Tofacitinib (Xeljanz) Special Investigation for Rheumatoid Arthritis

Completed

• Conditions: Rheumatoid Arthritis
• Interventions: Drug: Etanercept, other Biologics, Disease-modifying antirheumatic drugs (DMARDs), etc; Drug: Tofacitinib (Xeljanz)

• Registration Number: NCT01932372
• Lead Sponsor: Pfizer

Brief Summary

The objective of this Surveillance is to verify the following subject matters concerning Tofacitinib (Xeljanz) under general practice.

1) Occurrence of adverse reactions, factors that may potentially affect safety and efficacy 2） Long-term safety (particularly, malignant tumors and serious infections) and efficacy

Occurrences of malignant tumors and serious infections will be compared with a control group.

Detailed Description

All the patients whom an investigator prescribes the Xeljanz or Standard of Care for rheumatoid arthritis should be registered consecutively until the number of subjects reaches target number in order to extract patients enrolled into the investigation at random.

Study Timeline

• Study Start: 2013-07-26
• Study First Submitted: 2013-07-30
• Study First Submitted QC: 2013-08-27
• Study First Posted: 2013-08-30
• Primary Completion: 2021-08-24
• Study Completion: 2021-08-24
• Results First Submitted: 2022-06-15
• Status Verified: 2024-07
• Results First Submitted QC: 2024-07-30
• Last Update Submitted: 2024-07-30
• Results First Posted: 2024-10-26
• Last Update Posted: 2024-10-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 9968

Inclusion Criteria

• All patients receiving Tofacitinib (Xeljanz)

Exclusion Criteria

Not Applicable

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Standard of Care	Etanercept, other Biologics, Disease-modifying antirheumatic drugs (DMARDs), etc	Standard of Care for Rheumatoid Arthritis
Tofacitinib (Xeljanz)	Tofacitinib (Xeljanz)	Tablets 5 mg BID

Primary Outcome Measures

Name	Time	Method
Percentage of Participants With Treatment-related Adverse Events or Serious Treatment-related Adverse Events in Participants Who Received XELJANZ	36 months	An adverse drug reaction (ADR) was any untoward medical occurrence attributed to XELJANZ in a participant who received XELJANZ. A serious adverse event/adverse drug reaction (SADR) was a treatment-related adverse event resulting in any of the following outcomes or deemed significant for any other reason: death; life-threatening experience (immediate risk of dying); initial or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Relatedness to XELJANZ was assessed by the physician.
Change in Disease Activity Based on Simplified Disease Activity Index (SDAI)	Baseline, 1, 6, 12, 24, 36 months	The SDAI is the numerical sum of five outcome parameters: tender joint count (TJC) and swollen joint count (SJC) based on a 28-joint assessment, patient global assessment (PtGA) and physician global assessment (PGA) assessed on 0-10 cm VAS, and C-reactive protein (CRP) (mg/dL). SDAI is defined as follows: ≤3.3, disease remission; \>3.3 to ≤11, low disease activity, \>11 to ≤26, moderate disease activity; and \>26, high disease activity. SDAI was assessed at each evaluation time point and mean change from the start of XELJANZ was calculated with the 95% CI.
Change in Disease Activity Score Based on 28-joints Count (DAS28)	Baseline, 1, 6, 12, 24, 36 months	DAS28 is the numerical sum of 4 outcome parameters: tender joint count (TJC) and swollen joint count (SJC) based on a 28-joint assessment, PtGA assessed on 0-10 cm VAS, and the erythrocyte sedimentation rate (ESR) (millimeters per hour \[mm/hour\]). DAS28 is defined as follows: \<2.6, disease remission; ≥2.6 to \<3.2, low disease activity, ≥3.2 to ≤5.1, moderate disease activity; and \>5.1, high disease activity. DAS28 was assessed at each evaluation time point and mean change from the start of XELJANZ was calculated with the 95% CI.

Secondary Outcome Measures

Name	Time	Method
Occurrence of Serious Infection Events	12 months	Comparison of time to serious infection between XELJANZ group and control group.; Hazard ratio (unadjusted): Baseline characteristics are unadjusted Hazard ratio (adjusted 1): Adjusted by propensity score estimated by logistic regression Hazard ratio (adjusted 2): Adjusted by propensity score estimated by random forest Hazard ratio (adjusted 3): Adjusted by propensity score estimated by logistic regression (with upper limit of weighting) Hazard ratio (adjusted 4): Adjusted by propensity score estimated by random forest (with upper limit of weighting)
Occurrence of Malignancy	36 months	Comparison of time to malignancy between XELJANZ group and control group. The standard incidence ratio (SIR) was calculated using the general Japanese population as the reference population.; Hazard ratio (unadjusted): Baseline characteristics are unadjusted Hazard ratio (adjusted 1): Adjusted by propensity score estimated by logistic regression Hazard ratio (adjusted 2): Adjusted by propensity score estimated by random forest Hazard ratio (adjusted 3): Adjusted by propensity score estimated by logistic regression (with upper limit of weighting) Hazard ratio (adjusted 4): Adjusted by propensity score estimated by random forest (with upper limit of weighting)
Occurrence of Death	36 months	Comparison of time to death between XELJANZ group and control group. The standardized mortality ratio (SMR) was calculated using the general Japanese population as the reference population.; Hazard ratio (unadjusted): Baseline characteristics are unadjusted