The Sclerosing Cholangitis Outcomes in Pediatrics (SCOPE) Index: A Prognostic Tool for Children
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 s...
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| Veröffentlicht in: | Hepatology (Baltimore, Md.) Md.), 2021-03, Vol.73 (3), p.1074-1087 |
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| creator | Deneau, Mark R. Mack, Cara Perito, Emily R. Ricciuto, Amanda Valentino, Pamela L. Amin, Mansi Amir, Achiya Z. Aumar, Madeleine Auth, Marcus Broderick, Annemarie DiGuglielmo, Matthew Draijer, Laura G. Tavares Fagundes, Eleonora Druve El‐Matary, Wael Ferrari, Federica Furuya, Katryn N. Gupta, Nitika Hochberg, Jessica T. Homan, Matjaz Horslen, Simon Iorio, Raffaele Jensen, M. Kyle Jonas, Maureen M. Kamath, Binita M. Kerkar, Nanda Kim, Kyung Mo Kolho, Kaija‐Leena Koot, Bart G.P. Laborda, Trevor J. Lee, Christine K. Loomes, Kathleen M. Martinez, Mercedes Miethke, Alexander Miloh, Tamir Mogul, Douglas Mohammad, Saeed Mohan, Parvathi Moroz, Stacy Ovchinsky, Nadia Palle, Sirish Papadopoulou, Alexandra Rao, Girish Rodrigues Ferreira, Alexandre Sathya, Pushpa Schwarz, Kathleen B. Shah, Uzma Shteyer, Eyal Singh, Ruchi Smolka, Vratislav Soufi, Nisreen Tanaka, Atsushi Varier, Raghu Vitola, Bernadette Woynarowski, Marek Zerofsky, Melissa Zizzo, Andréanne Guthery, Stephen L. |
| description | 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 |
| doi_str_mv | 10.1002/hep.31393 |
| format | Article |
| fullrecord | <record><control><sourceid>proquest_pubme</sourceid><recordid>TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_8557635</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>2501878152</sourcerecordid><originalsourceid>FETCH-LOGICAL-c4083-834d031d1df3f85476e6406b2810286fd447b0eb37c45b44cfbe348247e8b44e3</originalsourceid><addsrcrecordid>eNp1kV1rFDEUhoModlu98A9IwJv2YtqTj5lkvCiUZbWFwi50vQ4zmTO7KbPJmsyo_fembi0qeBUO5-HhzXkJecfgnAHwiy3uzwUTtXhBZqzkqhCihJdkBlxBUefFETlO6R4Aasn1a3IkuKykgmpGzHqL9M4OGENyfkPn2zA0fuNGl-hyGm3YYaLO0xV2rhmjs4me3s2Xq8UZvfEd_vhIr-gqho0PaXSWrkMYaB9i9rihi-jfkFd9MyR8-_SekC-fFuv5dXG7_Hwzv7otrAQtCi1kB4J1rOtFr0upKqwkVC3XDLiu-k5K1QK2QllZtlLavkUhNZcKdR5RnJDLg3c_tTvsLPoxNoPZR7dr4oMJjTN_b7zbmk34ZnRZqkqUWXD6JIjh64RpNDuXLA75GhimZHjOyepaKJ3RD_-g92GKPn_P8BKYVjqXkKmzA2XzaVPE_jkMA_NYm8m1mV-1Zfb9n-mfyd89ZeDiAHx3Az7832SuF6uD8iedt6A1</addsrcrecordid><sourcetype>Open Access Repository</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>2501878152</pqid></control><display><type>article</type><title>The Sclerosing Cholangitis Outcomes in Pediatrics (SCOPE) Index: A Prognostic Tool for Children</title><source>MEDLINE</source><source>Wiley Online Library Journals Frontfile Complete</source><source>Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals</source><creator>Deneau, Mark R. ; Mack, Cara ; Perito, Emily R. ; Ricciuto, Amanda ; Valentino, Pamela L. ; Amin, Mansi ; Amir, Achiya Z. ; Aumar, Madeleine ; Auth, Marcus ; Broderick, Annemarie ; DiGuglielmo, Matthew ; Draijer, Laura G. ; Tavares Fagundes, Eleonora Druve ; El‐Matary, Wael ; Ferrari, Federica ; Furuya, Katryn N. ; Gupta, Nitika ; Hochberg, Jessica T. ; Homan, Matjaz ; Horslen, Simon ; Iorio, Raffaele ; Jensen, M. Kyle ; Jonas, Maureen M. ; Kamath, Binita M. ; Kerkar, Nanda ; Kim, Kyung Mo ; Kolho, Kaija‐Leena ; Koot, Bart G.P. ; Laborda, Trevor J. ; Lee, Christine K. ; Loomes, Kathleen M. ; Martinez, Mercedes ; Miethke, Alexander ; Miloh, Tamir ; Mogul, Douglas ; Mohammad, Saeed ; Mohan, Parvathi ; Moroz, Stacy ; Ovchinsky, Nadia ; Palle, Sirish ; Papadopoulou, Alexandra ; Rao, Girish ; Rodrigues Ferreira, Alexandre ; Sathya, Pushpa ; Schwarz, Kathleen B. ; Shah, Uzma ; Shteyer, Eyal ; Singh, Ruchi ; Smolka, Vratislav ; Soufi, Nisreen ; Tanaka, Atsushi ; Varier, Raghu ; Vitola, Bernadette ; Woynarowski, Marek ; Zerofsky, Melissa ; Zizzo, Andréanne ; Guthery, Stephen L.</creator><creatorcontrib>Deneau, Mark R. ; Mack, Cara ; Perito, Emily R. ; Ricciuto, Amanda ; Valentino, Pamela L. ; Amin, Mansi ; Amir, Achiya Z. ; Aumar, Madeleine ; Auth, Marcus ; Broderick, Annemarie ; DiGuglielmo, Matthew ; Draijer, Laura G. ; Tavares Fagundes, Eleonora Druve ; El‐Matary, Wael ; Ferrari, Federica ; Furuya, Katryn N. ; Gupta, Nitika ; Hochberg, Jessica T. ; Homan, Matjaz ; Horslen, Simon ; Iorio, Raffaele ; Jensen, M. Kyle ; Jonas, Maureen M. ; Kamath, Binita M. ; Kerkar, Nanda ; Kim, Kyung Mo ; Kolho, Kaija‐Leena ; Koot, Bart G.P. ; Laborda, Trevor J. ; Lee, Christine K. ; Loomes, Kathleen M. ; Martinez, Mercedes ; Miethke, Alexander ; Miloh, Tamir ; Mogul, Douglas ; Mohammad, Saeed ; Mohan, Parvathi ; Moroz, Stacy ; Ovchinsky, Nadia ; Palle, Sirish ; Papadopoulou, Alexandra ; Rao, Girish ; Rodrigues Ferreira, Alexandre ; Sathya, Pushpa ; Schwarz, Kathleen B. ; Shah, Uzma ; Shteyer, Eyal ; Singh, Ruchi ; Smolka, Vratislav ; Soufi, Nisreen ; Tanaka, Atsushi ; Varier, Raghu ; Vitola, Bernadette ; Woynarowski, Marek ; Zerofsky, Melissa ; Zizzo, Andréanne ; Guthery, Stephen L.</creatorcontrib><description>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.</description><identifier>ISSN: 0270-9139</identifier><identifier>ISSN: 1527-3350</identifier><identifier>EISSN: 1527-3350</identifier><identifier>DOI: 10.1002/hep.31393</identifier><identifier>PMID: 32464706</identifier><language>eng</language><publisher>United States: Wolters Kluwer Health, Inc</publisher><subject>Adolescent ; Bile ; Bilirubin ; Bilirubin - blood ; Biopsy ; Child ; Children ; Cholangiography ; Cholangitis ; Cholangitis, Sclerosing - diagnosis ; Cholangitis, Sclerosing - mortality ; Cholangitis, Sclerosing - pathology ; Cholangitis, Sclerosing - surgery ; Clinical trials ; Decision making ; Disease Progression ; Female ; Fibrosis ; gamma-Glutamyltransferase - blood ; Hepatology ; Humans ; Liver Transplantation ; Male ; Patients ; Pediatrics ; Platelet Count ; Prognosis ; Retrospective Studies ; Risk Factors ; Serum Albumin - analysis</subject><ispartof>Hepatology (Baltimore, Md.), 2021-03, Vol.73 (3), p.1074-1087</ispartof><rights>2020 by the American Association for the Study of Liver Diseases.</rights><rights>2021 by the American Association for the Study of Liver Diseases.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c4083-834d031d1df3f85476e6406b2810286fd447b0eb37c45b44cfbe348247e8b44e3</citedby><cites>FETCH-LOGICAL-c4083-834d031d1df3f85476e6406b2810286fd447b0eb37c45b44cfbe348247e8b44e3</cites><orcidid>0000-0001-6128-0155 ; 0000-0001-6350-2057 ; 0000-0002-8227-2004 ; 0000-0003-0459-9404 ; 0000-0002-6171-4731 ; 0000-0001-9538-3005</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1002%2Fhep.31393$$EPDF$$P50$$Gwiley$$H</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1002%2Fhep.31393$$EHTML$$P50$$Gwiley$$H</linktohtml><link.rule.ids>230,314,776,780,881,1411,27901,27902,45550,45551</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/32464706$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Deneau, Mark R.</creatorcontrib><creatorcontrib>Mack, Cara</creatorcontrib><creatorcontrib>Perito, Emily R.</creatorcontrib><creatorcontrib>Ricciuto, Amanda</creatorcontrib><creatorcontrib>Valentino, Pamela L.</creatorcontrib><creatorcontrib>Amin, Mansi</creatorcontrib><creatorcontrib>Amir, Achiya Z.</creatorcontrib><creatorcontrib>Aumar, Madeleine</creatorcontrib><creatorcontrib>Auth, Marcus</creatorcontrib><creatorcontrib>Broderick, Annemarie</creatorcontrib><creatorcontrib>DiGuglielmo, Matthew</creatorcontrib><creatorcontrib>Draijer, Laura G.</creatorcontrib><creatorcontrib>Tavares Fagundes, Eleonora Druve</creatorcontrib><creatorcontrib>El‐Matary, Wael</creatorcontrib><creatorcontrib>Ferrari, Federica</creatorcontrib><creatorcontrib>Furuya, Katryn N.</creatorcontrib><creatorcontrib>Gupta, Nitika</creatorcontrib><creatorcontrib>Hochberg, Jessica T.</creatorcontrib><creatorcontrib>Homan, Matjaz</creatorcontrib><creatorcontrib>Horslen, Simon</creatorcontrib><creatorcontrib>Iorio, Raffaele</creatorcontrib><creatorcontrib>Jensen, M. Kyle</creatorcontrib><creatorcontrib>Jonas, Maureen M.</creatorcontrib><creatorcontrib>Kamath, Binita M.</creatorcontrib><creatorcontrib>Kerkar, Nanda</creatorcontrib><creatorcontrib>Kim, Kyung Mo</creatorcontrib><creatorcontrib>Kolho, Kaija‐Leena</creatorcontrib><creatorcontrib>Koot, Bart G.P.</creatorcontrib><creatorcontrib>Laborda, Trevor J.</creatorcontrib><creatorcontrib>Lee, Christine K.</creatorcontrib><creatorcontrib>Loomes, Kathleen M.</creatorcontrib><creatorcontrib>Martinez, Mercedes</creatorcontrib><creatorcontrib>Miethke, Alexander</creatorcontrib><creatorcontrib>Miloh, Tamir</creatorcontrib><creatorcontrib>Mogul, Douglas</creatorcontrib><creatorcontrib>Mohammad, Saeed</creatorcontrib><creatorcontrib>Mohan, Parvathi</creatorcontrib><creatorcontrib>Moroz, Stacy</creatorcontrib><creatorcontrib>Ovchinsky, Nadia</creatorcontrib><creatorcontrib>Palle, Sirish</creatorcontrib><creatorcontrib>Papadopoulou, Alexandra</creatorcontrib><creatorcontrib>Rao, Girish</creatorcontrib><creatorcontrib>Rodrigues Ferreira, Alexandre</creatorcontrib><creatorcontrib>Sathya, Pushpa</creatorcontrib><creatorcontrib>Schwarz, Kathleen B.</creatorcontrib><creatorcontrib>Shah, Uzma</creatorcontrib><creatorcontrib>Shteyer, Eyal</creatorcontrib><creatorcontrib>Singh, Ruchi</creatorcontrib><creatorcontrib>Smolka, Vratislav</creatorcontrib><creatorcontrib>Soufi, Nisreen</creatorcontrib><creatorcontrib>Tanaka, Atsushi</creatorcontrib><creatorcontrib>Varier, Raghu</creatorcontrib><creatorcontrib>Vitola, Bernadette</creatorcontrib><creatorcontrib>Woynarowski, Marek</creatorcontrib><creatorcontrib>Zerofsky, Melissa</creatorcontrib><creatorcontrib>Zizzo, Andréanne</creatorcontrib><creatorcontrib>Guthery, Stephen L.</creatorcontrib><title>The Sclerosing Cholangitis Outcomes in Pediatrics (SCOPE) Index: A Prognostic Tool for Children</title><title>Hepatology (Baltimore, Md.)</title><addtitle>Hepatology</addtitle><description>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.</description><subject>Adolescent</subject><subject>Bile</subject><subject>Bilirubin</subject><subject>Bilirubin - blood</subject><subject>Biopsy</subject><subject>Child</subject><subject>Children</subject><subject>Cholangiography</subject><subject>Cholangitis</subject><subject>Cholangitis, Sclerosing - diagnosis</subject><subject>Cholangitis, Sclerosing - mortality</subject><subject>Cholangitis, Sclerosing - pathology</subject><subject>Cholangitis, Sclerosing - surgery</subject><subject>Clinical trials</subject><subject>Decision making</subject><subject>Disease Progression</subject><subject>Female</subject><subject>Fibrosis</subject><subject>gamma-Glutamyltransferase - blood</subject><subject>Hepatology</subject><subject>Humans</subject><subject>Liver Transplantation</subject><subject>Male</subject><subject>Patients</subject><subject>Pediatrics</subject><subject>Platelet Count</subject><subject>Prognosis</subject><subject>Retrospective Studies</subject><subject>Risk Factors</subject><subject>Serum Albumin - analysis</subject><issn>0270-9139</issn><issn>1527-3350</issn><issn>1527-3350</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2021</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNp1kV1rFDEUhoModlu98A9IwJv2YtqTj5lkvCiUZbWFwi50vQ4zmTO7KbPJmsyo_fembi0qeBUO5-HhzXkJecfgnAHwiy3uzwUTtXhBZqzkqhCihJdkBlxBUefFETlO6R4Aasn1a3IkuKykgmpGzHqL9M4OGENyfkPn2zA0fuNGl-hyGm3YYaLO0xV2rhmjs4me3s2Xq8UZvfEd_vhIr-gqho0PaXSWrkMYaB9i9rihi-jfkFd9MyR8-_SekC-fFuv5dXG7_Hwzv7otrAQtCi1kB4J1rOtFr0upKqwkVC3XDLiu-k5K1QK2QllZtlLavkUhNZcKdR5RnJDLg3c_tTvsLPoxNoPZR7dr4oMJjTN_b7zbmk34ZnRZqkqUWXD6JIjh64RpNDuXLA75GhimZHjOyepaKJ3RD_-g92GKPn_P8BKYVjqXkKmzA2XzaVPE_jkMA_NYm8m1mV-1Zfb9n-mfyd89ZeDiAHx3Az7832SuF6uD8iedt6A1</recordid><startdate>202103</startdate><enddate>202103</enddate><creator>Deneau, Mark R.</creator><creator>Mack, Cara</creator><creator>Perito, Emily R.</creator><creator>Ricciuto, Amanda</creator><creator>Valentino, Pamela L.</creator><creator>Amin, Mansi</creator><creator>Amir, Achiya Z.</creator><creator>Aumar, Madeleine</creator><creator>Auth, Marcus</creator><creator>Broderick, Annemarie</creator><creator>DiGuglielmo, Matthew</creator><creator>Draijer, Laura G.</creator><creator>Tavares Fagundes, Eleonora Druve</creator><creator>El‐Matary, Wael</creator><creator>Ferrari, Federica</creator><creator>Furuya, Katryn N.</creator><creator>Gupta, Nitika</creator><creator>Hochberg, Jessica T.</creator><creator>Homan, Matjaz</creator><creator>Horslen, Simon</creator><creator>Iorio, Raffaele</creator><creator>Jensen, M. 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Kyle ; Jonas, Maureen M. ; Kamath, Binita M. ; Kerkar, Nanda ; Kim, Kyung Mo ; Kolho, Kaija‐Leena ; Koot, Bart G.P. ; Laborda, Trevor J. ; Lee, Christine K. ; Loomes, Kathleen M. ; Martinez, Mercedes ; Miethke, Alexander ; Miloh, Tamir ; Mogul, Douglas ; Mohammad, Saeed ; Mohan, Parvathi ; Moroz, Stacy ; Ovchinsky, Nadia ; Palle, Sirish ; Papadopoulou, Alexandra ; Rao, Girish ; Rodrigues Ferreira, Alexandre ; Sathya, Pushpa ; Schwarz, Kathleen B. ; Shah, Uzma ; Shteyer, Eyal ; Singh, Ruchi ; Smolka, Vratislav ; Soufi, Nisreen ; Tanaka, Atsushi ; Varier, Raghu ; Vitola, Bernadette ; Woynarowski, Marek ; Zerofsky, Melissa ; Zizzo, Andréanne ; Guthery, Stephen L.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c4083-834d031d1df3f85476e6406b2810286fd447b0eb37c45b44cfbe348247e8b44e3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2021</creationdate><topic>Adolescent</topic><topic>Bile</topic><topic>Bilirubin</topic><topic>Bilirubin - blood</topic><topic>Biopsy</topic><topic>Child</topic><topic>Children</topic><topic>Cholangiography</topic><topic>Cholangitis</topic><topic>Cholangitis, Sclerosing - diagnosis</topic><topic>Cholangitis, Sclerosing - mortality</topic><topic>Cholangitis, Sclerosing - pathology</topic><topic>Cholangitis, Sclerosing - surgery</topic><topic>Clinical trials</topic><topic>Decision making</topic><topic>Disease Progression</topic><topic>Female</topic><topic>Fibrosis</topic><topic>gamma-Glutamyltransferase - blood</topic><topic>Hepatology</topic><topic>Humans</topic><topic>Liver Transplantation</topic><topic>Male</topic><topic>Patients</topic><topic>Pediatrics</topic><topic>Platelet Count</topic><topic>Prognosis</topic><topic>Retrospective Studies</topic><topic>Risk Factors</topic><topic>Serum Albumin - analysis</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Deneau, Mark R.</creatorcontrib><creatorcontrib>Mack, Cara</creatorcontrib><creatorcontrib>Perito, Emily R.</creatorcontrib><creatorcontrib>Ricciuto, Amanda</creatorcontrib><creatorcontrib>Valentino, Pamela L.</creatorcontrib><creatorcontrib>Amin, Mansi</creatorcontrib><creatorcontrib>Amir, Achiya Z.</creatorcontrib><creatorcontrib>Aumar, Madeleine</creatorcontrib><creatorcontrib>Auth, Marcus</creatorcontrib><creatorcontrib>Broderick, Annemarie</creatorcontrib><creatorcontrib>DiGuglielmo, Matthew</creatorcontrib><creatorcontrib>Draijer, Laura G.</creatorcontrib><creatorcontrib>Tavares Fagundes, Eleonora Druve</creatorcontrib><creatorcontrib>El‐Matary, Wael</creatorcontrib><creatorcontrib>Ferrari, Federica</creatorcontrib><creatorcontrib>Furuya, Katryn N.</creatorcontrib><creatorcontrib>Gupta, Nitika</creatorcontrib><creatorcontrib>Hochberg, Jessica T.</creatorcontrib><creatorcontrib>Homan, Matjaz</creatorcontrib><creatorcontrib>Horslen, Simon</creatorcontrib><creatorcontrib>Iorio, Raffaele</creatorcontrib><creatorcontrib>Jensen, M. Kyle</creatorcontrib><creatorcontrib>Jonas, Maureen M.</creatorcontrib><creatorcontrib>Kamath, Binita M.</creatorcontrib><creatorcontrib>Kerkar, Nanda</creatorcontrib><creatorcontrib>Kim, Kyung Mo</creatorcontrib><creatorcontrib>Kolho, Kaija‐Leena</creatorcontrib><creatorcontrib>Koot, Bart G.P.</creatorcontrib><creatorcontrib>Laborda, Trevor J.</creatorcontrib><creatorcontrib>Lee, Christine K.</creatorcontrib><creatorcontrib>Loomes, Kathleen M.</creatorcontrib><creatorcontrib>Martinez, Mercedes</creatorcontrib><creatorcontrib>Miethke, Alexander</creatorcontrib><creatorcontrib>Miloh, Tamir</creatorcontrib><creatorcontrib>Mogul, Douglas</creatorcontrib><creatorcontrib>Mohammad, Saeed</creatorcontrib><creatorcontrib>Mohan, Parvathi</creatorcontrib><creatorcontrib>Moroz, Stacy</creatorcontrib><creatorcontrib>Ovchinsky, Nadia</creatorcontrib><creatorcontrib>Palle, Sirish</creatorcontrib><creatorcontrib>Papadopoulou, Alexandra</creatorcontrib><creatorcontrib>Rao, Girish</creatorcontrib><creatorcontrib>Rodrigues Ferreira, Alexandre</creatorcontrib><creatorcontrib>Sathya, Pushpa</creatorcontrib><creatorcontrib>Schwarz, Kathleen B.</creatorcontrib><creatorcontrib>Shah, Uzma</creatorcontrib><creatorcontrib>Shteyer, Eyal</creatorcontrib><creatorcontrib>Singh, Ruchi</creatorcontrib><creatorcontrib>Smolka, Vratislav</creatorcontrib><creatorcontrib>Soufi, Nisreen</creatorcontrib><creatorcontrib>Tanaka, Atsushi</creatorcontrib><creatorcontrib>Varier, Raghu</creatorcontrib><creatorcontrib>Vitola, Bernadette</creatorcontrib><creatorcontrib>Woynarowski, Marek</creatorcontrib><creatorcontrib>Zerofsky, Melissa</creatorcontrib><creatorcontrib>Zizzo, Andréanne</creatorcontrib><creatorcontrib>Guthery, Stephen L.</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Immunology Abstracts</collection><collection>Nucleic Acids Abstracts</collection><collection>Oncogenes and Growth Factors Abstracts</collection><collection>Virology and AIDS Abstracts</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>MEDLINE - Academic</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Hepatology (Baltimore, Md.)</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Deneau, Mark R.</au><au>Mack, Cara</au><au>Perito, Emily R.</au><au>Ricciuto, Amanda</au><au>Valentino, Pamela L.</au><au>Amin, Mansi</au><au>Amir, Achiya Z.</au><au>Aumar, Madeleine</au><au>Auth, Marcus</au><au>Broderick, Annemarie</au><au>DiGuglielmo, Matthew</au><au>Draijer, Laura G.</au><au>Tavares Fagundes, Eleonora Druve</au><au>El‐Matary, Wael</au><au>Ferrari, Federica</au><au>Furuya, Katryn N.</au><au>Gupta, Nitika</au><au>Hochberg, Jessica T.</au><au>Homan, Matjaz</au><au>Horslen, Simon</au><au>Iorio, Raffaele</au><au>Jensen, M. Kyle</au><au>Jonas, Maureen M.</au><au>Kamath, Binita M.</au><au>Kerkar, Nanda</au><au>Kim, Kyung Mo</au><au>Kolho, Kaija‐Leena</au><au>Koot, Bart G.P.</au><au>Laborda, Trevor J.</au><au>Lee, Christine K.</au><au>Loomes, Kathleen M.</au><au>Martinez, Mercedes</au><au>Miethke, Alexander</au><au>Miloh, Tamir</au><au>Mogul, Douglas</au><au>Mohammad, Saeed</au><au>Mohan, Parvathi</au><au>Moroz, Stacy</au><au>Ovchinsky, Nadia</au><au>Palle, Sirish</au><au>Papadopoulou, Alexandra</au><au>Rao, Girish</au><au>Rodrigues Ferreira, Alexandre</au><au>Sathya, Pushpa</au><au>Schwarz, Kathleen B.</au><au>Shah, Uzma</au><au>Shteyer, Eyal</au><au>Singh, Ruchi</au><au>Smolka, Vratislav</au><au>Soufi, Nisreen</au><au>Tanaka, Atsushi</au><au>Varier, Raghu</au><au>Vitola, Bernadette</au><au>Woynarowski, Marek</au><au>Zerofsky, Melissa</au><au>Zizzo, Andréanne</au><au>Guthery, Stephen L.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>The Sclerosing Cholangitis Outcomes in Pediatrics (SCOPE) Index: A Prognostic Tool for Children</atitle><jtitle>Hepatology (Baltimore, Md.)</jtitle><addtitle>Hepatology</addtitle><date>2021-03</date><risdate>2021</risdate><volume>73</volume><issue>3</issue><spage>1074</spage><epage>1087</epage><pages>1074-1087</pages><issn>0270-9139</issn><issn>1527-3350</issn><eissn>1527-3350</eissn><abstract>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.</abstract><cop>United States</cop><pub>Wolters Kluwer Health, Inc</pub><pmid>32464706</pmid><doi>10.1002/hep.31393</doi><tpages>14</tpages><orcidid>https://orcid.org/0000-0001-6128-0155</orcidid><orcidid>https://orcid.org/0000-0001-6350-2057</orcidid><orcidid>https://orcid.org/0000-0002-8227-2004</orcidid><orcidid>https://orcid.org/0000-0003-0459-9404</orcidid><orcidid>https://orcid.org/0000-0002-6171-4731</orcidid><orcidid>https://orcid.org/0000-0001-9538-3005</orcidid></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0270-9139 |
| ispartof | Hepatology (Baltimore, Md.), 2021-03, Vol.73 (3), p.1074-1087 |
| issn | 0270-9139 1527-3350 1527-3350 |
| language | eng |
| recordid | cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_8557635 |
| source | MEDLINE; Wiley Online Library Journals Frontfile Complete; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals |
| subjects | Adolescent Bile Bilirubin Bilirubin - blood Biopsy Child Children Cholangiography Cholangitis Cholangitis, Sclerosing - diagnosis Cholangitis, Sclerosing - mortality Cholangitis, Sclerosing - pathology Cholangitis, Sclerosing - surgery Clinical trials Decision making Disease Progression Female Fibrosis gamma-Glutamyltransferase - blood Hepatology Humans Liver Transplantation Male Patients Pediatrics Platelet Count Prognosis Retrospective Studies Risk Factors Serum Albumin - analysis |
| title | The Sclerosing Cholangitis Outcomes in Pediatrics (SCOPE) Index: A Prognostic Tool for Children |
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