Bilary Atresia Prognosis After 2-Year Survival with Native Liver

A recent study (M Witt et al. JPGN 2018; 689-94) indicates that among patients with biliary atresia who reached 2 years of life with native liver survival (NLS), they continued to be at risk for progressive liver failure.

Key findings:

  • Upon a median follow-up of 16.4 years, NLS rates at 5, 10, 15, 18 years of age were 89%, 72%, 60%, 54%, respectively.
  • Corresponding overall survival rates were 98%, 90%, 87%, 87%, respectively
  • NLS ended in 37% by liver transplantation (LTx) and in 6% by (pre-transplant) mortality.
  • Abstract Link: Prognosis of Biliary Atresia After 2-year Survival With Native Liver

My take: This data provides more precise information for families about prognosis and reinforces the need for careful followup.

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Parker Ridge, near Banff

 

 

The Half Empty Glass: Rumination Outcomes

Briefly noted:

A Alioto, C DiLorenzo. JPGN 2018; 66: 21-25.  In this study based on patient follow-up questionnaires, among 47 adolescents with rumination syndome who received inpatient treatment, Key findings:

  • ~20% reported complete cessation of rumination for at least 6 months; though, even in this group, 73% had at least some recurrent symptoms.
  • 40% reported a reduction in rumination intensity following discharge and ~80% reported having at least one day with no rumination.
  • Triggers for recurrence of rumination symptoms included stress (51.4%), illness (27%), menstruation (10.8%), and certain foods (18.9%).
  • Treatment of rumination syndrome helped eliminate the need for supplemental tube feedings in the “vast majority of patients.”

One important limitation of this study is the patient selection; this group of inpatients with rumination syndrome at a specialized center likely had more severe rumination syndrome.

My take: Like many GI conditions, the expectation for rumination syndrome should probably be improvement/management rather than resolution/cure.

Signage on Bright Angel Trail, Grand Canyon

Helpful Review on Biliary Atresia

Biliary atresia (BA) remains the leading cause of pediatric liver transplantation and a frequent cause of cholestasis in newborns.  A recent review (AG Feldman, CL Mack. JPGN 2015; 61: 167-75) provides a helpful update. The article begins with a review on pathogenesis, though this remains unknown and continues to be an area of speculation.

The section on evaluation includes a suggested diagnostic algorithm for neonatal cholestasis.  In short, for a 2 week old with jaundice , the authors recommend (STEP 1) fractionating the bilirubin.  The infant is considered cholestasis if the direct bilirubin is ≥1 mg/dL (if total bilirubin is <5 mg/dL) or if direct bilirubin ≥20% of total bilirubin (if total bilirubin is >5 mg/dL).

Among cholestatic infants, the authors recommend (STEP 2) next checking ultrasound and alpha-one antitrypsin (A1AT) (level & phenotype).  The text implies that the authors would check a GGTP.  While this is not in their algorithm, many would suggest checking urine reducing substances, coags, serum glucose, and consideration of sepsis evaluation; these tests can identify issues that are more urgent than identifying biliary atresia.

STEP 3: If U/S and A1AT, are not diagnostic, consider urine culture, urine reducing substances, urine succinylacetone, and additional infectious studies.

STEP 4: Proceed with liver biopsy. If findings of biliary atresia (eg. bile plugs, bile duct proliferation, portal fibrosis), proceed with intraoperative cholangiogram.

Other points:

  • “It is rare for an infant with BA to have a GGTP level <200 U/L.” If low GGTP, consider PFIC, inborn error of bile acid metabolism, and panhypopituitarism.
  • Extensive differential diagnosis table given ((Table 1)
  • “Late diagnosis of BA remains a problem in the United States. The average age of HPE [hepatoportoenterostomy] is 61 days and 44% of patients still undergo HPE after 60 days of life.”  The authors indicate a goal for HPE of taking place  at <45 days of life.
  • Successful HPE can occur even with late diagnosis. 10% to 20% of children who undergo HPE after 100 to 120 days of life still have success in restoring bile flow.”
  • Early/successful HPE is helpful in increasing 10-year transplant-free rate.  Early on, 3 months after HPE, those with a total bilirubin <2 mg/dL compared with those with a total bilirubin of >6 mg/dL have a much lower likelihood of liver transplantation by 2 years of age: 84% vs. 16%.
  • Recommends checking a pulse oximetry at routine followup visits following HPE to look for the possibility of hepatopulmonary syndrome.
  • The article reviews complications including ascites, portal hypertension/GI bleeding, cholangitis, malignancy, and hepatopulmonary syndrome/portopulmonary hypertension.
  • Outcomes: With HPE, “up to two-thirds of patients with BA have short-term clearance of jaundice.” Yet, “80% of patients with biliary atresia will require liver transplantation during childhood.”

Also noted:

“Biliary Atresia is Associated with Hypertension” JPGN 2015; 61: 182-86.

“Pathogenesis of biliary atresia: defining biology to understand clinical phenotypes” A Asai, A Miethke, JA Bezerra. Nat Rev Gastroenterol Hepatol 2015; 12: 343-52.  This review provides in-depth review examines more precise phenotyping, influencing factors (eg. cytomegalovirus), and potential mechanisms.

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From Mt Washburn, Yellowstone

From Mt Washburn, Yellowstone

Inequality in Pediatric Health Care

“Of all the forms of inequality, injustice in health care is the most shocking and inhuman.”

-Martin Luther King, Jr

This quote is part of an editorial (Flores G, “Dead Wrong: The Growing List of Racial/Ethnic Disparities in Childhood Mortality” J Pediatr 2015; 166: 790-3). The author discusses the disparities among African-American (AA) and Latino children in comparison to white children.

Key points:

  • AA children and young adults had ~6 times the death rate for drowning in swimming pools, 4 times more likely of dying after liver transplant, and about twice the likelihood of dying due to acute lymphoblastic leukemia.
  • Latino children have higher cancer death rates with about twice the likelihood of dying due to acute lymphoblastic leukemia and increased drowning death rate as well.
  • One new study (pages 812-8) shows that black children have increased in-hospital mortality (OR 1.66) after complications following congenital heart surgery and that hispanic children have an increased complication rate following surgery (OR 1.13). This was a retrospective study using the Kids’ Inpatient Database with approximately 3 million discharge abstracts for three separate years.
  • A second study (pages 819-26) with a data set of 98,833 children shows that birth defects resulted in higher 8-year adjusted hazards of death for black, latino, and Asian/Pacific Islander children.

Recognizing these disparities inevitable leads to the question of why. Dr. Flores postulates several factors.

  • Genetic differences.  For example, some ethnicities have more difficult to treat cancers, either due to genetic mutations or due to metabolism of medications.
  • Delays in diagnosis and treatment.  Patients who present at a later stage of diagnosis often have lower cure/response rates. The author notes that black children receive a diagnosis of autism a mean of 1.4 years later than white children.
  • Barriers to specialty care.  Specialty care can result in improved outcomes.
  • Bias in healthcare delivery, both conscious and unintentional.

Bottomline: The problems of racial inequality is not just a matter of relationships between the police and the community.  It is clear that more needs to be done to improve outcomes in healthcare as well.

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Unrelated Link: Surgeon General Tells Elmo to Get His Vaccines