Rare Blood Disorder Initiative, Blood Surveillance Sept 30, 2006

1. Rare Blood Disorder Initiative, Blood Surveillance Sept 30, 2006 Bruce Ritchie Hematology, University of Alberta, Edmonton, Alberta

2. Disclosure Honoraria: Bayer, Baxter, Sanofi, Astra-Zeneca, Novo-Nordisk, Aventis-Behring, ZLB, Pharmacia, Pfizer - all go towards studentships Studentships: Pharmacia, Baxter, Sanofi, Novo-Nordisk, Bayer Travel Support: Bayer, Baxter, Astra-Zeneca, Novo-Nordisk, Pharmacia, Canadian Hemophilia Society, Canadian Hereditary AngioEdema Society, Canadian Immunodeficiency Patient Organization, Alberta Association of Radiologists, Research Funding: Bayer, Baxter, Novartis, NovoNordisk, CHS, Health Canada/Public Health Agency of Canada.

3. Outline of my presentation Rare Blood Disorder Initiative What is it? What is needed? How do we get it? Treatment surveillance Networks in place in Canada Sample collection/testing Inhibitors, HIV BBPSP

4. Rare Blood Disorders Initiative What is it? It is: specialized care for rare disorders including: specialized diagnostics specialized management - immunizations, … specialized tracking of outcomes surveillance

5. Rare Blood Disorders InitiativeHemophilia Untreated Hemophilia - median survival of 25 Optimal management - near normal life span Currently we have close to optimal management Available: Clinic for early diagnosis, treatment, tracking, surveillance

6. Rare Blood Disorders InitiativeSickle Cell Anemia Untreated Sickle cell anemia - median survival of 25 Optimal management - near normal life span Currently we have something in between. Needs: Better and earlier diagnosis, treatment Better tracking Better transfusion management Better chelation management

7. Exchange transfusionSickle Cell Anemia Exchange Transfusion More effective to lower % of sickle cells (<30%) Less iron overload Requires specialized equipment and staff

8. Rare Blood Disorders InitiativeHereditary AngioEdema Undiagnosed HAE common Mistreated HAE common - antihistamines, steroids, adrenalin don’t work Optimal management - near normal life span Currently we have something in between. Needs: Better and earlier diagnosis, treatment Better tracking Home management

9. Home RxHemophilia, HAE, PID

10. Rare Blood Disorders InitiativePrimary Immnodeficiency Undiagnosed PID common Mistreated PID common Optimal management - near normal life span Currently we have something in between. Needs: Better and earlier diagnosis, treatment Better tracking Home management - IgSC

11. Hypogqammaglobulinemias The IgG Molecule Kazatchkine, M. D. et al. N Engl J Med 2001;345:747-755

12. IgIV Outcomes IgSC Immunoglobulin Headache, Aseptic Meningitis Allergy, anaphylaxis Acute Renal Failure in the elderly, diabetes Blood Borne Pathogens - hepatitis C in mid 1990s 16% IgSC solutions licensed in Europe (ZLB, CSL), IgIm preparation licensed here (Talecris) Unlicensed (SAP) vs “Off-label” use of IgIM or IGIV for SC

13. Subgam - Patient Satisfaction Survey • After 6 months:

14. Year 1 Adult Drug Cost Routine Consultations and Tests Patient Training Syringe Drivers Maintenance Total Year 1 SCIG Home Based 10,752 673 365 1,762 804 14,356 IVIG/SCIG Cost Comparison (£) IVIG Hospital Based 10,752 673 n/a n/a 4,812 16,237

15. Rare Blood Disorders InitiativeBone Marrow Failure Undiagnosed mild disease common, but there is associated morbidity Specific treatment not readily available except in trials Currently we have something in between. Needs: Better and earlier diagnosis, treatment Better tracking Better transfusion management Better chelation therapy - ?Exjade

16. Rare Blood Disorders Initiative What is needed? A clinic with dedicated staff consisting of: 1. nurse, admin assistant, data entry person, physician 2. space - offices, stuff, storage, computers 3. Oversight - Multidisciplinary group (patients)

17. Rare Blood Disorders Initiative How do we get it? Lobbying - governments don’t listen to phsycians, so patients must lobby on their own behalf Organize meet, talk, argue, reach consensus establish policy - Rx, safety, … Network of Rare Blood Disorder Organizations,

18. Hemophilia & HIV FVIII Concentrates purified from up to 100,000 donors

19. http://www.hc-sc.gc.ca/english/protection/krever/

20. Human TSE Classical CJD, GSS, FFI, SP Kuru, New Variant CJD Rodent TSE Hamster, Mouse Bovine TSE BSE Sheep TSE Scrapie Deer, Elk TSE CWD Mink TSE TME Cat TSE FSE Transmissible Spongiform Encephalopathies (TSEs) PrPC PrPSC • Associated with infectivity • Proteinase K partial resistance • 33–35 kD MW • Normal host protein • Proteinase K sensitivity • 33–35 kD MW

21. Treatment surveillance Networks in place in Canada Quebec Blood Surveillance Network Health Canada AHCDC - BBPSP Sample collection/testing Inhibitors, HIV BBPSP - Blood Borne Pathogens Surveillance Project

23. Canadian Hemophilia Assessment and Resource Management information System (CHARMS) & NACHC Drug Company Palm Pilot systems Product distribution (CBS) Clinical outcomes (clinic) Genotyping (AHCDC) Sample archive CBS, HQ Product use/outcomes Provincial Governments CenterPoint server, Hamilton Adverse events Health Canada Manufacturers CBS, QBS

24. on handheld device Views - Messages PDA systems • Dialog - Bayer (formerly HemaScan) • Began as a PDA based inventory system • Barcodes generated by system • Now includes internet module • Currently in trial in Hamilton, Ste. Justine, Edmonton • Data management Arrowhead • Advoy - Baxter • Began as a web based management system • Will use consensus barcodes • Now includes PDAs • Currently in trial in Toronto, Edmonton • Data management 3rd party

25. Machine Readable Labels

27. Freezer Map

28. Freezer Map

29. The Inventory Database

30. Bruce Ritchie

31. Bruce Ritchie

32. Bruce Ritchie

33. Bruce Ritchie

34. Gene Therapy for point mutations • Genotyping • Chimeroplasty