CLINICAL DATA

Retrospective Studies

van Lierop et al (2025)^2,8 conducted a multicenter, retrospective cohort study in adult patients with moderate to severe UC to evaluate the long-term effectiveness and safety of STELARA with DE vs without DE.

Study Design/Methods

• This study included adult patients diagnosed with UC who were initiated on STELARA at 3 tertiary inflammatory bowel disease (IBD) care centers and received at least 1 STELARA intravenous (IV) induction dose between January 1, 2016, and November 1, 2021.
• Patients received STELARA weight-based IV loading dose (~6mg/kg) according to the labeling, followed by an 8-week maintenance therapy of STELARA 90 mg SC.
• The primary endpoint was corticosteroid-free clinical remission (CFCR) at the end of follow-up.
  • CFCR was defined as a partial Mayo (pMayo) score ≤ 2 (based on stool frequency, rectal bleeding, and Physician Global Assessment [PGA] scores) without systemic corticosteroids.
• Secondary endpoints included clinical remission; biochemical remission (C-reactive protein [CRP] <8 mg/L and/or fecal calprotectin [FCP] <250 μg/g); combined clinical and biochemical remission; endoscopic remission (mayo endoscopic score [MES] ≤1, with MES ranging from 0 to 3); combined clinical, biochemical, and endoscopic remission; and subsequent STELARA DEs.
• DEs were defined chronologically: first DE refers to the initial attempt at optimizing therapy (either through interval shortening or IV reloading), and subsequent DEs (second and third) represent chronologically ordered additional attempts to optimize treatment.
• Primary nonresponders were defined as patients not achieving response within 8 to 16 weeks after STELARA initiation.
• Secondary loss of response (LOR) was defined as an initial clinical response to STELARA induction therapy with a subsequent LOR to STELARA maintenance therapy.

Results

• Of the 121 patients with moderate to severe UC who were initiated on STELARA, 81 patients underwent DE and 40 patients were in the non-DE group.
• The median follow-up was 130 weeks for the DE group (interquartile range [IQR], 83-156) and 135 weeks for the non-DE group (IQR, 19-177).
• The median treatment duration until the first DE was 30.7 weeks (IQR, 19.8-53.8).
• Ten patients (12.3%) underwent the first DE before 16 weeks of STELARA treatment, and 60 patients (74.0%) required the first DE before 1 year of STELARA treatment.
• The median time to DE was 0.83 years (IQR, 0.45-2.87). The primary reasons for the first DE included no clinical response (n=36; 44.4%); LOR (n=25; 30.2%); and partial response (n=21; 25.9%), such as elevated FCP or residual endoscopic inflammation.
• DE methods and patient distribution across DEs are presented in Table: Patient Distribution Across DEs.

• Overall, 22 patients (27.2%) in the DE group vs 23 patients (57.5%) in the non-DE group achieved and maintained CFCR throughout the duration of follow-up.
• Data on patients achieving remission through the end of the follow-up period are presented in Table: Proportion of Patients in Remission at 1 Year, 2 Years, and End of Follow-up in the DE and Non-DE Groups.

• A total of 44 patients in the DE group and 18 patients in the non-DE group discontinued STELARA treatment.
• Discontinuation reasons are presented in Table: STELARA Discontinuation Reasons.

• At the end of the data collection period, 37 patients (45.7%) in the DE group and 22 patients (55.0%) in the non-DE group continued STELARA.
• Of the 62 patients who discontinued STELARA, 48 patients were switched to other biologic therapies, 7 patients (12.3%) underwent surgery, 2 patients (3.5%) remained on 5-aminosalicylic acid and low-dose prednisone, and 2 patients (3.5%) were switched to no maintenance medications (endpoints were missing for 5 patients).
• Of 19 hospitalizations, 6 hospitalizations were reported in the DE group (5 after DE and 1 before DE) and 13 hospitalizations were reported in the non-DE group.
  • Reasons for hospitalization included UC flare (8/19; 42.1%), colorectal surgery (9/19; 47.4%), febrile neutropenia (1/19; 5.3%), and severe abdominal pain (1/19; 5.3%).
• Nine patients (3 in the DE group and 6 in the non-DE group) underwent UC-related surgery during the follow-up; of these, 4 patients underwent a total proctocolectomy and 5 patients underwent a sub total colectomy.
  • Reasons for undergoing a UC-related surgery were refractory disease activity despite STELARA treatment (5/9 [55.5%]), STELARA DE (2/9 [22.2%]), and a diagnosis of colorectal cancer (CRC; 2/9 [22.2%]).
• Other than 2 cases of CRC, no malignancies were reported.

Yarur et al (2025)³ conducted a multicenter, retrospective cohort study to evaluate the effectiveness of STELARA in adult patients with UC from the REal World Biologics/Small mOlecules OuTcomes in IBD (REBOOT-IBD) consortium across the United States.

Study Design/Methods

• This study included adult patients diagnosed with UC who were taking STELARA between August 2017 and March 2021 and had at least 1 subsequent follow-up visit.
• Patients received STELARA-weight based IV loading dose (~6mg/kg) according to the Food and Drug Administration (FDA) label.
• The study also included endpoints (clinical response and endoscopic remission) among patients who were dose escalated.

Results

• Among the 245 patients enrolled, 94 patients underwent DE to STELARA 90 mg every 4 weeks (q4w).
• The median time between STELARA induction and STELARA DE was 24 weeks (IQR, 14-41).
• Clinical response data were available for 73 patients; 21 patients (28.4%) did not respond, 36 patients (49.3%) experienced partial response, and 16 patients (21.6%) were able to achieve remission.
• Among 35 patients who underwent endoscopic assessment following DE, 8 patients (22.9%) achieved endoscopic remission (defined as Mayo endoscopic score of 0).

Chaparro et al (2024)⁴ conducted a multicenter, retrospective cohort study in patients with UC to evaluate the short- and long-term effectiveness and safety of STELARA.

Study Design/Methods

• Enrolled patients were those who received the first STELARA dose at least 16 weeks before inclusion and were followed up from the first STELARA dose to treatment discontinuation or the last visit.
• Effectiveness analysis included only patients with active disease (pMayo score >2) at STELARA initiation. Clinical effectiveness was based on the pMayo score.
• Patients who discontinued STELARA before their last visit were considered not in remission at subsequent time points.

Results

• In total, 620 patients were included; 78% of patients received STELARA 90 mg q8w.
• During the median follow-up period of 12 months, 155 patients (25%) discontinued STELARA.
  • STELARA discontinuation was due to primary nonresponse (39%) and LOR (35%).
• STELARA dose was escalated in 72% of patients with LOR. Among these, 80% of patients experienced an improved response and 67% of patients achieved remission.

Lim et al (2024)⁵ conducted a retrospective, observational study to evaluate the long-term effectiveness and persistence rate of STELARA DE in adult patients with Crohn’s disease (CD) or UC from a Southeast Asian IBD center.

Study Design/Methods

• Adult patients diagnosed with CD or UC who were initiated on STELARA between September 2017 and October 2022 were included.
• Outcomes reported among patients with dose escalation were response, CRP response, and treatment persistence.
• Steroid-free clinical remission was defined as the absence of oral or IV corticosteroid use.
• Persistence of STELARA treatment was defined as the time between a patient’s first STELARA prescription and STELARA discontinuation or the end of the study period (October 26, 2022).

Results

• Of the 63 patients enrolled, 42 underwent DE (CD=34, UC=8).
• Patients with UC underwent STELARA DE to either q4w (n=4) or every 6 weeks (q6w; n=4).
• The median duration of STELARA treatment was 108 weeks (IQR, 44.7-185.9), and the median time to STELARA DE was 31.1 weeks (IQR, 17.8-65.7).
• This study did not report data specific to patients with UC who required DE.
• The 42 patients underwent STELARA DE due to suboptimal clinical response (42.9%), failure to achieve biochemical remission (16.7%), failure to achieve endoscopic healing (7.1%), failure to achieve transmural healing (9.5%), and a combination of all of these indications (23.8%).
• Of the 24 patients who continued STELARA DE, 16 patients (CD, n=13; UC, n=3) achieved the primary endpoint.
• Among the 42 patients who underwent STELARA DE, 23/42 patients (54.8%) achieved steroid-free clinical remission, 19/29 patients (65.5%) achieved biochemical remission, 3/5 patients (60%) achieved endoscopic healing, and 1/6 patients (16.7%) achieved transmural healing.
• Eight patients who did not achieve the study endpoints persisted with STELARA DE due to lack of alternative treatment options.
• There were 13 patients (CD, n=10; UC, n=3) in the DE group who received STELARA reinduction, but only 4 patients achieved the primary endpoint (P=0.51).
• Patients in the DE group did not exhibit a statistically significant response at 3 months after DE (n=36; P=0.209); however, a statistically significant CRP response was observed among these patients 6 months after DE (n=29; P=0.018).
• The overall drug persistence rate at 1 year was 75.7% (95% CI, 62.9-84.6) and at 2 years was 63.5% (95% CI, 49.9-74.3).
• The 1-year drug persistence among patients with and without DE was 73.5% (95% CI, 57.3-84.4) and 80.4% (95% CI, 55.9-92.2), respectively.
• Among patients who did not undergo DE, 7 patients (33.3%) discontinued STELARA treatment.
  • The reasons were self-discontinuation of treatment (n=5; 71.4%), financial reasons (n=1; 14.3%), and non-tuberculous mycobacterium infection (n=1; 14.3%).
• The median duration of STELARA discontinuation was 42.0 weeks (IQR, 30.0-86.0) in patients who did not undergo DE and 47.1 weeks (IQR, 29.8-87.8) in those who underwent DE.

Dalal et al (2023)⁶ conducted a retrospective cohort study to evaluate the clinical and endoscopic endpoints up to 2 years after STELARA DE to q4w or q6w in adult patients with UC or CD. Results specific to UC are described below.

Study Design/Methods

• Electronic records were manually reviewed.
• The primary endpoint was CFCR at 24±2 months (CFCR, defined as a HBI of <5 or Simple Clinical Colitis Activity Index [SCCAI] of ≤2 [or per provider global assessment if scores were not documented]) and no oral or IV corticosteroid use within 30 days preceding assessment.
• The secondary endpoint was CFCR at 12±2 months.
• Additionally, endoscopic response and remission were also assessed. Endoscopic response was defined as a reduction in the Mayo endoscopic subscore for UC by ≥1 point or per endoscopist assessment when compared with the most recent pre-dose escalation assessment. Endoscopic remission was defined as a Mayo endoscopic subscore of ≤1 or per endoscopist assessment.

Results

• Of the 40 patients with UC who underwent STELARA DE, 26 (65.0%) received q4w dosing and 14 (35.0%) received q6w dosing.
• The median time to DE from induction was 157 days (IQR, 88-279).
• At 24 months, among 15 patients with sufficient documentation, 6 (40.0%) achieved CFCR.
• At 12 months, among 40 patients with sufficient documentation, 21 (52.5%) achieved CFCR.
• Among 6 patients who were in CFCR at 12 months and had sufficient documentation at 24 months, 5 (83.3%) maintained CFCR at 24 months.
• Among 22 patients with available data, 13 (59.1%) achieved endoscopic response at a median of 314 days (IQR, 194-449) after STELARA DE.
  • Twelve (54.5%) patients achieved endoscopic remission.
• During follow-up, bowel resection surgery occurred in 1 (2.5%) patient.
• Twelve (30.0%) patients discontinued STELARA, and the most common reason was LOR (100%). AEs did not lead to treatment discontinuation in any patient.

Dalal et al (2022)⁷ conducted a multicenter, retrospective cohort study in adult patients with UC to determine predictors and endpoints of dose optimization with STELARA in UC over a 4-year period.

Study Design/Methods

• Adults with UC initiating STELARA at 2 centers in the United States were included.
• Data was analyzed by reviewing electronic health records and disease activity was assessed using the pMayo score or the SCCAI.
  • The pMayo score combines point totals for stool frequency (0-3 points), rectal bleeding (0-3 points), and PGA (0-3 points).
  • SCCAI combines point totals for daytime bowel frequency (0-3 points), nighttime bowel frequency (0-2 points), urgency of defecation (0-3 points), blood in stool (0-3 points), general well-being (0-4 points), and extracolonic manifestations (1 point per manifestation).
• The primary endpoint was CFCR defined as an SCCAI/Mayo score <3 points (remission) and no oral/IV corticosteroid use for ≥4 weeks at evaluation 12-16 weeks after DE.
• Secondary endpoints included clinical response (defined as a reduction in SCCAI/Mayo score by ≥3 points from baseline) at 12-16 weeks and time-to-dose escalation.
• DE was defined as reducing the dosing interval to q4w or q6w.

Results

• Of the 108 patients with UC initiating STELARA, 91.7% and 39.8% had prior anti-tumor necrosis factor (anti-TNF) and >2 prior biologic exposures, respectively. Additionally, 57.4% of patients were taking oral corticosteroids.
  • Among these patients, 39.6% (40/101 with SCCAI/Mayo data) achieved remission 12-16 weeks after induction.
• Dose escalation was required in 42.6% (46/108) of patients after a median of 95 days (IQR, 65-208 days) most commonly due to a lack of/minimal response to induction (22/46) or an LOR (20/46):
  • Of these patients, 33 and 13 patients required DE to q4w and q6w, respectively.
• For patients with SCCAI/Mayo data, 55% (22/40) and 67.5% (27/40) of patients achieved CFCR and clinical response, respectively, at 12-16 weeks after DE.
  • Drug discontinuation/colectomy occurred in 30% (12/40) of patients within 16 weeks after DE.
    • Of these 12 patients, no/minimal response to induction and an LOR occurred in 10 and 2 patients, respectively.
• Improvement in endoscopic inflammation and FCP was reported in 56.3% (9/16 with pre-dose escalation/post-dose escalation data) of patients.
• Hospitalization related to IBD and AEs (urinary tract infection and Clostridium difficile infection) occurred in 10% (4/40) and 5% (2/40) of patients, respectively, after a median follow-up of 230 days after intensification.
• A multivariable analysis showed that bowel frequency (hazard ratio [HR], 1.1; 95% CI, 1.02-1.2) and >2 previous biologic exposures (HR, 2.5; 95% CI, 1.1-5.8) were associated with time-to-dose escalation, whereas no/minimal response to induction was inversely associated with remission after dose escalation (OR, 0.2; 95% CI, 0.04-0.7).

Literature Search

A literature search of MEDLINE^®, EMBASE^®, BIOSIS Previews^®, and DERWENT^® (and/or other resources, including internal/external databases) was conducted on 25 March 2025. Summarized in this response are relevant data from retrospective studies.