesPediatric

Identifiers

PMID42288684

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.