Upadacitinib for Induction of Remission in Paediatric Crohn's Disease: An International Multicentre Retrospective Study

Shlomi Cohen; Elizabeth A Spencer; Michael T Dolinger; David L Suskind; Katarina Mitrova; Ondrej Hradsky; Máire A Conrad; Judith R Kelsen; Holm H Uhlig; Christos Tzivinikos; Paul Henderson; Magdalena Wlazlo; Lukas Hackl; Dror S Shouval; Matteo Bramuzzo; Darja Urlep; Christine Olbjørn; Giulia D'Arcangelo; Gemma Pujol-Muncunill; Dotan Yogev; Ben Kang; Marco Gasparetto; Christine Rungoe; Kaija-Leena Kolho; Iva Hojsak; Lorenzo Norsa; Firas Rinawi; Naire Sansotta; Ramit Magen Rimon; Maya Granot; Luca Scarallo; Eunice Trindade; Marta Velasco Rodríguez-Belvís; Dan Turner; Anat Yerushalmy-Feler

doi:10.1111/apt.70016

Upadacitinib for Induction of Remission in Paediatric Crohn's Disease: An International Multicentre Retrospective Study

Shlomi Cohen, Elizabeth A Spencer, Michael T Dolinger, David L Suskind, Katarina Mitrova, Ondrej Hradsky, Máire A Conrad, Judith R Kelsen, Holm H Uhlig, Christos Tzivinikos, Paul Henderson, Magdalena Wlazlo, Lukas Hackl, Dror S Shouval, Matteo Bramuzzo, Darja Urlep, Christine Olbjørn, Giulia D'Arcangelo, Gemma Pujol-Muncunill, Dotan YogevBen Kang, Marco Gasparetto, Christine Rungoe, Kaija-Leena Kolho, Iva Hojsak, Lorenzo Norsa, Firas Rinawi, Naire Sansotta, Ramit Magen Rimon, Maya Granot, Luca Scarallo, Eunice Trindade, Marta Velasco Rodríguez-Belvís, Dan Turner, Anat Yerushalmy-Feler

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Abstract

BACKGROUND: There are scarce data available on upadacitinib in children with Crohn's disease (CD).

AIM: To evaluate the effectiveness and safety of upadacitinib as an induction therapy in paediatric CD.

METHODS: This was a multicentre retrospective study between 2022 and 2024 of children treated with upadacitinib for induction of remission of active CD conducted in 30 centres worldwide affiliated with the IBD Interest and Porto group of the ESPGHAN. We recorded demographic, clinical and laboratory data and adverse events (AEs) at week 8 post-induction. The analysis of the primary outcome was based upon the intention-to-treat (ITT) principle.

RESULTS: We included 100 children (median age 15.8 [interquartile range 14.3-17.2]). All were previously treated with biologic therapies including 89 with ≥ 2 biologics. At the end of the 8-week induction period, we observed clinical response, clinical remission and corticosteroid- and exclusive enteral nutrition-free clinical remission (CFR) in 75%, 56% and 52%, respectively. By the end of induction, 68% had achieved normalisation of C-reactive protein, and 58% had faecal calprotectin (FC) < 150 mcg/g. There was combined CFR and FC remission in 13/31 children with available data at 8 weeks (13% of the ITT population). AEs were recorded in 24 children; the most frequent was acne in 12. Two AEs (severe acne and hypertriglyceridemia) led to discontinuation of therapy.

CONCLUSION: Upadacitinib is an effective induction therapy for refractory paediatric CD. Efficacy should be weighed against the potential risks of AEs.

Originalsprog	Engelsk
Tidsskrift	Alimentary Pharmacology and Therapeutics
Vol/bind	61
Udgave nummer	8
Sider (fra-til)	1372-1380
Antal sider	9
ISSN	0269-2813
DOI	https://doi.org/10.1111/apt.70016
Status	Udgivet - apr. 2025

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10.1111/apt.70016Licens: CC BY-NC-ND

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Cohen, S., Spencer, E. A., Dolinger, M. T., Suskind, D. L., Mitrova, K., Hradsky, O., Conrad, M. A., Kelsen, J. R., Uhlig, H. H., Tzivinikos, C., Henderson, P., Wlazlo, M., Hackl, L., Shouval, D. S., Bramuzzo, M., Urlep, D., Olbjørn, C., D'Arcangelo, G., Pujol-Muncunill, G., ... Yerushalmy-Feler, A. (2025). Upadacitinib for Induction of Remission in Paediatric Crohn's Disease: An International Multicentre Retrospective Study. Alimentary Pharmacology and Therapeutics, 61(8), 1372-1380. https://doi.org/10.1111/apt.70016

Cohen, S, Spencer, EA, Dolinger, MT, Suskind, DL, Mitrova, K, Hradsky, O, Conrad, MA, Kelsen, JR, Uhlig, HH, Tzivinikos, C, Henderson, P, Wlazlo, M, Hackl, L, Shouval, DS, Bramuzzo, M, Urlep, D, Olbjørn, C, D'Arcangelo, G, Pujol-Muncunill, G, Yogev, D, Kang, B, Gasparetto, M, Rungoe, C, Kolho, K-L, Hojsak, I, Norsa, L, Rinawi, F, Sansotta, N, Rimon, RM, Granot, M, Scarallo, L, Trindade, E, Rodríguez-Belvís, MV, Turner, D & Yerushalmy-Feler, A 2025, 'Upadacitinib for Induction of Remission in Paediatric Crohn's Disease: An International Multicentre Retrospective Study', Alimentary Pharmacology and Therapeutics, bind 61, nr. 8, s. 1372-1380. https://doi.org/10.1111/apt.70016

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title = "Upadacitinib for Induction of Remission in Paediatric Crohn's Disease: An International Multicentre Retrospective Study",

abstract = "BACKGROUND: There are scarce data available on upadacitinib in children with Crohn's disease (CD).AIM: To evaluate the effectiveness and safety of upadacitinib as an induction therapy in paediatric CD.METHODS: This was a multicentre retrospective study between 2022 and 2024 of children treated with upadacitinib for induction of remission of active CD conducted in 30 centres worldwide affiliated with the IBD Interest and Porto group of the ESPGHAN. We recorded demographic, clinical and laboratory data and adverse events (AEs) at week 8 post-induction. The analysis of the primary outcome was based upon the intention-to-treat (ITT) principle.RESULTS: We included 100 children (median age 15.8 [interquartile range 14.3-17.2]). All were previously treated with biologic therapies including 89 with ≥ 2 biologics. At the end of the 8-week induction period, we observed clinical response, clinical remission and corticosteroid- and exclusive enteral nutrition-free clinical remission (CFR) in 75%, 56% and 52%, respectively. By the end of induction, 68% had achieved normalisation of C-reactive protein, and 58% had faecal calprotectin (FC) < 150 mcg/g. There was combined CFR and FC remission in 13/31 children with available data at 8 weeks (13% of the ITT population). AEs were recorded in 24 children; the most frequent was acne in 12. Two AEs (severe acne and hypertriglyceridemia) led to discontinuation of therapy.CONCLUSION: Upadacitinib is an effective induction therapy for refractory paediatric CD. Efficacy should be weighed against the potential risks of AEs.",

keywords = "Humans, Retrospective Studies, Crohn Disease/drug therapy, Male, Female, Adolescent, Remission Induction, Heterocyclic Compounds, 3-Ring/therapeutic use, Treatment Outcome, Child",

author = "Shlomi Cohen and Spencer, {Elizabeth A} and Dolinger, {Michael T} and Suskind, {David L} and Katarina Mitrova and Ondrej Hradsky and Conrad, {M{\'a}ire A} and Kelsen, {Judith R} and Uhlig, {Holm H} and Christos Tzivinikos and Paul Henderson and Magdalena Wlazlo and Lukas Hackl and Shouval, {Dror S} and Matteo Bramuzzo and Darja Urlep and Christine Olbj{\o}rn and Giulia D'Arcangelo and Gemma Pujol-Muncunill and Dotan Yogev and Ben Kang and Marco Gasparetto and Christine Rungoe and Kaija-Leena Kolho and Iva Hojsak and Lorenzo Norsa and Firas Rinawi and Naire Sansotta and Rimon, {Ramit Magen} and Maya Granot and Luca Scarallo and Eunice Trindade and Rodr{\'i}guez-Belv{\'i}s, {Marta Velasco} and Dan Turner and Anat Yerushalmy-Feler",

note = "{\textcopyright} 2025 The Author(s). Alimentary Pharmacology & Therapeutics published by John Wiley & Sons Ltd.",

year = "2025",

month = apr,

doi = "10.1111/apt.70016",

language = "English",

volume = "61",

pages = "1372--1380",

journal = "Alimentary Pharmacology and Therapeutics",

issn = "0269-2813",

publisher = "John Wiley and Sons Inc.",

number = "8",

}

TY - JOUR

T1 - Upadacitinib for Induction of Remission in Paediatric Crohn's Disease

T2 - An International Multicentre Retrospective Study

AU - Cohen, Shlomi

AU - Spencer, Elizabeth A

AU - Dolinger, Michael T

AU - Suskind, David L

AU - Mitrova, Katarina

AU - Hradsky, Ondrej

AU - Conrad, Máire A

AU - Kelsen, Judith R

AU - Uhlig, Holm H

AU - Tzivinikos, Christos

AU - Henderson, Paul

AU - Wlazlo, Magdalena

AU - Hackl, Lukas

AU - Shouval, Dror S

AU - Bramuzzo, Matteo

AU - Urlep, Darja

AU - Olbjørn, Christine

AU - D'Arcangelo, Giulia

AU - Pujol-Muncunill, Gemma

AU - Yogev, Dotan

AU - Kang, Ben

AU - Gasparetto, Marco

AU - Rungoe, Christine

AU - Kolho, Kaija-Leena

AU - Hojsak, Iva

AU - Norsa, Lorenzo

AU - Rinawi, Firas

AU - Sansotta, Naire

AU - Rimon, Ramit Magen

AU - Granot, Maya

AU - Scarallo, Luca

AU - Trindade, Eunice

AU - Rodríguez-Belvís, Marta Velasco

AU - Turner, Dan

AU - Yerushalmy-Feler, Anat

PY - 2025/4

Y1 - 2025/4

N2 - BACKGROUND: There are scarce data available on upadacitinib in children with Crohn's disease (CD).AIM: To evaluate the effectiveness and safety of upadacitinib as an induction therapy in paediatric CD.METHODS: This was a multicentre retrospective study between 2022 and 2024 of children treated with upadacitinib for induction of remission of active CD conducted in 30 centres worldwide affiliated with the IBD Interest and Porto group of the ESPGHAN. We recorded demographic, clinical and laboratory data and adverse events (AEs) at week 8 post-induction. The analysis of the primary outcome was based upon the intention-to-treat (ITT) principle.RESULTS: We included 100 children (median age 15.8 [interquartile range 14.3-17.2]). All were previously treated with biologic therapies including 89 with ≥ 2 biologics. At the end of the 8-week induction period, we observed clinical response, clinical remission and corticosteroid- and exclusive enteral nutrition-free clinical remission (CFR) in 75%, 56% and 52%, respectively. By the end of induction, 68% had achieved normalisation of C-reactive protein, and 58% had faecal calprotectin (FC) < 150 mcg/g. There was combined CFR and FC remission in 13/31 children with available data at 8 weeks (13% of the ITT population). AEs were recorded in 24 children; the most frequent was acne in 12. Two AEs (severe acne and hypertriglyceridemia) led to discontinuation of therapy.CONCLUSION: Upadacitinib is an effective induction therapy for refractory paediatric CD. Efficacy should be weighed against the potential risks of AEs.

AB - BACKGROUND: There are scarce data available on upadacitinib in children with Crohn's disease (CD).AIM: To evaluate the effectiveness and safety of upadacitinib as an induction therapy in paediatric CD.METHODS: This was a multicentre retrospective study between 2022 and 2024 of children treated with upadacitinib for induction of remission of active CD conducted in 30 centres worldwide affiliated with the IBD Interest and Porto group of the ESPGHAN. We recorded demographic, clinical and laboratory data and adverse events (AEs) at week 8 post-induction. The analysis of the primary outcome was based upon the intention-to-treat (ITT) principle.RESULTS: We included 100 children (median age 15.8 [interquartile range 14.3-17.2]). All were previously treated with biologic therapies including 89 with ≥ 2 biologics. At the end of the 8-week induction period, we observed clinical response, clinical remission and corticosteroid- and exclusive enteral nutrition-free clinical remission (CFR) in 75%, 56% and 52%, respectively. By the end of induction, 68% had achieved normalisation of C-reactive protein, and 58% had faecal calprotectin (FC) < 150 mcg/g. There was combined CFR and FC remission in 13/31 children with available data at 8 weeks (13% of the ITT population). AEs were recorded in 24 children; the most frequent was acne in 12. Two AEs (severe acne and hypertriglyceridemia) led to discontinuation of therapy.CONCLUSION: Upadacitinib is an effective induction therapy for refractory paediatric CD. Efficacy should be weighed against the potential risks of AEs.

KW - Humans

KW - Retrospective Studies

KW - Crohn Disease/drug therapy

KW - Male

KW - Female

KW - Adolescent

KW - Remission Induction

KW - Heterocyclic Compounds, 3-Ring/therapeutic use

KW - Treatment Outcome

KW - Child

UR - http://www.scopus.com/inward/record.url?scp=85217224125&partnerID=8YFLogxK

U2 - 10.1111/apt.70016

DO - 10.1111/apt.70016

M3 - Journal article

C2 - 39921898

SN - 0269-2813

VL - 61

SP - 1372

EP - 1380

JO - Alimentary Pharmacology and Therapeutics

JF - Alimentary Pharmacology and Therapeutics

IS - 8

ER -

Upadacitinib for Induction of Remission in Paediatric Crohn's Disease: An International Multicentre Retrospective Study

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