Neurodevelopmental Outcome of Young Children with Biliary Atresia and Native Liver: Results from the ChiLDReN Study

To assess neurodevelopmental outcomes among participants with biliary atresia with their native liver at ages 12 months (group 1) and 24 months (group 2), and to evaluate variables predictive of neurodevelopmental impairment. Participants enrolled in a prospective, longitudinal, multicenter study un...

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Published inThe Journal of pediatrics Vol. 196; pp. 139 - 147.e3
Main Authors Ng, Vicky L., Sorensen, Lisa G., Alonso, Estella M., Fredericks, Emily M., Ye, Wen, Moore, Jeff, Karpen, Saul J., Shneider, Benjamin L., Molleston, Jean P., Bezerra, Jorge A., Murray, Karen F., Loomes, Kathleen M., Rosenthal, Philip, Squires, Robert H., Wang, Kasper, Arnon, Ronen, Schwarz, Kathleen B., Turmelle, Yumirle P., Haber, Barbara H., Sherker, Averell H., Magee, John C., Sokol, Ronald J., Hertel, Paula M., Harpavat, Sanjiv, Brandt, Mary L., Leung, Daniel H., Karnsakul, Wikrom, Torrance, Rebecca, Hall, Sherry, Doo, Edward, Hoofnagle, Jay H., Whitington, Peter, Bass, Lee, Miethke, Alexander G., Heubi, James E., Setchell, Kenneth, Bove, Kevin E., Tiao, Greg, Mack, Cara L., Narkewicz, Michael R., Feldman, Amy G., Sundaram, Shikha S., Suchy, Frederick J., Karrer, Frederick M., Lovell, Mark, Van Hove, Johan L., Rand, Elizabeth B., Squires, James E., Venkat, Veena L., Sindhi, Rakesh, Ranganathan, Sarangarajan, Bull, Laura, Teckman, Jeffrey, Bozic, Molly, Subbarao, Girish, Horslen, Simon, Hsu, Evelyn, Finn, Laura, Healey, Patrick, Kohli, Rohit, Thomas, Danny, Soufi, Nisreen, Michail, Sonia, Clifton, Matt, Gupta, Nitika, Romero, Rene, Vos, Miriam, Caltharp, Shelley, Kamath, Binita M., Ling, Simon C., Gold, Anna, Fecteau, Annie, Guthery, Stephen L., Jensen, Kyle, Meyers, Rebecka, Lowichik, Amy, Book, Linda, Merion, Robert M., Spino, Cathie, Jones, Karen
Format Journal Article
LanguageEnglish
Published United States Elsevier Inc 01.05.2018
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Abstract To assess neurodevelopmental outcomes among participants with biliary atresia with their native liver at ages 12 months (group 1) and 24 months (group 2), and to evaluate variables predictive of neurodevelopmental impairment. Participants enrolled in a prospective, longitudinal, multicenter study underwent neurodevelopmental testing with either the Bayley Scales of Infant Development, 2nd edition, or Bayley Scales of Infant and Toddler Development, 3rd edition. Scores (normative mean = 100 ± 15) were categorized as ≥100, 85-99, and <85 for χ2 analysis. Risk for neurodevelopmental impairment (defined as ≥1 score of <85 on the Bayley Scales of Infant Development, 2nd edition, or Bayley Scales of Infant and Toddler Development, 3rd edition, scales) was analyzed using logistic regression. There were 148 children who completed 217 Bayley Scales of Infant and Toddler Development, 3rd edition, examinations (group 1, n = 132; group 2, n = 85). Neurodevelopmental score distributions significantly shifted downward compared with test norms at 1 and 2 years of age. Multivariate analysis identified ascites (OR, 3.17; P = .01) and low length z-scores at time of testing (OR, 0.70; P < .04) as risk factors for physical/motor impairment; low weight z-score (OR, 0.57; P = .001) and ascites (OR, 2.89; P = .01) for mental/cognitive/language impairment at 1 year of age. An unsuccessful hepatoportoenterostomy was predictive of both physical/motor (OR, 4.88; P < .02) and mental/cognitive/language impairment (OR, 4.76; P = .02) at 2 years of age. Participants with biliary atresia surviving with native livers after hepatoportoenterostomy are at increased risk for neurodevelopmental delays at 12 and 24 months of age. Those with unsuccessful hepatoportoenterostomy are >4 times more likely to have neurodevelopmental impairment compared with those with successful hepatoportoenterostomy. Growth delays and/or complications indicating advanced liver disease should alert clinicians to the risk for neurodevelopmental delays, and expedite appropriate interventions. Clinicaltrials.gov: NCT00061828 and NCT00294684.
AbstractList OBJECTIVESTo assess neurodevelopmental outcomes among participants with biliary atresia with their native liver at ages 12 months (group 1) and 24 months (group 2), and to evaluate variables predictive of neurodevelopmental impairment.STUDY DESIGNParticipants enrolled in a prospective, longitudinal, multicenter study underwent neurodevelopmental testing with either the Bayley Scales of Infant Development, 2nd edition, or Bayley Scales of Infant and Toddler Development, 3rd edition. Scores (normative mean = 100 ± 15) were categorized as ≥100, 85-99, and <85 for χ2 analysis. Risk for neurodevelopmental impairment (defined as ≥1 score of <85 on the Bayley Scales of Infant Development, 2nd edition, or Bayley Scales of Infant and Toddler Development, 3rd edition, scales) was analyzed using logistic regression.RESULTSThere were 148 children who completed 217 Bayley Scales of Infant and Toddler Development, 3rd edition, examinations (group 1, n = 132; group 2, n = 85). Neurodevelopmental score distributions significantly shifted downward compared with test norms at 1 and 2 years of age. Multivariate analysis identified ascites (OR, 3.17; P = .01) and low length z-scores at time of testing (OR, 0.70; P < .04) as risk factors for physical/motor impairment; low weight z-score (OR, 0.57; P = .001) and ascites (OR, 2.89; P = .01) for mental/cognitive/language impairment at 1 year of age. An unsuccessful hepatoportoenterostomy was predictive of both physical/motor (OR, 4.88; P < .02) and mental/cognitive/language impairment (OR, 4.76; P = .02) at 2 years of age.CONCLUSIONParticipants with biliary atresia surviving with native livers after hepatoportoenterostomy are at increased risk for neurodevelopmental delays at 12 and 24 months of age. Those with unsuccessful hepatoportoenterostomy are >4 times more likely to have neurodevelopmental impairment compared with those with successful hepatoportoenterostomy. Growth delays and/or complications indicating advanced liver disease should alert clinicians to the risk for neurodevelopmental delays, and expedite appropriate interventions.TRIAL REGISTRATIONClinicaltrials.gov: NCT00061828 and NCT00294684.
To assess neurodevelopmental outcomes among participants with biliary atresia with their native liver at ages 12 months (group 1) and 24 months (group 2), and to evaluate variables predictive of neurodevelopmental impairment. Participants enrolled in a prospective, longitudinal, multicenter study underwent neurodevelopmental testing with either the Bayley Scales of Infant Development, 2nd edition, or Bayley Scales of Infant and Toddler Development, 3rd edition. Scores (normative mean = 100 ± 15) were categorized as ≥100, 85-99, and <85 for χ analysis. Risk for neurodevelopmental impairment (defined as ≥1 score of <85 on the Bayley Scales of Infant Development, 2nd edition, or Bayley Scales of Infant and Toddler Development, 3rd edition, scales) was analyzed using logistic regression. There were 148 children who completed 217 Bayley Scales of Infant and Toddler Development, 3rd edition, examinations (group 1, n = 132; group 2, n = 85). Neurodevelopmental score distributions significantly shifted downward compared with test norms at 1 and 2 years of age. Multivariate analysis identified ascites (OR, 3.17; P = .01) and low length z-scores at time of testing (OR, 0.70; P < .04) as risk factors for physical/motor impairment; low weight z-score (OR, 0.57; P = .001) and ascites (OR, 2.89; P = .01) for mental/cognitive/language impairment at 1 year of age. An unsuccessful hepatoportoenterostomy was predictive of both physical/motor (OR, 4.88; P < .02) and mental/cognitive/language impairment (OR, 4.76; P = .02) at 2 years of age. Participants with biliary atresia surviving with native livers after hepatoportoenterostomy are at increased risk for neurodevelopmental delays at 12 and 24 months of age. Those with unsuccessful hepatoportoenterostomy are >4 times more likely to have neurodevelopmental impairment compared with those with successful hepatoportoenterostomy. Growth delays and/or complications indicating advanced liver disease should alert clinicians to the risk for neurodevelopmental delays, and expedite appropriate interventions. Clinicaltrials.gov: NCT00061828 and NCT00294684.
To assess neurodevelopmental outcomes among participants with biliary atresia with their native liver at ages 12 months (group 1) and 24 months (group 2), and to evaluate variables predictive of neurodevelopmental impairment. Participants enrolled in a prospective, longitudinal, multicenter study underwent neurodevelopmental testing with either the Bayley Scales of Infant Development, 2nd edition, or Bayley Scales of Infant and Toddler Development, 3rd edition. Scores (normative mean = 100 ± 15) were categorized as ≥100, 85-99, and <85 for χ2 analysis. Risk for neurodevelopmental impairment (defined as ≥1 score of <85 on the Bayley Scales of Infant Development, 2nd edition, or Bayley Scales of Infant and Toddler Development, 3rd edition, scales) was analyzed using logistic regression. There were 148 children who completed 217 Bayley Scales of Infant and Toddler Development, 3rd edition, examinations (group 1, n = 132; group 2, n = 85). Neurodevelopmental score distributions significantly shifted downward compared with test norms at 1 and 2 years of age. Multivariate analysis identified ascites (OR, 3.17; P = .01) and low length z-scores at time of testing (OR, 0.70; P < .04) as risk factors for physical/motor impairment; low weight z-score (OR, 0.57; P = .001) and ascites (OR, 2.89; P = .01) for mental/cognitive/language impairment at 1 year of age. An unsuccessful hepatoportoenterostomy was predictive of both physical/motor (OR, 4.88; P < .02) and mental/cognitive/language impairment (OR, 4.76; P = .02) at 2 years of age. Participants with biliary atresia surviving with native livers after hepatoportoenterostomy are at increased risk for neurodevelopmental delays at 12 and 24 months of age. Those with unsuccessful hepatoportoenterostomy are >4 times more likely to have neurodevelopmental impairment compared with those with successful hepatoportoenterostomy. Growth delays and/or complications indicating advanced liver disease should alert clinicians to the risk for neurodevelopmental delays, and expedite appropriate interventions. Clinicaltrials.gov: NCT00061828 and NCT00294684.
Author Loomes, Kathleen M.
Ng, Vicky L.
Healey, Patrick
Ling, Simon C.
Merion, Robert M.
Miethke, Alexander G.
Kohli, Rohit
Hall, Sherry
Karnsakul, Wikrom
Karpen, Saul J.
Bove, Kevin E.
Feldman, Amy G.
Hsu, Evelyn
Squires, Robert H.
Arnon, Ronen
Ranganathan, Sarangarajan
Rosenthal, Philip
Sherker, Averell H.
Romero, Rene
Torrance, Rebecca
Venkat, Veena L.
Thomas, Danny
Finn, Laura
Bozic, Molly
Suchy, Frederick J.
Lovell, Mark
Murray, Karen F.
Fecteau, Annie
Molleston, Jean P.
Hoofnagle, Jay H.
Gupta, Nitika
Book, Linda
Bezerra, Jorge A.
Sindhi, Rakesh
Whitington, Peter
Setchell, Kenneth
Alonso, Estella M.
Bass, Lee
Sundaram, Shikha S.
Bull, Laura
Fredericks, Emily M.
Leung, Daniel H.
Soufi, Nisreen
Subbarao, Girish
Vos, Miriam
Moore, Jeff
Schwarz, Kathleen B.
Haber, Barbara H.
Clifton, Matt
Jones, Karen
Narkewicz, Michael R.
Heubi, James E.
Michail, Sonia
Rand, Elizabeth B.
Spino, Cathie
Van Hove, Johan L.
Mack, Cara L.
Hertel, Paula M.
Gold, Anna
Karrer, Frederick M.
Horslen, Simon
Squires, James E.
Meyers, Rebecka
Jensen, Kyle
Doo, Edward
Caltharp, Shell
AuthorAffiliation 17 Washington University School of Medicine, St. Louis, MO, USA
5 Department of Biostatistics, University of Michigan, Ann Arbor, MI, USA
20 Department of Surgery, University of Michigan Medical School, Ann Arbor, MI, USA
9 Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA
16 Johns Hopkins School of Medicine, Baltimore, MD, USA
11 Division of Gastroenterology, Hepatology, and Nutrition, Children's Hospital of Philadelphia, Philadelphia, PA, USA
2 Department of Child & Adolescent Psychiatry, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL, USA
8 Section of Pediatric Gastroenterology, Hepatology, and Nutrition, Indiana University School of Medicine, Rylie Hospital for Children, Indianapolis, IN, USA
13 Children's Hospital of Pittsburgh, Pittsburgh, PA, USA
3 Division of Gastroenterology, Hepatology, and Nutrition, Ann & Robert H. Lurie Children
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– name: 17 Washington University School of Medicine, St. Louis, MO, USA
– name: 11 Division of Gastroenterology, Hepatology, and Nutrition, Children's Hospital of Philadelphia, Philadelphia, PA, USA
– name: 6 Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, Emory University School of Medicine/Children's Healthcare of Atlanta, Atlanta, GA, USA
– name: 21 Section of Pediatric Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, University of Colorado School of Medicine, Children's Hospital Colorado, Aurora, CO, USA
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– name: 9 Division of Pediatric Gastroenterology, Hepatology, and Nutrition, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA
– name: 14 Division of Pediatric Surgery, Children's Hospital Los Angeles, University of Southern California, Los Angeles, CA, USA
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– name: 5 Department of Biostatistics, University of Michigan, Ann Arbor, MI, USA
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– name: 8 Section of Pediatric Gastroenterology, Hepatology, and Nutrition, Indiana University School of Medicine, Rylie Hospital for Children, Indianapolis, IN, USA
– name: 15 Division of Gastroenterology, Hepatology and Nutrition, Mount Sinai Hospital, New York, New York, USA
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– name: 13 Children's Hospital of Pittsburgh, Pittsburgh, PA, USA
– name: 18 Division of Gastroenterology, Hepatology, and Nutrition, Children's Hospital of Philadelphia, Philadelphia, PA, USA
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  givenname: Ronald J.
  surname: Sokol
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  organization: Section of Pediatric Gastroenterology, Hepatology, and Nutrition, Department of Pediatrics, University of Colorado School of Medicine, Children's Hospital Colorado, Aurora, CO
– sequence: 23
  givenname: Paula M.
  surname: Hertel
  fullname: Hertel, Paula M.
  organization: Baylor College of Medicine, Texas Children's Hospital, Houston, TX
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  surname: Harpavat
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  organization: Baylor College of Medicine, Texas Children's Hospital, Houston, TX
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– sequence: 36
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– sequence: 38
  givenname: Greg
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  givenname: Frederick M.
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  organization: Children's Hospital of Philadelphia, Philadelphia, PA
– sequence: 48
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– sequence: 49
  givenname: Veena L.
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  givenname: Rakesh
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  fullname: Sindhi, Rakesh
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  surname: Bull
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  organization: Genetics Core Lab, UCSF Institute for Human Genetics, UCSF Benioff Children's Hospital, San Francisco, CA
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  givenname: Jeffrey
  surname: Teckman
  fullname: Teckman, Jeffrey
  organization: Saint Louis University School of Medicine, Saint Louis University/Cardinal, Glennon Children's Medical Center, St. Louis, MO
– sequence: 54
  givenname: Molly
  surname: Bozic
  fullname: Bozic, Molly
  organization: Pediatric Gastroenterology, Riley Hospital for Children, Indianapolis, IN
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  givenname: Girish
  surname: Subbarao
  fullname: Subbarao, Girish
  organization: Pediatric Gastroenterology, Hepatology and Nutrition, Riley Hospital for Children, Indianapolis, IN
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  givenname: Simon
  surname: Horslen
  fullname: Horslen, Simon
  organization: Division of Gastroenterology and Hepatology, Seattle Children's Hospital, Seattle, WA
– sequence: 57
  givenname: Evelyn
  surname: Hsu
  fullname: Hsu, Evelyn
  organization: Division of Gastroenterology and Hepatology, Seattle Children's Hospital, Seattle, WA
– sequence: 58
  givenname: Laura
  surname: Finn
  fullname: Finn, Laura
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BackLink https://www.ncbi.nlm.nih.gov/pubmed/29519540$$D View this record in MEDLINE/PubMed
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ContentType Journal Article
Contributor Healey, Patrick
Guthery, Stephen L
Kohli, Rohit
Hall, Sherry
Karnsakul, Wikrom
Venkat, Veena L
Merion, Robert M
Sundaram, Shikha S
Hsu, Evelyn
Kamath, Binita M
Ranganathan, Sarangarajan
Romero, Rene
Torrance, Rebecca
Thomas, Danny
Finn, Laura
Bozic, Molly
Ling, Simon C
Lovell, Mark
Fecteau, Annie
Gupta, Nitika
Book, Linda
Sindhi, Rakesh
Rand, Elizabeth B
Whitington, Peter
Setchell, Kenneth
Narkewicz, Michael R
Bass, Lee
Bull, Laura
Soufi, Nisreen
Subbarao, Girish
Vos, Miriam
Clifton, Matt
Jones, Karen
Suchy, Frederick J
Leung, Daniel H
Michail, Sonia
Spino, Cathie
Mack, Cara L
Feldman, Amy G
Gold, Anna
Horslen, Simon
Meyers, Rebecka
Hertel, Paula M
Jensen, Kyle
Doo, Edward
Miethke, Alexander G
Caltharp, Shelley
Heubi, James E
Bove, Kevin E
Tiao, Greg
Karrer, Frederick M
Lowichik, Amy
Harpavat, Sanjiv
Brandt, Mary L
Hoofnagle, Jay H
Van Hove, Johan L
Squires, James E
Teckman, Jeffrey
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  organization: Baylor College of Medicine, Texas Children's Hospital, Houston, TX
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  organization: Baylor College of Medicine, Texas Children's Hospital, Houston, TX
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  surname: Brandt
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  organization: Baylor College of Medicine, Texas Children's Hospital, Houston, TX
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  organization: Baylor College of Medicine, Texas Children's Hospital, Houston, TX
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  organization: Johns Hopkins School of Medicine, Baltimore, MD
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  organization: Bile Acid Core, Gastroenterology, Hepatology and Nutrition, Cincinnati Children's Hospital, Cincinnati, OH
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  organization: Pathology, Division of Pediatric Pathology and Laboratory Medicine, Cincinnati Children's Hospital, Cincinnati, OH
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  organization: Department of Biostatistics, University of Michigan, Ann Arbor, MI
Copyright 2018 Elsevier Inc.
Copyright © 2018 Elsevier Inc. All rights reserved.
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CorporateAuthor Childhood Liver Disease Research Network (ChiLDReN)
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ISSN 0022-3476
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Fri Dec 06 06:33:28 EST 2024
Sat Nov 02 12:16:08 EDT 2024
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Keywords BSID-II
motor
cognitive
Bayley-III
chronic liver disease
START
Kasai
HPE
ChiLDReN
PROBE
TB
Language English
License Copyright © 2018 Elsevier Inc. All rights reserved.
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content type line 23
List of additional members of the Childhood Liver Disease Research Network (ChiLDReN) is available at www.jpeds.com (Appendix)
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Snippet To assess neurodevelopmental outcomes among participants with biliary atresia with their native liver at ages 12 months (group 1) and 24 months (group 2), and...
OBJECTIVESTo assess neurodevelopmental outcomes among participants with biliary atresia with their native liver at ages 12 months (group 1) and 24 months...
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SubjectTerms Biliary Atresia - complications
Biliary Atresia - therapy
Child, Preschool
chronic liver disease
Cognition
cognitive
Developmental Disabilities - diagnosis
Developmental Disabilities - etiology
Female
Humans
Infant
Kasai
Liver - physiology
Longitudinal Studies
Male
motor
Motor Skills
Multivariate Analysis
Neuropsychological Tests
Observational Studies as Topic
Prospective Studies
Randomized Controlled Trials as Topic
Regression Analysis
Risk
Treatment Outcome
Vulnerable Populations
Title Neurodevelopmental Outcome of Young Children with Biliary Atresia and Native Liver: Results from the ChiLDReN Study
URI https://dx.doi.org/10.1016/j.jpeds.2017.12.048
https://www.ncbi.nlm.nih.gov/pubmed/29519540
https://www.proquest.com/docview/2012918477
https://pubmed.ncbi.nlm.nih.gov/PMC5924604
Volume 196
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