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Why do we want “Best Standard Treatments”?

Development of Evidence Based “Best Standard Treatments” in Pediatric Rheumatology Carol A. Wallace MD Professor of Pediatrics University of Washington School of Medicine Seattle Children’s Hospital and Research Institute Immediate Past Chair of CARRA.

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Why do we want “Best Standard Treatments”?

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  1. Development of Evidence Based“Best Standard Treatments” in Pediatric RheumatologyCarol A. Wallace MDProfessor of PediatricsUniversity of Washington School of MedicineSeattle Children’s Hospital and Research InstituteImmediate Past Chair of CARRA

  2. Why do we want “Best Standard Treatments”? • Standardized care results in decreased variation in patient care, which has been shown to result in improved quality and outcomes • Enable comparative effectiveness research to identify the safest, most effective and most cost effective treatments • Prevent complications by use of safest possible treatments • Disseminated widely to improve overall standards of patient care nationwide

  3. 8 6 4 ( ) IRSG NWTSG 2 SWOG Pediatric Division POG CALGB Pediatric Division CCG The National Childhood Cancer Mortality Rate and the Pediatric Cooperative Groups Annual USA Cancer Mortality Rate Children <15 Years Mortality per 100,000, Age- Adjusted COG 1950 1960 1970 1980 1990 2000 2005

  4. How do we develop evidence based Best Standard Treatments? • Analyze data from clinical trials • Analyze data from large prospective cohorts followed from initial presentation with detailed information on treatment and outcomes We have neither available data sources…

  5. In the absence of data, one approach is to start with development of “Consensus Treatment Plans” • Consensus formed; with broad input • Reasonable, common and practical • Once developed, these can facilitate the development of evidence based BestStandard Care by: • Decreasing treatment variation • Enabling analysis of prospectively collected data of presenting features, treatments and outcomes

  6. Comparative Effectiveness Research in Pediatric Rheumatic Diseases: Leveraging CARRA for Improved Child Health “ ” CARRA- In response to NIAMS RFA 05-AR-102

  7. ChildhoodArthritisandRheumatologyResearchAlliance

  8. CARRA is: A research net work ofNorth American Pediatric Rheumatologists and Health Care Professionals dedicated toimproving the treatment and health related outcomes of children with rheumatic diseases through clinical research.

  9. CARRA Site Map

  10. CARRA ORGANIZATIONAL STRUCTURE Elected Steering Committee Chairs of the Disease Committees Small and Large Center Reps, Finance Chair, Past Chair Chair, Vice Chair, JIA Research Committee SLE Research Committee Small Center Rep Large Center Rep JDM Research Committee 304 members (71 trainees) Pain Research Committee Vasculitis/Scleroderma Research Committee Finance Committee Translational Research and Technology Committee 92 SITES www.carragroup.org

  11. What makes CARRA a Network? Member Participation • Disease Specific Committee work • Conduct of Research • Education, training and mentoring • Annual Meeting • Collaborations with other organizations

  12. A Research Network Can… Improve the standard of care through clinical studies Promote a “culture” of research at Pediatric Rheumatology sites Develop Best Standard Treatments for better patient outcomes

  13. Specific Aims CARRA- • To develop multiple standardized treatment plans for use in new onset patients with: JIA, SLE, JDM and localized scleroderma • Use of consensus methodology • Specify of outcome measures, data collection details and time points • Develop mechanisms to enable rapid dissemination and use of the consensus developed treatment plans • Develop analysis plans for the data collected to determine the clinical efficacy and safety of patients treated with these plans

  14. Overall Concept of CARRA- • When a new patient presents, a physician would choose to use one of the standardized treatment plans that most fits what they would normally do. • Data would be collected on the patients treated with these standardized treatments • Data would be analyzed to determine the most efficacious treatment with the least side effects • Thru this process, we would create evidence based “Best Standard Treatment”

  15. CARRA- Carol Wallace (contact PI) Norm Ilowite (PI) JDM Module Ann Reed - Leader Linear Scl Module Rob Fuhlbrigge-Leader Adam Huber Co-leader Suzanne Li Co-Leader JIA Module Yuki Kimura- Leader SLE Module Emily Von Scheven Leader Esi Morgan DeWitt Co-leader Hermine Brunner Co-leader

  16. CARRA- Advisory Board Chair- Edward Giannini Dan Solomon, Brian Feldman, Pamela Weiss, Peter Boehm, Vincent Del Gaizo Dan Solomon Overall Grant Consultant Carol Wallace (contact PI) Norm Ilowite (PI) JDM Module Ann Reed -Leader Linear Scl Module Rob Fuhlbrigge-Leader Adam Huber Co-leader Suzanne Li Co-Leader JIA Module YukiKimura-Leader SLE Module Emily Von Scheven Leader Esi Morgan DeWitt Co-leader Hermine Brunner Co-leader

  17. CARRA- Advisory Board Chair- Edward Giannini Dan Solomon, Brian Feldman, Pamela Weiss, Peter Boehm, Vincent Del Gaizo Dan Solomon Overall Grant Consultant Carol Wallace (contact PI) Norm Ilowite (PI) JDMModule Ann Reed - Leader Linear Scl Module Rob Fuhlbrigge -Leader Adam Huber Co-leader Suzanne Li Co-Leader JIA Module Yuki Kimura-Leader SLE Module Emily Von Scheven Leader Lay Members Lay Members Esi Morgan DeWitt Co-leader Hermine Brunner Co-leader Lay Members Lay Members

  18. Consensus Methodology Choices • Convene a panel of experts • NIH model: • Public discussion of questions and evidence before an expert panel, followed by private unstructured deliberations of the panel • Delphi Questionnaire Technique • Nominal Group Technique • Combination of the Delphi Questionnaires and Nominal Group Technique

  19. Development process for Consensus Treatment Plans ***** Workgroup*****

  20. Development process for Consensus Treatment Plans Initial CARRA wide surveys JIA: new onset systemic JIA SLE: new onset nephritis JDM: new onset moderate to severe disease Localized scleroderma: new onset ***** Workgroup*****

  21. Development process for Consensus Treatment Plans Review of literature ***** Workgroup*****

  22. Treatment of Linear Scleroderma: Survey of Current Practice Case 1:4yo F with 2 mo history of linear scleroderma of lower leg. Lesion crosses ankleand initially had erythematous- violaceousborder. Now warm with SQ tissue loss. Labs show ESR

  23. Treatment of Linear Scleroderma: Survey of Current Practice 4% use MTX only Case 1:4yo F with 2 mo history of linear scleroderma of lower leg. Lesion crosses ankleand nitiallyhad erythematous- violaceousborder. Now warm with SQ tissue loss. Labs show ESR 96% use methotrexate (MTX) + corticosteroid (CS) This looks pretty good. But what about treatment specifics? Li et al. J Rheum 2010;37: 175-81

  24. What type of CS were chosen? Case 1: 4 yo F with linear scleroderma of leg 4% do not use CS How about specific CS doses and dosing regimens?

  25. CS use in LS: Dosing regimens and duration 3 main oral CS regimens: -1 mg/kg/d (28) -2 mg/kg/d (18) -0.5 mg/kg/d (11) 4 IV CS regimens: -3d/mo (33) -1/wk (19) -3d/wk (19) -1/mo (5)

  26. MTX Treatment Regimens in LS 13 different initial doses 27 different initial doses and routes

  27. Development process for Consensus Treatment Plans Review of literature ***** Workgroup*****

  28. CARRA- • Consensus methodologies facilitated and promoted the highest quality team work • However, this is REALLY hard and time consuming!

  29. CARRA-Guiding Principles • Evidence-based when possible • Considered the best available literature • Compatible with the current practice • Assessed by the surveys • Consensus agreement • Delphi Questionnaires • Nominal group technique

  30. Development of ConsensusStandardized Treatments • Distill what we do- using collective judgment and consensus processes • Forced us to: • Think critically about what we do • Use facts when present, not beliefs or “this is how I always do this” • Emphasis on decreased variation

  31. Development of Consensus Standardized Treatments • Not rigorous protocols- but rather menus of standardized treatments • Not perfect or exactly what each physician might do, but what he/she can live with • The goal is to reduce variation so we can treat in a standardized fashion, collect data and compare patient outcomes

  32. Common Module Work • Define the patient characteristics and exclusions for use of the treatment plans • What to do if better, worse or adverse event • Essential data to be collected • Essential labs to be collected • Key data collection time points

  33. Challenges • Steroids • How to standardize dosages and routes • Standardization of steroid taper rate • JIA: • Patient characteristics: how early to allow treatment - may need to treat before fulfilling ILAR criteria for JIA • Include a new medication not yet approved? • SLE: Induction and maintenance, or just induction? • Linear scleroderma: • Lack of outcome measures • Lack of definitions for improvement, worsening

  34. SLE Breakout sessions

  35. Localized Scleroderma Consensus Treatment Plans

  36. JIA Module • Developed 4 treatment plans: • Steroid only • Methotrexate • Anakinra • Tocilizumab • Possible steroid plans : • IV 30/kg/day X 3 days • Oral • 1 mg/kg/day – taper over 1, 3 or 6 months • 2 mg/kg/day – taper over 1, 3 or 6 months

  37. Systemic JIA data collection

  38. Development process for Consensus Treatment Plans Review of literature ***** Workgroup*****

  39. Consensus Treatment Plans developed for new onset: • Systemic JIA • Steroids • MTX +/- steroids • Anakinra +/steroids • Tocilizumab +/steroids • Juvenile Dermatomyositis • Pred + MTX • Pred + MTX + IV methylpred • Pred + MTX + IV methylpred + IVIG • Renal disease in SLE • Cell cept induction • IV cytoxan induction • Linear Scleroderma • MTX • MTX +Oral steroid • MTX + IV methyl pred • All Modules have submitted the Treatment Plans and their • development for publication

  40. Next Steps • Parallel to this effort has been the development of a large foundational pediatric rheumatology registry: CARRAnet • 60 participating pediatric rheumatology sites • 6,000 patients enrolled • Plan is that new onset patients treated with one of the Consensus Treatment Plans will have specific data collected using the CARRAnetplatform

  41. Consensus Treatment Plans • CARRA website Publication progress: • JDM – AC&R • SLE – AC&R • Systemic JIA – revision AC&R • Localized scleroderma – revision AC&R

  42. Implementation of Consensus Treatment Plans • Protocol for the CARRA Registry and Consent form allows for data collection that will cover the basic registry and additional sub-studies. • Actual additional data collection will occur at sites participating in the pilot studies. • All CARRA members are encouraged to use the CTP on new patients, regardless of participation in the pilot studies

  43. Consensus Treatment Plans: Pilot Studies • JDM: Friends of CARRA and CUREJM • 10 sites/ 40 patients • Systemic JIA: Arthritis Foundation • 15 sites/ 30 patients • SLE: Lupus Foundation of America • 15 sites/ 40 patients • Localized Scleroderma: Arthritis Foundation • 11 sites/ 50 patients

  44. Future Steps • Analysis of the data collected through the CARRAnet platform will allow for comparisons between treatments for determination of effectiveness, outcomes and side effects • Evidence based Best Standard Treatments can then be identified and widely disseminated • Over time these can be improved upon in an iterative fashion to improve the outcomes of children with rheumatic diseases • Development of standardized treatment plans for additional phases/categories of disease are underway

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