Trial Outcomes & Findings for An Observational Study of RoActemra/Actemra (Tocilizumab) As Monotherapy in Rheumatoid Arthritis Patients in Routine Clinical Practice (NCT NCT01474291)

Last Updated: 2016-10-28

Results Overview

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.

Recruitment status

COMPLETED

Target enrollment

608 participants

Primary outcome timeframe

Day 1

Results posted on

2016-10-28

Participant Flow

Participants were not allocated to study arms but were separated according to therapy regimen post hoc for efficacy and safety analyses. Of the 608 participants enrolled 5 were not eligible for analysis (1 was found to be duplicate and 4 did not receive tocilizumab infusion).

Participant milestones

Participant milestones
Measure	All Tocilizumab Tocilizumab (RoActemra/Actemra) administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Overall Study STARTED	603
Overall Study Evaluable for Efficacy	577
Overall Study COMPLETED	383
Overall Study NOT COMPLETED	220

Reasons for withdrawal

Reasons for withdrawal
Measure	All Tocilizumab Tocilizumab (RoActemra/Actemra) administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Overall Study Not Evaluable for Efficacy	26
Overall Study Lack of Treatment Efficacy	88
Overall Study Adverse Event	53
Overall Study Lost to Follow-up	29
Overall Study Patient No Longer Wanted to Participate	12
Overall Study No Reason Reported	10
Overall Study Death	2

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Tocilizumab Monotherapy n=228 Participants Tocilizumab administered as monotherapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=349 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Total n=577 Participants Total of all reporting groups
Age, Continuous	59.1 years STANDARD_DEVIATION 12.7 • n=5 Participants	55.3 years STANDARD_DEVIATION 12.5 • n=7 Participants	56.8 years STANDARD_DEVIATION 12.7 • n=5 Participants
Sex: Female, Male Female	180 Participants n=5 Participants	274 Participants n=7 Participants	454 Participants n=5 Participants
Sex: Female, Male Male	48 Participants n=5 Participants	75 Participants n=7 Participants	123 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 1

Population: Efficacy population, defined as all participants who received at least one infusion of tocilizumab and who met all inclusion and exclusion criteria.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=577 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Number of Participants Assigned Tocilizumab Monotherapy Versus Tocilizumab as Part of Combination Therapy at Study Inclusion Monotherapy	228 participants	—
Number of Participants Assigned Tocilizumab Monotherapy Versus Tocilizumab as Part of Combination Therapy at Study Inclusion Combination Therapy	349 participants	—

SECONDARY outcome

Timeframe: Day 1 (assessment of discontinuations within prior 2 years)

Population: Participants in the Tocilizumab Monotherapy group (Efficacy population) who discontinued MTX and with available data.

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.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=206 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Methotrexate (MTX) Therapeutic escape	12.6 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Methotrexate (MTX) Cytopenia	4.4 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Methotrexate (MTX) Hepatic Cytolysis	20.4 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Methotrexate (MTX) Other Intolerance	46.1 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Methotrexate (MTX) Patient's Choice	4.9 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Methotrexate (MTX) Primary Failure	8.7 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Methotrexate (MTX) Remission	0.5 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Methotrexate (MTX) Reason Not Specified	2.4 percentage of participants	—

SECONDARY outcome

Timeframe: Day 1 (assessment of discontinuations within prior 2 years)

Population: Participants in the Tocilizumab Monotherapy group (Efficacy population) who discontinued leflunomide and with available data.

The percentage of participants who discontinued leflunomide treatment prior to being assigned to tocilizumab monotherapy is presented by reason for discontinuation.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=34 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Leflunomide Therapeutic escape	17.6 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Leflunomide Intolerance	47.1 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Leflunomide Patient's Choice	2.9 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Leflunomide Primary Failure	32.4 percentage of participants	—

SECONDARY outcome

Timeframe: Day 1 (assessment of discontinuations within prior 2 years)

Population: Participants in the Tocilizumab Monotherapy group (Efficacy population) who discontinued sulfasalazine and with available data.

The percentage of participants who discontinued sulfasalazine treatment prior to being assigned to tocilizumab monotherapy is presented by reason for discontinuation.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=17 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Sulfasalazine Therapeutic escape	23.5 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Sulfasalazine Intolerance	41.2 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Sulfasalazine Primary failure	35.3 percentage of participants	—

SECONDARY outcome

Timeframe: Day 1 (assessment of discontinuations within prior 2 years)

Population: Participants in the Tocilizumab Monotherapy group (Efficacy population) who discontinued hydroxychloroquine and with available data.

The percentage of participants who discontinued hydroxychloroquine treatment prior to being assigned to tocilizumab monotherapy is presented by reason for discontinuation.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=10 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Hydroxychloroquine Therapeutic escape	30.0 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Hydroxychloroquine Primary failure	50.0 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Hydroxychloroquine Reason Not Specified	20.0 percentage of participants	—

SECONDARY outcome

Timeframe: Day 1 (assessment of discontinuations within prior 2 years)

Population: Participants in the Tocilizumab Monotherapy group (Efficacy population) who discontinued treatment with unspecified csDMARDs and with available data.

The percentage of participants who discontinued treatment with unspecified csDMARDs prior to being assigned to tocilizumab monotherapy is presented by reason for discontinuation.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=79 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Unspecified Conventional Synthetic Disease-modifying Antirheumatic Drugs (csDMARDs) Therapeutic escape	26.6 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Unspecified Conventional Synthetic Disease-modifying Antirheumatic Drugs (csDMARDs) Intolerance	38.0 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Unspecified Conventional Synthetic Disease-modifying Antirheumatic Drugs (csDMARDs) Patient's Choice	1.3 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Unspecified Conventional Synthetic Disease-modifying Antirheumatic Drugs (csDMARDs) Primary Failure	27.8 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Unspecified Conventional Synthetic Disease-modifying Antirheumatic Drugs (csDMARDs) Remission	1.3 percentage of participants	—
Percentage of Participants Receiving Tocilizumab Monotherapy Who Discontinued Unspecified Conventional Synthetic Disease-modifying Antirheumatic Drugs (csDMARDs) Reason Not Specified	5.1 percentage of participants	—

SECONDARY outcome

Timeframe: Up to 30 months

Population: Efficacy population, defined as all participants who received at least one infusion of tocilizumab and who met all inclusion and exclusion criteria.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=228 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=349 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Mean Number of Tocilizumab Infusions Over the Study Period	9.1 infusions Standard Deviation 4.3	9.7 infusions Standard Deviation 4.0

SECONDARY outcome

Timeframe: Up to 13.4 months

Population: Efficacy population, defined as all participants who received at least one infusion of tocilizumab and who met all inclusion and exclusion criteria.

The percentage of participants who received infusions is presented by category of total infusions received over the study period.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=228 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=349 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants Who Received Tocilizumab Infusions Over the Study Period 1 Infusion	5.7 percentage of participants	4.6 percentage of participants
Percentage of Participants Who Received Tocilizumab Infusions Over the Study Period 2 Infusions	4.8 percentage of participants	4.0 percentage of participants
Percentage of Participants Who Received Tocilizumab Infusions Over the Study Period 3 Infusions	7.0 percentage of participants	2.6 percentage of participants
Percentage of Participants Who Received Tocilizumab Infusions Over the Study Period 4 Infusions	3.5 percentage of participants	4.0 percentage of participants
Percentage of Participants Who Received Tocilizumab Infusions Over the Study Period 5 Infusions	4.8 percentage of participants	5.7 percentage of participants
Percentage of Participants Who Received Tocilizumab Infusions Over the Study Period 6 Infusions	3.9 percentage of participants	5.4 percentage of participants
Percentage of Participants Who Received Tocilizumab Infusions Over the Study Period 7 Infusions	6.6 percentage of participants	2.6 percentage of participants
Percentage of Participants Who Received Tocilizumab Infusions Over the Study Period 8 Infusions	3.5 percentage of participants	2.6 percentage of participants
Percentage of Participants Who Received Tocilizumab Infusions Over the Study Period 9 Infusions	2.6 percentage of participants	5.4 percentage of participants
Percentage of Participants Who Received Tocilizumab Infusions Over the Study Period 10 Infusions	4.4 percentage of participants	5.4 percentage of participants
Percentage of Participants Who Received Tocilizumab Infusions Over the Study Period 11 Infusions	7.9 percentage of participants	6.3 percentage of participants
Percentage of Participants Who Received Tocilizumab Infusions Over the Study Period 12 Infusions	16.7 percentage of participants	22.3 percentage of participants
Percentage of Participants Who Received Tocilizumab Infusions Over the Study Period 13 Infusions	17.1 percentage of participants	18.3 percentage of participants
Percentage of Participants Who Received Tocilizumab Infusions Over the Study Period 14 Infusions	11.0 percentage of participants	9.7 percentage of participants
Percentage of Participants Who Received Tocilizumab Infusions Over the Study Period 15 Infusions	0.4 percentage of participants	0.9 percentage of participants

SECONDARY outcome

Timeframe: Up to 30 months

Population: Efficacy population, defined as all participants who received at least one infusion of tocilizumab and who met all inclusion and exclusion criteria.

The percentage of participants with no modifications (dose modification or discontinuation) is presented.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=228 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=349 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants With No Modification of Tocilizumab Treatment Over the Study Period	48.2 percentage of participants	56.2 percentage of participants

SECONDARY outcome

Timeframe: Up to 30 months

Population: Participants in Efficacy population who received at least 1 infusion of tocilizumab, who met all inclusion/exclusion criteria, and with no permanent discontinuation of tocilizumab treatment over the study period. For efficacy criteria with response/non response values, participants with non-evaluable response were considered as non-responders.

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.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=158 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=256 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants With at Least One csDMARD Intensification During the Study	8.2 percentage of participants	4.7 percentage of participants

SECONDARY outcome

Timeframe: Month 12

Population: Efficacy population, defined as all participants who received at least one infusion of tocilizumab and who met all inclusion and exclusion criteria.

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.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=228 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=349 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
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	41.2 percentage of participants	44.4 percentage of participants

SECONDARY outcome

Timeframe: Month 12

Population: Efficacy population, defined as all participants who received at least one infusion of tocilizumab and who met all inclusion and exclusion criteria.

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.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=228 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=349 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants With DAS28-ESR Remission at Month 12	34.6 percentage of participants	35.5 percentage of participants

SECONDARY outcome

Timeframe: Month 12

Population: Efficacy population, defined as all participants who received at least one infusion of tocilizumab and who met all inclusion and exclusion criteria.

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.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=228 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=349 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants With Clinical Disease Activity Index (CDAI) LDA at Month 12	31.1 percentage of participants	23.5 percentage of participants

SECONDARY outcome

Timeframe: Month 12

Population: Efficacy population, defined as all participants who received at least one infusion of tocilizumab and who met all inclusion and exclusion criteria.

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.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=228 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=349 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants With CDAI Remission at Month 12	9.6 percentage of participants	8.6 percentage of participants

SECONDARY outcome

Timeframe: Month 12

Population: Efficacy population, defined as all participants who received at least one infusion of tocilizumab and who met all inclusion and exclusion criteria.

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.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=228 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=349 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants With Simplified Disease Activity Index (SDAI) LDA at Month 12	30.7 percentage of participants	23.2 percentage of participants

SECONDARY outcome

Timeframe: Month 12

Population: Efficacy population, defined as all participants who received at least one infusion of tocilizumab and who met all inclusion and exclusion criteria.

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.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=228 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=349 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants With SDAI Remission at Month 12	10.1 percentage of participants	9.7 percentage of participants

SECONDARY outcome

Timeframe: Month 12

Population: Efficacy population, defined as all participants who received at least one infusion of tocilizumab and who met all inclusion and exclusion criteria.

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.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=228 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=349 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants With American College or Rheumatology (ACR)20, ACR50, and ACR70 at Month 12 ACR20	32.5 percentage of participants	26.4 percentage of participants
Percentage of Participants With American College or Rheumatology (ACR)20, ACR50, and ACR70 at Month 12 ACR50	21.9 percentage of participants	16.9 percentage of participants
Percentage of Participants With American College or Rheumatology (ACR)20, ACR50, and ACR70 at Month 12 ACR70	9.6 percentage of participants	9.7 percentage of participants

SECONDARY outcome

Timeframe: Month 12

Population: Efficacy population, defined as all participants who received at least one infusion of tocilizumab and who met all inclusion and exclusion criteria.

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

Outcome measures

Outcome measures
Measure	All Tocilizumab n=228 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=349 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants With Good or Moderate European League Against Rheumatism (EULAR) Response at Month 12 Good Response	40.4 percentage of participants	38.7 percentage of participants
Percentage of Participants With Good or Moderate European League Against Rheumatism (EULAR) Response at Month 12 Moderate Response	9.6 percentage of participants	10.9 percentage of participants

SECONDARY outcome

Timeframe: Baseline; Month 6; Month 12

Population: Participants in the Efficacy population who received at least one infusion of tocilizumab, who met all inclusion and exclusion criteria, and with available data at the respective time point.

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.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=228 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=349 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Mean Change From Baseline in Health Assessment Questionnaire Disability Index (HAQ-DI) Score Change at Month 12 (n = 74, 106)	-0.47 units on a scale Standard Deviation 0.68	-0.45 units on a scale Standard Deviation 0.62
Mean Change From Baseline in Health Assessment Questionnaire Disability Index (HAQ-DI) Score Baseline (n = 171, 251)	1.62 units on a scale Standard Deviation 0.67	1.47 units on a scale Standard Deviation 0.66
Mean Change From Baseline in Health Assessment Questionnaire Disability Index (HAQ-DI) Score Change at Month 6 (n = 85, 121)	-0.45 units on a scale Standard Deviation 0.64	-0.44 units on a scale Standard Deviation 0.62

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=228 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=349 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Mean Change From Baseline in Rheumatoid Arthritis Impact of Disease (RAID) Score Change at Month 6 (n = 82, 122)	-2.11 units on a scale Standard Deviation 2.23	-2.07 units on a scale Standard Deviation 2.47
Mean Change From Baseline in Rheumatoid Arthritis Impact of Disease (RAID) Score Change at Month 12 (n = 72, 108)	-2.42 units on a scale Standard Deviation 2.29	-2.15 units on a scale Standard Deviation 2.34
Mean Change From Baseline in Rheumatoid Arthritis Impact of Disease (RAID) Score Baseline (n = 170, 252)	6.47 units on a scale Standard Deviation 1.98	5.92 units on a scale Standard Deviation 1.89

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=228 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=349 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants With Acceptable Health State Assessed by the Patient Acceptable Symptom State (PASS) Questionnaire. Baseline (n = 168, 246)	25.6 percentage of participants	34.1 percentage of participants
Percentage of Participants With Acceptable Health State Assessed by the Patient Acceptable Symptom State (PASS) Questionnaire. Month 6 (n = 101, 153)	69.3 percentage of participants	73.2 percentage of participants
Percentage of Participants With Acceptable Health State Assessed by the Patient Acceptable Symptom State (PASS) Questionnaire. Month 12 (n = 84, 132)	84.5 percentage of participants	79.5 percentage of participants

SECONDARY outcome

Timeframe: Up to 30 months

Population: Safety population, defined as participants who received at least one infusion of tocilizumab.

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.

Outcome measures

Outcome measures
Measure	All Tocilizumab n=234 Participants Tocilizumab administered as monotherapy or in combination with other standard of care therapy according to prescribing information and normal clinical practice.	Tocilizumab Combination Therapy n=369 Participants Tocilizumab administered in combination with other standard of care therapy according to prescribing information and normal clinical practice.
Percentage of Participants With Adverse Events	54.7 percentage of participants	53.4 percentage of participants

Adverse Events

Tocilizumab Monotherapy

Serious events: 31 serious events

Other events: 112 other events

Deaths: 0 deaths

Tocilizumab Combination Therapy

Serious events: 43 serious events

Other events: 182 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Medical Communications

Hoffmann-La Roche

Phone: 800 821-8590

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
Publication restrictions are in place

Restriction type: OTHER