Validation of the OMERACT-OARSI Responder Index:

Responders Have Better Overall Health Status than Non-responders

Marc C. Hochberg, Barker Bausell, Kevin Frick, Donald Steinwachs and Brian BermanUniversity of Maryland School of Medicine and The Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland, USA

Objective

To validate the OMERACT-OARSI Responder Index

Test the hypothesis that patients with knee OA participating in a clinical trial who fulfil the OMERACT-OARSI Responder Index will have better overall health status as measured by both arthritis-specific and general measures.

Comparison of Outcomes by OMERACT-OARSI Response 236 (41.4%) of 570 patients randomized

achieved an OMERACT-OARSI Response at the end of study– 61% of 386 completers

WOMAC Scores by OMERACT-OARSI Response

05

10

15

2025

30

35

40

45

WOMAC Pain WOMAC Function WOMAC Total

Responders Nonresponders

HAQ Scores by OMERACT-OARSI Response

0

0.2

0.4

0.6

0.8

1

1.2

1.4

1.6

HAQ Pain HAQ Disability

Responders Nonresponders

EuroQoL Scores by OMERACT-OARSI Response

01020

304050

607080

90

EQ-5D EQ-VAS

Responders Nonresponders

SF-36 Scores by OMERACT-OARSI Response

010

20

3040

50

60

7080

90

PF RP Pain GH MH V RE SA

Responders Nonresponders

Conclusion

These data validate the OMERACT-OARSI Responder Index.– Results unchanged when analysis

performed only with completers (N = 386) OMERACT-OARSI Responder Index

should be considered as primary outcome for clinical trials of symptomatic therapies in patients with OA.

State Measures - 2

Minimal Clinically Important Improvement (MCII)– Smallest change in measurement that signifies an

important improvement in a patient’s symptom– 75%ile of distribution of change score among

those who had good or excellent improvement with therapy

Tubach F et al: Ann Rheum Dis 2005;64:29-33

State Measures - 2

Patient Acceptable Symptom State (PASS)– Value in a measurement of a patient’s

symptom beyond which the patient considers herself well

– 75%ile of distribution of absolute score among those who are satisfied with their current state after therapy

Tubach F et al: Ann Rheum Dis 2005;64:34-7

State Measures in OA Patients

MCII– Pain 177 (33%)– Function 220 (41%)– Global 136 (25%)

PASS– Pain 241 (44%)– Function 221 (41%)– Global 177 (33%)

Highly significant association between achieving an OMERACT-OARSI Response and having either an MCII or a PASS for each of the 3 domains, especially pain and function.

Knee Rating Scales for Athletic Patients Modified Lysholm Scale Cincinnati Knee Rating System AAOS Sports Knee Rating Scale ADL Scale of the Knee Outcome Survey Single Assessment Numeric Evaluation Knee Injury and OA Outcome Score QoL Outcome Measure for Chronic ACL Def International Knee Documentation Committee

Marx RG: Arthroscopy 2003;19:1103-8

KOOS

Evaluates both short- and long-term consequences of knee injury

42 items in 5 separately scored domains– Pain– Other symptoms– Function in daily living– Function in sport and recreation– Knee-related quality of life

KOOS

Validated in several populations– Surgical reconstruction of ACL– Knee arthroscopy– Meniscectomy 16 years previously– Total knee arthroplasty– Autologous cartilage transplantation

KOOS

Reliable Responsive

– Effect sizes > 1.0 for all 5 subscales in patients undergoing arthroplasty and tibial osteotomy

– Effect sizes > 0.5 for all 5 subscales in patients undergoing ACL reconstruction and meniscectomy

KOOS vs. WOMAC

KOOS contains WOMAC pain, function and stiffness subscales (Likert v3.0)

KOOS adds 18 questions covering sport and recreational function, knee-related quality of life and other symptoms

Larger effect sizes with KOOS– Younger subjects with knee injury– Older subjects with total knee arthroplasty

Summary

There are numerous options for assessing clinically relevant outcomes in trials of products used for cartilage repair

KOOS is the recommended self-report measure of pain, function and QoL– “http://www.koos.nu”

Prevention of the Occurrence of Incident OA Definition of incident OA

– Structural• Arthroscopy• Radiography• MRI

– Symptomatic

ACI Compared with Microfracture in the Knee RCT: 80 patients followed for 2 years Similar clinical outcomes

– Lysholm and VAS pain score

– Significant difference in SF-36 PCS favoring microfracture group

Similar structural outcomes– Arthroscopy

– Histology of cartilage biopsiesKnutsen et al: J Bone Jt Surg 2004;86-A:455-64

Thank you for your time and attention.