S J Lobritto

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Researchers developed a "discrepancy score" to quantify the gap between how children and their caregivers rate health-related quality of life after liver transplant, testing it across seven centers. Children who reported lower quality of life had larger discrepancies with their caregivers' assessments, suggesting the gap itself is a meaningful clinical signal. Closing that gap — by aligning caregiver and child perceptions — may be a practical target for improving care after pediatric liver transplantation.

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This study measured how much immunosuppression practices vary within individual pediatric liver transplant centers and whether that variability affects one-year outcomes, using data from 2,542 transplants across 67 centers. Centers with more inconsistent induction regimens had nearly three times the odds of acute rejection in the first year, though the variability did not affect graft or patient survival. Standardizing induction practices within centers could reduce rejection rates, and understanding why variation exists is the next step.

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The GUARDIAN study tested whether genome sequencing is a feasible addition to standard newborn screening in a racially and ethnically diverse New York City population, enrolling 4,000 newborns across six hospitals. Testing succeeded in over 99% of cases, 72% of families consented, and 3.7% of newborns had a positive screen — including for treatable conditions not currently covered by standard tests. These results show genome sequencing can be implemented at scale in diverse communities, expanding the window for early intervention.

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Analyzing over 2,500 pediatric liver transplants from national registries, this study found that immunosuppression choices vary enormously between centers — with steroid use and mycophenolate ranging from 0% to 100% — and that the type of induction therapy produced conflicting results across two different data sources. T-cell depleting antibody use was linked to better survival in one registry but worse outcomes in another, highlighting that existing data cannot reliably identify the best approach. Collaborative prospective research is needed to establish evidence-based immunosuppression standards.

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This single-center study assessed health literacy in adolescent liver transplant recipients and their caregivers, finding limited health literacy in 57% of patients and 47% of caregivers. Patients with adequate health literacy showed significantly higher readiness to transition to adult care, while health literacy did not significantly affect adherence or clinical outcomes in this sample. Routinely measuring health literacy before the transfer to adult care could identify teenagers who need extra support to make that transition successfully.

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Using the SPLIT pediatric liver transplant registry, this study compared long-term outcomes in children who had successfully stopped immunosuppression for at least one year against matched controls still on medications. Children off immunosuppression had similar rejection rates, growth, lab values, and overall composite outcomes compared to controls, with no graft losses. While the small sample limits broad conclusions, the findings suggest that carefully selected children can safely remain off immunosuppression without worse long-term outcomes.

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This multicenter study examined whether live vaccines for measles, mumps, rubella, and varicella are safe and effective in children who had received solid organ transplants, including 281 recipients from 18 centers. The majority of children developed protective antibody levels after vaccination, most maintained protection at one year, and serious adverse events were rare — with only five mild varicella cases and no graft rejections following vaccination. The results support reconsidering blanket restrictions on live vaccines for selected, stable pediatric transplant recipients.

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A survey of pediatric liver transplant centers in the SPLIT registry documented wide variation in routine monitoring practices — including lab frequency, protocol biopsies, and imaging — after the first post-transplant year. All centers reduced monitoring over time, but practices differed substantially, and half performed routine surveillance biopsies while half did not, often because results would not change management. This snapshot of practice variation sets the stage for studies linking specific monitoring strategies to outcomes.

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This multicenter analysis from the SPLIT registry examined survival after liver transplantation for pediatric liver cancers, covering 157 hepatoblastoma and 18 hepatocellular carcinoma cases. Three-year event-free survival was 81% for hepatoblastoma and 62% for hepatocellular carcinoma, with older age and larger tumor size predicting worse outcomes in HCC, while even salvage transplants for hepatoblastoma had comparable results to primary transplants. The data support an aggressive transplant approach for unresectable pediatric liver tumors and call for careful patient selection to distinguish truly unresectable tumors.

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This joint position paper from two major pediatric hepatology societies synthesized evidence on how COVID-19 affects children with chronic liver disease and liver transplant recipients. Children with liver disease can develop acute liver injury from COVID-19, and those on immunosuppression may face hepatic decompensation, though severe COVID-19 is less common in children than adults. The paper provides evidence-based guidance on prevention and treatment for this vulnerable population.

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A survey of pediatric liver transplant centers in the SPLIT registry found that while core practices are similar — all use written protocols, tacrolimus for maintenance, and steroids for induction — significant variation exists in how centers handle ABO-incompatible grafts, presume rejection, and treat acute rejection. The survey establishes a baseline picture of current immunosuppression practices across institutions. Connecting these practice patterns to outcomes in future research will help identify best practices.

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This multicenter trial systematically withdrew immunosuppression over 36-48 weeks in 88 stable, long-term pediatric liver transplant recipients to identify those who had developed tolerance. Thirty-eight percent achieved operational tolerance — meaning their grafts functioned normally without any immunosuppression for at least one year. Even patients who rejected during withdrawal had reversal of rejection with treatment, and no long-term graft damage occurred in either tolerant or non-tolerant patients, supporting further investigation of immunosuppression minimization strategies.

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This phase 2/3 trial tested a pediatric mini-tablet formulation of a four-drug hepatitis C regimen (ombitasvir, paritaprevir, ritonavir, and dasabuvir) plus ribavirin in 26 children aged 3-11 years with genotype 1a infection. The sustained virologic response rate was 96%, with drug exposures comparable to adult doses and no treatment-related serious adverse events. The results support this regimen as a highly effective and well-tolerated treatment option for young children with chronic hepatitis C.

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This case report describes a Sudanese child with Crigler-Najjar syndrome type 1 — a severe inherited inability to process bilirubin — in whom standard genetic testing failed to find a known mutation. A novel homozygous gene variant was identified, and laboratory testing confirmed it completely blocks the relevant enzyme's function. The case highlights that genetic diversity across populations means known mutation panels miss some patients, arguing for broader inclusion of diverse populations in genomic research.

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This phase 2/3 study evaluated a combination hepatitis C regimen (ombitasvir/paritaprevir/ritonavir with or without dasabuvir and ribavirin) in 38 adolescents aged 12-17 years with genotype 1 or 4 infection. All 38 patients achieved a sustained virologic response at 12 weeks post-treatment — a 100% cure rate — with no serious adverse events or treatment discontinuations. The treatment was safe and highly effective in adolescents, with drug levels matching those in adults.

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This cross-sectional study biopsied 157 apparently healthy pediatric liver transplant recipients with consistently normal liver tests to look for hidden chronic graft injury. Subclinical inflammation or fibrosis was found in most patients, with those showing interface activity on biopsy having a gene expression pattern linked to T-cell-mediated rejection. The findings challenge the assumption that normal blood tests mean a healthy graft, and suggest silent immune activity may be damaging livers even when children appear well.

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This case report describes a 12-year-old boy who had received a liver transplant as an infant and presented years later with an ischemic bowel due to an enterolith — a stone — blocking his Roux-en-Y biliary limb, a rare complication of biliary reconstruction. Surgical resection and revision of the biliary-intestinal connection resolved the problem. The case alerts clinicians to consider enteroliths causing bowel obstruction in long-term liver transplant survivors with biliary reconstructions.

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This study of 64 patients with portal vein thrombosis and no cirrhosis found that these patients had significantly shrunken livers — about 20-26% smaller than expected — due to reduced portal blood flow. Surgical shunts that preserved blood flow into the liver led to significant liver regrowth, while shunts that diverted blood away did not. Choosing the right type of shunt surgery can reverse liver atrophy and restore normal liver size in patients with portal vein thrombosis.

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This single-center study compared long-term outcomes in 241 children who received liver transplants from living donors versus deceased donors, following them for years after transplant. Living donor recipients had significantly lower rates of acute rejection, chronic rejection, and graft loss, and were more likely to be on minimal immunosuppression — with maternal grafts performing especially well. The data make a strong case for living donor liver transplantation in children, particularly from mothers, both for graft quality and immune advantages.

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This case report describes an 18-month-old girl with three simultaneous rare conditions — autosomal recessive polycystic kidney disease, Caroli syndrome, and hepatoblastoma — successfully treated with surgical resection alone without chemotherapy. Nine years later she remained tumor-free with stable kidney function. The case adds to a limited literature on managing concurrent pediatric liver tumors and cystic kidney disease.

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This case report describes the first attempt to combine living donor liver transplantation and stem cell transplantation from the same parent donor in a child with advanced liver cancer and lung metastases, aiming to achieve both a graft-vs-tumor effect and immune tolerance. The liver engrafted successfully and no tumor recurrence was found at autopsy, but the patient died from a fungal infection unrelated to the tumor. The case demonstrates the technical feasibility of this combined approach while highlighting the need to optimize infection prevention.

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This study found that in pediatric liver transplant recipients who develop de novo autoimmune hepatitis — an unexpected immune attack on their transplanted liver — the regulatory T cells (Tregs) that normally suppress inflammation become dysfunctional. Monocytes in the liver of affected patients produce high levels of IL-12 and IL-6, which convert Tregs into pro-inflammatory cells that lose their protective function. Blocking the inflammatory molecule IFN-gamma partially restored Treg suppression, pointing to a potential therapeutic target.

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This article reviews postoperative imaging findings in children who underwent auxiliary partial orthotopic liver transplantation, a specialized technique used in acute liver failure where a donor graft is placed alongside the native liver rather than replacing it entirely. The complex anatomy after this procedure makes imaging interpretation challenging, and awareness of normal postoperative appearances is essential for radiologists and treating clinicians. If the native liver recovers function, long-term immunosuppression can be stopped entirely.

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This case report describes an adolescent with Still's disease who developed acute liver failure while being treated with anakinra, an anti-inflammatory drug that blocks interleukin-1. The liver failure resolved completely after stopping the drug, making this the first reported case of such severe liver toxicity from anakinra in a young patient. Clinicians using anakinra for inflammatory conditions should be aware of this potentially serious hepatic side effect and monitor liver function accordingly.

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This long-term follow-up study tracked 38 children from a randomized trial of pegylated interferon treatment for hepatitis C, reassessing them 5-7 years after they achieved a sustained virologic response. All 21 children who had cleared the virus in the original trial remained virus-free at follow-up, confirming that treatment response is durable in children. The favorable long-term outcome, combined with the finding that a specific genetic variant (IL28B CC) predicts treatment success, supports treating hepatitis C in childhood when possible.

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This study characterized 49 patients with lysosomal acid lipase deficiency — a rare inherited enzyme deficiency causing fat accumulation in the liver and blood vessels — finding that disease typically begins in childhood and progresses steadily. Most patients had elevated liver enzymes, fatty liver, and abnormal cholesterol, with more than half showing liver fibrosis on biopsy, and 13% eventually requiring a liver transplant. The results establish a clear natural history for this predominantly pediatric disease and reinforce the need for early diagnosis and treatment.

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This study analyzed 20 children enrolled in an immunosuppression withdrawal trial for pediatric liver transplant, focusing on whether stopping these medications affected their risk of metabolic problems like obesity, high blood pressure, and abnormal cholesterol. Despite successfully stopping immunosuppression, the majority of tolerant children still developed multiple components of metabolic syndrome by five years, similar to controls on medication. Immunosuppression withdrawal alone does not appear to eliminate the metabolic risks that accumulate after pediatric liver transplantation.

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This seven-year single-center study evaluated serial transverse enteroplasty (STEP) — a surgery that lengthens and narrows a dilated, dysfunctional small intestine — in 20 infants and children with short bowel syndrome. Median tolerance for tube feeding increased from 22% to 61% of caloric needs within six months of surgery, and five patients eventually achieved full enteral independence. STEP is an effective surgical option for children with intestinal failure who are struggling to advance from intravenous nutrition.

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This large registry study analyzed viral testing results in 820 children who developed acute liver failure not caused by acetaminophen, finding a confirmed viral cause in only about 20% of those tested. Herpes simplex virus was the most important viral culprit, particularly in infants, yet was not tested in over half of cases. The findings highlight that viral testing for acute liver failure in children is often incomplete, and routine HSV testing — especially in infants — should be standard practice.

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This study monitored autoantibodies in 114 children with hepatitis C being treated with pegylated interferon with or without ribavirin, finding that nearly one in five already had autoantibodies before treatment began, and that the rate did not rise significantly during or after treatment. Although three children developed autoimmune diseases (diabetes and hypothyroidism) during treatment, their autoantibody levels had not predicted this. Routine autoantibody screening during pediatric hepatitis C treatment did not reliably predict who would develop clinical autoimmune disease.

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This review provides a comprehensive overview of biliary atresia — a newborn liver disease where bile ducts are progressively destroyed — covering its clinical presentation, diagnostic evaluation, and surgical management. The Kasai procedure, performed urgently in affected infants, is the initial treatment and can delay or prevent the need for liver transplantation. Liver biopsy plays a central role in the diagnostic workup, and a coordinated multidisciplinary team approach produces the best outcomes.

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This case series describes three patients with abdominal tumors that wrapped around the celiac and superior mesenteric arteries — major blood vessels that normally make such tumors unresectable — who were treated with a radical technique borrowed from multivisceral transplantation. All three had complete tumor removal with clear margins, and all were alive without recurrence at 17-38 months of follow-up. Multivisceral ex vivo surgery offers a potentially curative option for locally invasive abdominal tumors that cannot be removed by standard approaches.

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This pilot trial enrolled 20 stable pediatric liver transplant recipients from living parent donors and gradually withdrew their immunosuppression over at least nine months. Sixty percent remained off all immunosuppression for at least one year with normal graft function, and all eight who rejected responded to treatment without lasting damage. The study established feasibility and identified pre-withdrawal liver biopsy and later timing of withdrawal as predictors of success, paving the way for larger tolerance trials.

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This prospective study measured quality of life, behavior, and cognitive function in 114 children with hepatitis C before, during, and after a year of treatment with pegylated interferon with or without ribavirin. Physical quality of life scores dipped modestly during treatment but stayed within normal range, and no significant treatment-related changes occurred in behavioral, emotional, or cognitive functioning. Children and families can be reassured that this treatment regimen does not meaningfully impair psychological or cognitive wellbeing during the treatment period.

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This retrospective study of 45 pediatric solid organ transplant recipients who developed post-transplant lymphoproliferative disease found that whether the tumor expressed the cell-surface marker CD20 strongly predicted survival. Children with CD20-positive tumors had five-year event-free survival of 84% and overall survival of 96%, compared to 29% and 56% in those with CD20-negative tumors. Because most CD20-positive lymphoproliferative diseases can be treated with rituximab (which targets CD20), this marker helps explain survival differences and should guide treatment selection.

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This randomized controlled trial in 114 children aged 5-17 with chronic hepatitis C compared pegylated interferon plus ribavirin against pegylated interferon plus placebo. The combination therapy achieved a sustained viral clearance rate of 53% versus only 21% with interferon alone, and the benefit was maintained through two years of follow-up. Adding ribavirin more than doubles the cure rate for hepatitis C in children and adolescents, establishing the combination as the standard of care for this age group.

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This study examined outcomes in 93 children with intestinal failure and liver disease related to long-term intravenous nutrition, treated at a dedicated intestinal rehabilitation center using a structured program including adjusted nutrition, bowel stimulation, and surgery when needed. Among the 76 children who initially had elevated bilirubin indicating liver disease, 75% achieved normalization — with those whose liver disease resolved having only 5% mortality compared to 58% in those who did not improve. A specialized intestinal rehabilitation program can reverse liver damage in most children with intestinal failure, dramatically reducing the need for transplantation.

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This single-center review of 71 children who received liver transplants for biliary atresia — the most common reason for pediatric liver transplantation — examined what factors affect timing and outcomes. Overall patient survival was 94% at a median follow-up of nearly five years, and living donor transplantation was used in nearly half of cases with equivalent outcomes. Children with higher disease severity scores (PELD ≥10) had slightly worse outcomes, suggesting these scores should trigger timely transplant discussions.

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This pharmacokinetic study measured blood levels of mycophenolate mofetil in eight stable pediatric liver transplant recipients receiving it alongside cyclosporine, finding that drug exposure was significantly lower than in adults on the same dose per body surface area. Modeling suggested that children on cyclosporine need roughly 740 mg/m² twice daily — more than twice the typical adult dose — to achieve comparable drug levels. The results call for a dedicated dosing study to confirm the appropriate regimen in pediatric liver transplant recipients on this drug combination.

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This study described a pattern of unusual microscopic inclusions in liver cells — resembling those seen in hepatitis B but occurring in patients without that infection — identified in ten patients including transplant recipients, diabetics, and a child on long-term intravenous nutrition. Electron microscopy showed the inclusions were made of abnormal glycogen, and the authors proposed they represent a newly recognized liver finding associated with altered glycogen metabolism or certain medications. Recognizing this pattern prevents misdiagnosis as hepatitis B and prompts investigation for underlying metabolic or drug-related causes.

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This case report describes a premature newborn with multiple skin hemangiomas and liver vascular lesions who underwent liver transplantation after failing steroids and chemotherapy, only to have the liver pathology reveal angiosarcoma — a malignant vascular tumor — rather than the presumed benign hemangioma. Skin biopsies confirmed the cutaneous lesions were benign infantile hemangiomas, not metastatic cancer. The case highlights that not all liver vascular lesions in children are benign and underscores the importance of histopathological evaluation.

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This series compared hepatic artery reconstruction outcomes in 28 children during liver transplantation using either operating microscope magnification or high-power loupes, after the surgeons switched between techniques. Neither approach resulted in any hepatic artery thrombosis — the main surgical complication — and outcomes were equivalent between the two groups. High-power loupe magnification (6x) can achieve the same results as operative microscopy in pediatric liver transplant hepatic artery reconstruction, offering more flexibility without sacrificing safety.

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This single-center comparison of 45 living donor liver transplants in adults versus children found that while donor outcomes were equivalent, adult recipients had significantly worse graft survival (65% versus 91% at one year), more biliary leaks, and more hepatic vein complications. Problems unique to adults included small-for-size syndrome and late-diagnosed biliary leaks that were fatal in some cases. These findings shaped the field by demonstrating that techniques developed in children require modification before being safely extended to adult recipients.

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This retrospective review identified four patients who developed serious lung disease — including bronchiolitis obliterans and interstitial pneumonitis — while taking interferon and ribavirin for hepatitis C as part of clinical trials. All four recovered after stopping the drugs, with or without steroids, and one case represented the first reported pulmonary toxicity from the then-new long-acting pegylated interferon. Pulmonary symptoms that progress or are more than mild in patients on interferon-ribavirin therapy should prompt investigation for drug-induced lung disease, even though the complication is rare.

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This review covers the use of endoscopy in children with chronic liver disease, discussing how pediatric-specific equipment, anesthetic agents, and specialized units have made this a safe and effective diagnostic and therapeutic tool. Endoscopy serves an important role in managing complications of portal hypertension, including variceal bleeding, in pediatric patients. Ongoing technical advances continue to expand what can be achieved endoscopically in this demanding patient population.