STELARA - Treatment of Pediatric Crohn’s Disease

UNITED STATES PRESCRIBING INFORMATION

Recommended Pediatric Dosage in Crohn’s Disease (CD)

Intravenous (IV) Induction Pediatric Dosage Regimen

• The recommended induction dosage in pediatric patients weighing 10 kg and above is a single IV infusion administered using the weight-based dosage regimen as described in the Table: Initial IV Induction Dose of STELARA in Pediatric Patients (Weighing 10 kg or More) with CD.¹⁷

Subcutaneous (SC) Maintenance Pediatric Dosage Regimen

• The recommended maintenance dosage in pediatric patients using the weight-based dosage is¹⁷:
  • 10 kg to 35 kg: a SC 2.5 mg/kg dose administered 8 weeks after the initial IV dose, then every 8 weeks (Q8W) thereafter
  • Greater than 35 kg: a SC 90 mg dose administered 8 weeks after the initial IV dose, then Q8W thereafter

Clinical Study - Pediatric CD

• Of the 101 pediatric patients enrolled in the open-label IV 8-week induction phase followed by the 44-week double-blind maintenance phase, 93 patients were included in the efficacy analyses.¹⁷
• Patients received induction treatment with a single STELARA IV dose of approximately 6 mg/kg (patients weighing 40 kg or more) or 250 mg/m² (patients weighing less than 40 kg, based on body surface area [BSA]), followed by a randomized double-blind SC maintenance regimen of 90 mg STELARA (patients weighing 40 kg or more) or 60 mg/m² STELARA (patients weighing less than 40 kg, based on BSA) Q8W or another SC dosage regimen.
• The recommended dosage for pediatric patients weighing 10 kg to less than 40 kg differed from the BSA-based dosage in this study. There are no anticipated clinically relevant differences in efficacy between the recommended and studied pediatric STELARA dosages.
• At week 8, the proportion of patients achieving clinical remission (defined as Pediatric Crohn’s Disease Activity Index [PCDAI] score ≤10) was 46% (43/93) and those achieving clinical response (defined reduction from baseline in PCDAI score of >12.5 points with a total PCDAI score ≤30) was 87% (81/93).
• Of the 81 patients who received IV induction treatment and achieved clinical response or remission at week 8, 39 patients received SC maintenance dosage Q8W. Efficacy endpoints assessed at week 52 are described in Table: Proportion of Pediatric Patients 2 Years of Age or Older with Moderately to Severely Active CD Meeting Efficacy Endpoints at Week 52 From Initiation of Induction.

• At week 52, the proportion of patients in endoscopic response (defined as reduction in the Simple Endoscopic Score for Crohn’s Disease [SES-CD] score of >50% or SES-CD score ≤2, in patients with baseline SES-CD score ≥3) was 26% (10/39) and those achieving corticosteroid (CS)-free clinical remission (PCDAI score ≤10 points and not receiving CS for ≤90 days prior to week 52) was 49% (19/39).

Clinical Trial Experience in Pediatric Patients ≥2 Years of Age with CD

• The safety of STELARA has been studied in 101 pediatric patients (2 to 17 years of age) with moderately to severely active CD and 48 patients received the recommended maintenance dosage.¹⁷
• In general, adverse reactions reported in the clinical trial of pediatric patients with CD were similar to those reported in adult patients with CD in UNITI-1, UNITI-2, and IM-UNITI.
• Other adverse reactions reported in at least 10% of pediatric patients were upper respiratory tract infection (13% during induction and 17% during maintenance),
  COVID-19 (17% during maintenance), and headache (10% during maintenance).
• One pediatric patient discontinued treatment with STELARA after developing anaphylactic shock within the first 2 minutes of initiation of the first IV infusion and was stabilized with medical intervention.

CLINICAL DATA

PIVOTAL PHASE 3 STUDY-UNITI Jr

Turner et al (2026)¹ evaluated the efficacy and safety of STELARA in pediatric patients with moderately to severely active CD.

Study Design/Methods

• Patients weighing ≥40 kg, having a PCDAI score of >30, and ileocolonoscopic ulceration (as assessed by SES-CD) and receiving a single, weight-tiered IV induction dose of STELARA dose matching the adult posology were included in the study.
• After 8 weeks of induction therapy, patients were randomized 1:1 to receive a single SC 90 mg maintenance dose of STELARA either Q8W or every 12 weeks (Q12W).
• The primary endpoint was clinical remission (PCDAI score ≤10) at week 8 and clinical remission at week 52 among randomized induction responders.

Results

• The study population included 101 patients; 42.6% were biologic-naïve, and the median (interquartile range, IQR) PCDAI score was 40.0 (35.0-45.0).
• At week 8, 46.5% (n=47) of patients achieved clinical remission. Among them, 68.1% (32/47) maintained clinical remission at week 52.
• At week 8, 84.2% (n=85) of patients achieved clinical response. Among them, 54.1% (46/85) achieved clinical remission and 52.9% (45/85) achieved CS-free clinical remission at week 52.
• At week 52, remission rates were higher in patients without prior biologic failure and were similar across all weight subgroups from week 8 to week 52, see Table: Subgroup Analysis of Remission.

Safety

• The rate of adverse events (AEs) was reported to be similar across the treatment groups.¹
• Serious adverse events (SAEs) were reported in 16.8% (17/101) of patients, with CD exacerbation (5/17) being the most frequent SAE. Serious infections occurred in 5.9% of patients.

Clinical, Endoscopic, and Histologic Outcomes

De Greef et al (2026)³ conducted a substudy evaluating the efficacy of STELARA on endoscopic and histologic outcomes in patients with active CD (UNITI Jr study).

Study Design/Methods

• Patients underwent ileocolonoscopies at screening; week 16 and week 52.
• The endoscopic (SES-CD scores) and histologic (Robarts Histopathology Index [RHI]) response and remission were evaluated in a blinded manner at week 16 and at week 52 among induction responders.

Results

• Of the 97 randomized patients, 57 had ileocolonic disease, 9 had ileal-only disease, and 17 had colonic-only disease.
• The median (IQR) baseline SES-CD score was 12.0 (7.0-19.0), and 97.9% (n=94) had a baseline SES-CD score of ≥3.
• At baseline, RHI was assessed for 86.3% (82/95) of patients.
• Endoscopic, histologic, and combined histo-endoscopic index scores at week 16 and week 52 are summarized in Table: Endoscopic, Histologic, and Combined Histo-Endoscopic Index Scores for Response and Remission.
• Week 52 outcomes stratified by weight category (<30 kg, ≥30 to <40 kg, ≥40 kg) are summarized in Table: Endoscopic and Histo-Endoscopic Response and Remission Rates at Weeks 16 and 52 by Patient Weight.

Dose Escalation

Russel et al (2026)⁴ evaluated dose escalation in participants with primary/secondary loss of response to conventional STELARA dosing in the Exposure Optimization Study (EOS) of UNITI Jr study.

Study Design/Methods

• Patients from the UNITI Jr study with ustekinumab trough levels (UTLs) <1.4 μg/mL who were nonresponders at week 16 of induction or who lost response after week 16 were included in an additional 16-week EOS with Q4W dosing.

Results

• A total of 26 patients (26.8%) were included in the EOS; 80.8% (21/26) had prior biologic failure, and 53.8% (14/26) had a baseline PCDAI >40 at baseline of EOS.
• A total of 21 patients (80.8%) completed ≥16 weeks of EOS treatment.
• At week 16 of the EOS, 20 patients completed the short form of PCDAI (sPCDAI) assessment.
  • Of those patients, 50% (10/20) achieved clinical remission and 95% (19/20) achieved clinical response.
• Median (IQR) serum ustekinumab concentration increased from 0.71 μg/mL (0.37-1.02) at the start of EOS to 3.92 μg/mL (2.08-4.74) at week 16, falling within the observed week 16 range of adults with CD who received STELARA Q8W (median, 2.05 μg/mL [1-3.70]).
• Laboratory parameters of inflammation and fecal lactoferrin levels improved from EOS baseline to week 16 and were similar to those at week 52 of the main study, see Table: Mean Laboratory Values (range) at EOS Week 16 and Changes from EOS Baseline vs Values at Week 52.

• Overall, 73.1% of patients reported AEs (38.5% gastrointestinal-related), and 11.5% of patients reported SAEs. No new safety issues were reported.

Health-Related Quality of Life

Griffiths et al (2026)⁵ evaluated the effect of STELARA on disease-specific symptoms using the IMPACT-III metric in patients with CD from the UNITI Jr study.

Study Design/Methods

• IMPACT-III is a disease-specific health-related quality of life (HRQoL) metric used in pediatric clinical trials.
• Patients ≥10 years of aged at week 0 and week 52 were included for the IMPACT-III questionnaire.
• Symptoms were evaluated using both the original 6‑domain IMPACT‑III and a modified 4‑domain version, in which the “Well‑being” subdomain reflects only bowel and systemic symptoms. To minimize overlapping content with the PCDAI, abdominal pain item was also removed from “Well-being” subdomain.
• Clinical meaningfulness was assessed using distribution-based methods (0.5 SD threshold).

Results

• Among 77 patients, the mean (SD) baseline IMPACT-III total score was 101.7 (23.89).
• At week 52, the mean change from baseline (SD) in the transformed 6-domain IMPACT-III total score was 13.7 (19.99).
• The most responsive subdomains (mean change from baseline [SD]) were “bowel symptoms” (20.2 [26.91]) and “systemic symptoms” (19.3 [27.80]).
• For the modified 4-domain IMPACT-III, the well-being subdomain “symptom facets” showed a mean change from baseline (SD) of 21.7 (28.32). Change from baseline (improvement) exceeded the 0.5 SD threshold.

Pharmacokinetics and Exposure-Response Relationships

Adedokun et al (2026)⁶ evaluated the pharmacokinetics (PK) and exposure-response (ER) relationship of ustekinumab in pediatric patients with moderate to severely active CD enrolled in the UNITI Jr study.

Study Design/Methods

• PK and ER parameters assessed through week 52 in the UNITI Jr study were compared to those in the adult population from the UNITI1, UNITI2, and IMUNITI studies.

Results

• Overall, 99 patients received ≥1 dose of STELARA, and PK was evaluated using ≥1 blood sample for each patient.
• Following induction and maintenance phases of therapy, median serum ustekinumab concentrations were similar between pediatric and adult patients.
• A positive association between serum ustekinumab concentrations and efficacy outcomes was observed during both induction and maintenance phases in pediatric and adult patients.
• At week 8, a positive ER relationship between serum ustekinumab concentrations and clinical remission was observed in both pediatric and adult patients, with a higher probability of clinical remission among pediatric patients.
• At week 52, following maintenance therapy, the ER trend between steady-state ustekinumab concentrations and clinical remission was similar between pediatric and adult patients.
• Similar to adult patients, at a given serum concentration, a higher proportion of pediatric patients achieved clinical remission in the anti-tumor necrosis factor (TNF) nonfailure population compared with the anti-TNF failure population.
• No effect of age or body weight on the ER relationship was observed in pediatric patients in this study.
• No association was observed between serum ustekinumab concentrations and safety events, including infections, serious infections, or SAEs. The ER safety pattern was similar between pediatric and adult patients.
• The incidence of anti-STELARA antibodies through approximately 1 year in pediatric patients 3 (N=91; 3.3%) was similar to that in adult patients 9 (N=287; 3.1%).

Phase 1 Clinical Study

Rosh et al (2021)⁷ evaluated the PK, efficacy and safety of STELARA in pediatric patients with moderately to severely active CD or fistulizing CD for ≥3 months in a phase 1, multicenter, 16-week, double-blind induction dose-ranging study (UNISTAR).

Study Design/Methods

• Patients with moderately to severely active CD who were 2 to <18 years of age (body weight ≥10 kg) were included. Additionally, patients also had a PCDAI score >30 and at least an abnormal C-reactive protein (CRP; >0.3 mg/dL) or fecal calprotectin (FCP; >250 µg/g), or ulcerations in the ileum and/or colon.
• All patients received previous or current treatment for CD, including oral CSs and/or immunomodulators (eg, azathioprine, 6-mercaptopurine, methotrexate); however, they were required to be stable prior to study start. Patients who failed or were intolerant to anti-TNF therapy were also allowed to participate.
• Randomization (1:1) was performed for induction to 1 of 2 weight-based IV doses:
  • Lower IV induction dose: 3 mg/kg if body weight 10 kg to <40 kg, or 130 mg if body weight ≥40 kg
  • Higher IV induction dose: 9 mg/kg if body weight 10 kg to <40 kg, or 390 mg if body weight ≥40 kg
• At week 8, patients received a single SC maintenance dose of STELARA 2 mg/kg if body weight 10 kg to <40 kg, or 90 mg if body weight ≥40 kg.

Results

Baseline Characteristics

• A total of 44 patients were randomized to either the lower dose STELARA IV induction (n=23) or the higher dose STELARA IV induction (n=21). The median age was 13 years (IQR, 12-16); 59% of patients had a body weight ≥40 kg and 91% had prior exposure to biologics (infliximab, adalimumab, and vedolizumab).
• A total of 73% (32/44) of patients were receiving ≥1 concomitant medications for CD at baseline which included immunomodulators (39%), oral CSs (32%), oral aminosalicylates (21%), and antibiotics (5%).
• Through week 16, 9.1% (4/44) of patients discontinued STELARA due to AEs (worsening of CD [n=2] and lack of efficacy per the investigator [n=2]).

Pharmacokinetics

• Mean serum ustekinumab concentrations were 51.3 μg/mL, 7.7 μg/mL, 3.0 μg/mL, and 1.6 μg/mL at weeks 0 (after infusion), 3, 6, and 8, respectively, for the lower induction dose group. Mean serum ustekinumab concentrations for the higher induction dose group at the same time points were 149.0 μg/mL, 23.7 μg/mL, 9.1 μg/mL, and 4.8 μg/mL, respectively.

Clinical and Endoscopic Outcomes

• Clinical and endoscopic outcomes (clinical response, clinical remission, endoscopic response, and endoscopic remission) from week 3, week 8, and week 16 are shown in Table: Summary of Clinical Outcomes at Weeks 3, 8, and 16.

CS Use

• Steroid use from baseline to week 16 decreased in both lower (30% to 13%) and higher dose groups (33% to 19%).
  • At weeks 8 and 16, 22% (5/23) of patients in the lower dose group were in steroid-free clinical remission; in the higher dose group, 10% (2/21) and 24% (5/21) of patients were in steroid-free clinical remission at weeks 8 and 16, respectively.

Inflammatory Biomarkers

• In the lower dose group, 16.7% (3/18) of patients had normalized CRP levels at weeks 8 and 16, while in the higher dose group, 28.6% (4/14) and 21.4% (3/14) of patients had normalized CRP levels at weeks 8 and 16, respectively.
• Median (IQR) change in CRP concentration from baseline at week 8 vs change from baseline at week 16 was -0.7 (-8.0 to 0.1) mg/L vs 0 (-9.3 to 0) mg/L in the lower dose group and -0.3 (-14.4 to 0.3) mg/L vs -0.8 (-8.7 to 0) mg/L in the higher dose group, respectively.
• Median (IQR) change in FCP concentration from baseline at week 8 vs change from baseline at week 16 was 0 (-2395.0 to 418.0) mg/kg vs 0 (-3438.0 to 190.0) mg/kg in the lower dose group and -37.0 (-1347.0 to 553.0) mg/kg vs 0 (-1126.0 to 654.0) mg/kg in the higher dose group, respectively.

Clinical Outcomes and PK

• Clinical response at week 8 was achieved in 63.2% (12/19) of patients in the higher serum ustekinumab concentration group (>1.38 μg/mL) and 45% (9/20) of patients in the lower serum ustekinumab concentration group (≤1.38 μg/mL).
• Median improvement from baseline in PCDAI score at week 8 was 20 for patients in the higher serum ustekinumab concentration group (>1.4 μg/mL) and 17.5 for patients in the lower serum ustekinumab concentration group (≤1.4 μg/mL).
• There was no observable correlation between serum ustekinumab concentration and clinical remission at week 8.

Safety

• A total of 73% of patients reported ≥1 AE through week 16 which included 83% in the lower dose group and 62% in the higher dose group.
• SAEs were reported in 16% of patients (lower dose group, 26%; higher dose group, 5%) with CD exacerbation being the most frequent (lower dose group, 9%; higher dose group, 5%).
• Infections (upper respiratory tract infection, anal abscess, Clostridium difficile, eczema infected, gastroenteritis, gastroenteritis viral, and nasopharyngitis) were reported in 39% of patients (lower dose: 39%, n=9; higher dose: 38%, n=8).
• A total of 2 patients discontinued treatment due to AEs (n=1, each dosing group) and no malignancies, deaths, injection-site reactions, anaphylaxis, serum sickness-like events, opportunistic infections, or antibodies to STELARA were reported through week 16.

Long-Term Extension of UNISTAR

Turner et al (2024)⁸ evaluated the PK, immunogenicity, efficacy, and safety of STELARA in pediatric patients with moderately to severely active CD in the long-term extension (LTE) of the UNISTAR study through week 240.

Study Design/Methods

• Patients who responded to STELARA, at the opinion of the investigator, at week 16 entered LTE and were continued on SC STELARA maintenance therapy Q8W up to week 240.
• Clinical efficacy and inflammatory biomarkers were evaluated from enrollment in LTE (week 16 of UNISTAR) through week 224.
  • Clinical remission was evaluated using PCDAI (PCDAI score ≤10 points), which was assessed Q8W from weeks 24 to 48 and every 24 weeks from week 56 through the end of the study.
  • CRP was evaluated Q8W from weeks 24 to 48 and approximately every 24 weeks thereafter.
  • Stool FCP and lactoferrin were evaluated every 24 weeks from week 24.
• Serum ustekinumab concentration and antibodies were measured Q8W through week 56, every 24 weeks thereafter, and at early termination and safety follow‐up visits.

Results

Baseline Characteristics

• Among the patients (N=44) who received STELARA, 34 were enrolled in LTE at week 16 and 8 completed the LTE.

Clinical Outcomes

• At weeks 16 and 48, 25% (11/44) and 41.2% (14/34) of patients achieved clinical remission, respectively.
• At week 48, 38.2% (13/34) of patients were in CS-free clinical remission and 90.0% (9/10) of patients who were in clinical remission at week 8 were still in clinical remission.

Inflammatory Biomarkers

• Among 17 patients with abnormal CRP values at week 0 and available CRP data at week 48, 41.2% (7/17) achieved normalization of CRP at week 48.
• Among 24 patients with abnormal CRP values at week 0 and assuming that patients with missing data did not achieve CRP normalization, 29.2% (7/24) achieved normalization of CRP at week 48.
• Among patients with abnormal FCP values at week 0 and available data at week 48, 36.8% (7/19) achieved normalization of FCP and 30.0% (6/20) achieved normalization of fecal lactoferrin at week 48.

HRQoL

• The mean (SD) IMPACT-III score at week 0 was 107.9 (17.2). The mean (SD) IMPACT-III score at week 48 was 131.1 (22.8), showing a mean (SD) change of 23.9 (25.4) from baseline.

PK and Immunogenicity

• Antibodies to STELARA were observed in one patient randomized to the lower induction dose with a peak titer of 1:100 and tested positive for neutralizing antibodies.

Safety

• Safety events from enrollment in the LTE through the final safety follow-up at week 240 is presented in Table: Safety Events in the LTE Period through Week 240.

Prospective Study

Keller et al (2023)⁹ evaluated the association between UTLs and sonographic transmural healing in pediatric patients with inflammatory bowel disease (IBD) treated with STELARA.

Study Design/Methods

• The study included patients with IBD (aged ≤18 years) receiving maintenance STELARA treatment, who underwent intestinal ultrasound (IUS) examination and serum UTLs at a tertiary center.
• Patients with IUS and UTL performed on 2 events >6 months apart were included in a per-event analysis.
• The primary outcome was the comparison of UTL between patients with and without transmural healing (defined as bowel wall thickness <3 mm and no hyperemia [bowels with hyperemia] on color Doppler, using the Mann-Whitney U test).
• Secondary outcome was the comparison of UTL for each IUS parameter.

Results

• Among 44 children with IBD, 34 had CD.
• The baseline characteristics of patients with CD were as follows:
  • Median age: 14.9 years (IQR, 12.9-16.6)
  • Median disease duration: 3.83 years (IQR, 1.76-5.07)
  • Median time between UTL and IUS: 0 days (IQR, 0-26.5)
• Overall, 18 (53%) patients with CD achieved transmural healing.
  • Higher UTL was associated with transmural healing (11.7 [5.3-17.0] µg/mL) vs without transmural healing (5.5 [4.2-11.0] µg/mL; P=0.013).
• Optimal UTL cut-point for transmural healing detection was 11.4 ug/mL for patients with CD (area under the receiver operating curve [AUROC], 0.661; sensitivity, 59%; specificity, 79%).

Retrospective Studies

Mitchel et al (2025)¹⁰evaluated the real-world effectiveness, safety, and long-term durability of STELARA in the treatment of pediatric patients with CD in a retrospective, longitudinal cohort study.

Study Design/Methods

• Children with active CD based on sPCDAI score ≥15, CRP ≥1.5 mg/dL, and/or FCP ≥250 mcg/g at the time of STELARA initiation were included.
• The primary outcome was the rate of steroid-free clinical remission at 1 year.
  • Steroid-free remission was defined as sPCDAI score <15 without concomitant steroids at any dose in the past 30 days.
• Secondary outcomes were the rates of clinical remission, biochemical remission, drug failure, and drug escalation at 1 year and the last follow-up.
  • Clinical remission was defined as sPCDAI score <15.
  • Biochemical remission was defined as CRP <1.0 mg/dL and FCP <250 mcg/g when both markers were available; individual values were considered separately if only 1 was available at the time point of interest.

Results

Baseline Characteristics

• A total of 101 patients with a median age of 16.38 years (IQR, 13.78-18.07) at the time of STELARA initiation were included.
• The median time of follow-up was 16.6 months (IQR, 8.71-31.2).
• The median baseline sPCDAI score was 20.0 (IQR, 7.5-27.5).
• Eighty-six (85%) patients had previously received ≥1 anti-TNF agent, whereas 14 (14%) patients were biologic naïve.

Effectiveness

• Steroid-free clinical remission was achieved by 79.73% (59/74) of patients at the 1-year follow-up.
• Clinical remission was achieved by 79.73% (59/74) of patients at 1 year; furthermore, 59% (38/64) patients with available CRP and/or FCP values achieved biochemical remission at 1 year.
• A significant improvement in sPCDAI, CRP, erythrocyte sedimentation rate, albumin, and hemoglobin was observed at 1 year (P<0.01, P<0.01, P=0.07, P<0.01, and P<0.01, respectively).
• The time to steroid-free clinical remission was 2.7 months for 50% of patients.
• Within the first year of being initiated on STELARA, 18 patients required hospitalization and 22 patients underwent surgical procedures.
• Among biologic-naïve patients, 86% achieved both clinical and steroid-free remission, 86% achieved clinical remission, and 36% achieved biochemical remission at 1 year.
• At the last follow-up, 67 (66%) patients were in steroid-free clinical remission and 68 (67%) patients were in clinical remission. Of the 75 patients with available CRP and/or FCP data, 43 (57%) achieved biochemical remission.
• A total of 20 patients required hospitalization, and 23 patients underwent surgery through the last follow-up period.
• Surgical procedures included ileocecectomy (n=5), subtotal colectomy (n=4), ileal diversion (n=5), perianal surgery (n=4), and anorectal stricture dilation (n=4).
• Among biologic-naïve patients, 93% achieved steroid-free clinical remission and 29% achieved biochemical remission at the last follow-up.

Safety

• AEs were reported in 6.9% (7/101) of patients on STELARA after a median duration of 7.3 months (IQR, 1.9-11.2).
  • Infections: 2% (2/101)
  • Headaches: 2% (2/101)
    • One occurred following IV induction and resolved spontaneously without intervention.
    • The other was a tension-type headache lasting 1 month and resolved with treatment.
  • Hypersensitivity reactions after SC administration: 1.9% (2/101)
  • Postauricular eczematous rash: 1% (1/101), which resolved with topical CSs
• None of the patients discontinued STELARA due to these AEs.
• No SAEs were observed at the time of the final follow-up.

Pujol-Muncunill et al (2024)¹¹ evaluated the efficacy and safety of STELARA in pediatric patients with CD in a multicenter, retrospective study (STEP-CD Study) from the pediatric IBD Porto group of the European Society of Pediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN).

Study Design/Methods

• Children with CD (2-18 years of age) from 23 centers worldwide who were treated with STELARA monotherapy or with STELARA combined with other medications were included in the study.
• The primary outcome was the proportion of patients who achieved CS- and exclusive enteral nutrition (EEN)-free clinical remission (defined by weighted Pediatric Crohn’s Disease Activity Index [wPCDAI] <12.5) or clinical remission by Physician Global Assessment (PGA) at week 12 without the need for any IV, oral, or topical CS at the time of assessment or ≥25% of the daily energy intake from a partial exclusive nutrition formula.
• Secondary outcomes were CS- and EEN-free sustained remission at week 52; CS-, EEN-, and surgery-free remission at week 52; rate of response (defined by wPCDAI >17.5) or remission (defined by wPCDAI <12.5) after STELARA dose optimization; rate of deep remission (defined as clinical remission, FCP <100 µg/g, and CRP <5 mg/L).

Results

Baseline Characteristics

• A total of 101 patients with a mean age of 15.4 years (IQR, 12.7-17.2) were included.
• The median wPCDAI at treatment initiation was 38.7 (IQR, 25-57.5).
• Baseline patient characteristics are presented in Table: Select Baseline Demographics and Clinical Characteristics.

• The recommended induction and maintenance dosing for STELARA in adult patients (~6 mg/kg) was used in 87% (88 patients) and 79% of patients, respectively.
• The intention-to-treat CS- and EEN-free response rate at week 12 was 72.9% (95% confidence interval [CI], 61.9-81.7). Among these patients (n=74), remission was observed in 30 patients (40.5%; 95% CI, 30.1-51.9) and complete deep remission was observed in 8 patients (10.8%; 95% CI, 5.6-19.9).
• At week 52, 30 of 74 patients were in CS- and EEN-free clinical remission (40.5%; 95% CI, 30.1-51.9), and 16 of 74 patients were in sustained CS-, EEN-, and surgery-free remission (21.6%; 95% CI, 13.7-32.2).
• The remission rate was similar between patients on combination therapy (CSs and/or thiopurines) and those on monotherapy at weeks 12 (27.6% vs 47.2%; P=0.086) and 52 (58.8% vs 48.4%; P=0.349).
• Data on predictive factors associated with STELARA-induced clinical remission at week 12 are presented in Table: Multivariate Analyses of STELARA Response.

• At 52 weeks of follow-up, 51 of 74 patients (68.9%) remained on STELARA treatment (median duration of 10.6 months [IQR, 6.0-12.0]).
• A total of 23 patients (22.7%) were discontinued from the STELARA treatment due to lack of response, n=21; lingual vasculitis as a suspected AE of STELARA, n=1; and death nonrelated to STELARA therapy, n=1.
• STELARA treatment was ceased in 4 patients (5.4%) and 23 patients (31.1%) within 12 weeks and 1 year, respectively.
• Six patients (6%) required surgical treatment during the 12 months of treatment (intestinal resection, n=4; endoscopic dilatation, n=1; and surgical treatment for perianal disease, n=1) at a median time of 6.9 months (IQR, 3.5-9.6). All the patients continued on STELARA treatment after surgery.
• At 12 months of follow-up, 7 mild to moderate AEs (probably drug related) were reported: 3 cases of infections (herpes zoster, axillary abscess, and appendicitis), 2 cases of abnormal blood monitoring tests (1 case of hepatic enzyme elevation and 1 case of lymphopenia not requiring discontinuation), 1 case of eczema, and 1 case of lingual vasculitis. No malignancies or serious drug-related AEs were reported.

Koudsi et al (2023)¹² conducted a retrospective, multicenter study assessing the effectiveness and safety of STELARA in pediatric patients with CD at pediatric IBD centers in France affiliated with Groupe d’Etude Thérapeutique des Affections Inflammatoires du tube Digestif (GETAID).

Study Design/Methods

• The study included patients aged ≤18 years who had received STELARA.
• All patients received the initial STELARA IV dose (6 mg/kg), followed by a 90 mg SC dose after 8 weeks.
• Disease activity was assessed using PCDAI, categorized as remission (<10), mild (<30), moderate (≥30-40), and severe (≥40) disease. A clinically significant response to therapy was defined as a score change of ≥12.5 points.

Results

Baseline Characteristics

• A total of 53 children with IBD were included, of whom 48 (90.6%) patients had CD.
• The median follow-up duration was 10.5 months (range, 1-51).
• Among the 48 patients with CD, 30 (65.2%) had ileocolonic disease, 11 (23.9%) had colonic disease, and 5 (10.9%) had an isolated ileal inflammation.
• Mean PCDAI at baseline was 28.7 in 35 patients, <15 in 9 patients, and >45 in 6 patients.

Effectiveness

• At 3 months postinduction, compared to baseline among CD and ulcerative colitis (UC) patients:
  • Mean weight increased significantly from 45 kg (21.5-70.5) to 49 kg (23.3-73), P<0.001.
  • Body mass index (BMI) increased from 19 kg/m² (13-32.6) to 20 kg/m² (15.7-33.6), P<0.001.
  • CRP levels significantly decreased at 3 months (P<0.05) and at the last follow-up (P<0.001).
  • Blood albumin levels significantly increased at 3 months (P<0.005) and at the last follow-up (P<0.001).
  • The erythrocyte sedimentation rate was significantly lower (P<0.001) and hematocrit rate was significantly higher (P<0.005) at the last follow-up.
• PCDAI score significantly decreased at 3 months (P<0.05) with 17 (48%) patients achieving a PCDAI <15, increasing to 20 (57%) patients at the last follow-up.
• Among CD and UC patients at baseline, 9 (16%) patients were steroid-free, increasing to 31 (58%) at 3 months and 33 (62%) at the last follow-up.
• Among the 20 (42%) patients with perineal CD at baseline, 9 had active perineal lesions at STELARA induction, with 1 patient developing perineal inflammation during treatment. Improvement in lesions was observed in 5 (50%) patients with STELARA treatment.
• Among 20 patients with perianal and nonperianal CD, the mean PCDAI at induction was 25.6 (range, 5-55) and 30.8 (range, 5-85), respectively.
  • After 3 months of treatment, these values were 22.5 (range, 2.5-75) and 14.3 (range, 0-37.5), respectively (P=0.17).
  • At the last follow-up, the values were 19 (range, 0-60) and 8.5 (range, 0-35), respectively (P=0.04).

Safety

• At 13-month follow-up among CD and UC patients, 9 patients experienced AEs, with fatigue (n=3 [3%]) and headache (n=3 [3%]) being more frequent. Treatment was discontinued in 1 patient due to recurrent respiratory tract infections.

Registry-Based Studies

REALITI

Adler et al (2024),¹³ Shehzad et al (2024),¹⁴ and Steiner et al (2024)¹⁵ reported results from a real-world evidence study (REALITI) that evaluated the effectiveness and safety of STELARA in pediatric patients with CD using data from ImproveCareNow (ICN) registry.

Study Design/Methods

• The study included pediatric patients, aged 2 to <18 years, with reference data from young adults (aged 18-25 years) with CD.
• Three readers independently scored reports blinded to clinical information and each other's scores.¹³
• Demographic and clinical data, recorded prospectively in ICN, were summarized, and compared between patients with and without endoscopy.¹³
• Clinical remission was defined as sPCDAI ≤10 and endoscopic remission as Simplified Endoscopic Mucosal Assessment of Crohn’s Disease (SEMA-CD) ≤1.

Results

Baseline Characteristics

• A total of 479 patients with CD treated with STELARA were included (n=348, pediatric patients; n=131, young adults).

Assessment of Mucosal Inflammation

• A total of 114 pediatric patients with moderate to severe CD (sPCDAI ≥30) were analyzed at week 52 (n=77, with endoscopic assessment; n=37, without endoscopic assessment).¹³
• At baseline, the median age for all patients was 16.0 years (IQR, 15-16).¹³
• Patients with endoscopic assessments vs patients without were predominantly female (56% vs 41%) and had more ileocolonic disease activity (73% vs 54%), perianal disease (39% vs 35%), and stricturing phenotype (B2; 10% vs 5%), respectively.¹³
• At baseline, the median sPCDAI was 40 (IQR, 35-50) in all 114 patients and the median SEMA-CD was 8.5 (IQR, 5-13) in 38 patients.¹³
• A total of 77 patients had ≥1 report during the study and 20 patients had a report at week 52.¹³
• At week 52, endoscopic remission was achieved by 4/20 (20%) patients (95% CI, 8.1-41.6) with lower remission rates were observed in patients with baseline endoscopic assessments (n=17/77 [22.1%]; 95% CI, 14.3-32.5) compared to those without (n=10/37 [27.0%]; 95% CI, 15.4-43.0).¹³

Pediatric Patients vs Young Adults

• An analysis of pediatric patients (n=348) compared to young adults (n=131) was conducted.¹⁴
• At baseline, the majority of pediatric patients (98.9%) and young adults (95.4%) had received prior biologic therapy, with approximately half (47.1% and 50.4%, respectively) also receiving CSs.¹⁴
• A total of 49.7% of pediatric patients, compared to 44.8% of young adults had moderate to severe CD (sPCDAI ≥30).¹⁴
• At week 52, clinical remission was achieved in 105/348 (30.2%) pediatric patients (95% CI, 25.6-35.2) vs 37/131 (28.2%) young adults (95% CI, 21.2-36.5).¹⁴
• At week 52, the rates of discontinuation were similar in pediatric patients (21.0%) and young adults (22.9%).¹⁴
• When comparing pediatric patients to young adults, IBD-related hospitalizations were reported in 31.9% vs 19.8% of patients, IBD-related surgery occurred in 16.1% vs 9.2% of patients, serious infections occurred in 7.5% vs 3.8% of patients, and the rates of opportunistic infections were 1.4% vs 0%.¹⁴
• There were no events of tuberculosis, malignancy, or anaphylaxis requiring STELARA discontinuation.¹⁴
• One death was reported among pediatric patients, which was considered as unrelated to IBD or STELARA treatment by investigators; no deaths were reported among young adults.¹⁴

Pediatric Patients vs Young Adults Weighing >40 kg

• An analysis was conducted on 114 pediatric patients who weighed >40 kg with a sPCDAI ≥30, compared to 51 young adults with moderately to severely active CD.¹⁵
• All pediatric patients were treatment refractory (99.1%, no response to prior biologics; <1%, biologic naïve).¹⁵
• At week 52, clinical remission was achieved in 26/114 (22.8%) pediatric patients (95% CI, 16.1-31.3) vs 11/51 (21.6%) young adults (95% CI, 12.5-34.6).¹⁵
• At week 52, the rates of discontinuation were similar in pediatric patients (25.4%) and young adults (25.5%).¹⁵
• When comparing pediatric patients to young adults, IBD-related hospitalizations were reported in 36.0% vs 21.6% of patients, IBD-related surgery occurred in 14.0% vs 11.8% of patients, serious infections occurred in 9.6% vs 3.9% of patients, and the rates of opportunistic infections were 1.8% vs 0%.¹⁵
• There were no reports of tuberculosis, malignancy, or anaphylaxis requiring STELARA discontinuation. No deaths were reported in either group.¹⁵

DEVELOP

Koletzko et al (2024)¹⁶ assessed STELARA safety in pediatric patients with CD using data from DEVELOP, a multicenter, global, prospective, observational, longitudinal registry of long-term safety of infliximab (and other treatments) in pediatric patients with IBD.

Study Design/Methods

• The study included pediatric patients aged <18 years.
• Patients were categorized into 2 groups based on weight: <40 kg group and ≥40 kg group.

Results

Baseline Characteristics

• A total of 150 patients were included (n=31, <40 kg group; n=119, ≥40 kg group).
• Most of the treated patients were between 12 and 17 years old (92.0%). About 91% of patients initially received STELARA Q8W. At the last known dose, 64% received STELARA Q8W, and 22% received STELARA every 4 weeks.
• Overall, 65.3% of patients had prior IBD surgery, 98% received prior biologic therapy and 72% received ≥2 biologics prior to STELARA.

Safety

• During follow-up, 37 patients (24.7%) discontinued STELARA, with a mean (SD) time to discontinuation of 16.7 (17.00) months, while 84 patients (56.0%) were exposed to STELARA for ≥24 months.
• Rates (events per 100 patient-years) of AEs, SAEs, serious infections, CD-related hospitalizations, and IBD-related surgeries are shown in Table: Rates of AEs, SAEs, Serious Infections, CD-Related Hospitalizations, and IBD-Related Surgeries Among STELARA Treated Patients.
• One malignancy (malignant carcinoid tumor) was reported in an 18-year-old female in the ≥40 kg group, with a history of prior therapy with vedolizumab, methotrexate, and 6-mercaptopurine. There were no reports of deaths or opportunistic infections.

LITERATURE SEARCH

A literature search of MEDLINE^®, EMBASE^®, BIOSIS Previews^®, and DERWENT^® (and/or other resources, including internal/external databases) was conducted on 14 April 2026.

Summarized in this response are data from UNITI Jr, UNISTAR, prospective study, retrospective studies with a sample size of ≥50 patients and registries.