An Observational Study of RoActemra/Actemra (Tocilizumab) As Monotherapy in Rheumatoid Arthritis Patients in Routine Clinical Practice

Completed

• Conditions: Rheumatoid Arthritis
• Interventions: Biological: Tocilizumab

• Registration Number: NCT01474291
• Lead Sponsor: Hoffmann-La Roche

Brief Summary

This prospective, multi-center, observational study will evaluate factors influencing the use of tocilizumab (RoActemra/Actemra) as monotherapy in rheumatoid arthritis patients in real life setting. Data will be collected from participants for 12 months following initiation of tocilizumab treatment.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2011-11-15
• Study First Submitted QC: 2011-11-15
• Study First Posted: 2011-11-18
• Study Start: 2012-01
• Primary Completion: 2014-09
• Study Completion: 2014-09
• Status Verified: 2016-09
• Results First Submitted: 2016-09-08
• Results First Submitted QC: 2016-09-08
• Last Update Submitted: 2016-09-08
• Results First Posted: 2016-10-28
• Last Update Posted: 2016-10-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 608

Inclusion Criteria

• Adult participants, >/= 18 years of age
• Patients with rheumatoid arthritis for whom the rheumatologist decides to start tocilizumab in combination with DMARD or as monotherapy

Read More

Exclusion Criteria

• Current participation in a clinical trial in rheumatoid arthritis

Read More

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Tocilizumab	Tocilizumab	Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.

Primary Outcome Measures

Name	Time	Method
Number of Participants Assigned Tocilizumab Monotherapy Versus Tocilizumab as Part of Combination Therapy at Study Inclusion	Day 1	The number of participants assigned to tocilizumab monotherapy versus tocilizumab combination therapy is reported. A multivariate analysis was performed to search for predictive factors for the initiation of tocilizumab in monotherapy.

Secondary Outcome Measures

Name	Time	Method
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Unspecified Conventional Synthetic Disease-modifying Antirheumatic Drugs (csDMARDs)	Day 1 (assessment of discontinuations within prior 2 years)	The percentage of participants who discontinued treatment with unspecified csDMARDs prior to being assigned to tocilizumab monotherapy is presented by reason for discontinuation.
Percentage of Participants With No Modification of Tocilizumab Treatment Over the Study Period	Up to 30 months	The percentage of participants with no modifications (dose modification or discontinuation) is presented.
Percentage of Participants With Clinical Disease Activity Index (CDAI) LDA at Month 12	Month 12	CDAI was calculated from the number of swollen joints and tender joints using the 28-joint count and the patient's global assessment of disease activity and physician's global assessment of disease activity; CDAI scores range from 0 to 76, where lower scores indicate less disease activity. A score of ≤10 was considered to be CDAI LDA. Participants with missing data were considered to have failed to achieve the outcome.
Percentage of Participants With CDAI Remission at Month 12	Month 12	CDAI was calculated from the number of swollen joints and tender joints using the 28-joint count and the patient's global assessment of disease activity and physician's global assessment of disease activity; CDAI scores range from 0 to 76, where lower scores indicate less disease activity. A score of ≤2.8 was considered to be CDAI remission. Participants with missing data were considered to have failed to achieve the outcome.
Percentage of Participants With Simplified Disease Activity Index (SDAI) LDA at Month 12	Month 12	SDAI was calculated from the number of swollen joints and tender joints using the 28-joint count, C-reactive protein (CRP) (milligrams per liter (mg/L)) per , and the patient's global assessment of disease activity and physician's global assessment of disease activity; SDAI scores range from 0 to 86, where lower scores indicate less disease activity. A score of ≤11 was considered to be SDAI LDA. Participants with missing data were considered to have failed to achieve the outcome.
Percentage of Participants With SDAI Remission at Month 12	Month 12	SDAI was calculated from the number of swollen joints and tender joints using the 28-joint count, CRP (mg/L), and the patient's global assessment of disease activity and physician's global assessment of disease activity; SDAI scores range from 0 to 86, where lower scores indicate less disease activity. A score of ≤3.3 was considered to be SDAI remission. Participants with missing data were considered to have failed to achieve the outcome.
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Methotrexate (MTX)	Day 1 (assessment of discontinuations within prior 2 years)	The percentage of participants who discontinued MTX treatment prior to being assigned to tocilizumab monotherapy is presented by reason for discontinuation.; Reason for discontinuation "Other Intolerance" = intolerance other than cytopenia or hepatic cytolysis.
Mean Number of Tocilizumab Infusions Over the Study Period	Up to 30 months
Percentage of Participants With at Least One csDMARD Intensification During the Study	Up to 30 months	csDMARD intensification was defined as an addition of a csDMARD without suppression of other csDMARD, dose increase of a csDMARD, switch (addition and suppression) of a csDMARD without intolerance, biological abnormality or symptom improvement to the suppressed csDMARD, or modification of the MTX administration route (from oral route to intramuscular/subcutaneous) with dose increase or maintenance.
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Leflunomide	Day 1 (assessment of discontinuations within prior 2 years)	The percentage of participants who discontinued leflunomide treatment prior to being assigned to tocilizumab monotherapy is presented by reason for discontinuation.
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Sulfasalazine	Day 1 (assessment of discontinuations within prior 2 years)	The percentage of participants who discontinued sulfasalazine treatment prior to being assigned to tocilizumab monotherapy is presented by reason for discontinuation.
Mean Change From Baseline in Rheumatoid Arthritis Impact of Disease (RAID) Score	Baseline; Month 6, Month 12	The RAID questionnaire is a participant-reported outcome measure evaluating the impact of rheumatoid arthritis on participant quality of life. This composite index score ranges from 0 (best) to 10 (worst) and includes questions regarding 7 domains (pain, functional disability assessment, fatigue, sleep, physical well-being, emotional well-being, coping). A decrease in score corresponds to improvement in participant-assessed health state.
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Hydroxychloroquine	Day 1 (assessment of discontinuations within prior 2 years)	The percentage of participants who discontinued hydroxychloroquine treatment prior to being assigned to tocilizumab monotherapy is presented by reason for discontinuation.
Percentage of Participants Who Received Tocilizumab Infusions Over the Study Period	Up to 13.4 months	The percentage of participants who received infusions is presented by category of total infusions received over the study period.
Percentage of Participants in Disease Activity Score Based on 28-joint Count and Erythrocyte Sedimentation Rate (DAS28-ESR) Low Disease Activity (LDA) at Month 12	Month 12	DAS28-ESR was calculated from the number of swollen joints and tender joints using the 28-joint count, ESR (mm/hour) and patient's global assessment of disease activity; scores range from 0 to 10, where lower scores indicate less disease activity. A score of ≤3.2 was considered to be DAS28-ESR LDA. Participants with missing data were considered to have failed to achieve the outcome.
Percentage of Participants With DAS28-ESR Remission at Month 12	Month 12	DAS28-ESR was calculated from the number of swollen joints and tender joints using the 28-joint count, ESR (mm/hour) and patient's global assessment of disease activity; scores range from 0 to 10, where lower scores indicate less disease activity. A score of \<2.6 was considered to be DAS28-ESR remission. Participants with missing data were considered to have failed to achieve the outcome.
Percentage of Participants With Acceptable Health State Assessed by the Patient Acceptable Symptom State (PASS) Questionnaire.	Baseline; Month 6; Month 12	Participants were asked: "If you were to remain in the same condition for the next few months as you have been over the last 8 days, would this be 1) acceptable, 2) unacceptable?" The percentage of participants who responded "acceptable" at each time point is presented.
Percentage of Participants With American College or Rheumatology (ACR)20, ACR50, and ACR70 at Month 12	Month 12	ACR20/50/70 response was calculated as improvement (from baseline) of at least 20/50/70% (respectively) of tender and of swollen joints, and improvement from baseline of least 20/50/70% (respectively) in at least 3 of the 5 following parameters: participant's pain assessment, patient's global assessment of disease activity, physician's global assessment of disease activity, health assessment questionnaire disability index (HAQ-DI) score, and ESR (mm/hour) or CRP (mg/L). Participants with missing data were considered to have failed to achieve the outcome.
Percentage of Participants With Good or Moderate European League Against Rheumatism (EULAR) Response at Month 12	Month 12	EULAR response was categorized as good or moderate response and was calculated as the difference between DAS28-ESR scores at baseline and Month 12. DAS28-ESR was calculated from the number of swollen joints and tender joints using the 28-joint count, ESR (mm/hour) and patient's global assessment of disease activity; scores range from 0 to 10, where lower scores indicate less disease activity.; * If diminution from baseline \>1.2 and score ≤3.2 at Month 12 = good response; * If diminution from baseline \>1.2 and score \>3.2 at Month 12 = moderate response; * If diminution from baseline \>0.6 and ≤1.2, and score ≤5.1 at Month 12 = moderate response; * If diminution from baseline \>0.6 and ≤1.2, and score \>5.1 at Month 12 = non-response; * If diminution from baseline ≤1.2 at Month 12 = non-response; * Participants with missing data were considered as non-response
Mean Change From Baseline in Health Assessment Questionnaire Disability Index (HAQ-DI) Score	Baseline; Month 6; Month 12	The HAQ-DI is a participant-reported assessment of ability to perform daily living activities. This composite index score ranges from 0 (normal) to 3 (total functional disability) and includes questions regarding 8 domains (dress/groom; arise; eat; walk; reach; grip; hygiene; and common activities over past week). A decrease in score corresponds to improvement in participant-assessed health state.
Percentage of Participants With Adverse Events	Up to 30 months	An adverse event was defined as any unfavorable and unintended sign (including an abnormal laboratory finding if accompanied by clinical symptoms, results in a change in study treatment, results in a medical intervention or a change in concomitant therapy or clinically significant in the investigator's judgment), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.