Preventing Parenteral Nutrition-Associated Liver Disease in Children

46 Questions

What is the incidence of short bowel syndrome in neonates?

24.5 per 100,000 live births

What percentage of patients with intestinal failure will develop PNALD?

Two-thirds

What is the case fatality rate of short bowel syndrome in neonates?

37.5%

What is the purpose of this clinical guideline?

To develop recommendations for the care of children with PN-dependent intestinal failure

What is the name of the working group that developed the concepts used to evaluate the evidence in this guideline?

The Grading of Recommendations, Assessment, Development, and Evaluation (GRADE) Working Group

How many questions were used to develop the recommendations in this guideline?

What is the term used to describe PNALD in this guideline?

Parenteral nutrition–associated liver disease (PNALD)

What percentage of patients with PNALD traditionally progress to end-stage liver disease?

25%

What percentage of long-term survival does PNALD have?

70%–90%

What is the nature of PNALD?

Cholestatic

What is the primary reason for using liver biopsies as endpoints?

To depict a picture of cholestasis and varying degrees of fibrosis

What type of fatty acids are predominantly found in soy-based fat emulsions?

ω-6 long-chain polyunsaturated fatty acids

What is a risk factor for PNALD in premature babies?

Imature livers with incompletely expressed enzymatic activity

What is the effect of endotoxin on bile transport proteins?

It impairs bile transport proteins

What is the goal of therapy in patients with PNALD?

To optimize intestinal function and promote gut adaptation

What is a common complication in patients with PN-dependent intestinal failure?

Catheter-related bloodstream infection (CLABSI)

What is the consequence of frequent CLABSIs in patients with PNALD?

Increased risk of PNALD

Why are children with intestinal failure at risk of CLABSIs?

They have feeding enterostomies, stomas, and overgrowth of intestinal bacteria

What is the primary objective of instilling 70% ethanol as a lock solution into the PN catheter?

To prevent CLABSI and reduce catheter replacements

What is the minimum concentration of ethanol required to penetrate and break down biofilm in laboratory studies?

30%

What is the recommended dwell time for 70% ethanol lock solution to be effective in vivo?

2 hours

What type of catheters have been tested with ethanol lock therapy in children?

Only silicone

What is the association between IV SOE and PNALD in mixed adult and pediatric home PN cohorts?

Positive association

What is the purpose of adding fish oil emulsion (FOE) to the treatment of children with HPN who develop PNALD?

To reduce the dose of SOE

What is the primary mechanism of ursodeoxycholic acid (UDCA) in treating cholestatic liver disease?

Correcting bile acid deficiency and improving bile flow

What is the purpose of multidisciplinary nutrition support teams or intestinal rehabilitation programs in children with intestinal failure?

To optimize the management of children with intestinal failure who require HPN

What is the frequency of planned revisions for this clinical guideline?

In 2018

What is the recommendation grade for the use of ethanol lock to prevent CLABSI and reduce catheter replacements in children at risk of PNALD?

Weak

What is the recommended dose of SOE to treat cholestasis in children with PNALD?

≤1 g/kg/d

What is the status of FOE in the United States?

Available under a compassionate use protocol

What is the primary outcome of RCTs studying SMOF in preterm infants?

Plasma phospholipid profiles and adverse events

What is the current status of fat emulsions containing a blend of refined olive and soybean oil for use in pediatric patients?

Approved for use in adult patients receiving PN

What is the recommended treatment for elevated liver enzymes in children with PNALD?

Enteral UDCA

What is the concern with restricting lipids in pediatric patients with PNALD?

Poor growth and development

What is the benefit of managing patients with PNALD by a multidisciplinary intestinal rehabilitation team?

Improved PNALD outcomes

What is the limitation of the studies on UDCA for the treatment of PNALD?

Confounded by concurrent lipid dose reduction and advancement of enteral feedings

What is the current recommendation for the use of SMOF in pediatric patients with PNALD?

No recommendation can be made

What is the grading of the recommendation for the use of UDCA in pediatric patients with PNALD?

Weak

What is the quality of the evidence supporting the recommendation to refer patients with PN-dependent intestinal failure to multidisciplinary intestinal rehabilitation programs?

Very low

What is the primary reason for the weak recommendation to refer patients with PN-dependent intestinal failure to multidisciplinary intestinal rehabilitation programs?

The evidence is of very low quality

What is the main limitation of the studies comparing clinical outcomes before and after the establishment of multidisciplinary intestinal rehabilitation programs?

Historical controls

What is the relative risk of survival from intestinal failure in patients treated with multidisciplinary intestinal rehabilitation programs compared to historical controls?

1.22 (95% CI, 1.06-1.40)

What is the main reason for the heterogeneity of patient populations in the studies on multidisciplinary intestinal rehabilitation programs?

All of the above

What is the primary improvement needed in the literature on multidisciplinary intestinal rehabilitation programs?

Consensus on definitions of key clinical outcomes

What is the likely impact of further research on the recommendation to refer patients with PN-dependent intestinal failure to multidisciplinary intestinal rehabilitation programs?

Further research is likely to change this recommendation

What is the primary outcome of interest in the meta-analysis by Stanger et al?

Survival from intestinal failure

Study Notes

Parenteral Nutrition-Associated Liver Disease (PNALD)

PNALD is a life-threatening complication associated with parenteral nutrition (PN) dependence.
Incidence of short bowel syndrome in neonates: 24.5 per 100,000 live births with a case fatality rate of 37.5%.
Two-thirds of patients with intestinal failure will develop PNALD.
Prevention of PNALD can improve the quality of life of children and their families.

Characteristics of PNALD

Cholestatic in nature with a spectrum of disease, ranging from mild cholestasis to cirrhosis and liver failure.
No standardized definition of PNALD, but often described by hyperbilirubinemia (direct or total).
Liver biopsy is invasive and not practical for routine care, and is prone to sampling error.

Risk Factors of PNALD

Premature babies have an increased risk for PNALD due to immature livers with incompletely expressed enzymatic activity.
Recurrent sepsis increases the risk of cholestasis.
Lack of enteral feeding impairs the enterohepatic circulation and bile acid secretion/absorption, leading to mucosal atrophy and bacterial translocation.

CLABSIs are associated with an increased risk of PNALD when they occur frequently.
The instillation of 70% ethanol as a lock solution into the PN catheter has been examined as a strategy to prevent CLABSI.

Fat Emulsion Strategies

High doses of soy-based fat emulsions (SOEs) may promote cholestasis.
Reduced doses of SOEs, addition of fish oil emulsion (FOE), and fat emulsions designed with a mixture of soy oil, medium-chain triglycerides, olive oil, and fish oil (SMOF) have been considered as potential therapies in children with PN-dependent intestinal failure.

Ursodeoxycholic Acid (UDCA)

UDCA is a bile acid that has been given orally to treat cholestatic liver disease in adults.
The mechanism of UDCA's effects is not fully established, but may correct bile acid deficiency, improve bile flow, displace cytotoxic bile acids, or provide immunomodulatory protection.

Multidisciplinary Intestinal Rehabilitation Team

Multidisciplinary nutrition support teams or intestinal rehabilitation programs have been developed to optimize the management of children with intestinal failure who require HPN.
The impact of these programs on PNALD outcomes has been examined, with studies suggesting improved survival and clinical outcomes.

This quiz covers clinical guidelines for the care of children with intestinal failure dependent on parenteral nutrition, focusing on preventing or treating parenteral nutrition-associated liver disease. It's based on a systematic review of the best available evidence.

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