The document discusses the comprehensive care practices for hemophilia, highlighting historical developments, myths, and current treatment approaches. It emphasizes the importance of collaboration among patients, healthcare providers, and researchers, as well as the establishment of a national patient registry and treatment centers. Innovations in therapy and management tools, such as gene therapy and a modern registry system, are also noted, with ongoing challenges in providing adequate care for all patients.

1. Best Practice Case
Comprehensive Care for Hemophilia
CORD/RDI Conference
Toronto, May 11, 2019
David Page, National Director of Health Policy,
Canadian Hemophilia Society

2. Myths about hemophilia
§ We’re all descended from royalty
§ Hemophilia affects only males
§ People with hemophilia bleed profusely
from minor cuts

3. What is hemophilia?
§ Inherited bleeding disorder
§ Caused by a deficiency in one of the proteins
(factor VIII or IX) needed for blood coagulation
§ Life-long, no cure (???)
§ Symptoms: Bleeding into joints, muscles, soft
tissues and, sometimes, vital organs

4. Bleeding disorders are rare
Incidence Expected
cases in
world
Diagnosed
cases in
world*
Expected
cases in
Canada
Diagnosed
cases in
Canada**
Hemophilia
A
1 in
10,000
770,000 149,764 3600 3018
Hemophilia
B
1 in
50,000
154,000 29,712 720 669
von
Willebrand
disease
1 in 1000
7.7
million
71,648 36,000 4321
Other rare
inherited
bleeding
disorders
1 in
100,000
to 1 in
1,000,000
? 39,495 ? 2057
* 2016 World Federation of Hemophilia Global Survey
** 2018 Canadian Bleeding Disorder Registry

5. Bleeding disorders are rare
Incidence Expected
cases in
world
Diagnosed
cases in
world*
Expected
cases in
Canada
Diagnosed
cases in
Canada**
Hemophilia
A
1 in
10,000
770,000 149,764 3600 3018
Hemophilia
B
1 in
50,000
154,000 29,712 720 669
von
Willebrand
disease
1 in 1000
7.7
million
71,648 36,000 4321
Other rare
inherited
bleeding
disorders
1 in
100,000
to 1 in
1,000,000
? 39,495 ? 2057
* 2016 World Federation of Hemophilia Global Survey
** 2018 Canadian Bleeding Disorder Registry

6. Bleeding disorders are rare
Incidence Expected
cases in
world
Diagnosed
cases in
world*
Expected
cases in
Canada
Diagnosed
cases in
Canada**
Hemophilia
A
1 in
10,000
770,000 149,764 3600 3018
Hemophilia
B
1 in
50,000
154,000 29,712 720 669
von
Willebrand
disease
1 in 1000
7.7
million
71,648 36,000 4321
Other rare
inherited
bleeding
disorders
1 in
100,000
to 1 in
1,000,000
? 39,495 ? 2057
* 2016 World Federation of Hemophilia Global Survey
** 2018 Canadian Bleeding Disorder Registry

7. Bleeding disorders are rare
Incidence Expected
cases in
world
Diagnosed
cases in
world*
Expected
cases in
Canada
Diagnosed
cases in
Canada**
Hemophilia
A
1 in
10,000
770,000 149,764 3600 3018
Hemophilia
B
1 in
50,000
154,000 29,712 720 669
von
Willebrand
disease
1 in 1000
7.7
million
71,648 37,000 4321
Other rare
inherited
bleeding
disorders
1 in
100,000
to 1 in
1,000,000
? 39,495 ? 2057
* 2016 World Federation of Hemophilia Global Survey
** 2018 Canadian Bleeding Disorder Registry

8. Current situation in the world
75% of people with hemophilia
receive little or no treatment
§ Life expectancy: 20 years
§ Those who survive suffer
terrible pain and become
physically disabled

9. Hemophilia in the 1950s: Before effective care
In 1953, a small group of
hemophiliacs, their families and
physicians founded the Canadian
Hemophilia Society in Montreal.
Their dream? To improve the
quality of life and to find a cure.
Success factor # 1: close
collaboration among patients,
health care providers and
researchers

10. Beginning of a network of centres
The best treatment was limited to
a few hospitals in major centres
like Montreal, Toronto and
Vancouver.
The Canadian Hemophilia Society
created a Medical and Scientific
Advisory Committee to begin to
build a national network.

11. National (and international) collaboration
Success factor #
2:
National (and
indeed
international)
collaboration
among health
care
professionals
and patient
organizations

12. Hemophilia home care cuts costs,
hospitalizations (MD of Canada, 1972)

13. Data kicks!
1972: Prospective, randomized study comparing
home care vs. hospital care in 40 patients
§ 85% reduction in hospitalizations
§ 87% reduction in costs
§ 50% reduction in “time to treat”
§ 2.5 days lost from school per hemorrhage compared to
6.8 days
§ 97% of patients and families preferred home infusion
Success factor # 3: Self/family administration
of therapeutics

14. Systematic family education
Success factor #4: Education of patients, families and
health care providers

15. Home infusion
Today, more than
90% of clotting
factor concentrates
are administered in
the home by the
patient or his/her
caregiver.
A specialized
nurse coordinator
is key!

16. Next challenge: A registry
In the summer of 1977, the
CHS hired young people with
hemophilia to go across the
country to register other
people with hemophilia. The
first registry was created. We
could now make a case to
government to set up
“hemophilia treatment
centres.”
Success factor # 5: A
national patient registry

17. A Network of HTCs
Between 1978 and 1980, 24 hemophilia treatment
centres were set up across the country. They included:
§ A physician
§ A nurse coordinator
§ A physiotherapist
§ A social worker
§ Access to an orthopedic surgeon and dentist
§ Other specialties as needed
Success factor # 6: Comprehensive, inter-disciplinary
care (integrated care)
NHF-McMaster Guideline on Care Models for Haemophilia Management
Haemophilia (2016), 22 (Suppl. 3), 6–16 DOI: 10.1111/hae.13008

18. Other features of comprehensive care
Success factor # 7:
Provincial designation
Success factor # 8:
Decentralization through outreach
Success factor # 9:
Flexibility in organization

19. Too good to be true

20. Success factor # 10: collaborative research
Between 1990 and
2018, CHS invested
$8,000,000 in…
§ Fundamental
research
§ Clinical research
§ Fellowships and
studentships

21. Benefits of research agenda
§ New knowledge
§ Improved care
§ Increased interest
among clinicians/
researchers
§ Closer links between
patient organization
and physicians
§ Hope for community

22. Success factor # 11: Standards of care
In 2007, the patient,
physician and allied
health care provider
groups adopted …

23. Standards of care
Standards include:
§ Diagnostic and therapeutic principles of
care
§ Populations served
§ Description of the care team
§ Services, responsibilities of a treatment
centre
§ Scope of care (& key indicators)
§ Quality measures (& key indicators)
§ Therapeutic services (& key indicators)

24. Program evaluation and accreditation
In 2014, the CHS evaluated 25 of the 26 HTCs
across the country, using…
§ Individual interviews with clinic staff
§ A patient satisfaction survey
Success factor # 12, program evaluation,
remains to be fully achieved

25. How far we’ve come

26. … but we’re not there yet
Innovative therapies
Factor VIII and IX therapies with 1.5 to
5 times the half-life
2016
Emicizumab (a monoclonal antibody
that mimics factor VIII)
Thursday (in
Quebec)
Fitusiran (a RNAi therapy that down
regulates anti-thrombin to re-balance
hemostasis), anti-TFPIs
2021?
Gene therapy using a AAV vector to
deliver the gene to make factor VIII and
IX
2021?

27. … but we’re not there yet
21st-century tools to better manage
care

29. Today’s Canadian Bleeding Disorder Registry
§ Total registered: 10,065 patients
§ Anonymous (unique identifier)
§ Run by physician group
§ Public website (www.ahcdc.ca/cbdr)
4 purposes
§ Typical disease registry
§ Electronic patient record for the treatment
centres
§ Patient home reporting tool via computer,
tablet, smartphone for bleeds and infusions
§ Database for research