MedPath

Observational Study to Determine How Physicians Make Treatment Decisions in Patients Treated With Tofacitinib for Moderate to Severe Active Rheumatoid Arthritis

Completed
Conditions
Arthritis, Rheumatoid
Registration Number
NCT03387423
Lead Sponsor
Pfizer
Brief Summary

This non-interventional study aims to identify key factors that are driving treatment decisions by rheumatologists in the treatment of rheumatoid arthritis (RA) patients starting treatment with Tofacitinib in a real world setting.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
1459
Inclusion Criteria

Patients aged ≥ 18 years

Confirmed Diagnosis of Rheumatoid Arthritis by rheumatologist

Patient is eligible for Tofacitinib treatment according to Summary of Product Characteristics (SmPC)

Evidence of a personally signed and dated informed consent document indicating that the patient (or a legally acceptable representative) has been informed of all pertinent aspects of the study.

Exclusion Criteria

Contraindications according to Xeljanz® SmPC

Receipt of any investigational drug within 3 months before study inclusion

Patients who have received any previous treatment with Tofacitinib or other JAK inhibitors

Patients who are investigational site staff members or patients who are Pfizer employees directly involved in the conduct of the study.

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Number of Participants With Treatment EscalationsFrom date of first prescription of tofacitinib up to 24 Months

Treatment escalations was defined as participants who switched to another disease modifying antirheumatic drug (DMARD) or combination of DMARDs when compared to the last visit. The following three types of escalations were reported: treatment termination, treatment step-up/switch and treatment step-down. Treatment termination was defined as the termination of a DMARD (or multiple when on combination therapy) without starting a new DMARD therapy. Treatment step-up/switch was defined as an increase from the current treatment regime towards e.g.a combination of DMARDs and treatment step-down, was defined as de-escalation from the current treatment regime, example (e.g.) from combination therapy to monotherapy. One participant can fall into more than 1 type of escalation.

Time to Treatment EscalationFrom date of first prescription of tofacitinib up to 24 Months

Time to treatment escalation was defined as time in days from escalation visit date to next escalation visit date. The following three types of escalations were reported: treatment termination, treatment step-up/switch and treatment step-down. Treatment termination was defined as the termination of a DMARD (or multiple when on combination therapy) without starting a new DMARD therapy. Treatment step-up/switch was defined as an increase from the current treatment regime (monotherapy) towards a combination of DMARDs and treatment step-down, was defined as de-escalation from the current treatment regime, e.g. from combination therapy to monotherapy. Number of participants with total number of each type of treatment escalations during the study (step-up/switch, step-down, treatment termination along with all escalations) are reported in this outcome measure.

Secondary Outcome Measures
NameTimeMethod
Time to First Step Up Treatment EscalationFrom date of first tofacitinib prescription up to date of first step-up treatment escalation (maximum up to 24 months)

Time to first step-up treatment escalation was defined for participants who experienced a treatment step-up and was measured as date of first treatment step up escalation minus (-) date of first prescription of tofacitinib. Treatment step-up was defined as an increase from the current treatment regime (monotherapy) towards a combination of DMARDs.

Change From Baseline in DAS28-4 ESR at Months 3, 6, 9, 12, 15, 18, 21 and 24Baseline, Months 3, 6, 9, 12, 15, 18, 21 and 24

DAS28 is a measure of disease activity in participants with rheumatoid arthritis. DAS28-4 (ESR) was calculated using the following formula: 0.56\*square root of (TJC28) + 0.28\*square root of (SJC28) + 0.70\*In (ESR in mm/ hour) + 0.014\*PtGA in cm. PtGA was assessed on 0-10 cm VAS scale (scores ranging from 0 cm \[very well\] to 10 cm \[worst\]), where higher scores indicated greater affliction due to disease activity). Total DAS28-4 (ESR) score range: 0 (none) to 9.4 (extreme disease activity), higher score indicated more disease activity.

Number of Participants Achieving Low Disease Activity (LDA) as Assessed by Simplified Disease Activity Index (SDAI) at Months 3, 6, 9, 12, 15, 18, 21 and 24Months 3, 6, 9, 12, 15, 18, 21 and 24

SDAI was calculated using the following formula: SDAI = Tender Joint Count (TJC) (using 28 joints) + Swollen Joint Count (SJC) (using 28 joints) + Patient Global Assessment of Arthritis (PtGA) (0-10 centimetre \[cm\] scale) + Physician's Global Assessment of Arthritis (PhGA) (0-10 cm scale) + C-reactive protein (CRP) (milligram per decilitre \[mg/dL\]). TJC28 joints included shoulders, elbows, wrists, metacarpophalangeal (MCP) and proximal interphalangeal (PIP) joints, and knees whereas SJC28 included shoulders, elbows, wrists, MCP, PIP and knees (excluding artificial joints). PtGA and PhGA both were assessed on 0-10 cm visual analogue scale (VAS) scale (0 cm \[very well\] to 10 cm \[worst\], where higher scores indicated greater affliction due to disease activity). SDAI total score ranged from 0 to 86, where higher scores indicated higher disease activity. SDAI score of less than or equal to (\<=11) indicates LDA.

Number of Participants Achieving LDA as Assessed by Clinical Disease Activity Index (CDAI) at Months 3, 6, 9, 12, 15, 18, 21 and 24Months 3, 6, 9, 12, 15, 18, 21 and 24

CDAI was calculated using the following formula: CDAI = (28TJC) + (28SJC) + (PhyGA in cm) + (PtGA in cm). TJC28 joints included shoulders, elbows, wrists, MCP and proximal PIP joints, and knees whereas SJC28 included shoulders, elbows, wrists, MCP, PIP and knees (excluding artificial joints). PtGA and PhGA both were assessed on 0-10 cm VAS scale (0 cm \[very well\] to 10 cm \[worst\], where higher scores indicated greater affliction due to disease activity). CDAI total score ranged from 0 to 76, where higher scores indicated higher disease activity. CDAI score of \<= 10 indicates LDA.

Number of Participants Achieving LDA Based on Disease Activity Score (DAS28-4) Erythrocyte Sedimentation Rate (ESR) at Months 3, 6, 9, 12, 15, 18, 21 and 24Months 3, 6, 9, 12, 15, 18, 21 and 24

DAS28 is a measure of disease activity in participants with rheumatoid arthritis. DAS28-4 (ESR) was calculated using the following formula: 0.56\* square root of (TJC28) + 0.28\* square root of (SJC28) + 0.70\* natural log (ln) (ESR in \[millimeter per hour \[mm/ hour\]) + 0.014\*(PtGA in cm). PtGA was assessed on 0-10 cm VAS scale (scores ranging from 0 cm \[very well\] to 10 cm \[worst\]), where higher scores indicated greater affliction due to disease activity). Total DAS28-4 (ESR) score range: 0 (none) to 9.4 (extreme disease activity), higher score indicated more disease activity. DAS28-4 ESR \<= 3.2 indicates LDA.

Number of Participants Achieving LDA as Based on DAS28-4 C-Reactive Protein (CRP) at Months 3, 6, 9, 12, 15, 18, 21 and 24Months 3, 6, 9, 12, 15, 18, 21 and 24

DAS28 is a measure of disease activity in participants with rheumatoid arthritis. DAS28-4 (CRP) was calculated using the following formula: 0.56\* square root of (TJC28) + 0.28\* square root of (SJC28) + 0.36\*ln (CRP in mg/l +1) + 0.014\*(PtGA in mm)+ 0.96. PtGA was assessed on 0-10 cm VAS scale (scores ranging from 0 cm \[very well\] to 10 cm \[worst\]), where higher scores indicated greater affliction due to disease activity). Total DAS28-4 (CRP) score range: 0 (none) to 9.4 (extreme disease activity), higher score indicated more disease activity. DAS28-4 CRP \<= 3.2 indicates LDA.

Number of Participants Achieving Remission as Assessed by American College of Rheumatology (ACR)-EULAR Boolean Remission Criteria at Months 3, 6, 9, 12, 15, 18, 21 and 24Months 3, 6, 9, 12, 15, 18, 21 and 24

ACR-EULAR Boolean remission criteria was defined as when participant had ACR remission =1 if; TJC28 \<=1, SJC28 \<=1, CRP \<=1mg/dL and PtGA \<= 2 cm on a 0 to 10 cm scale, where higher values indicate greater affection due to disease activity.

Number of Participants Achieving Remission as Assessed by SDAI at Months 3, 6, 9, 12, 15, 18, 21 and 24Months 3, 6, 9, 12, 15, 18, 21 and 24

SDAI was calculated using the following formula: SDAI = (TJC28) + (SJC28) + (PtGA in cm) + (PhGA in cm) + (CRP in mg/dL). TJC28 joints included shoulders, elbows, wrists, MCP and PIP joints, and knees whereas SJC28 included shoulders, elbows, wrists, MCP, PIP and knees (excluding artificial joints). PtGA and PhGA both were assessed on 0-10 cm VAS scale (0 cm \[very well\] to 10 cm \[worst\], where higher scores indicated greater affliction due to disease activity). SDAI total score ranged from 0 to 86, where higher scores indicated higher disease activity. SDAI score of \<=3.3 indicates remission.

Number of Participants Achieving Remission as Assessed by CDAI at Months 3, 6, 9, 12, 15, 18, 21 and 24Months 3, 6, 9, 12, 15, 18, 21 and 24

CDAI was calculated using the following formula: CDAI = (28TJC) + (28SJC) + (PhyGA in cm) + (PtGA in cm). TJC28 joints included shoulders, elbows, wrists, MCP and proximal PIP joints, and knees whereas SJC28 included shoulders, elbows, wrists, MCP, PIP and knees (excluding artificial joints). PtGA and PhGA both were assessed on 0-10 cm VAS scale (0 cm \[very well\] to 10 cm \[worst\], where higher scores indicated greater affliction due to disease activity). CDAI total score ranged from 0 to 76, where higher scores indicated higher disease activity. CDAI score of \<=2.8 indicates remission.

Number of Participants Achieving Remission as Assessed by DAS-28 ESR at Months 3, 6, 9, 12, 15, 18, 21 and 24Months 3, 6, 9, 12, 15, 18, 21 and 24

DAS28 is a measure of disease activity in participants with rheumatoid arthritis. DAS28-4 (ESR) was calculated using the following formula: 0.56\*square root of (TJC28) + 0.28\*square root of (SJC28) + 0.70\*In (ESR in mm/ hour) + 0.014\*PtGA in cm. PtGA was assessed on 0-10 cm VAS scale (scores ranging from 0 cm \[very well\] to 10 cm \[worst\]), where higher scores indicated greater affliction due to disease activity). Total DAS28-4 (ESR) score range: 0 (none) to 9.4 (extreme disease activity), higher score indicated more disease activity. DAS28-4 ESR score of \<2.6 indicates remission.

Number of Participants Achieving Remission as Assessed by DAS28-4 CRP at Months 3, 6, 9, 12, 15, 18, 21 and 24Months 3, 6, 9, 12, 15, 18, 21 and 24

DAS28 is a measure of disease activity in participants with rheumatoid arthritis. DAS28-4 (CRP) was calculated using the following formula: 0.56\*square root of (TJC28) + 0.28\*square root of (SJC28) + 0.36\*ln (CRP in mg/l +1) + 0.014\*(PtGA in cm)+ 0.96. PtGA was assessed on 0-10 cm VAS scale (scores ranging from 0 cm \[very well\] to 10 cm \[worst\]), where higher scores indicated greater affliction due to disease activity). Total DAS28-4 (CRP) score range: 0 (none) to 9.4 (extreme disease activity), higher score indicated more disease activity. DAS28-4 CRP score of \<2.6 indicates remission.

Change From Baseline in DAS28-4 CRP at Months 3, 6, 9, 12, 15, 18, 21 and 24Baseline, Months 3, 6, 9, 12, 15, 18, 21 and 24

DAS28 is a measure of disease activity in participants with rheumatoid arthritis. DAS28-4 (CRP) was calculated using the following formula: 0.56\*square root of(TJC28) + 0.28\*square root of(SJC28) + 0.36\*ln (CRP in mg/l +1) + 0.014\*(PtGA in cm)+ 0.96. PtGA was assessed on 0-10 cm VAS scale (scores ranging from 0 cm \[very well\] to 10 cm \[worst\]), where higher scores indicated greater affliction due to disease activity). Total DAS28-4 (CRP) score range: 0 (none) to 9.4 (extreme disease activity), higher score indicated more disease activity.

Change From Baseline in Duration of Morning Stiffness at Months 3, 6, 9, 12, 15, 18, 21 and 24Baseline, Months 3, 6, 9, 12, 15, 18, 21 and 24

The duration of morning stiffness was determined by asking the following questions: "When you wake up in the morning, do you currently suffer from morning stiffness?" "How long does the morning stiffness last from the time you wake up?". The change from baseline in duration of morning stiffness was calculated as: morning stiffness at time point -morning stiffness at baseline.

Change From Baseline in the Functional Ability Questionnaire Hannover (FFbH) at Months 3, 6, 9, 12, 15, 18, 21 and 24Baseline, Months 3, 6, 9, 12, 15, 18, 21 and 24

The FFbH for RA is a German short questionnaire for the assessment of patientive functional capacity in the context of basic everyday activities (range: 0-100% functional capacity, where higher percentage indicates more capacity for everyday activities). The FFbH questionnaire consisted of 18 questions with 3 possible responses (Yes; Yes, but with effort; No or only with outside help). The total score was the sum of the scores of all 18 questions, where for each question (Yes = 2 points; Yes, but with effort = 1 point; No or only with outside help = 0 points).The functional capacity (%) is calculated by (total score\*100) divided by (2\*number of valid responses). The change from baseline was calculated at each time point as functional capacity at timepoint - functional capacity at baseline.

Number of Participants Achieving Functional Remission in FFbH at Months 3, 6, 9, 12, 15, 18, 21 and 24Months 3, 6, 9, 12, 15, 18, 21 and 24

The FFbH for RA is a German short questionnaire for the assessment of patientive functional capacity in the context of basic everyday activities (range: 0-100% functional capacity, where higher percentage indicates more capacity for everyday activities). The FFbH questionnaire consisted of 18 questions with 3 possible responses (Yes; Yes, but with effort; No or only with outside help). The total score was the sum of the scores of all 18 questions, where for each question (Yes = 2 points; Yes, but with effort = 1 point; No or only with outside help = 0 points). The functional capacity \[%\] is calculated by (total score\*100)/ (2\*number of valid responses). Functional remission in FFbH was defined as functional capacity \> 83 %.

Change From Baseline in European Quality of Life (EuroQoL) Group EuroQoL- 5 Dimensions- 3 Levels (EQ-5D-3L) Total Scores at Months 3, 6, 9, 12, 15, 18, 21 and 24Baseline, Months 3, 6, 9, 12, 15, 18, 21 and 24

The EQ-5D-3L is a standardised instrument used to measure quality of life. It is based on five dimensions: mobility, self-care, usual activities, pain and discomfort, and anxiety and depression with each dimension having three levels of function: 1 indicates no problem; 2 indicates some problem; 3 indicates extreme problem. Scoring formula developed by EuroQol Group (German weights) assigns a utility value for each domain in the profile. Score was transformed and results in a total score range of 0.05 to 1.00; higher scores indicating a better health state.

Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)- Fatigue Scale at Months 3, 6, 9, 12, 15, 18, 21 and 24Baseline, Months 3, 6, 9, 12, 15, 18, 21 and 24

The FACIT-F Scale is a participant completed questionnaire consisting of 13 items that assess fatigue. Participants responded to each item on a 5-point scale based on their experience of fatigue during the past 7 days (0 = not at all; 1 = a little bit; 2 =somewhat; 3 = quite a bit; 4 = very much). Instrument scoring yielded a range from 0 to 52 (negatively worded items were reversed during analysis), with higher scores representing better participant status (less fatigue).

Mean Number of Days of Drug Survival at Months 12 and 24At 12 and 24 months

Drug survival was assessed using Kaplan-Meier methodology. For participants who terminated tofacitinib, the drug survival status was recorded as (status = 1 and time = termination date tofacitinib - date of initiation on tofacitinib) and for participants who do not terminate tofacitinib (status = 0 and time = date of final visit - date of initiation on tofacitinib.

Number of Participants Per Categories of Satisfaction With Tofacitinib Treatment at Months 3, 6, 9, 12, 15, 18, 21 and 24Months 3, 6, 9, 12, 15, 18, 21 and 24

Satisfaction with treatment was assessed on a 5-point Likert scale (where 0 = extremely dissatisfied, 1= dissatisfied, 2 = neither satisfied nor dissatisfied, 3 = satisfied and 4 = extremely satisfied) in response to the question "How satisfied are you with the drugs that you have received for your arthritis since your last visit?".

Trial Locations

Locations (87)

Private Practice Richter

🇩🇪

Rostock, Germany

Private Practice

🇩🇪

Templin, Germany

Private Praxis

🇩🇪

Duesseldorf, Nordrhein-westfalen, Germany

Private Practice Kupka

🇩🇪

Altenburg, Germany

Private Practise Boehm

🇩🇪

Altenholz, Germany

Private Practice Marycz

🇩🇪

Amberg, Germany

Private Practice Manger

🇩🇪

Bamberg, Germany

MFZ Medizinisches Forschungszentrum Weserbergland

🇩🇪

Bad Pyrmont, Germany

Private Practice Ochs

🇩🇪

Bayreuth, Germany

Private Practice Schmitt-Haendle

🇩🇪

Bayreuth, Germany

Private Practice Bozorg

🇩🇪

Berlin, Germany

Private Practice Brandt-Juergens

🇩🇪

Berlin, Germany

Privat Practice Remstedt

🇩🇪

Berlin, Germany

Private Practice Zinke

🇩🇪

Berlin, Germany

private practise Herzberg

🇩🇪

Berlin, Germany

Private Practice Seifert

🇩🇪

Berlin, Germany

Private Practice Thiele

🇩🇪

Berlin, Germany

Private Practise

🇩🇪

Neubrandenburg, Germany

Immanuel Klinikum Bernau

🇩🇪

Bernau, Germany

Private Practice Lorenz

🇩🇪

Chemnitz, Germany

Kreiskranenhaus Demmin GmbH

🇩🇪

Demmin, Germany

Private Practice Steinmueller

🇩🇪

Ehringshausen, Germany

Private Practice Kaestner

🇩🇪

Erfurt, Germany

Private Practice Koch

🇩🇪

Erfurt, Germany

Private Practice Mueller

🇩🇪

Freiberg, Germany

Private Practice Haeckel

🇩🇪

Frankenberg/Sa., Germany

Private Practice Abahji

🇩🇪

Germering, Germany

Private Practice Sensse

🇩🇪

Gifhorn, Germany

private practise Kühne

🇩🇪

Haldensleben, Germany

Private Practice Semmler

🇩🇪

Guestrow, Germany

Private Practice Dahmen

🇩🇪

Hamburg, Germany

Private Practice Liebhaber

🇩🇪

Halle, Germany

private practise Aries

🇩🇪

Hamburg, Germany

private practise Heilig

🇩🇪

Heidelberg, Germany

Medius Kliniken gGmbH

🇩🇪

Kirchheim unter Teck, Germany

Kreiskrankenhaus Langenau

🇩🇪

Langenau, Germany

Private Practice Kudela

🇩🇪

Magdeburg, Germany

Private Practice Hamann

🇩🇪

Leipzig, Germany

private practise Sieburg

🇩🇪

Magdeburg, Germany

Private Practice Schwarze

🇩🇪

Leipzig, Germany

Private Practice Rossbach

🇩🇪

Mansfeld, Germany

Private Practice Harmuth

🇩🇪

Marktredwitz, Germany

Private Practice Worsch

🇩🇪

Muehlhausen, Germany

Private Practice Raub

🇩🇪

Muenster, Germany

Private Practice Berger

🇩🇪

Naunhof, Germany

Private Practice Krueger

🇩🇪

Muenchen, Germany

Praxis Dr.med. Holger Krauel Facharzt für Innere Medizin und Rheumatologie

🇩🇪

Naumburg (Saale), Germany

Private Practice Volberg

🇩🇪

Neuss, Germany

MVZ Dialysezentrum Schweinfurt

🇩🇪

Schweinfurt, Germany

MVZ Klinikum Straubing

🇩🇪

Straubing, Germany

Private Practice Melzer

🇩🇪

Seesen, Germany

Private Practice Alliger

🇩🇪

Zwiesel, Germany

MED Bayern OST GmbH

🇩🇪

Burghausen, Germany

Private Practice Fuchs

🇩🇪

Augsburg, Germany

private practise Gause

🇩🇪

Bad Bramstedt, Germany

Private Practice Menne

🇩🇪

Dortmund, Germany

ACURA Kliniken Rheinland-Pfalz AG

🇩🇪

Bad Kreuznach, Germany

Ambulantes Gesundheitszentrum der Charité

🇩🇪

Berlin, Germany

Private Practice Kirrstetter

🇩🇪

Deggendorf, Germany

Private Practice Fischer

🇩🇪

Dresden, Germany

private practise Pech

🇩🇪

Eberswalde, Germany

Private Practice Luethke

🇩🇪

Dresden, Germany

SRH Krankenhaus Waltershausen-Friedrichroda GmbH

🇩🇪

Friedrichroda, Germany

Private Practice Holst

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Glaisin, Germany

Private Practice Zeh

🇩🇪

Goeppingen, Germany

Asklepios MVZ Nord SH GmbH, c/o AK St. Georg

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Elmshorn, Germany

Private Practice Kremers

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Juelich, Germany

private practise Wernicke

🇩🇪

Hohen Neuendorf, Germany

Private Practice Stille

🇩🇪

Hannover, Germany

Private Practice Baerlecken

🇩🇪

Koeln, Germany

MCN Medic Center Nuernberg GmbH

🇩🇪

Nuernberg, Germany

Private Practice Goettl

🇩🇪

Passau, Germany

Private Practice Wassenberg, Koehler, Weier

🇩🇪

Ratingen, Germany

Private Practice Baumann

🇩🇪

Plauen, Germany

Private Practice Graessler

🇩🇪

Pirna, Germany

private practise Biewer

🇩🇪

Saarbrücken, Germany

Knappschaftsklinikum Soor, Klinik fuer Rheumatologie und Klinische Immunologie

🇩🇪

Puettlingen, Germany

Private Practice Pyra

🇩🇪

Torgelow, Germany

Private Practice Moebius

🇩🇪

Schwerin, Germany

Private Practice Haas

🇩🇪

Tuebingen, Germany

private practise Ständer

🇩🇪

Schwerin, Germany

Klinik an der Weissenburg GmbH

🇩🇪

Uhlstaedt-Kirchhasel, Germany

Private Practice Rinaldi

🇩🇪

Ulm, Germany

Private Practice Woerth

🇩🇪

Wiesbaden, Germany

Klinikverbund St. Antonius und St. Josef GmbH

🇩🇪

Wuppertal, Germany

Rheuma Praxis Barmen Dres. med. Demirel / Hruschka

🇩🇪

Wuppertal, Germany

Private Practice Fricke-Wagner

🇩🇪

Zwickau, Germany

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