Detail
Article
Online article
FT
Medvik - BMC
  • Something wrong with this record ?

The Sclerosing Cholangitis Outcomes in Pediatrics (SCOPE) Index: A Prognostic Tool for Children

MR. Deneau, C. Mack, ER. Perito, A. Ricciuto, PL. Valentino, M. Amin, AZ. Amir, M. Aumar, M. Auth, A. Broderick, M. DiGuglielmo, LG. Draijer, ED. Tavares Fagundes, W. El-Matary, F. Ferrari, KN. Furuya, N. Gupta, JT. Hochberg, M. Homan, S....

. 2021 ; 73 (3) : 1074-1087. [pub] 20201219

Language English Country United States

Document type Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't

Persistent link   https://www.medvik.cz/link/bmc21026018

Grant support
UL1TR002538 NCATS NIH HHS - United States
UL1 RR025764 NCRR NIH HHS - United States
KL2 TR001065 NCATS NIH HHS - United States
UL1 TR000105 NCATS NIH HHS - United States

BACKGROUND AND AIMS: Disease progression in children with primary sclerosing cholangitis (PSC) is variable. Prognostic and risk-stratification tools exist for adult-onset PSC, but not for children. We aimed to create a tool that accounts for the biochemical and phenotypic features and early disease stage of pediatric PSC. APPROACH AND RESULTS: We used retrospective data from the Pediatric PSC Consortium. The training cohort contained 1,012 patients from 40 centers. We generated a multivariate risk index (Sclerosing Cholangitis Outcomes in Pediatrics [SCOPE] index) that contained total bilirubin, albumin, platelet count, gamma glutamyltransferase, and cholangiography to predict a primary outcome of liver transplantation or death (TD) and a broader secondary outcome that included portal hypertensive, biliary, and cancer complications termed hepatobiliary complications (HBCs). The model stratified patients as low, medium, or high risk based on progression to TD at rates of <1%, 3%, and 9% annually and to HBCs at rates of 2%, 6%, and 13% annually, respectively (P < 0.001). C-statistics to discriminate outcomes at 1 and 5 years were 0.95 and 0.82 for TD and 0.80 and 0.76 for HBCs, respectively. Baseline hepatic fibrosis stage was worse with increasing risk score, with extensive fibrosis in 8% of the lowest versus 100% with the highest risk index (P < 0.001). The model was validated in 240 children from 11 additional centers and performed well. CONCLUSIONS: The SCOPE index is a pediatric-specific prognostic tool for PSC. It uses routinely obtained, objective data to predict a complicated clinical course. It correlates strongly with biopsy-proven liver fibrosis. SCOPE can be used with families for shared decision making on clinical care based on a patient's individual risk, and to account for variable disease progression when designing future clinical trials.

1st Department of Pediatrics University of Athens Children's Hospital Agia Sofia Athens Greece

Alder Hey Children's Hospital Liverpool United Kingdom

Amsterdam University Medical Center Amsterdam The Netherlands

Boston Children's Hospital and Harvard Medical School Boston MA

Children's Health Ireland at Crumlin and University College Dublin Dublin Ireland

Children's Hospital at Montefiore Albert Einstein College of Medicine Bronx NY

Children's Hospital of Philadelphia Philadelphia PA

Children's National Medical Center Washington DC

Cincinnati Children's Hospital Medical Center Cincinnati OH

Columbia University New York NY

Emory University School of Medicine Atlanta GA

Faculty of Medicine and Health Sciences UJK Kielce Kielce Poland

Federal University of Minas Gerais Belo Horizonte Brazil

Indiana University Indianapolis IN

Johns Hopkins University Baltimore MD

London Health Sciences Center Western University London Ontario Canada

Lurie Children's Hospital Chicago IL

Massachusetts General Hospital Harvard Medical School Boston MA

Medical College of Wisconsin Milwaukee WI

Memorial University St John's Newfoundland Canada

Nemours Alfred 1 duPont Hospital for Children Wilmington DE

Northwest Pediatric Gastroenterology LLC Portland OR

Oklahoma University Oklahoma City OK

Palacky University Olomouc Czech Republic

Phoenix Children's Hospital Phoenix AZ

Sapienza University of Rome Rome Italy

Shaare Zedek Medical Center Jerusalem Israel

Teikyo University School of Medicine Tokyo Japan

The Dana Dwek Children's Hospital The Tel Aviv Medical Center Tel Aviv University Tel Aviv Israel

University of California San Diego San Diego CA

University of California San Francisco San Francisco CA

University of Colorado School of Medicine Aurora CO

University of Helsinki Hospital and Tampere University Helsinki Finland

University of Lille CHU Lille Lille France

University of Ljubljana Ljubljana Slovenia

University of Manitoba Winnipeg Manitoba Canada

University of Miami Miami FL

University of Naples Federico 2 Naples Italy

University of Rochester Medical Center Rochester NY

University of Southern California Los Angeles CA

University of Toronto Toronto Ontario Canada

University of Ulsan Seoul South Korea

University of Utah and Intermountain Primary Children's Hospital Salt Lake City UT

University of Washington Seattle WA

University of Wisconsin Madison School of Medicine and Public Health Madison WI

Yale University School of Medicine New Haven CT

References provided by Crossref.org

000      
00000naa a2200000 a 4500
001      
bmc21026018
003      
CZ-PrNML
005      
20211026133255.0
007      
ta
008      
211013s2021 xxu f 000 0|eng||
009      
AR
024    7_
$a 10.1002/hep.31393 $2 doi
035    __
$a (PubMed)32464706
040    __
$a ABA008 $b cze $d ABA008 $e AACR2
041    0_
$a eng
044    __
$a xxu
100    1_
$a Deneau, Mark R $u University of Utah and Intermountain Primary Children's Hospital, Salt Lake City, UT
245    14
$a The Sclerosing Cholangitis Outcomes in Pediatrics (SCOPE) Index: A Prognostic Tool for Children / $c MR. Deneau, C. Mack, ER. Perito, A. Ricciuto, PL. Valentino, M. Amin, AZ. Amir, M. Aumar, M. Auth, A. Broderick, M. DiGuglielmo, LG. Draijer, ED. Tavares Fagundes, W. El-Matary, F. Ferrari, KN. Furuya, N. Gupta, JT. Hochberg, M. Homan, S. Horslen, R. Iorio, MK. Jensen, MM. Jonas, BM. Kamath, N. Kerkar, KM. Kim, KL. Kolho, BGP. Koot, TJ. Laborda, CK. Lee, KM. Loomes, M. Martinez, A. Miethke, T. Miloh, D. Mogul, S. Mohammad, P. Mohan, S. Moroz, N. Ovchinsky, S. Palle, A. Papadopoulou, G. Rao, A. Rodrigues Ferreira, P. Sathya, KB. Schwarz, U. Shah, E. Shteyer, R. Singh, V. Smolka, N. Soufi, A. Tanaka, R. Varier, B. Vitola, M. Woynarowski, M. Zerofsky, A. Zizzo, SL. Guthery
520    9_
$a BACKGROUND AND AIMS: Disease progression in children with primary sclerosing cholangitis (PSC) is variable. Prognostic and risk-stratification tools exist for adult-onset PSC, but not for children. We aimed to create a tool that accounts for the biochemical and phenotypic features and early disease stage of pediatric PSC. APPROACH AND RESULTS: We used retrospective data from the Pediatric PSC Consortium. The training cohort contained 1,012 patients from 40 centers. We generated a multivariate risk index (Sclerosing Cholangitis Outcomes in Pediatrics [SCOPE] index) that contained total bilirubin, albumin, platelet count, gamma glutamyltransferase, and cholangiography to predict a primary outcome of liver transplantation or death (TD) and a broader secondary outcome that included portal hypertensive, biliary, and cancer complications termed hepatobiliary complications (HBCs). The model stratified patients as low, medium, or high risk based on progression to TD at rates of <1%, 3%, and 9% annually and to HBCs at rates of 2%, 6%, and 13% annually, respectively (P < 0.001). C-statistics to discriminate outcomes at 1 and 5 years were 0.95 and 0.82 for TD and 0.80 and 0.76 for HBCs, respectively. Baseline hepatic fibrosis stage was worse with increasing risk score, with extensive fibrosis in 8% of the lowest versus 100% with the highest risk index (P < 0.001). The model was validated in 240 children from 11 additional centers and performed well. CONCLUSIONS: The SCOPE index is a pediatric-specific prognostic tool for PSC. It uses routinely obtained, objective data to predict a complicated clinical course. It correlates strongly with biopsy-proven liver fibrosis. SCOPE can be used with families for shared decision making on clinical care based on a patient's individual risk, and to account for variable disease progression when designing future clinical trials.
650    _2
$a mladiství $7 D000293
650    _2
$a bilirubin $x krev $7 D001663
650    _2
$a biopsie $7 D001706
650    _2
$a dítě $7 D002648
650    _2
$a cholangiografie $7 D002758
650    _2
$a sklerozující cholangitida $x diagnóza $x mortalita $x patologie $x chirurgie $7 D015209
650    _2
$a progrese nemoci $7 D018450
650    _2
$a ženské pohlaví $7 D005260
650    _2
$a lidé $7 D006801
650    _2
$a transplantace jater $7 D016031
650    _2
$a mužské pohlaví $7 D008297
650    _2
$a počet trombocytů $7 D010976
650    _2
$a prognóza $7 D011379
650    _2
$a retrospektivní studie $7 D012189
650    _2
$a rizikové faktory $7 D012307
650    _2
$a sérový albumin $x analýza $7 D012709
650    _2
$a gama-glutamyltransferasa $x krev $7 D005723
655    _2
$a časopisecké články $7 D016428
655    _2
$a Research Support, N.I.H., Extramural $7 D052061
655    _2
$a práce podpořená grantem $7 D013485
700    1_
$a Mack, Cara $u University of Colorado School of Medicine, Aurora, CO
700    1_
$a Perito, Emily R $u University of California, San Francisco, San Francisco, CA
700    1_
$a Ricciuto, Amanda $u University of Toronto, Toronto, Ontario, Canada
700    1_
$a Valentino, Pamela L $u Yale University School of Medicine, New Haven, CT
700    1_
$a Amin, Mansi $u Phoenix Children's Hospital, Phoenix, AZ
700    1_
$a Amir, Achiya Z $u The Dana-Dwek Children's Hospital, The Tel-Aviv Medical Center, Tel-Aviv University, Tel Aviv, Israel
700    1_
$a Aumar, Madeleine $u University of Lille, CHU Lille, Lille, France
700    1_
$a Auth, Marcus $u Alder Hey Children's Hospital, Liverpool, United Kingdom
700    1_
$a Broderick, Annemarie $u Children's Health Ireland at Crumlin & University College Dublin, Dublin, Ireland
700    1_
$a DiGuglielmo, Matthew $u Nemours Alfred I. duPont Hospital for Children, Wilmington, DE
700    1_
$a Draijer, Laura G $u Amsterdam University Medical Center, Amsterdam, The Netherlands
700    1_
$a Tavares Fagundes, Eleonora Druve $u Federal University of Minas Gerais, Belo Horizonte, Brazil
700    1_
$a El-Matary, Wael $u University of Manitoba, Winnipeg, Manitoba, Canada
700    1_
$a Ferrari, Federica $u Sapienza University of Rome, Rome, Italy
700    1_
$a Furuya, Katryn N $u University of Wisconsin-Madison School of Medicine and Public Health, Madison, WI
700    1_
$a Gupta, Nitika $u Emory University School of Medicine, Atlanta, GA
700    1_
$a Hochberg, Jessica T $u University of Miami, Miami, FL
700    1_
$a Homan, Matjaz $u University of Ljubljana, Ljubljana, Slovenia
700    1_
$a Horslen, Simon $u University of Washington, Seattle, WA
700    1_
$a Iorio, Raffaele $u University of Naples Federico II, Naples, Italy
700    1_
$a Jensen, M Kyle $u University of Utah and Intermountain Primary Children's Hospital, Salt Lake City, UT
700    1_
$a Jonas, Maureen M $u Boston Children's Hospital and Harvard Medical School, Boston, MA
700    1_
$a Kamath, Binita M $u University of Toronto, Toronto, Ontario, Canada
700    1_
$a Kerkar, Nanda $u University of Rochester Medical Center, Rochester, NY
700    1_
$a Kim, Kyung Mo $u University of Ulsan, Seoul, South Korea
700    1_
$a Kolho, Kaija-Leena $u University of Helsinki Hospital and Tampere University, Helsinki, Finland
700    1_
$a Koot, Bart G P $u Amsterdam University Medical Center, Amsterdam, The Netherlands
700    1_
$a Laborda, Trevor J $u University of Utah and Intermountain Primary Children's Hospital, Salt Lake City, UT
700    1_
$a Lee, Christine K $u Boston Children's Hospital and Harvard Medical School, Boston, MA
700    1_
$a Loomes, Kathleen M $u Children's Hospital of Philadelphia, Philadelphia, PA
700    1_
$a Martinez, Mercedes $u Columbia University, New York, NY
700    1_
$a Miethke, Alexander $u Cincinnati Children's Hospital Medical Center, Cincinnati, OH
700    1_
$a Miloh, Tamir $u University of Miami, Miami, FL
700    1_
$a Mogul, Douglas $u Johns Hopkins University, Baltimore, MD
700    1_
$a Mohammad, Saeed $u Lurie Children's Hospital, Chicago, IL
700    1_
$a Mohan, Parvathi $u Children's National Medical Center, Washington, DC
700    1_
$a Moroz, Stacy $u University of Southern California, Los Angeles, CA
700    1_
$a Ovchinsky, Nadia $u Children's Hospital at Montefiore, Albert Einstein College of Medicine, Bronx, NY
700    1_
$a Palle, Sirish $u Oklahoma University, Oklahoma City, OK
700    1_
$a Papadopoulou, Alexandra $u First Department of Pediatrics, University of Athens, Children's Hospital Agia Sofia, Athens, Greece
700    1_
$a Rao, Girish $u Indiana University, Indianapolis, IN
700    1_
$a Rodrigues Ferreira, Alexandre $u Federal University of Minas Gerais, Belo Horizonte, Brazil
700    1_
$a Sathya, Pushpa $u Memorial University, St. John's, Newfoundland, Canada
700    1_
$a Schwarz, Kathleen B $u Johns Hopkins University, Baltimore, MD $u University of California San Diego, San Diego, CA
700    1_
$a Shah, Uzma $u Massachusetts General Hospital, Harvard Medical School, Boston, MA
700    1_
$a Shteyer, Eyal $u Shaare Zedek Medical Center, Jerusalem, Israel
700    1_
$a Singh, Ruchi $u Cincinnati Children's Hospital Medical Center, Cincinnati, OH
700    1_
$a Smolka, Vratislav $u Palacky University, Olomouc, Czech Republic
700    1_
$a Soufi, Nisreen $u University of Southern California, Los Angeles, CA
700    1_
$a Tanaka, Atsushi $u Teikyo University School of Medicine, Tokyo, Japan
700    1_
$a Varier, Raghu $u Northwest Pediatric Gastroenterology LLC, Portland, OR
700    1_
$a Vitola, Bernadette $u Medical College of Wisconsin, Milwaukee, WI
700    1_
$a Woynarowski, Marek $u Faculty of Medicine and Health Sciences, UJK Kielce, Kielce, Poland
700    1_
$a Zerofsky, Melissa $u University of California San Francisco, San Francisco, CA
700    1_
$a Zizzo, Andréanne $u London Health Sciences Center, Western University, London, Ontario, Canada
700    1_
$a Guthery, Stephen L $u University of Utah and Intermountain Primary Children's Hospital, Salt Lake City, UT
773    0_
$w MED00009806 $t Hepatology (Baltimore, Md.) $x 1527-3350 $g Roč. 73, č. 3 (2021), s. 1074-1087
856    41
$u https://pubmed.ncbi.nlm.nih.gov/32464706 $y Pubmed
910    __
$a ABA008 $b sig $c sign $y p $z 0
990    __
$a 20211013 $b ABA008
991    __
$a 20211026133301 $b ABA008
999    __
$a ok $b bmc $g 1714894 $s 1146525
BAS    __
$a 3
BAS    __
$a PreBMC
BMC    __
$a 2021 $b 73 $c 3 $d 1074-1087 $e 20201219 $i 1527-3350 $m Hepatology $n Hepatology $x MED00009806
GRA    __
$a UL1TR002538 $p NCATS NIH HHS $2 United States
GRA    __
$a UL1 RR025764 $p NCRR NIH HHS $2 United States
GRA    __
$a KL2 TR001065 $p NCATS NIH HHS $2 United States
GRA    __
$a UL1 TR000105 $p NCATS NIH HHS $2 United States
LZP    __
$a Pubmed-20211013

Find record

Citation metrics

Archiving options