Validation of predictive models for disease outcomes in paediatric ulcerative colitis: A multicentre prospective inception cohort

Ohad Atia; Renz C.W. Klomberg; Lissy de Ridder; Polychronis Kemos; Frank M. Ruemmele; Ben Kang; Sujin Choi; Byung Ho Choe; Youra Kang; Dror S. Shouval; Gili Focht; Oren Ledder; Raffi Lev-Tzion; Natalie Carmon; Tal David Berger; Dan Turner; Nicholas M. Croft; Esther Orlanski-Meyer

doi:10.1111/apt.17544

Validation of predictive models for disease outcomes in paediatric ulcerative colitis: A multicentre prospective inception cohort

Ohad Atia, Renz C.W. Klomberg, Lissy de Ridder^*, Polychronis Kemos, Frank M. Ruemmele, Ben Kang, Sujin Choi, Byung Ho Choe, Youra Kang, Dror S. Shouval, Gili Focht, Oren Ledder, Raffi Lev-Tzion, Natalie Carmon, Tal David Berger, Dan Turner, Nicholas M. Croft, Esther Orlanski-Meyer

^*Corresponding author for this work

Faculty of Medicine

Research output: Contribution to journal › Article › peer-review

3 Scopus citations

Abstract

Background: Several studies have proposed models to predict disease outcomes in paediatric ulcerative colitis (UC), notably PROTECT, Schechter and PIBD-ahead, but none has been validated by external cohorts. Aim: To explore these models in a prospective multicentre inception cohort. Methods: Children newly diagnosed with UC in 17 centres were followed at disease onset and 3 and 12 months thereafter, as well as at last visit. Outcomes included steroid-free remission (SFR) and acute severe colitis (ASC). Results: Of the 223 included children, 74 (34%), 97 (43%) and 52 (23%) presented with mild, moderate and severe disease, respectively. SFR rate was 35% at 3 months and 47% at 12 months (62% of those with mild disease at diagnosis vs. 41% in moderate–severe disease; p = 0.01). Thirty-six (16%) children developed ASC during the first month after diagnosis, and 53 (24%) during the first year. The AUC of the PROTECT model for predicting SFR at 3 and 12 months was 0.78 [95% CI 0.65–0.92] and 0.57 [95% CI 0.47–0.66], respectively. The sensitivity/specificity/PPV/NPV of Schechter's criteria to predict sustained SFR at 12 months was 50%/60%/35%/74%. ASC was predicted only by the PUCAI score at diagnosis and at 3 months. Conclusions: The PROTECT model had a good predictive utility for SFR at 3 months, but not at 12 months. The other predictive models did not achieve sufficient accuracy, which was far from that reported in the original studies. This highlights the necessity for external validation of any prediction model prior to its implementation into clinical practice.

Original language	English
Pages (from-to)	182-190
Number of pages	9
Journal	Alimentary Pharmacology and Therapeutics
Volume	58
Issue number	2
DOIs	https://doi.org/10.1111/apt.17544
State	Published - Jul 2023

Bibliographical note

Funding Information:
OA, RK, OK, FMR, SC, BHC, YK, OL, RLT, NC and TDB—None. LdR—Collaborations (such as involvement in industry‐sponsored studies, investigator‐initiated studies, consultancy) with AbbVie, Lilly, Takeda, Janssen and Pfizer. BK—In the last 3 years received speaker fee, consultation fee or research grant from Celltrion, Janssen, Eisai, AbbVie, Takeda, Yuhan, Yungjin and JW Pharmaceutical. DSS—Research grant from Takeda; consulting and lecturing fees from Jansen and AbbVie. GF—Received consultation fees from AbbVie and Lilly in the last 3 years. DT—In the last 3 years received consultation fee(s), research grant(s), royalties or honoraria from Janssen, Pfizer, Hospital for Sick Children, Ferring, AbbVie, Takeda, Atlantic Health, Shire, Celgene, Lilly, Roche, Thermo Fisher, BMS and SorrisoPharma. NMC—AbbVie, Shire, Takeda, Lilly and Pfizer for lectures and research fees (trials) all paid to NMC's employer. EOM—Lecture fees were received in the last 3 years from Takeda and Abbott.

Publisher Copyright:
© 2023 The Authors. Alimentary Pharmacology & Therapeutics published by John Wiley & Sons Ltd.

Access to Document

10.1111/apt.17544

Cite this

Atia, O., Klomberg, R. C. W., de Ridder, L., Kemos, P., Ruemmele, F. M., Kang, B., Choi, S., Choe, B. H., Kang, Y., Shouval, D. S., Focht, G., Ledder, O., Lev-Tzion, R., Carmon, N., Berger, T. D., Turner, D., Croft, N. M., & Orlanski-Meyer, E. (2023). Validation of predictive models for disease outcomes in paediatric ulcerative colitis: A multicentre prospective inception cohort. Alimentary Pharmacology and Therapeutics, 58(2), 182-190. https://doi.org/10.1111/apt.17544

@article{6741fc5ef6bd4e2b9f070be75d399ea0,

title = "Validation of predictive models for disease outcomes in paediatric ulcerative colitis: A multicentre prospective inception cohort",

abstract = "Background: Several studies have proposed models to predict disease outcomes in paediatric ulcerative colitis (UC), notably PROTECT, Schechter and PIBD-ahead, but none has been validated by external cohorts. Aim: To explore these models in a prospective multicentre inception cohort. Methods: Children newly diagnosed with UC in 17 centres were followed at disease onset and 3 and 12 months thereafter, as well as at last visit. Outcomes included steroid-free remission (SFR) and acute severe colitis (ASC). Results: Of the 223 included children, 74 (34%), 97 (43%) and 52 (23%) presented with mild, moderate and severe disease, respectively. SFR rate was 35% at 3 months and 47% at 12 months (62% of those with mild disease at diagnosis vs. 41% in moderate–severe disease; p = 0.01). Thirty-six (16%) children developed ASC during the first month after diagnosis, and 53 (24%) during the first year. The AUC of the PROTECT model for predicting SFR at 3 and 12 months was 0.78 [95% CI 0.65–0.92] and 0.57 [95% CI 0.47–0.66], respectively. The sensitivity/specificity/PPV/NPV of Schechter's criteria to predict sustained SFR at 12 months was 50%/60%/35%/74%. ASC was predicted only by the PUCAI score at diagnosis and at 3 months. Conclusions: The PROTECT model had a good predictive utility for SFR at 3 months, but not at 12 months. The other predictive models did not achieve sufficient accuracy, which was far from that reported in the original studies. This highlights the necessity for external validation of any prediction model prior to its implementation into clinical practice.",

author = "Ohad Atia and Klomberg, {Renz C.W.} and {de Ridder}, Lissy and Polychronis Kemos and Ruemmele, {Frank M.} and Ben Kang and Sujin Choi and Choe, {Byung Ho} and Youra Kang and Shouval, {Dror S.} and Gili Focht and Oren Ledder and Raffi Lev-Tzion and Natalie Carmon and Berger, {Tal David} and Dan Turner and Croft, {Nicholas M.} and Esther Orlanski-Meyer",

note = "Publisher Copyright: {\textcopyright} 2023 The Authors. Alimentary Pharmacology & Therapeutics published by John Wiley & Sons Ltd.",

year = "2023",

month = jul,

doi = "10.1111/apt.17544",

language = "אנגלית",

volume = "58",

pages = "182--190",

journal = "Alimentary Pharmacology and Therapeutics",

issn = "0269-2813",

publisher = "John Wiley and Sons Inc.",

number = "2",

}

Atia, O, Klomberg, RCW, de Ridder, L, Kemos, P, Ruemmele, FM, Kang, B, Choi, S, Choe, BH, Kang, Y, Shouval, DS, Focht, G, Ledder, O, Lev-Tzion, R, Carmon, N, Berger, TD, Turner, D, Croft, NM & Orlanski-Meyer, E 2023, 'Validation of predictive models for disease outcomes in paediatric ulcerative colitis: A multicentre prospective inception cohort', Alimentary Pharmacology and Therapeutics, vol. 58, no. 2, pp. 182-190. https://doi.org/10.1111/apt.17544

TY - JOUR

T1 - Validation of predictive models for disease outcomes in paediatric ulcerative colitis

T2 - A multicentre prospective inception cohort

AU - Atia, Ohad

AU - Klomberg, Renz C.W.

AU - de Ridder, Lissy

AU - Kemos, Polychronis

AU - Ruemmele, Frank M.

AU - Kang, Ben

AU - Choi, Sujin

AU - Choe, Byung Ho

AU - Kang, Youra

AU - Shouval, Dror S.

AU - Focht, Gili

AU - Ledder, Oren

AU - Lev-Tzion, Raffi

AU - Carmon, Natalie

AU - Berger, Tal David

AU - Turner, Dan

AU - Croft, Nicholas M.

AU - Orlanski-Meyer, Esther

PY - 2023/7

Y1 - 2023/7

N2 - Background: Several studies have proposed models to predict disease outcomes in paediatric ulcerative colitis (UC), notably PROTECT, Schechter and PIBD-ahead, but none has been validated by external cohorts. Aim: To explore these models in a prospective multicentre inception cohort. Methods: Children newly diagnosed with UC in 17 centres were followed at disease onset and 3 and 12 months thereafter, as well as at last visit. Outcomes included steroid-free remission (SFR) and acute severe colitis (ASC). Results: Of the 223 included children, 74 (34%), 97 (43%) and 52 (23%) presented with mild, moderate and severe disease, respectively. SFR rate was 35% at 3 months and 47% at 12 months (62% of those with mild disease at diagnosis vs. 41% in moderate–severe disease; p = 0.01). Thirty-six (16%) children developed ASC during the first month after diagnosis, and 53 (24%) during the first year. The AUC of the PROTECT model for predicting SFR at 3 and 12 months was 0.78 [95% CI 0.65–0.92] and 0.57 [95% CI 0.47–0.66], respectively. The sensitivity/specificity/PPV/NPV of Schechter's criteria to predict sustained SFR at 12 months was 50%/60%/35%/74%. ASC was predicted only by the PUCAI score at diagnosis and at 3 months. Conclusions: The PROTECT model had a good predictive utility for SFR at 3 months, but not at 12 months. The other predictive models did not achieve sufficient accuracy, which was far from that reported in the original studies. This highlights the necessity for external validation of any prediction model prior to its implementation into clinical practice.

AB - Background: Several studies have proposed models to predict disease outcomes in paediatric ulcerative colitis (UC), notably PROTECT, Schechter and PIBD-ahead, but none has been validated by external cohorts. Aim: To explore these models in a prospective multicentre inception cohort. Methods: Children newly diagnosed with UC in 17 centres were followed at disease onset and 3 and 12 months thereafter, as well as at last visit. Outcomes included steroid-free remission (SFR) and acute severe colitis (ASC). Results: Of the 223 included children, 74 (34%), 97 (43%) and 52 (23%) presented with mild, moderate and severe disease, respectively. SFR rate was 35% at 3 months and 47% at 12 months (62% of those with mild disease at diagnosis vs. 41% in moderate–severe disease; p = 0.01). Thirty-six (16%) children developed ASC during the first month after diagnosis, and 53 (24%) during the first year. The AUC of the PROTECT model for predicting SFR at 3 and 12 months was 0.78 [95% CI 0.65–0.92] and 0.57 [95% CI 0.47–0.66], respectively. The sensitivity/specificity/PPV/NPV of Schechter's criteria to predict sustained SFR at 12 months was 50%/60%/35%/74%. ASC was predicted only by the PUCAI score at diagnosis and at 3 months. Conclusions: The PROTECT model had a good predictive utility for SFR at 3 months, but not at 12 months. The other predictive models did not achieve sufficient accuracy, which was far from that reported in the original studies. This highlights the necessity for external validation of any prediction model prior to its implementation into clinical practice.

UR - http://www.scopus.com/inward/record.url?scp=85161219969&partnerID=8YFLogxK

U2 - 10.1111/apt.17544

DO - 10.1111/apt.17544

M3 - ???researchoutput.researchoutputtypes.contributiontojournal.article???

C2 - 37248642

AN - SCOPUS:85161219969

SN - 0269-2813

VL - 58

SP - 182

EP - 190

JO - Alimentary Pharmacology and Therapeutics

JF - Alimentary Pharmacology and Therapeutics

IS - 2

ER -

Validation of predictive models for disease outcomes in paediatric ulcerative colitis: A multicentre prospective inception cohort

Abstract

Bibliographical note

Access to Document

Other files and links

Fingerprint

Cite this