A Study to Evaluate Disease Control, Treatment Patterns, Burden of Disease and Quality of Life in Participants With Moderate to Severe Inflammatory Bowel Disease (IBD) (RISE AR)

Real-world Data of Moderate to Severe Inflammatory Bowel Disease in Argentina: A Non-interventional, Multicenter Study to Evaluate Disease Control, Treatment Patterns, Burden of Disease and Quality of Life

The purpose of this study is to evaluate the percentage of moderate to severe IBD participants with active disease at Day 1.

Study Overview

• Status: Completed
• Conditions: Crohn Disease; Colitis, Ulcerative; Inflammatory Bowel Disease

Detailed Description

This is a retrospective, cross-sectional, and non-interventional study of participants with IBD. The study will have a cross-sectional evaluation on Day 1 to provide the real-world data of disease activity, treatment patterns, burden of disease and quality of life in participants with moderate to severe CD or UC.

The study will involve an additional retrospective review of medical charts of participants of previous 3 years to describe the IBD treatments and use of other healthcare resources related with management of IBD.

The study will enroll approximately 246 participants. All participants will be enrolled in one observational cohort.

This multi-center trial will be conducted in Argentina. The overall time for data collection in the study will be approximately 3 years before the start of the study (Day 1).

• Study Type: Observational
• Enrollment (Actual): 246

Contacts and Locations

Study Locations

• Argentina
  • Cordoba, Argentina
    • Hospital Privado de Cordoba
  • Buenos Aires
    • Caba, Buenos Aires, Argentina
      • Centro de Educacion Medica e Investigaciones Clinicas Norberto Quirno (CEMIC)
    • Caba, Buenos Aires, Argentina
      • Hospital Britanico de Buenos Aires
    • Caba, Buenos Aires, Argentina
      • Hospital de Gastroenterología Dr. Carlos Bonorino Udaondo
    • Caba, Buenos Aires, Argentina
      • Sanatorio Mater Dei
    • La Plata, Buenos Aires, Argentina
      • Hospital San Martin
  • Santa Fe
    • Rosario, Santa Fe, Argentina
      • Hospital Provincial del Centenario

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Participants diagnosed with moderate to severe CD or UC will be observed.

Description

Inclusion Criteria:

1. Diagnosis of moderate to severe CD or UC for at least 6 months prior to Day 1 appointment based on clinical or endoscopic or image criteria.

Exclusion Criteria:

1. Indeterminate or not classified colitis.
2. Presenting mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

All Participants; Participants diagnosed with moderate to severe UC or CD from the 7 participating sites will be observed on Day 1 for cross-sectional evaluation of disease activity, treatment patterns, burden of disease and quality of life along with retrospective data collection for previous 3 years from the date of UC or CD diagnosis until Day 1 to assess the IBD treatments, medical history and comorbidities, treatment patterns and use of health resources.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Crohn's Disease: Percentage of Participants With Active CD at Day 1	Percentage of participants with active CD were observed, where active CD was defined as Harvey Bradshaw index (HBI) greater than or equal to (>=) 8 or Crohn's disease active index (CDAI) >= 220 points. CDAI assessed CD based on clinical signs and symptoms such as number of liquid stools, intensity of abdominal pain, general wellbeing, presence of comorbid conditions, use of antidiarrheal, physical examination and laboratory findings. Total score ranged from 0 to 600 points. Higher score indicated more severe disease. HBI consisted of 5 clinical parameters: general well-being, abdominal pain, number of liquid stools per day, abdominal mass, and complications. Total score was sum of individual parameters. Score ranges from a minimum score of 0 to no pre-specified maximum score as it depends on number of liquid stools, where higher scores indicated more severe disease.	Day 1
Ulcerative Colitis: Percentage of Participants With Active UC at Day 1	Percentage of participants with active disease UC disease were observed, where UC was defined as 9-point Partial Mayo Score (pMayo score) >=5. The Mayo score was composed of four categories (bleeding, stool frequency, physician assessment, and endoscopic appearance) rated from 0 to 3 that are summed to give a total score that ranges from 0 to 12. The pMayo score was previously compared with the full Mayo score and categorized participants as being in remission (score of 0 to 2), having mild disease (pMayo of 3 or 4) or moderate to severe disease (pMayo of >=5).	Day 1

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Moderate to Severe CD or UC Stratified by Steroid Behavior		Day 1
Number of Participants With Moderate to Severe CD or UC Stratified by Anthropometric Information	Anthropometric information included height, weight and body mass index (BMI). BMI ranges: underweight (BMI less than [<] 18.50 kilogram per square meter (kg/m^2), normal range (BMI between 18.50 and 24.99 kg/m^2), overweight (BMI between 25.00 and 29.99 kg/m^2), obese (BMI >=30.00 kg/m^2).	Day 1
Number of Participants With Moderate to Severe CD or UC Based on Medical History, Comorbidities or Extra Intestinal Manifestation (EIM)	Medical history was defined as an EIM or non-EIM according to the investigator's judgement.	Day 1
Crohn's Disease: Number of Participants With Moderate to Severe CD Stratified by Clinical Characterization	The Montreal classification index for CD was used to classify the extent of the disease activity. It consisted of two parameters: location and behavior of the disease activity. There were four different disease locations presented: Location 1 (L1) is ileum, Location 2 (L2) is colonic disease, Location 3 (L3) is ileocolon and Location 4 (L4) is isolated upper gastrointestinal (GI) tract disease. The first three categories (L1-L3) was combined with L4 where disease sites coexisted. There were 4 different categories for the behavior of the disease activity: Behavior 1 (B1) was non stenosing or non penetrating; Behavior 2 (B2) was stenosing; Behavior 3 (B3) was penetrating and p as perianal disease (p). The first 3 categories (B1 to B3) could be added with p to indicate coexisting perianal disease. Perianal disease (p) was defined as the presence of perianal abscesses or fistulae.	Day 1
Ulcerative Colitis: Number of Participants With Moderate to Severe CD Stratified by Clinical Characterization	Montreal classification index for UC was used to classify the extent and severity of the disease activity. There were three subgroups of UC defined by extent of inflammation: Extent 1 (E1) =Ulcerative distal UC proctitis and proctosigmoiditis, Extent 2 (E2) =Left-sided mucosa inflammation extending up to splenic flexure and Extent 3 (E3) =Pancolitis mucosa inflammation up to proximal transverse colon and beyond. Severity of UC as S0=Clinical remission (asymptomatic),S1 =Mild UC (passage of four or fewer stools/day [with or without blood],absence of any systemic illness, and normal inflammatory markers),S2 =Moderate UC (passage of more than four stools per day but with minimal signs of systemic toxicity),S3 =Severe UC (passage of at least 6 bloody stools daily, pulse rate of at least 90 beats per minute, temperature of at least 37.5 degree Celsius, hemoglobin of <10.5 gram per 100 milliliter (g/100mL), and erythrocyte sedimentation rate (ESR) of at least 30 millimeter per hour (mm/h).	Day 1
Number of Participants With Moderate to Severe CD or UC Who Used Various Types of Therapies for IBD in Previous 3 Years	Various types of therapies for IBD included salicylic derivatives, corticosteroids, immunosuppressors, biologic therapy, antibiotics, nutrition support and others. One participant could use more than one therapy.	Within previous 3 years including Day 1
Duration of IBD Treatment During Previous 3 Years	Time between the beginning of IBD treatment until the end of treatment. For participants with ongoing IBD treatment at Day 1, stop date was considered as Day 1 date visit. Various types of therapies for IBD included salicylic derivates, corticosteroids, immunosuppressors, biologic therapy, antibiotics, nutrition support and others. One participant could use more than one therapy.	Within previous 3 years including Day 1
Number of Participants With Moderate to Severe CD or UC Treated With Biologic Therapy During Previous 3 Years	Biologic therapies included infliximab, adalimumab, vedolizumab, certolizumab, golimumab, and ustekimumab. For participants with ongoing biologic therapy at Day 1, stop date was considered as Day 1 date visit.	Within previous 3 years including Day 1
Number of Participants With Moderate to Severe CD or UC Who Have Not Responded Previously to Biologic Therapies With Reasons		Within previous 3 years including Day 1
Number of Participants With Moderate to Severe CD or UC Who Had IBD Treatment Initiation on Day 1		Day 1
Number of Participants With Moderate to Severe and No or Mild Activity of CD or UC Stratified by Socio-demographic Variables	Participants were stratified based on gender, professional status, educational level and income.	Day 1
Quality of Life Assessed by European Quality of Life 5-dimension (EQ-5D) Health States Visual Analogue Scale (VAS) Score at Day 1	EQ-5D considered five attributes of quality of life evaluation, that is, mobility, self-care, usual activity, pain/discomfort, and anxiety/depression. Each dimension had five possible levels: 1 (no problems); 2 (slight problems); 3 (moderate problems); 4 (severe problems), and; 5 (extreme problems). EQ-5D also included an additional visual analogic scale (EQ-VAS), which ranged from 0 to 100, where 0 indicated worst imaginable health state and 100 was best imaginable health state.	Day 1
Quality of Life as Assessed by 36-item Short Form Health Survey (SF-36) Component Score at Day 1	The SF-36 evaluated 8 health dimensions: physical functioning, bodily pain, role physical (limitations due to physical problems), role emotional (limitations due to personal or emotional problems), mental health, social functioning, vitality, and general health perceptions. Based on these 4 scales (physical functioning, role-physical, bodily pain, general health), the physical component summary (PCS) score was generated which ranged between 0 and 100, with higher scores indicating a better quality of life. Based on these 4 scales (vitality, social functioning, role-emotional, and mental health), the mental component summary (MCS) score was generated which ranged between 0 and 100, with higher scores indicating a better quality of life.	Day 1
Inflammatory Bowel Disease Questionnaire (IBDQ) Total Score at Day 1	The IBDQ was a 32-item questionnaire that measured 4 dimensions: bowel function, emotional status, systemic symptoms, and social function. Within dimensions, each question presented seven possible answers/points. Each domain score was the sum of 8 responses each ranging from 1 to 7, where 1 indicated worst function and 7 the best. The sub-score ranged from 8 to 56 and thus the total score ranged from 32 to 224, where higher score indicating better quality of life.	Day 1
Inflammatory Bowel Disease Questionnaire (IBDQ) Domain Score at Day 1	IBDQ was a 32-item questionnaire that measured 4 dimensions: bowel systems, emotional health, systemic systems, and social function. Within dimensions, each question presented seven possible answers/points. Each domain score was the average of 8 responses each ranging from 1 to 7, where 1 indicated worst function and 7 the best function.	Day 1
Mean of Percentage of Total Work Productivity Impairment (TWPI) Due to CD or UC as Assessed by Work Productivity and Activity Impairment Questionnaire (WPAI)	WPAI assessed impact of IBD on work productivity and daily activities in past 7 days. WPAI questions were: if currently employed, 2) hours (hrs) missed due to disease, 3) hrs missed other reasons, 4) hrs actually worked, 5) degree disease affected productivity while working, 6) degree disease affected regular activities. WPAI generates 4 component scores: percentage (%) of work time missed (absenteeism), % of impairment while working (presenteeism), % of overall work impairment (absenteeism and presenteeism combined), and % of activity impairment. TWPI was calculated based on 3 items: (Q2) number of hrs missed from work due to health problems in the past 7 days; (Q4) number of actual work hrs in past 7 days; and (Q5) to what degree did disease impair productivity while working in past 7 days. Data was calculated by: Q2/(Q2+Q4)+([1-(Q2/[Q2+Q4])*(Q5/10])*100 (converted to %). Data is presented as impairment percentage, with higher numbers indicating greater impairment/less productivity.	Day 1
Mean of Percentage of Work Time Missed Due to CD or UC as Assessed by WPAI at Day 1	WPAI assessed impact of IBD on work productivity and daily activities in past 7 days. WPAI questions were: if currently employed, 2) hours missed due to disease, 3) hours missed other reasons, 4) hours actually worked, 5) degree disease affected productivity while working, 6) degree disease affected regular activities. WPAI generates 4 component scores: percentage of work time missed (absenteeism), percentage of impairment while working (presenteeism), percentage of overall work impairment (absenteeism and presenteeism combined), and percentage of activity impairment. Work time missed was calculated based on 2 items: (Q2) number of hours missed from work due to health problems in the past 7 days; and (Q4) number of actual work hours in past 7 days. Data was calculated by following formula:Q2/(Q2+Q4)*100 (converted to %), where higher numbers indicated greater impairment and less productivity.	Day 1
Mean of Percentage of Impairment While Working Due to CD or UC as Assessed by WPAI at Day 1	WPAI assessed impact of IBD on work productivity and daily activities in past 7 days. WPAI questions were: if currently employed, 2) hours missed due to disease, 3) hours missed other reasons, 4) hours actually worked, 5) degree disease affected productivity while working, 6) degree disease affected regular activities. WPAI generates 4 component scores: percentage of work time missed (absenteeism), percentage of impairment while working (presenteeism), percentage of overall work impairment (absenteeism and presenteeism combined), and percentage of activity impairment. Percentage of impairment while working was calculated based on 1 item: (Q5) to what degree did disease impair productivity while working in past 7 days. The item was measured on a scale from 0 (no effect) to 10 (completely prevented from doing regular activities/ working). Data was calculated by following formula: Q5/10*100 (converted to percent), where higher numbers indicated greater impairment and less productivity.	Day 1
Mean of Percentage of Total Activity Impairment Due to CD or UC as Assessed by WPAI at Day 1	WPAI assessed impact of IBD on work productivity and daily activities in past 7 days. WPAI questions were: if currently employed, 2) hours (hrs) missed due to disease, 3) hrs missed other reasons, 4) hrs actually worked, 5) degree disease affected productivity while working, 6) degree disease affected regular activities. WPAI generates 4 component scores: percentage (%) of work time missed (absenteeism), % of impairment while working (presenteeism), % of overall work impairment (absenteeism and presenteeism combined), and % of activity impairment. % of total activity impairment was calculated based on: (Q6) in past 7 days, how much did your health problems affect your ability to do your regular daily activities, other than work at a job. Item was measured on scale from 0 (no effect) to 10 (completely prevented from doing regular activities/ working). Data was calculated by: Q6/10*100 (converted to percent), where higher numbers indicated greater impairment and less productivity.	Day 1
Percentage of Participants Who Quit Job Due to IBD and Have Not Been Able to Return to Work		Day 1
Number of Participants With Moderate to Severe CD or UC Who Used Healthcare Resources Over the Previous 3 Years	Healthcare resources included drug therapies, imaging and laboratory testing, surgeries, hospitalizations and consultations.	Within previous 3 years including Day 1

Collaborators and Investigators

• Sponsor: Takeda

Publications and helpful links

General Publications

• P600 Real-world clinical characteristics and therapeutic strategies in patients with moderate-to-severe Inflammatory Bowel Disease in Argentina: Data from the RISE AR study. J S Lasa, A Sambuelli, I Zubiaurre, G J Correa, P Lubrano, D C Balderramo, O Ruffinengo, L Brion, D B Leonardi, J El-Hakeh, P N Guimaraens, P Olivera Sendra. Journal of Crohn's and Colitis, Volume 15, Issue Supplement_1, May 2021, Pages S546-S547, https://doi.org/10.1093/ecco-jcc/jjab076.721
• P426 Health-Related Quality of Life and Work Productivity in patients with moderate-to-severe Inflammatory Bowel Disease in Argentina: Data from the RISE AR study. G J Correa, M Yantorno, P Olivera Sendra, J S Lasa, P Lubrano, D C Balderramo, I Zubiaurre, O Ruffinengo, L Brion, D B Leonardi, J El-Hakeh, P N Guimaraens, A Sambuelli. Journal of Crohn's and Colitis, Volume 15, Issue Supplement_1, May 2021, Pages S430-S431, https://doi.org/10.1093/ecco-jcc/jjab076.550

Study record dates

Study Major Dates

• Study Start (ACTUAL): December 12, 2018
• Primary Completion (ACTUAL): May 31, 2019
• Study Completion (ACTUAL): May 31, 2019

Study Registration Dates

• First Submitted: March 27, 2018
• First Submitted That Met QC Criteria: April 3, 2018
• First Posted (ACTUAL): April 4, 2018

Study Record Updates

• Last Update Posted (ACTUAL): February 8, 2022
• Last Update Submitted That Met QC Criteria: February 1, 2022
• Last Verified: February 1, 2022

More Information

Terms related to this study

• Keywords: Drug therapy
• Additional Relevant MeSH Terms: Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Colonic Diseases; Colitis; Inflammatory Bowel Diseases; Crohn Disease; Intestinal Diseases; Colitis, Ulcerative
• Other Study ID Numbers: IBD-5004; U1111-1207-4110 (OTHER: WHO)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Takeda makes patient-level, de-identified data sets and associated documents available for all interventional studies after applicable marketing approvals and commercial availability have been received (or program is completely terminated), an opportunity for the primary publication of the research and final report development has been allowed, and other criteria have been met as set forth in Takeda's Data Sharing Policy (see www.TakedaClinicalTrials.com for details). To obtain access, researchers must submit a legitimate academic research proposal for adjudication by an independent review panel, who will review the scientific merit of the research and the requestor's qualifications and conflict of interest that can result in potential bias. Once approved, qualified researchers who sign a data sharing agreement are provided access to these data in a secure research environment.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No