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STELARA® (ustekinumab)

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STELARA - Treatment of Adult Patients with Ulcerative Colitis - Real-World Evidence

Last Updated: 12/12/2024

SUMMARY

Summarized in this response are data focused on the clinical efficacy and safety from observational and retrospective studies and registries.¹^-²⁰

Real-world data in adult patients with uLCERATIVE cOLITIS

Real-world evidence in adults with ulcerative colitis treated with STELARA is described in the Table: Summary of Real-World Studies in Adult Patients with UC.

Summary of Real-World Studies in Adult Patients with UC

Study Description and Select Baseline Characteristics	Results
Observational Study
Iborra et al (2024)¹ evaluated the efficacy and safety of STELARA in Spanish patients with refractory UC through an observational, multicenter, open-label, cohort study. Patients who received ≥1 IV infusion (6 mg/kg) were included. Of the 108 patients included in the study, 2 received only the first IV dose. The median (IQR) age at baseline was 44.2 (36.4-56.6) years, and the median (IQR) disease duration was 10.1 (6.01-14.9) years. At baseline, the median (IQR) PMS score was 5.00 (3.00-7.00). The median (IQR) FCP and CRP levels were 1260 (461-2560) µg/g and 4.25 (1.03-11.4) mg/L, respectively.	Efficacy
	Rates of clinical remission (PMS ≤2 with a combined rectal bleeding and stool frequency subscore of ≤1) at 8, 16, 24, and 52 weeks were 59%, 56.5%, 57%, and 69%, respectively, and at 18 and 24 months, clinical remission was maintained in 65% (28/43) and 80% (20/25), respectively. FCP level decreased to normal (<250 µg/g) at 8, 16, 24, and 52 weeks in 40%, 38%, 41%, and 51% of patients, respectively, and at 18 and 24 months in 61% and 58% of patients, respectively. CRP level decreased to normal (<5 mg/L) at 8, 16, 24, and 52 weeks in 77%, 82%, 80%, and 76.5% of patients, respectively, and at 18 and 24 months in 72.5% and 70% of patients, respectively. Among patients (n=17) with available endoscopy data before and after treatment with STELARA, 53% (n=9) reported endoscopic improvement (Mayo endoscopic subscore of 0 or 1) after a median (IQR) treatment time of 53 (31-61) weeks.
	Safety
	AEs were reported in 4.6% (n=5) of patients. Hospitalizations occurred in 18.5% (n=20) of patients. Of these patients, 8 were hospitalized due to a severe flare at the beginning of treatment. Colectomy was performed in 9.3% (n=10) of patients. Up to the last follow-up, 5 AEs were reported in 5 patients, which included asthenia, arthralgia, C. diff infection, relapsed uveitis and venous thromboembolism. Patients who experienced relapsed uveitis and venous thromboembolism discontinued STLEARA. In total, 21% (n=23) of patients discontinued STELARA.
Retrospective Studies
Chaparro et al (2024)² evaluated the durability, efficacy, and safety of STELARA in patients with UC through a retrospective, multicenter study (ULISES). Patient included were those had active disease (PMS >2) and received the first dose of STELARA ≥16 weeks prior. Overall, 620 patients were included in the study.	Efficacy
	Steroid-free remission was reported in 20% (N=561), 34% (N=561), and 40% (N=561) of patients at weeks 4, 8, and 16, respectively. Steroid-free remission was reported in 50% (N=402), 49% (N=169), and 49% (N=78) of patients at months 12, 24, and 36, respectively.
	Safety
	AEs were reported in 28% (n=176) of patients (infections were the most common AE, [11%]). The main reasons for discontinuation were primary nonresponse (39%) and loss of response (35%).
Yarur et al (2024)³ evaluated the efficacy and safety of STELARA in patients with UC through a retrospective, multicenter study as part of the REBOOT-IBD (REal World Biologics/Small mOlecules OuTcomes in IBD). The primary outcome was the cumulative rate of steroid-free clinical and biochemical remission (PMS <2 while off steroids, with a normal CRP [<0.5 mg/dL to <1 mg/dL] and/or FCP [<150 ug/g]). Secondary outcomes were endoscopic remission (Mayo endoscopic score=0) and clinical remission (PMS <2 independent of biomarkers or steroid use).	Efficacy
	Throughout follow-up, steroid-free clinical and biochemical remission was reported in 37.1% (n=91) of patients. Endoscopic remission was reported in 28.7% (39/136) of patients who underwent a follow-up endoscopic assessment. At 12 months, the rates of steroid-free clinical and biochemical remission, clinical remission, and endoscopic remission were 23.8%, 54.3%, and 31.0%, respectively. Colectomy was performed in 4.9% (n=12) of patients at the end of follow-up.
	Safety
	AEs attributed to STELARA were reported in 5.7% (n=14) of patients and included headaches (n=3), infection (n=3), arthralgias (n=6), and skin rash (n=2). One patient who experienced arthralgia was required to discontinue STELARA. No SAEs or thrombotic or cardiovascular events were reported.
Ando et al (2024)⁴ evaluated the efficacy and safety of STELARA among patients with UC through a multicenter, observational, cohort study in Japan. Patients who received STELARA for induction of remission were included. Among the 125 patients included, the mean duration of disease was 75 months.	Efficacy
	Clinical response rates (clinical remission plus clinical improvement) in the overall population at weeks 8, 16, 32, and 56 were 72.8% (91/125), 71.1% (86/121), 71.7% (71/99), and 73.2% (60/82), respectively. Clinical remission rates (partial Mayo score of ≤2 with a stool score of 0 or 1 and a rectal bleeding score of 0) in the overall population at weeks 8, 16, 32, and 56 were 56% (70/125), 53.7% (65/121), 60.6% (60/99), and 63.4% (52/82), respectively.
	Safety
	AEs were reported in 6.4% (n=8) of patients. AEs included 1 case each of pulmonary embolism, arthritis, paranasal sinusitis and COVID-19, fatigue and headache, herpes zoster, infectious colitis, CMV colitis, and pneumocystis pneumonia.
Parra et al (2024)⁵ evaluated the efficacy and safety of STELARA in Brazilian patients with moderate to severe UC through an observational, retrospective, multicenter study. Patients included were those with a total Mayo score of 6-12 and endoscopic subscore of 2 or 3. The coprimary endpoints included clinical remission (defined as a total Mayo score of ≤2 at 1 year with a combined rectal bleeding and stool frequency subscore of ≤1) and endoscopic remission (defined as an endoscopic Mayo subscore of 0) within 1 year from baseline. Overall, 50 patients with moderate to severe UC were included in the study. The mean (IQR) age was 42.8 (13-72) years, and the mean (IQR) duration of disease was 9.2 (1-27) years.	Efficacy
	At 1 year, clinical and endoscopic remission were achieved in 50% and 36% of patients, respectively. At weeks 12-16, clinical response, clinical remission, steroid-free clinical remission, and biochemical response were achieved in 56%, 18%, 16.3% (7/43), and 63.8% (30/47) of patients, respectively. At 1 year, endoscopic response, steroid-free clinical remission, and biochemical remission were achieved in 68%, 46.5% (20/43), and 53.2% (25/47) of patients, respectively.
	Safety
	A total of 26 AEs were reported, of which 15 were SAEs. During follow-up, 20% (n=10) of patients were hospitalized. Hospitalization due to disease worsening occurred in 18% (n=9) of patients (3 of whom required a colectomy). One patient was diagnosed with prostate cancer at week 12 of treatment with STELARA. One patient developed a cutaneous rash 24 hours after the first SC dose of STELARA, requiring hospitalization and discontinuation of STELARA. One patient experienced a severe headache requiring hospitalization after STELARA induction and during hospitalization, was diagnosed with cavernous sinus thrombosis. One patient had pulmonary embolism likely related to UC activity. No deaths were reported.
Sabhan et al (2024)⁶ evaluated the efficacy of STELARA in Swedish patients with UC using data from 4 hospitals through a long-term, retrospective, multicenter, cohort study (STOCUSTE). Primary outcomes were clinical steroid-free response and remission at 3, 6, 12, 24, and 36 months. Among the 96 patients included in the study, 92 (96%) had active disease and 4 were in remission at baseline.	Efficacy
	Remission was reported in 9/71 (74%), 25/42 (44%), and 28/37 patients at 3, 6, and 12 months, respectively. The steroid-free remission rates were 9.4% (N=35) and 29.2% (N=9) at 3 and 12 months, respectively.
	Safety
	Causes for discontinuation of treatment included primary non-response (20%), AEs or intolerance (8%), loss of response (6%), need for surgery (2%), and loss of follow-up (2%). No deaths or malignancies were reported.
Burbage et al (2024)⁷ evaluated the efficacy of STELARA in patients with UC through a retrospective, cross-sectional survey in the US (Adelphi Real World UC Disease Specific Programme™). Patients who received ≥1 STELARA dose for >1 day at the time of the visit were included. Remission was defined using the Mayo score and assessed in AT-naïve (STELARA as first-line AT) and AT-experienced (STELARA as second- or later-line AT) patients. Among the 160 patients included in the study from 50 physicians, the mean (SD) age was 39.7 (13.0) years and the median (IQR) duration of disease was 2.2 (0.5-4.5) years. Overall, 68.1% of patients were AT-naïve; among AT-experienced patients, 18.1% and 11.3% received STELARA as second- and third-line AT, respectively. The median (IQR) treatment duration for STELARA was 8.7 (4.1-18.6) months, and 83.9% of patients received STELARA for ≥3 months.	Efficacy
	At the time of the visit, remission was achieved in 53.6% and 58.3% of AT-naïve and AT-experienced patients, respectively.
	Safety
	Disease severity improved at the time of the visit compared with the severity prior to initiating STELARA (34.4% vs 90.9%, respectively; P<0.0001). At the time of the study visit, the proportions of patients with the following symptoms (as reported by the treating physician) were significantly lower vs before STELARA initiation: Abdominal pain, 30.2% vs 72.1%; P<0.0001 Bowel urgency, 22% vs 55.1%; P<0.0001 Bloody diarrhea, 17.6% vs 54.4%; P=0.0001 Fatigue, 32.7% vs 50.3%; P=0.0001 Nonbloody diarrhea, 21.4% vs 40.8%; P=0.0001 Nighttime bowel urgency, 9.4% vs 32%; P=0.0001
Tursi et al (2024)⁸ evaluated the efficacy and safety of STELARA in patients with treatment-refractory UC through a multicenter, retrospective, observational cohort study. Patients received a STELARA weight-based IV induction dose of ~6 mg/kg, followed by an SC maintenance dose of 90 mg q8w. The primary outcome was clinical remission (defined as a PMS of ≤1) at 8 and 24 weeks. Secondary outcomes included the following: Clinical response (defined as a reduction of ≥2 points in the PMS) at 8 and 24 weeks Steroid-free clinical remission (defined as a PMS of ≤1 plus absence of steroid treatment) at 8 and 24 weeks Mucosal healing (defined as a Mayo endoscopic subscore of ≤1) at 24 weeks Endoscopic response (defined as a change of ≥1 point in the MES at Week 24 compared with Week 0) at 24 weeks Overall, 256 patients who failed standard or biologic therapies or tofacitinib were included in the study. At baseline, the median (IQR) PMS, CRP level, and FCP level were 5.00 (4.00-6.00), 7 (3-19) mg/dL, and 663 (386-1425) μg/g, respectively.	Efficacy
	At 8 weeks, clinical remission, clinical response, and steroid-free clinical remission were achieved in 18.7% (44/235), 53.2% (125/235), and 18.3% (43/235) of patients, respectively. At 24 weeks, clinical remission, clinical response, and steroid-free clinical remission were achieved in 27.6% (42/152), 61.8% (94/152), and 26.3% (40/152) of patients, respectively, At 8 weeks, a statistically significant reduction from baseline was observed in CRP and FCP levels (P<0.0001 for both). At 24 weeks, mucosal healing and endoscopic response were achieved in 52% (26/50) and 69.38% (34/49) of patients, respectively.
	Safety
	AEs were reported in 2.34% (n=6) of patients. Four patients underwent colectomy (1 at 12 weeks, 2 at 24 weeks, and 1 at 48 weeks). One death due to severe COVID-19 pneumonia was reported in an 84-year-old patient with comorbid hypertension. One patient had CMV infection at the 8-week follow-up and another patient at 24 weeks. During the same timeframe, 1 case of itching post-STELARA administration, 2 cases of COVID-19 infection, and 1 case of cystitis were reported.
Komeda et al (2023)⁹ evaluated the short- and long-term efficacy and safety of STELARA in patients with steroid-resistant/dependent UC through a retrospective, observational study. Patients received a STELARA weight-based IV induction dose of ~6 mg/kg at baseline, followed by 90 mg SC at Week 8 and then every 8-12 weeks, per clinical evaluation. The primary outcomes were the short-term (at 8 weeks) and long-term (at 44 and 152 weeks) efficacies of STELARA. Clinical response was defined as a decrease in Mayo score of ≥30% from baseline and a score decrease of ≥3 points plus a Mayo rectal bleeding subscore of 1. Clinical remission was defined as a Mayo score of ≤2, with no subscores >1. Among the 30 patients included in the study, the median (IQR) age was 41.2 (26-56.5) years and the median (IQR) duration of disease was 8.7 (4-12.5) years.	Efficacy
	At 8 weeks, clinical response and remission were achieved in 73% and 70% of patients, respectively. At 44 and 152 weeks, respectively, clinical remission was achieved in 67% and 63% of patients and steroid-free remission was achieved in 63% of patients at each timepoint. At a mean follow-up of 86 weeks, the STELARA persistence rate was 67%. Clinical response was achieved in 84.6% and 58% of patients who failed up to 1 biologic (including those who were naive) and of patients who failed 2 or more biologics, respectively. A multivariate analysis demonstrated that PMS was the only parameter significantly associated with STELARA discontinuation (OR, 0.411; 95% CI, 0.197-0.858; P=0.018).
	Safety
	No SAEs or nonserious side effects were reported.
Holvoet et al (2023)¹⁰ evaluated the efficacy and safety of STELARA in patients with UC through a multicentric, retrospective, observational study in Belgium (SULTAN). The primary endpoint was steroid-free remission at Week 16 (defined as a PMS of ≤2 with no subscore of >1). The secondary endpoints included clinical response (defined as a reduction in the PMS of ≥3 points and ≥3 points plus a reduction in rectal bleeding score of ≥1 or an absolute rectal bleeding score of ≤1) and biochemical response (defined as a reduction of ≥50% in CRP and/or FCP levels). Overall, 107 patients with moderate to severe UC were included in the study. The median duration of disease was 10 (1-73) years.	Efficacy
	At Week 16, steroid-free clinical remission and clinical response were achieved in 33% (n=35) and 62.6% (n=67) of patients, respectively. A statistically significant reduction from baseline was observed at Week 16 for: PMS (6.0 vs 2.0; P<0.001), CRP levels (3.85 vs 3.15 mg/L; P<0.001), and FCP levels (1040 vs 300 mg/kg; P<0.001). Biochemical response was achieved in 38% (n=41) of patients.
	Safety
	In total, 0.9% (n=1) of patients reported severe infections. No other new safety signals were identified. At Week 52 (end of follow-up), 70% (n=75) of patients continued treatment with STELARA. Causes for the discontinuation of treatment included primary nonresponse (17%; n=18), loss of response (7.5%; n=8), and AEs (1.9%; n=2).
Uchida et al (2023)¹¹ evaluated the short- and long-term efficacy, safety, and risk factors for STELARA discontinuation in patients with UC through a retrospective, single-center, observational study. Patients received a STELARA weight-based IV induction dose of ~6 mg/kg, followed by 90 mg SC at Week 8. Subsequently, the maintenance dose could be given q8w or q12w based on the investigator's discretion. The primary outcome was clinical remission (defined as a Lichtiger score of ≤3) at Weeks 2-8 (induction phase) and Weeks 24-48 (maintenance phase). Among the 31 patients included in this study, the median (IQR) age was 32 (24-45.5) years and the median (IQR) time from diagnosis of UC was 65 (36-134.5) months. The median (IQR) Lichtiger score before STELARA treatment was 8.0 (7.0-9.5).	Efficacy
	At Weeks 2, 8, 24, and 48, clinical remission was achieved in 3, 9, 17, and 20 patients, respectively. Of the 11 patients who had no response at Week 2 or 8, 1 and 3 achieved clinical response and remission at Week 48, respectively. Endoscopic remission and response (per UCEIS) were achieved in 1 and 19 patients, respectively.
	Safety
	No AEs were reported during follow-up. After a median (IQR) treatment duration of 118 (28-325) days, 12 patients discontinued STELARA. Main reasons for discontinuation were primary nonresponse (9/12) and loss of response (3/12). A multivariate analysis demonstrated a significant association of high baseline CRP levels with STELARA discontinuation at 48 weeks (adjusted HR, 7.28; 95% CI, 2.2-24; P=0.00113).
Ando et al (2022)¹² evaluated the efficacy of STELARA and its predictive factors among patients with UC through a multicenter, observational study in Japan. Outcomes evaluated included clinical remission (PMS of ≤2), clinical improvement (change in the PMS of -3 points and decreasing to ≥-30%), and clinical response (remission+improvement). Among the 132 patients included in the study, the mean age was 41.3±16.2 years, and the mean duration of disease was 69.8±87.3 months.	Efficacy
	At Weeks 8, 16, 32, and 56, clinical remission was achieved in 62.5% (60/96), 50.7% (35/69), 61% (36/59), and 72.2% (26/36) of patients, respectively. At Weeks 8, 16, 32, and 56, clinical response was achieved in 82.3% (79/96), 69/6% (48/69), 72.8% (44/59), and 80.6% (29/36) of patients, respectively.
	Safety
	AEs were reported in 9.3% (n=7) of patients including 1 case each of pulmonary embolism, arthritis, paranasal sinusitis, COVID-19, fatigue and headache, herpes zoster, infectious colitis, and CMV colitis.
Honap et al (2022)¹³ evaluated the efficacy and safety of STELARA among patients with UC through a multicenter, observational study in the UK. Patients received STELARA at a dose of ∼6 mg/kg IV and then 90 mg SC at Week 8, followed by q8w-q12w per clinical assessment. The primary endpoints included corticosteroid-free remission (remission without steroid use at a timepoint, irrespective of steroid use at baseline) at Weeks 16 and 26 after STELARA induction. The secondary endpoints included clinical response (a reduction in SCCAI score by ≥3 points) and remission (an SCCAI score of ≤2) and endoscopic response (any improvement in MES or UCEIS score) and remission (MES of ≤1 or UCEIS score of ≤1) at Weeks 8, 16, and 26. Among the 110 patients included in the study, the median (range) age was 40 (18-89) years, and the median (IQR) duration of disease and follow-up was 7 (3-13) years and 28 (17-47) weeks, respectively.	Efficacy
	At Week 8, clinical response and remission were achieved in 58.2% (32/55) and 38.2% (21/55) of patients, respectively. At Weeks16, corticosteroid-free remission, clinical response and remission were achieved in 36.7% (18/49), 59.2% (29/49) and 46.9% (23/49) of patients, respectively. At Week 26, corticosteroid-free remission, clinical response, and remission were achieved in 33.3% (13/39), 59% (23/39), and 43.6% (17/39) of patients, respectively. Endoscopic response and remission were achieved in 43.8% (14/39) and 28.1% (9/39) of patients, respectively.
	Safety
	AEs were reported in 18% (n=20) of patients which included arthralgia (n=3) and worsening diarrhea (n=7). Three AEs led to treatment discontinuation: Nonanaphylactic infusion reaction Wide-spread urticarial rash 24 hours after infusion Marked inflammatory demyelinating polyneuropathy in a 47-year-old female patient after 5 days of STELARA induction therapy, per changes on electromyography. STELARA was discontinued and treatment with IV immunoglobulin led to complete symptom resolution and return of neurological function. SAEs were reported in 12% (n=13) of patients which included hospitalization for disease progression (7/9 required colectomy, and 2/9 required IV corticosteroids and STELARA dose escalation) and hospitalizations for nondrug-related or non-IBD-related reasons.
Chiappetta et al (2021)¹⁴ evaluated the efficacy and safety of STELARA among patients with moderate to severe active UC in Italy. Patients received weight-based initial IV infusion (~6 mg/kg) at Week 0, then a 90 mg SC induction dose at Week 8, followed by SC maintenance doses of 90 mg q8w or q12w (per treating physician’s discretion). The primary outcome was steroid-free remission (PMS of <2 points without use of any steroids) at Weeks 24 and 52. The secondary outcomes included clinical response (a reduction from baseline in PMS of ≥3 points), reduction in the concomitant use of steroids, reduction in the number of patients with elevated CRP levels at baseline. Among the 68 patients included in the study, the median (range) age was 42 (16-72) years, and the median (range) duration of disease was 8 (1-34) years.	Efficacy
	Week 24 Steroid-free remission and clinical response were achieved in 31% (19/61) and 84% (51/61) of patients, respectively. Twenty percent (n=12) of patients still received concomitant steroids and 30% (n=15) had persistently elevated CRP levels. Week 52 Thirty-eight patients had sufficient follow-up through Week 52. Steroid-free remission and clinical response were achieved in 50% (19/38) and 82% (31/38) of patients, respectively. Thirteen percent (n=5) of patients still received concomitant steroids and 19% (n=5) had elevated CRP levels. CRP reduction At all timepoints (Weeks 8, 24, and 52), the mean CRP levels decreased significantly from baseline (P<0.0001).
	Safety
	AE was reported in 1 patient who was diagnosed with pituitary adenoma after 12 months of treatment with STELARA.
Dalal et al (2021)¹⁵ evaluated baseline predicators of colectomy-free drug survival of STELARA among patients with UC through a multicenter retrospective cohort study. Clinical response (reduction from baseline in the SCCAI score of ≥2 points) at 12-16 weeks after induction and corticosteroid-free response were among the secondary outcomes assessed. Among the 108 patients included in the study, the median (IQR) age was 39 (30-56) years, median (IQR) disease duration was 9 (4-16) years, and the median (IQR) duration of follow-up was 290 (189-607) days after induction.	Efficacy
	At 12-16 weeks, clinical response and corticosteroid-free response were achieved in 65.3% (64/98) and 43.9% (43/98) of patients, respectively.
Ecker et al (2021)¹⁶ evaluated treatment response and characteristics of patients with UC receiving STELARA through a retrospective study. Among the 26 patients included in the study, the median age was 27 years, and the mean disease duration was 7.8 years. Disease assessment was measured using the Mayo subscore. Before treatment initiation with STELARA, the median endoscopic Mayo subscore was 3 points (n=20). A mean of 2 FCP levels prior to (median 7 weeks) and after (median 11 weeks) STELARA therapy was used to measure treatment response in 14 patients.	Efficacy
	After 11 weeks (IQR=7) of treatment initiation, FCP levels decreased by a mean of 58.4% (n=14). The clinical Mayo subscore decreased from 6 points at baseline to 3 points at the first patient contact in the outpatient clinic (n=17). The clinical Mayo subscore decreased by ≥3 points in 76.5% of patients.
	Safety
	There were no treatment discontinuations due to SAEs. Two patients switched therapy due to lack of improvement and 1 patient underwent colectomy due to colorectal cancer and discontinued STELARA. One patient had colonic reactivation of CMV but continued treatment.
Ochsenkühn et al (2020)¹⁷ evaluated the efficacy of STELARA as rescue treatment among patients with treatment-refractory or intolerant UC through a single-center, observational study in Germany. Remission was defined as a Lichtiger score (modified Truelove and Witts CAI) of ≤3 and partial response as a Lichtiger score of 4-10. Among the 19 patients included in the study, the median (range) age was 46 (25-81) years, and the median duration of disease was 5 years.	Efficacy
	Fourteen patients out of 19 continued therapy with STELARA at 12 months and underwent a total colonoscopy. The median CAI scores at baseline, 3, 6, 9, and 12 months were 8.5, 2.5, 2.5, 1.8, and 2.0, respectively. The median FCP levels at baseline, 3, 6, 9, and 12 months were 428 µg/g, 236 µg/g, 316 µg/g, 260 µg/g, and 244 µg/g, respectively. The median (range) Mayo endoscopy scores decreased from 2 (1-3) at the start of observation to 1 (1-3) at 12 months. At 12 months, 71% of patients (n=10) achieved a Mayo score of 1. Overall outcome At 1, 3, 6, 9, and 12 months, clinical remission was achieved in 37% (n=7), 58% (n=11), 58% (n=11), 53% (n=10), and 53% (n=10) of patients, respectively. Among patients who were in clinical remission at 1 year (n=10), the median FCP decreased from 516 µg/g to 186 µg/g. Of the 3 (16%) patients who were already in remission and intolerant to previous therapy, STELARA was able to maintain remission in 2 patients up to 12 months: Patient 1: CAI score decreased from 1 to 0. Patient 2: CAI score decreased from 3 to 1. Patient 3: stopped treatment. Patients with active disease Among patients with active disease at the start of observation (n=16), clinical remission (CAI score of <4) was achieved in 4 patients at 1 month and in 8 patients at 3, 6, 9, and 12 months, respectively.
Registries
Thunberg et al (2022)¹⁸ evaluated clinical outcomes among patients with UC without previous colectomy who received STELARA in a noninterventional cohort study using data from the SWIBREG registry. The outcomes assessed included: Clinical remission (a stool frequency subscore of ≤1 and a rectal bleeding subscore of 0 in the patient-reported Mayo score) Corticosteroid-free clinical remission among patients with concomitant corticosteroid use at baseline Biochemical remission (FCP level of <250 µg/g) CRP and FCP levels SHS (4 self-reported outcomes: symptom burden, functional status, disease-related worry, and general wellbeing) scores compared with those at baseline. Among the 133 patients included in the study, the median (IQR) age was 38 (28-48) years, and the median (IQR) duration of disease was 7 (3-12) years.	Efficacy
	Outcomes at Week 16 Clinical and biochemical remission were achieved in 17% (n=22) and 14% (n=19) of patients, respectively. Seventy-nine and 80 patients lacked data for clinical remission and biochemical remission, respectively. Among patients with concomitant corticosteroid use at baseline (n=23), 22% (n=5) were in corticosteroid-free clinical remission; clinical remission data for 10 of these patients was missing. Among patients with available data, the median (IQR) patient-reported Mayo scores decreased from 4 (3-5) at baseline to 3 (2-4; P=0.03; n=31). The median (IQR) FCP levels decreased from 875 (193-1561) µg/g at baseline to 239 (56-908) µg/g (P=0.04; n=28) at 16 weeks. The median (IQR) CRP levels did not demonstrate a statistically significant change from 2.8 (2.0-8.0) mg/L at baseline to 2.8 (1.0-4.5) mg/L (P=0.32; n=29). In the self-reported SHS, the “functional status” domain demonstrated a statistically significant improvement (P=0.02) from baseline. Outcomes at the last follow-up Information on clinical remission at the last follow-up was missing for 27/133 patients. Clinical and biochemical remission were achieved in 32% (n=42) and 23% (n=31) of patients, respectively. Data for biochemical remission in 39 patients were not available. Among patients with concomitant corticosteroid use at baseline (n=23), 48% (n=11) were in corticosteroid-free clinical remission. The patient-reported median (IQR) Mayo scores decreased from 4 (3-5) at baseline to 2 (2-4; P<0.01; n=47). The median (IQR) FCP levels decreased from 740 (278-1510) µg/g at baseline to 98 (32-663) µg/g (P<0.01; n=37). The median (IQR) CRP levels did not demonstrate a statistically significant change from 2.8 (1.0-6.1) mg/L at baseline to 2.8 (1.0-5.3) mg/L (n=43). In the self-reported SHS, all 4 domains demonstrated statistically significant improvements (n=46; P<0.01).
	Safety
	Patients discontinued STELARA treatment (data was not available in 6 patients) due to: Lack or loss of response (n=32) Intolerance (n=2) Other reasons (n=4)
Chaparro et al (2021)¹⁹ evaluated the efficacy and safety of STELARA in patients with UC through an observational multicenter study using data from the ENEIDA registry. A total of 95 patients were included in the study with a median (IQR) time of exposure to STELARA of 31 (18-59) weeks. Patients received an IV dose of STELARA (∼6 mg/kg) and then standard maintenance doses (q12w or q8w). Patients who discontinued STELARA due to lack of therapeutic effect, an AE, or worsening of UC prior to their last visit were considered as not having achieved the endpoint (remission or response) at subsequent timepoints and were considered failures.	Efficacy
	At Week 16, clinical remission and clinical response (including remission) were achieved in 35% (33/95) and 53% (50/95) of patients, respectively. At Week 24, clinical remission and corticosteroid-free clinical remission were achieved in 39% (32/84) and 30% (25/84) of patients, respectively. The corresponding values for Week 52 were 33% (18/54) and 32% (17/54), respectively.
	Safety
	Three AEs were reported which included: dry skin and itching (probably related to STELARA; did not lead to treatment discontinuation), pneumonia (probably not related to STELARA; did not lead to treatment discontinuation), and death of a 54-year-old male patient with extensive UC and no comorbidities who had been treated with STELARA (90 mg q6w) for 43 weeks and developed severe SARS-CoV-2 pneumonia.
Fumery et al (2021)²⁰ evaluated the efficacy and safety of STELARA maintenance therapy through a multicenter cohort study from GETAID. Patients received 6 mg/kg IV STELARA at Week 0 and then 90 mg SC q8w-q12w (per investigator’s discretion). The primary outcome was steroid-free clinical remission at 12 months. Clinical remission was defined as a PMS (including stool frequency, rectal bleeding, and PGA subscores) of ≤2, with a combined stool frequency and rectal bleeding subscore of ≤1. The secondary outcomes included Mayo clinic endoscopic subscore and UCEIS over the study period, colectomy, and occurrence of any AE or SAEs. Among the 103 patients included in the study, the median (IQR) age was 39.3 (29.1-52.3) years, and the median (IQR) duration of disease was 7.6 (3.6-12.9) years.	Efficacy
	The median (IQR) duration of follow-up was 12.0 (7.4-14.4) months. At Week 52, steroid-free clinical remission was achieved in 32% (n=33) of patients. Clinical remission was achieved in 34% (n=35) of patients. A rectal bleeding subscore of 0 and a stool frequency subscore of 0 was reported in 24.3% (n=25) of patients. A rectal bleeding subscore of 0 and a stool frequency subscore of 0 or 1 was reported in 33% (n=34) of patients. Among the 93 patients with a baseline assessment of endoscopic activity, 63.1% (n=65) of patients were also re-evaluated between Weeks 26 and 52. The mean±SD UCEIS score decreased from 5.0±1.1 at baseline to 3.6±1.1 between Weeks 26 and 52 (P<0.001). The mean Mayo endoscopic subscore decreased from 2.7±0.5 at baseline to 2.0±1.0 between Weeks 26 and 52. At Week 52, among patients with endoscopic evaluation (n=30), 16.6% (n=5) and 33% (n=10) had a UCEIS score of 0 or 1 and a Mayo endoscopic subscore of 0 or 1, respectively. After a median (IQR) duration of 6.7 (4.3-10.6) months after STELARA initiation, 9.7% (n=10) of patients needed a colectomy. After 3, 6, 9, and 12 months, the cumulative probabilities of colectomy were 1%, 5.2%, 7.7%, and 9.1%, respectively.
	Safety
	Through the induction phase, there were 8 and 4 AEs and SAEs reported, respectively. Between Weeks 12 and 52, 16 AEs were reported in 14.8% (n=15) of patients, which included SAEs in 3.9% (n=4; exacerbation of UC leading to hospitalization, n=3; death of a 62-year-old male due to myocardial infarction 4 months after STELARA initiation). No cases of cancer were reported. AEs that led to STELARA discontinuation were reported in 3.9% (n=4) of patients.
Abbreviations: AE, adverse event; AT, advanced therapy; CAI, Clinical Activity Index; CI, confidence interval; CMV, cytomegalovirus; COVID-19, coronavirus disease 2019; CRP, C-reactive protein; ENEIDA, Estudio Nacional en Enfermedad Inflamatoria Intestinal Sobre Determinantes Genéticos y Ambientales; FCP, fecal calprotectin; GETAID, Groupe d’Etude Thérapeutique des Affections Inflammatoires du Tube Digestif; HR, hazard ratio; IBD, inflammatory bowel disease; IQR, interquartile range; IV, intravenous; MES, Mayo endoscopic score; OR, odds ratio; PGA, Physician’s Global Assessment; PMS, partial Mayo score; q4w, every 4 weeks; q6w, every 6 weeks; q8w, every 8 weeks; q12w, every 12 weeks; SAE, serious adverse event; SARS-CoV-2, severe acute respiratory syndrome coronavirus 2; SC, subcutaneously; SCCAI, Simple Clinical Colitis Activity Index; SD, standard deviation; SHS, Short Health Scale; SWIBREG, Swedish Inflammatory Bowel Disease Register; UC, ulcerative colitis; UCEIS, Ulcerative Colitis Endoscopic Index of Severity; UK, United Kingdom; US, United States.

Literature Search

A literature search of MEDLINE^®, EMBASE^®, BIOSIS Previews^®, and DERWENT^® (and/or other resources, including internal/external databases) was conducted on 23 October 2024.

Summarized in this response are data focused on clinical efficacy and safety from observational and retrospective studies and registries.

References

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