rare blood disorder initiative, blood surveillance sept 30, 2006 bruce ritchie hematology,...

Rare Blood Disorder Initiative, Blood Surveillance Sept 30, 2006

Bruce Ritchie Hematology, University of Alberta, Edmonton, Alberta

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.

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

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

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

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

Exchange transfusionSickle Cell Anemia

Exchange TransfusionMore effective to lower % of sickle cells (<30%)Less iron overloadRequires specialized equipment and staff

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

Home RxHemophilia, HAE, PID

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

Kazatchkine, M. D. et al. N Engl J Med 2001;345:747-755

The IgG MoleculeHypogqammaglobulinemias

IgIV OutcomesHeadache, Aseptic Meningitis

Allergy, anaphylaxis

Acute Renal Failure in the elderly, diabetes

Blood Borne Pathogens - hepatitis C in mid 1990s

IgSC Immunoglobulin

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

Subgam - Patient Subgam - Patient Satisfaction SurveySatisfaction Survey After 6 months:

All patients(n=43)

P rior SCIG(n=14)

P rior IVIG(n=29)

Adults (>19 y)(n=23)

Children (<12y) (n=15)

Teenagers(12-19 y) (n=5)

0

10

20

30

40

50

60

70

Perc

enta

ge o

f patients

Much more More Same Not as much

IVIG/SCIG Cost Comparison IVIG/SCIG Cost Comparison ((£)£)

Year 1

•Adult Drug Cost•Routine

Consultations and Tests

•Patient Training•Syringe Drivers•Maintenance

•Total Year 1

SCIG Home Based

10,752

673365

1,762804

14,356

IVIG Hospital Based

10,752

673n/an/a

4,812

16,237

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

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)

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,

FVIII Concentrates purified from up to 100,000 donors

Hemophilia & HIV

QuickTime™ and aTIFF (LZW) decompressor

are needed to see this picture.

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

Transmissible Spongiform Encephalopathies (TSEs)

Rodent TSEHamster, Mouse

Sheep TSEScrapie

Bovine TSEBSE

Cat TSEFSE

Mink TSETME

Deer, Elk TSECWD

Human TSEClassical CJD, GSS, FFI, SPKuru, New Variant CJD

� Normal host protein

� Proteinase K sensitivity

� 33–35 kD MW

PrPC PrPSC

� Associated with infectivity

� Proteinase K partial resistance

� 33–35 kD MW

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

QuickTime™ and aTIFF (Uncompressed) decompressor

are needed to see this picture.

Canadian Hemophilia Assessment and Resource Management information System (CHARMS) & NACHC

Product distribution(CBS)

Clinical outcomes(clinic)

CBS, HQ

CenterPoint server, Hamilton

Genotyping(AHCDC)Sample archive

Health CanadaManufacturersCBS, QBS

Provincial Governments

Drug CompanyPalm Pilot systems

Adverseevents

Productuse/outcomes

PDA systemsViews- Messages

Q ui ckTi me™ and a TI FF (LZW) decompressor are needed to see thi s pi cture.

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

Machine Readable Labels

Freezer Map

Freezer Map

The Inventory Database

QuickTime™ and aPhoto - JPEG decompressor

are needed to see this picture.

Bruce Ritchie

Bruce Ritchie

QuickTime™ and aMotion JPEG OpenDML decompressor

are needed to see this picture.

QuickTime™ and aMotion JPEG OpenDML decompressor

are needed to see this picture.

Bruce Ritchie

Bruce Ritchie

Gene Therapy for point mutations

• Genotyping• Chimeroplasty