Transplantation for Cholestatic Liver Disease in Children

Ibrahim, Samar H.; Bezerra, Jorge A.; Balistreri, William F.

doi:10.1016/b978-1-4557-0268-8.00025-7

Cited by 2 publications

(10 citation statements)

References 159 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Two children in our cohort had undergone Kasai‐PE. Kasai‐PE in AGS may increase the degree of liver injury and progression to hepatic fibrosis and worsen overall outcomes . It may be difficult to differentiate from BA in early infancy as the dysmorphic features may not be evident and histopathological features may be evolving .…”

Section: Discussionmentioning

confidence: 99%

“…It may be difficult to differentiate AGS from BA in the neonatal period. Both can present with high gamma‐glutamyl transferase levels, absent or small gallbladder, and absent intestinal excretion in biliary scintigraphy . Diagnosis is further complicated by the fact that liver biopsy may not show bile duct paucity in 40% of patients with AGS before 6 months of age .…”

Section: Discussionmentioning

confidence: 99%

“…Clinical data of children with AGS who underwent LT between July 2009 and May 2019 in our unit were retrospectively analyzed. The diagnosis was based on modified criteria for AGS; the presence of mutation in JAG1 or NOTCH2, family history of AGS, or clinical signs were considered . Clinical criteria for diagnosis included consistent liver histology (bile duct paucity and/or cholestasis on explant histopathology), cardiac, renal, ocular, or skeletal involvement, structural vascular anomalies, and presence of characteristic facies .…”

Section: Methodsmentioning

confidence: 99%

“…The diagnosis was based on modified criteria for AGS; the presence of mutation in JAG1 or NOTCH2, family history of AGS, or clinical signs were considered . Clinical criteria for diagnosis included consistent liver histology (bile duct paucity and/or cholestasis on explant histopathology), cardiac, renal, ocular, or skeletal involvement, structural vascular anomalies, and presence of characteristic facies . Bile duct paucity is defined as absence or marked reduction in the number of bile ducts in the portal triads (<0.5 interlobular bile ducts per triad) in the presence of normal‐sized branches of the portal vein and hepatic artery …”

Section: Methodsmentioning

confidence: 99%

“…20%‐30% of children with AGS will require LT over their lifetime . The indications for LT in AGS are refractory pruritus with poor quality of life, end‐stage liver disease, portal hypertension, and recurrent femur fractures . LT in AGS can be complicated by the associated cardiac, renal, and vascular anomalies .…”

Section: Introductionmentioning

confidence: 99%

See 4 more Smart Citations

Living donor liver transplantation in Alagille syndrome—Single center experience from south Asia

Valamparampil¹,

Reddy²,

Shanmugam³

et al. 2019

Pediatric Transplantation

View full text Add to dashboard Cite

To analyze the clinical characteristics and the outcomes of living donor liver transplantation in children with Alagille syndrome (AGS). Clinical data of children with AGS who underwent liver transplantation between July 2009 and May 2019 in our unit were retrospectively analyzed. Primary end‐points were patient and graft survival. Ten children with AGS underwent living donor liver transplantation at a median age of 28 months (range, 12‐84 months). Jaundice was the most common initial symptom and was noted after a median duration of 20 days after birth (range, 7‐60 days). Two patients had undergone Kasai porto‐enterostomy for misdiagnosis of biliary atresia. The most common indication for transplantation was severe pruritus with poor quality of life. Explant livers in three children showed cirrhosis with early well‐differentiated hepatocellular carcinoma. We have 100% patient and graft survival at a mean follow‐up of 32 months (range 3‐72 months). The median z‐score for weight and height at liver transplantation was −2.66 (range: −6.44 to −0.9) and −3.6 (range: −7.96 to −0.93) while at follow‐up was −1.7 (range: −3.4 to −0.35) and −2.1 (range: −3.9 to −1.4), respectively. The estimated glomerular filtration rate was normal pretransplant and follow‐up. This is the first series of LDLT for Alagille syndrome in the Indian sub‐continent. We report excellent post‐transplant outcomes in contrast to outcomes reported from Western literature.

show abstract

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 3 more Smart Citations

Living donor liver transplantation in Alagille syndrome—Single center experience from south Asia

Valamparampil¹,

Reddy²,

Shanmugam³

et al. 2019

Pediatric Transplantation

View full text Add to dashboard Cite

show abstract

Cholestatic liver diseases of genetic etiology: Advances and controversies

et al. 2022

Self Cite

View full text Add to dashboard Cite

With the application of modern investigative technologies, cholestatic liver diseases of genetic etiology are increasingly identified as the root cause of previously designated “idiopathic” adult and pediatric liver diseases. Here, we review advances in the field enhanced by a deeper understanding of the phenotypes associated with specific gene defects that lead to cholestatic liver diseases. There are evolving areas for clinicians in the current era specifically regarding the role for biopsy and opportunities for a “sequencing first” approach. Risk stratification based on the severity of the genetic defect holds promise to guide the decision to pursue primary liver transplantation versus medical therapy or nontransplant surgery, as well as early screening for HCC. In the present era, the expanding toolbox of recently approved therapies for hepatologists has real potential to help many of our patients with genetic causes of cholestasis. In addition, there are promising agents under study in the pipeline. Relevant to the current era, there are still gaps in knowledge of causation and pathogenesis and lack of fully accepted biomarkers of disease progression and pruritus. We discuss strategies to overcome the challenges of genotype–phenotype correlation and draw attention to the extrahepatic manifestations of these diseases. Finally, with attention to identifying causes and treatments of genetic cholestatic disorders, we anticipate a vibrant future of this dynamic field which builds upon current and future therapies, real‐world evaluations of individual and combined therapeutics, and the potential incorporation of effective gene editing and gene additive technologies.

show abstract

Transplantation for Cholestatic Liver Disease in Children

Cited by 2 publications

References 159 publications

Living donor liver transplantation in Alagille syndrome—Single center experience from south Asia

Living donor liver transplantation in Alagille syndrome—Single center experience from south Asia

Cholestatic liver diseases of genetic etiology: Advances and controversies

Contact Info

Product

Resources

About