STELARA - Treatment of Pediatric Ulcerative Colitis

SUMMARY

• The company cannot recommend any practices, procedures, or usage that deviate from the approved labeling.
• UNIFI Jr (NCT04630028) is a phase 3 study evaluating the efficacy, safety, and pharmacokinetics (PK) of STELARA in pediatric patients with moderately to severely active ulcerative colitis (UC). Results through approximately 1 year of treatment are summarized below.^1,2
• The efficacy and safety of STELARA in pediatric patients with UC were also evaluated in a prospective study and retrospective studies.^3-9

clinical data

UNIFI Jr Phase 3 Study

Greef et al (2026)^1,2 evaluated the efficacy, safety, and PK of STELARA in pediatric patients with moderately to severely active UC.

Study Design/Methods

• An open-label, intravenous (IV) induction phase followed by a randomized, double-blind, subcutaneous (SC) STELARA maintenance phase.¹ 
• The study included 112 patients (2 to <18 years; weight ≥10 kg) with moderately to severely active UC (baseline Mayo score ≥6, Mayo endoscopy subscore ≥2), with inadequate response or intolerance to conventional/biologic therapy or corticosteroid dependent. 
• At week 8, 109 patients were randomized (1:1), stratified by weight (<40 kg/≥40 kg) and week 8 clinical response status (defined as a decrease from baseline of ≥30% and ≥2 points in the Modified Mayo score, with a decrease of ≥1 point in the rectal bleeding subscore or a rectal bleeding subscore of ≤1), to receive blinded SC STELARA maintenance therapy every 8 weeks (Q8W) or every 12 weeks (Q12W) for 44 weeks.
• STELARA dosing was based on body surface area (<40 kg) or weight-tier if ≥40 kg.
• The primary endpoint was clinical remission (defined as a Mayo stool frequency subscore of 0 or 1, with no increase from induction baseline; a rectal bleeding subscore of 0; and an endoscopy subscore of 0 or 1, with no friability present on endoscopy) at week 8 and at week 52 in those with response to induction at week 8.

Results

Induction and Maintenance Phase

Baseline Characteristics

• Among the 112 patients included, the median (interquartile range [IQR]) age was 14.0 (11.0-15.5) years; 54.5% of patients were female, and 60.7% of patients were biologic naïve.
• The median (IQR) Pediatric Ulcerative Colitis Activity Index (PUCAI) score was 55.0 (45.0-60.0), and the median (IQR) Mayo score was 8.0 (7.0-9.0); 91.7% of patients had moderate UC, and 67.0% of patients had extensive UC.

Efficacy

• At week 8, 79 patients achieved clinical response (induction responders).¹ 
• At week 52, 32/79 patients (40.5%; 95% confidence interval [CI], 30.4-51.5) achieved clinical remission; see Table: Clinical and Endoscopic Outcomes at Week 52.

• For a subgroup analysis of clinical remission by prior biologic use and body weight, see Table: Subgroup Analysis of Remission.

Safety

• During maintenance therapy, serious adverse events (SAEs) occurred in 6.4% (7/109) of patients, with gastrointestinal disorders being the most commonly reported SAEs.¹ 
  • Overall, the adverse event (AE) rate was similar between the Q8W and Q12W dosing groups.
    • Treatment-emergent SAEs occurred in 9.3% (5/54) and 3.6% (2/55) of patients receiving Q8W and Q12W dosing, respectively.

Exposure Optimization Substudy

Study Design/Methods

• Patients who experienced loss of response after week 16 or were week 16 induction nonresponders and had low steady-state trough STELARA levels (<1.4 μg/mL) were eligible to enroll in an optional exposure optimization substudy (EOS; ≥16 weeks) with every 4 week (Q4W) dosing.² 
• Clinical response (partial Mayo score) and clinical remission (PUCAI score) were evaluated at week 16.

Baseline Characteristics

• Among the 109 patients randomized to receive maintenance therapy, 97 were responders.
• A total of 21/109 (19.3%) patients received Q4W dosing in EOS, including 4/109 (3.7%) induction nonresponders at week 16 and 17/97 (17.5%) responders who experienced confirmed loss of response with low STELARA concentrations during the maintenance period.² 
• Key baseline demographics of the 21 patients treated in EOS: median (IQR) age was 14.0 (9.0, 15.0) years, and 12/21 (57.1%) patients experienced prior biologic failure.² See Table: Summary of Baseline Demographics and Disease Characteristics of Patients Treated in EOS.

Efficacy and PK

• The 16-week EOS was completed by 16/21 (76.2%) patients.²
• At EOS week 16, clinical response (as assessed using the partial Mayo score) was achieved by 11/13 (84.6%) patients, and clinical remission (as assessed using the PUCAI score) was achieved by 9/16 (56.3%) patients. See Figure: Percentage of Patients Achieving Clinical Remission Based on the PUCAI Score by Visit from EOS Initiation Over Time.
• The average PUCAI score and partial Mayo score decreased over time following EOS initiation.
• After Q4W dosing, the median (mean) STELARA concentration in pediatric patients increased from 0.38 (0.97) µg/mL to within the observed range of adults receiving STELARA 90 mg Q8W (serum trough STELARA concentration ranged from 0.86 to 7.18 µg/mL, at substudy week 12 in UNIFI Jr when this approximated steady state).

Safety

• AEs were reported in 81.0% of patients, including ≥1 infections in 57.1% of patients (none classified as serious); SAEs were reported in 9.5% of patients.²
• Safety with SC STELARA Q4W was consistent with that observed in the main study, with no new safety concerns identified.

Prospective Study

Dhaliwal et al (2021)³ evaluated the serum concentrations and efficacy of STELARA in children and adolescents (2-17 years of age) with UC who were refractory to treatment with biologics.

Study Design/Methods

• This was an open-label, prospective study of pediatric patients from 12 academic pediatric inflammatory bowel disease (IBD) centers identified through a Canadian database.
• Children were included in this intention-to-treat (ITT) analysis if they were treated with IV STELARA following the failure of infliximab.
• All patients received STELARA via IV induction without concomitant immunomodulators:
  • 260 mg for patients weighing 18.3-52.6 kg (n=13)
  • 390 mg for patients weighing 36-76.1 kg (n=11)
  • 520 mg for patients weighing 74.8 kg (n=1)
• SC injections of STELARA 90 mg were scheduled to begin at week 8, except for the smallest child who received 45 mg.
• The standard Q8W dosing frequency could be shortened after the first SC dose per the discretion of the treating physician based on continuing symptoms.
• Baseline disease activity was categorized using PUCAI. Endoscopic findings were documented using the Mayo endoscopic score (MES).
• Disease activity and medication usage was recorded as needed and routinely every 6 months.
• Ustekinumab serum levels were also measured by enzyme-linked immunosorbent assay (ELISA) prior to SC injections.
• The primary outcome of this study was steroid-free clinical remission (SFCR; PUCAI <10 and no corticosteroid usage for ≥4 weeks) on continued STELARA therapy with an intact colon at week 52.
• Secondary and other outcomes included:
  • SFCR at week 26
  • Sustained SFCR (no corticosteroid usage between weeks 26 and 52)
  • Endoscopic improvement (rectosigmoid Mayo score ≤1 in those with pre-STELARA score ≤2); fecal calprotectin (FCP) <250 µg/g was accepted as endoscopic improvement
  • Biomarker remission (FCP score <250 µg/g)
  • Colectomy rate at 1 year

Results

Baseline Characteristics

• There were 25 children with UC analyzed in this study; the median age was 14.8 years (IQR, 12.3-16.2) and the mean duration of UC was 2.3 years (IQR, 1.1-4.2) at STELARA initiation.
• Previous biologic failure to 1 class (anti-tumor necrosis factor [TNF] agent) was reported in 13 (56%) patients; 12 (44%) patients had previous biologic failure to 2 classes (anti-TNF agent and vedolizumab [VDZ]).
• Additionally, 17 patients were receiving concomitant corticosteroids at the time of STELARA initiation, and no patients were receiving a concomitant immunomodulator.
• The mean STELARA dose was 6.4 mg/kg (IQR, 5.5-7.5).

SFCR and Endoscopic Improvement

• SFCR was achieved in 11/25 (44%) children at week 52. This included 9 (69%) of 13 children with previous biologic failure to anti-TNF agents vs 2 (17%) children with previous biologic failure to anti-TNF agents and VDZ (P=0.008).
• For SFCR at months 6 and 12, sustained SFCR, and endoscopic improvement, see Table: Rates of SFCR and Endoscopic Improvement.

• A multivariate analysis that adjusted for age and sex found the odds ratio (OR) of achieving SFCR at week 52 following previous failure to only an anti-TNF agent vs anti-TNF agent and VDZ was 17.4 (95% CI, 1.6-190).
• Among 8 of the 11 patients who achieved SFCR at week 52 and had data >1 year at follow-up, remission had been maintained to ≥72 weeks, including 2 patients with previous failure to anti-TNF agents and VDZ and 5 patients with previous failure to anti-TNF agents only.
• There were 5 patients who discontinued treatment after receiving 1 IV dose of STELARA due to colectomy (n=4) and parental choice (n=1). Another 4 patients discontinued STELARA during maintenance due to colectomy (n=2) and initiation of other treatment (n=2).

Ustekinumab Concentrations and Antidrug Antibodies

• Of the 20 patients initiating maintenance therapy, 14 (70%) patients had serum ustekinumab levels and antidrug antibody levels measured at the median time of 30.9 weeks (IQR, 22.1-55.8).
• The median trough ustekinumab concentration was 5.2 µg/mL (IQR, 4.0-9.7); no patient developed detectable antibody levels to STELARA.
• Greater exposure to STELARA (dosing Q4W vs Q8W) was not associated with a superior rate of clinical remission.
• During the maintenance phase of treatment with STELARA, median ustekinumab levels were higher in patients with active colitis (all with dosing interval already empirically shortened to 4 weeks) than patients in clinical remission (9.5 µg/mL [IQR, 4.7-11.8] vs 3.9 µg/mL [IQR, 2.7-6.2], respectively; P=0.02).

Endoscopic Outcomes and Biomarker Remission

• Patients who achieved the primary endpoint (n=11) also underwent a colonoscopic reassessment (n=5) and/or FCP determination (n=9) to verify more than symptom resolution. Endoscopic improvement was evident in 7 of these 9 patients.
  • Of the 9 patients with stools examined at a median time of 51.1 weeks (IQR, 34.6-56.0), FCP was <250 µg/g in 5 patients (baseline median FCP, 863 µg/g [IQR, 759-2100]).
  • Of the 5 patients who underwent a colonoscopic reassessment at a median time of 51.4 weeks (IQR, 29.1-91.5), the MES was 0 or 1 in 4 of these patients (baseline MES ≥2).
• No AEs were reported with treatment of STELARA.

Retrospective Studies

Alhadab et al (2025)⁴ conducted a single-center retrospective cohort study on the clinical effectiveness and safety of STELARA in children and adolescents with IBD from a single tertiary hospital in Saudi Arabia.

Study Design/Methods

• This study included patients diagnosed with Crohn’s disease (CD) or UC at <16 years of age who had received the first dose of STELARA between January 2017 and February 2022 with a minimum follow-up of 1 year.
• Response to therapy was assessed by using clinical scores, laboratory markers, the need for corticosteroid therapy, and both endoscopic and pathologic measures.
• PUCAI was used to assess UC clinical activity.
• Clinical remission was defined as a PUCAI score of ≤10.
• Patients in clinical remission without corticosteroids were considered in SFCR.
• Biochemical remission was defined as FCP and C-reactive protein (CRP) levels ≤250 μg/g and 5 g/dL, respectively.
• Patients were divided into 2 groups based on clinical remission at week 52:
  • Group I: Patients who achieved clinical remission
  • Group II: Patients who did not achieve clinical remission
• Endoscopic findings were documented using MES, with endoscopic remission defined as an MES of 0.
• Primary nonresponse was defined as failure to achieve a response to induction therapy (clinical, biochemical, or endoscopic) despite reaching therapeutic levels.
• Secondary nonresponse referred to patients who had previously responded to a biologic but experienced deterioration or relapse while still on the same biologic therapy.
• All patients received a single IV induction dose:
  • 9 mg/kg for patients weighing 10 to <40 kg (maximum dose of 260 mg)
  • 390 mg for patients weighing >40 kg
• Maintenance doses were given SC:
  • 2-4 mg/kg for patients weighing 10 to <40 kg (minimum dose of 45 mg and maximum dose of 90 mg)
  • 90 mg for patients weighing >40 kg

Results

Baseline Characteristics

• Of the 13 patients included in this analysis, 5 (38.5%) had UC (group I, 4 [80%]; group II, 1 [20%]), with a median age of 13 years (IQR, 11.5-14).
• STELARA treatment was initiated after a median of 3 years (IQR, 2.3-7) of diagnosis.
• Five (41.7%) patients received corticosteroids at induction.
• All patients had prior exposure to ≥1 anti-TNF drug before the start of STELARA, with a median biologic treatment duration of 2 years (IQR, 1.3-5).

Clinical and Biochemical Response to STELARA

• Overall, 10 (76.9%) and 6 (46.2%) patients achieved clinical remission and biochemical remission, respectively; see Table: Changes in PCDAI/PUCAI Scores, Biochemical Remission, and SFCR.
• Three (60%) and 2 (40%) patients with UC had S0 and S1 disease severity, respectively, defined by PUCAI scores >65.
• CRP and Pediatric Crohn’s Disease Activity Index (PCDAI)/PUCAI scores decreased in both clinical responders and nonresponders, but the decrease was more in the clinical remission group (P=0.007 and P=0.371, respectively).
• Similarly, FCP levels and the need for corticosteroids after STELARA therapy significantly decreased in the clinical remission group (P=0.021 and P=0.014, respectively).

Endoscopic Remission to STELARA

• Endoscopy was performed on 4 patients with UC.
• MES scores for 3 patients with UC decreased from 3, 2, and 2 before STELARA to 1, 0, and 0 after treatment, respectively.

Safety Profile

• Among the 13 patients treated with STELARA, 2 reported infection related to treatment.
• One patient experienced acute Salmonella enteritis, which required hospitalization and IV antibiotics, whereas another patient developed cystitis.

Cohen et al (2024)⁵ conducted a retrospective cohort study to assess the effectiveness and safety of STELARA in children with UC and unclassified inflammatory bowel disease (IBDU).

Study Design/Methods

• This was a retrospective study from 16 centers affiliated with the Pediatric IBD Interest and Porto Groups of the European Society for Paediatric Gastroenterology Hepatology and Nutrition (ESPGHAN).
• Children and adolescents (≤18 years old) who received ≥1 dose of STELARA for UC or IBDU according to the revised Porto criteria and current guidelines were included.
• The primary outcome was corticosteroid-free clinical remission (CFCR) following STELARA initiation over 52 weeks.
• Secondary outcomes included:
  • Clinical remission and response at 16 weeks
    • Clinical remission was defined as a PUCAI score of <10.
    • Clinical response was defined as a reduction in PUCAI score of >20 points.
  • CRP levels below 5 mg/L and FCP levels under 150 µg/g
    • Laboratory remission was defined as CRP levels below 5 mg/L and FCP levels below 150 µg/g.
  • Endoscopic and radiologic remission
    • Radiologic remission was defined as complete remission on bowel ultrasound or magnetic resonance enterography.
    • Endoscopic remission was defined as an MES of 0 on colonoscopy during follow-up.
    • Endoscopic and radiologic remission were determined for the ITT group, using nonresponse imputation for children who discontinued therapy during the follow-up period.
  • Disease exacerbation
    • Defined as a PUCAI score of ≥10
  • Hospitalizations
  • Need for IBD-related surgery during follow-up
  • AEs
• The induction dose ranged from 130 mg to 520 mg with a median dose of 6.1 mg/kg (IQR, 5.5-7.7).⁶
• Maintenance dosage regimens used were: 45 mg Q4W, every 6 weeks, or Q8W; 90 mg Q4W, every 6 weeks, every 7 weeks, Q8W, or Q12W; or 130 mg Q4W.⁶

Results

Baseline Characteristics

• There were 58 children in the study, with 39 (67.2%) diagnosed with UC; the median age at STELARA initiation was 14.5 years (IQR, 11.5-16.5) and median duration of follow-up was 12.8 months (IQR, 5.1-26.3).
• Prior to STELARA therapy, 50 (86.2%) children had received treatment with immunomodulators.
• All 58 children were treated with biologic agents:
  • 20 (34.5%) had failed 1 biologic agent
  • 28 (48.3%) had failed 2 biologic agents
  • 10 (17.2%) had failed 3 biologic agents
• The median PUCAI score at the initiation of STELARA therapy was 45 (IQR, 31.3-55).
• Twenty-three (39.7%) children underwent escalation of STELARA dosage during the follow-up period, with 13 (57%) achieving clinical or biomarker response.

Clinical and Biochemical Response to STELARA

• CFCR was achieved in 27 (46.6%), 33 (56.9%), and 37 (63.8%) children at weeks 16, 26, and 52, respectively.
• Eighty-four percent of the children successfully weaned steroids within 3 months of STELARA initiation.
• Clinical remission was achieved in 29 (50%), 33 (56.9%), and 37 (63.8%) children at weeks 16, 26, and 52, respectively; clinical response was observed in 39 (67.2%) children at 16 weeks.
• Overall, CFCR was achieved in 19/31 (57.6%) children receiving corticosteroid therapy at baseline vs 18/27 (66.7%) children not treated with corticosteroids (P=0.671).
• The variables associated with achieving CFCR were:
  • Diagnosis of UC compared with IBDU (hazard ratio [HR], 2.24; 95% CI, 1.03-4.85; P=0.041)
  • Lack of prior VDZ therapy (HR, 2.18; 95% CI, 1.11-4.27; P=0.023)
  • History of corticosteroid-resistant disease (HR, 4.29; 95% CI, 1.58-11.65; P=0.004)
• Laboratory remission was achieved in 11 (19%), 16 (36.2%), and 25 (51.7%) children at weeks 16, 26, and 52, respectively.
• For longitudinal changes in laboratory parameters, see Table: Longitudinal Changes in Laboratory Parameters.

• Twenty-four (41.4%) children discontinued STELARA at a median duration of 5 (3.4-10.3) months after initiation. The reasons for discontinuation among children were, n (%):
  • Nonresponse: 21 (87.5)
  • AEs: 3 (12.5)
• Ten (41.7%) children underwent colectomy after discontinuation.
• Among the 6 (10.3%) children who underwent colectomy while on STELARA therapy, 3 had severe UC at STELARA initiation.
• Eleven (19%) children experienced IBD-related hospitalizations.
• Disease exacerbation occurred in 13 (37.1%) of children in remission.

Ustekinumab Serum Levels

• Ustekinumab serum levels were available for 22 (38%) children, with median (IQR) levels of 4.1 µg/mL (1.9-5.1), 2.7 µg/mL (1.6-6.8), and 2.6 µg/mL (2.1-5.4) at weeks 16, 26, and 52, respectively.
• None of the children showed evidence of antidrug antibodies.

Safety Profile

• Treatment-related AEs were reported in 6 (10.3%) children, leading to STELARA discontinuation in 3 children and death in 1.
• These events included interstitial nephritis, hypersensitivity reaction, breast abscess, injection-site reaction, cytomegalovirus infection, and 1 death attributed to an acute episode of severe diarrhea in an adolescent male who was also on corticosteroids and methotrexate while on STELARA therapy.

Patel et al (2024)⁷ conducted a retrospective cohort study using the ImproveCareNow (ICN) registry to compare the real-world effectiveness and safety of STELARA and VDZ in pediatric patients with UC who were refractory to anti-TNF therapy.

Study Design/Methods

• This analysis included pediatric patients (<21 years) with confirmed UC who had previously failed anti-TNF therapy and were subsequently treated with either STELARA or VDZ as second-line therapy.
• Patients with CD, IBDU, a history of colectomy, or prior exposure to STELARA or VDZ before anti-TNF therapy were excluded.
• Patients were required to have active disease prior to initiating STELARA or VDZ, which was defined as a PUCAI score ≥10, or a Physician Global Assessment (PGA) >1 (if PUCAI was unavailable), or current corticosteroid use, or elevated inflammatory markers (CRP ≥5 mg/L).
• The primary endpoint was CFCR at 6 months, defined as a PUCAI<10 or PGA=1, with no corticosteroid use, therapy discontinuation, IBD-related hospitalization, or surgery at the outcome visit.
• Secondary endpoints included clinical response, IBD-related hospitalizations and surgeries, growth, nutrition, infections, and malignancies.
• Clinical response was defined as a binary outcome that included patients in remission and those with a ≥20-point decrease in PUCAI or a PGA indicating mild or quiescent disease, as per STRIDE-II guidelines.

Results

Baseline Characteristics

• A total of 336 pediatric patients were included (STELARA, n=74; VDZ, n=262). For details, see Table: Select Baseline Characteristics After Imputation.

Primary Endpoint

• At month 6, 25.8% of patients treated with STELARA achieved CFCR, compared to 28.3% of patients treated with VDZ. An unadjusted OR showed no significant difference between the 2 groups (OR, 0.88; 95% CI, 0.47-1.63; P=0.68).

Secondary Endpoints

• For secondary endpoints including efficacy and safety outcomes, see Table: Secondary Endpoints.
• In a multivariate regression model adjusted for baseline differences in PUCAI scores and immunomodulator use, the odds of achieving clinical response remained nonsignificant (OR, 0.65; 95% CI, 0.36-1.15; P=0.14).
• No corticosteroid use at baseline was the only predictor of achieving clinical response (OR, 0.36; 95% CI, 0.21-0.63; P<0.001) and CFCR (OR, 0.33; 95% CI, 0.18-0.62; P<0.001) at 6 months.
• Treatment discontinuation was reported in 40% and 32% of patients treated with STELARA and VDZ, respectively, after 1 year of treatment.

Koudsi et al (2023)⁸ assessed the safety and efficacy of STELARA in a multicenter, retrospective study in pediatric patients with IBD using data from the Groupe d'Etudes Thérapeutiques des Affections Inflammatoires du Tube Digestif (GETAID), a French consortium.

• This analysis included patients <18 years of age treated with ≥1 STELARA injection for CD or UC from January 2016 to December 2019 at 9 university hospitals.
• Clinical, biological, and endoscopic data were retrospectively collected. Additionally, the analysis was based on information from electronic medical records such as patient’s baseline characteristics, clinical data, clinical disease activity index, disease phenotype before starting STELARA, treatment history, endoscopic findings, and laboratory parameters at the beginning of the treatment (induction), 3 months after induction and at the last follow-up.
• All patients included in this study were resistant to anti-TNF agents.
• Patients received weight-adjusted STELARA IV (6 mg/kg) and 90 mg SC after 8 weeks.
• Of the 53 patients included in this analysis, 5 (9.4%) patients had UC.
• Significant improvements in PUCAI were observed at 3 months (mean, 25 [15-40]) and at the last follow-up (mean, 18.3 [0-35]) compared to baseline (mean, 47 [35-65]).
• The mean CRP at STELARA induction was 15.8 mg/L (0.5-30). At 3 months, CRP normalization was observed in 75% of patients.
• Overall, STELARA was discontinued in 15/53 (28%) patients due to lack of efficacy in 8 patients, loss of response in 5 patients, and exacerbation of an associated Chronic Recurrent Multifocal Osteomyelitis in 1 patient. The treatment was discontinued in the first 3 months in 7 patients and after 3 months in 8 patients.
• No severe AEs were reported in this analysis.

Patel et al (2021)⁹ conducted a retrospective chart review of the use of STELARA in pediatric patients with UC at a single center.

• Clinical remission and steroid-free remission at weeks 26 and 52 of therapy were the primary outcomes.
• Of the twelve patients evaluated, the cohort was mostly female (83%) and ages ranged from 6-15 years.
• By week 26, 78% (7/9) of patients achieved both clinical and steroid-free remission, and 87% (6/7) of these patients were on STELARA monotherapy.
• Five patients had data at week 52; of those, 80% (4/5) had clinical and steroid-free remission on STELARA monotherapy.
• At week 26, 88% of patients were on intensified therapy compared to adult dosing recommendations based on therapeutic drug monitoring and clinical decision making; at week 52, 100% of patients were on intensified therapy.
• No AEs were observed in this cohort.

Literature Search

A literature search of MEDLINE^®, EMBASE^®, BIOSIS Previews^®, and DERWENT^® (and/or other resources, including internal/external databases) was conducted on 22 January 2026.

Summarized in this response are data from UNIFI Jr phase 3 study, prospective and retrospective studies on the use of STELARA in pediatric patients with UC.