Clinical trial NCT06184971: Biliary Atresia Research Network Northeast
Official title: Biliary Atresia Research Network Northeast (BARNN)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Biliary Atresia
Lead sponsor: Yale University
Sponsor class: OTHER
First posted: 2023-12-29
Enrollment: 150
Sex: ALL
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: RETROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Children 12 years and younger (at the time of the qualifying encounter for care) who were diagnosed with biliary atresia, underwent surgical intervention for biliary atresia, and/or had an encounter for care related to biliary atresia at a participating institution during the study period. Patients who did not undergo surgery will be included (e.g. patients who died before surgery).

Brief summary: This is a multi-center retrospective chart review to compile a data repository of the management and outcomes of children with biliary atresia. Overall, investigators aim to evaluate which specific factors contribute to improved patient outcomes, to help guide potential improvements in patient care and resource utilization.

Detailed description: Biliary atresia is quite rare, with only 0.73 cases per 10,000 births in the US. Due to this rarity and the geographical spread of the US, the small number of cases end up being dispersed amongst the various pediatric hospitals, which leads to certain hospitals only encountering an affected patient once per decade. This rarity and dispersion makes biliary atresia difficult for researchers to study: single-institution studies are limited by low power and only provide narrow snapshots, whereas large NIH-sponsored consortia report highly selected outcomes from only the largest or most dedicated centers and have largely excluded New England. This leaves a significant knowledge gap regarding the management and outcomes at more typical hospitals. The purpose of this study is to collect clinical data from all children with biliary atresia at all hospitals providing pediatric surgical care in the Northeast, even very-low-volume hospitals. This is a retrospective study, only involving chart review. There will be no interaction with subjects, intervention, or collection of specimens for the purposes of this study. The data will only include clinical information that was recorded during the normal course of patient care. The subjects will be de-identified before entry into a HIPAA-compliant data repository. This data repository will allow researchers to pool data, to yield adequate statistical power and assess differences in management and outcomes regarding this very rare condition.

Eligibility criteria: Inclusion Criteria:

* Children with a healthcare encounter related to biliary atresia at a participating institution during the study period (January 1st, 2012 through December 31st, 2021)
* Age 12 years and younger at the time of the qualifying encounter during the study period (encompassing patients born between January 2nd, 1999 through December 31st, 2021)
* Diagnosis of biliary atresia, based on one of the following ICD-10 codes:

  * Q44.2 Atresia of bile ducts
  * Q44.3 Congenital stenosis and stricture of bile ducts
  * Q44.4 Disorders of the biliary tract with major complication or comorbidity
  * Q44.5 Other congenital malformations of the bile ducts
  * Q44.6 Disorders of the biliary tract without major complication or comorbidity

Exclusion Criteria:

* Patients aged 13 years old or older during the study period (i.e. those born before January 2nd, 1999)
* Patients born and/or diagnosed with biliary atresia after the end of the study period (i.e. those born after December 31st, 2021)
* No diagnosis of biliary atresia
* History of biliary atresia without any episodes of care related to biliary atresia during the study period (e.g. a teenager with history of BA who underwent Kasai procedure as an infant and has no current issues related to their BA and is receiving care for an unrelated reason)

Primary outcomes:
measure: Death
timeFrame: 10 years
measure: Kasai procedure
timeFrame: 10 years
measure: Liver transplant
timeFrame: 10 years
Secondary outcomes:
measure: Clearance of jaundice
timeFrame: 6 months
measure: Bilirubin level
timeFrame: 10 years
measure: Cholangitis
timeFrame: 10 years
measure: Bleeding
timeFrame: 10 years
measure: Presentation to ED
timeFrame: 10 years
measure: Age at follow-up with providers
timeFrame: 10 years
Other outcomes:
Locations:
city: Hartford
country: United States
facility: Connecticut Children's Medical Center
state: Connecticut
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Bangor
country: United States
facility: Eastern Maine Medical Center
state: Maine
city: Portland
country: United States
facility: Maine Medical Center
state: Maine
city: Boston
country: United States
facility: Massachusetts General Hospital for Children
state: Massachusetts
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Springfield
country: United States
facility: Baystate
state: Massachusetts
city: Worcester
country: United States
facility: UMass Memorial Medical Center
state: Massachusetts
city: Lebanon
country: United States
facility: Dartmouth-Hitchcock Medical Center
state: New Hampshire
city: Albany
country: United States
facility: Albany Medical Center/Bernard & Millie Duke's Children's Hospital
state: New York
city: Buffalo
country: United States
facility: John R. Oishei Children's Hospital
state: New York
city: Rochester
country: United States
facility: University of Rochester Medical Center/Golisano Children's Hospital
state: New York
city: Syracuse
country: United States
facility: SUNY Upstate Medical University
state: New York
city: Providence
country: United States
facility: Rhode Island Hospital/Hasbro Children's Hospital
state: Rhode Island
Study officials:
affiliation: Yale University
name: Robert Cowles, MD
role: PRINCIPAL_INVESTIGATOR
References: