Workshop on increasing awareness concerning primary immunodeficiencies held in Florence, Italy in October 2012.

1. Esther
de
Vries
Anna
Sediva
e.d.vries@jbz.nl
anna.sediva@lfmotol.cuni.cz
esid@estherdevries.nl
www.estherdevries.nl
Increasing
awareness
of
PID
worldwide
Workshop
by
ESID-­‐INGID-­‐IPOPI
Jointly
organised
by
ESID
Working
Par7es
for
Educa7on
and
PID-­‐care
in
development

2. Workshop
program
• IntroducBon
&
example
by
Esther
de
Vries
• European
Immunoglobulin
Map
by
Anna
Sediva
• World
PI
Week
2012
by
Jose
Drabwell
• Ways
to
address
other
specialists
/
specialBes
by
Mikko
Seppänen
• “Care
Path
for
Immunodeficiency”
in
general
hospitals
by
Esther
de
Vries
• EducaBon
through
distance
learning
strategies
by
Peter
Vickers
• How
to
organize
awareness
with
limited
resources
by
Lokman
Mohd
Noh
• Let’s
work
together
to
define
suitable
signals
for
awareness
by
everyone

3. Faculty Disclosure
relevant issues dr. Esther de Vries
Company Nature of Affiliation
• Companies: CSL Behring,
• Honoraria, expenses
Sanquin
• Company: Baxter • Funded research
Off-Label Product Usage
• None

4. Electronic
voBng
–
QuesBon
1
(grouping
quesBon)
I
am
a?ending
the
meeAng
of
:
1.
ESID
2.
INGID
100
3.
IPOPI
75
48,6
4.
I
am
(related
to)
a
sponsor
50
25,7
25 17,1
8,6
0
1 2 3 4

5. Electronic
voBng
–
QuesBon
2
I
am
working
in
:
100 100
75 75
1.
paediatric
–
clinical
50 44,4 50
50
ONLY
27,8
25
30
25 16,7
11,1 10 10
0 0
0 0
2.
adult
–
clinical
ONLY
1 2 3 4 5 1 2 3 4 5
ESID INGID
3.
paediatric
–
clinical
AND
lab
/
research
100 100
75 75
50 50
4.
adult
–
clinical
AND
lab
/
50
33,3 33,3 33,3
50
research
25 25
0
0 0 0 0
0 0
1 2 3 4 5 1 2 3 4 5
5.
lab
/
research
ONLY
IPOPI SPONSOR

6. Electronic
voBng
–
QuesBon
3
100 100
83,3
75 75
58,3
I
have
a
:
50 41,7 50
25 25 16,7
1.
junior
/
trainee
0
1 2
0
1 2
posiBon
ESID INGID
2.
established
/
senior
100 100
posiBon
75 66,7 75
75
50 50
33,3
25
25 25
0 0
1 2 1 2
IPOPI SPONSOR

7. IntroducBon
&
example
Esther
de
Vries
ESID
-­‐
SAS

8. Awareness
of
PID
is
low
–
but
recogniBon
is
important
• Individual
PIDs
are
rare
• But
altogether
PIDs
make
up
a
substanBal
number
of
affected
paBents
in
Europe
and
worldwide
(esBmate:
1
in
8-­‐10,000
people)
• PID
paBents
have
more
infecBons,
but
‘normal’
people
also
suffer
from
infecBons
• PIDs
have
a
geneBc
basis
• When
you
come
from
a
PID
family,
the
chances
you
have
PID
too
are
not
small
• PIDs
can
be
treated
(anBbioBcs,
anBfungals,
anBvirals;
immunoglobulins;
G-­‐CSF;
C1-­‐inh;
SCT;
gene
therapy)
• The
prognosis
of
paBents
with
PID
depends
on
Bmely
recogniBon
&
treatment

9. Example
of
an
awareness
campaign:
www.alAjdziek.nl
in
the
Netherlands

10. Electronic
voBng
–
QuesBon
4
100 100
We
have
a
web-­‐ 75
55,6
75
55,6
based
awareness
50 50
campaign
for
PID
in
25
22,2 22,2
25
27,8
16,7
our
country
:
0 0
1 2 3 1 2 3
1.
yes
ESID INGID
100 100
2.
no
75 65,5 75 66,7
50 50
33,3
3.
I
don’t
know
25
27,6
25
6,9
0
0 0
1 2 3 1 2 3
IPOPI SPONSOR

11. Electronic
voBng
–
QuesBon
5
I
think
it
is
important
100 100
to
have
a
web-­‐based
72,2
75 75
awareness
campaign
50 50
for
PID
in
my
country
:
50 50
27,8
25 25
0 0 0 0
1.
yes,
very
important
0 0
1 2 3 4 1 2 3 4
ESID INGID
2.
it
would
be
nice
100 100
83,3
78,1
75 75
3.
not
really
50 50
25 18,8 25
4.
no,
it
is
useless,
a
16,7
3,1 0 0 0
waste
of
Bme
and
0
1 2 3 4
0
1 2 3 4
money
IPOPI SPONSOR

12. European
Immunoglobulin
Map
Anna
Sediva
ESID

13. European Immunoglobulin Map
Issues on access to treatment for PID
patients in Europe
Outcome of questionnaire / survey in
European countries
ESID - Anna Sediva, Klaus Warnatz, Helen Chapel

14. European Immunoglobulin Map
Goals
§ to create an overview on availability of EUROPE
Ig MAP 2006 Iceland
immunoglobulin and other PID treatments fully covered
Sweden
Finland
in European countries
no information
Norway
Estonia
not covered
Latvia
Denmark
Lithuania
Ireland
Netherlands
United
Belarus
Kingdom
§ to monitor progress and improvement
Poland
Germany
Belgium
Ukraine
Czech
Rep
Luxembourg Slovakia
France Moldova
Austria Hungary
Switzerland
Slovenia Romania
Liechtenstein Croatia
Bosnia Serbia
Portugal Italy and
§ to use the „European Immunoglobulin
Herzegovina Bulgaria
Spain
Macedonia
Greece Montenegro
Map“ as a tool for negotiations with EU/ Albania
national authorities in order to reach full
availibility of treatment for each PID
patient

15. EUROPE IMMUNOGLOBULIN MAP
IVIG 2011 SCIG 2011
80
40 20
40
50
30
5
40 70 1
20
No reply 35 10
75
available
Available
but not
used much Comments:
only one product N percentage of SCIG substitution
Unavailable

16. EUROPE IMMUNOGLOBULIN MAP 2012
IVIG 2012 SCIG 2012
full availibility
limitation
no availibility
no response

17. EUROPE 2012
IVIG/SCIG ratio
100%IVIG adults children
50%IVIG
10%IVIG
10/90 50/50 100/0 90/10 100/0
10/90 10/90
10/90
60/40
50/50
33/67 100/0
100/0 100/0
60/40 95/5 70/30 10/90 85/15 70/30
50/50 50/50
70/30 70/30 80/20
90/10 75/25 90/10
75/25 40/60 90/10 60/40
100/0 100/0 100/0 100/0
100/0 100/0
100/0 90/0
85/15 80/20 100/0 100/0 45/55 75/25 80/20 90/0 100/0
55/45

18. Electronic voting – question 6
I think 100 100
immunoglobulins will 75 75
53,3
be available for all 50
37,5
50
50
PID patients in 25
12,5
25
26,7
20
Europe by 2020: 0
0
0
0
1 2 3 4 1 2 3 4
1. yes, in Western
ESID INGID
Europe
100 100
2. yes, all over 75 75
Europe 50 50
50
32,3 33,3
29
22,6
25 16,1 25 16,7
3. no 0
0 0
1 2 3 4 1 2 3 4
4. I don‘t know
IPOPI SPONSOR

19. Electronic voting – question 7
100 100
I think 75 75
immunoglobulins 50 44,4 50
52,9
will -by 2020- 33,3
22,2
35,3
25 25
preferentially 0 0
11,8
be used as: 0
1 2 3 4
0
1 2 3 4
1. SCIG ESID INGID
100 100
2. IVIG 75 75
59,4
50
50 50
3. Equally 33,3
25 18,8 25 16,7
9,4 12,5
0
4. I don‘t know 0
1 2 3 4
0
1 2 3 4
IPOPI SPONSOR

20. World
PI
Week
2012
Jose
Drabwell
IPOPI

21. WORLD PRIMARY
IMMUNODEFICIENCY WEEK

22. World Primary Immunodeficiency Week
22 – 29 April 2011 and 2012
q Global awareness campaign
q All PID stakeholders
q Doctors, patients, nurses, industry
q 28 – 2011 and 32 – 2012 (13 and 17 IPOPI)
q Media outreach campaigns
q Medical & public awareness campaigns
q Advocacy campaigns
q Targeted events
q Publications / Scientific editorials
q Arabic, German, Italian, Polish, Portuguese,
Spanish, Serbian

23.
Participating Countries
Argentina
Australia Belgium
Brazil Canada Colombia
Czech Republic Egypt France
Germany Greece Hungary
India Iran Italy
Japan Mexico Morocco
Netherlands Peru Poland
Portugal Puerto Rico Serbia
Slovakia South Africa Spain
Sweden Turkey UK
USA Venezuela

24. WPIW Mission
Drive recognition of primary immunodeficiencies (PI) as an
increasingly important disease group;
Increase understanding amongst medical professionals,
researchers, nurses, patients, school teachers, day care
employees and the public;
Stimulate efforts to improve recognition, diagnosis, treatment
and quality of life of people living with PI world-wide;
Encourage health authorities to use awareness materials
produced by IPOPI and other stakeholders to promote timely
diagnosis and treatment;
Promote the model of combining physician education and
global awareness with the infrastructure to diagnose and treat PI.

25. WPIW 2012 Poster

26. Electronic voting – question 8
WHAT IF? 100 100 100 100
75 75
All the 50 50
stakeholders in 25 25
each country 0
0
0
0
collaborated in 1 2 1 2
this campaign?
ESID INGID
Would this then
make a real 100 93,5 100 100
difference 75 75
to raising 50 50
awareness? 25 25
6,5
0
0 0
1. YES 1 2 1 2
2. NO
IPOPI SPONSOR

27. Ways
to
address
other
specialists
/
specialBes
Mikko
Seppänen
ESID

28. ConsulBng
other
specialBes
–
do
our
colleagues
have
a
chance
to
be
aware
of
PIDDs?
• 16
standard
textbooks,
various
specialBes
• Some
textbooks
sBll
lack
chapters
on
PIDDs
– When
existed,
were
well
wrinen,
concise
and
accurate.
– With
a
few
posiBve
excepBons,
PIDDs
were
poorly
integrated
into
the
remaining
body
of
text
and
tables.
– No
textbook
covered
full
spectrum
of
CVIDs
syndromic
findings,
possibly
due
to
space
constraints?
– Inaccuracies
on
PIDD-­‐related
findings
were
frequent
in
texts
(outside
a
specific
chapter).

30. INFECTIONS AUTOIMMUNE DISEASES
Sinusitis Meningitis
Lungs
Eye
Otitis & conjuctivitis
Thyroid
Pneumonias
Liver and biliary Lymphatic tissue
Blood
Infectious diarrhea
Other Gastrointestinal
Skin
Other
Mikko Seppänen / HumanArt Helena Schmidt Tmi. Sponsored by Sanquin Finland, open domain

31. Electronic
voBng
–
QuesBon
9
The
syndromic
nature
and
variability
of
CVIDs
and
their
manifestaBons
100 100 92,9
may
not
be
well
covered
in
88,9
textbooks.
As
shown,
Early
diagnosis
75 75
of
even
the
most
common
life-­‐
threatening
PIDDs
(CVIDs)
may
be
50 50
delayed
and
PIDD
physicians
may
have
difficulBes
in
communicaBng
25 25
with
colleagues
in
other
fields
of
11,1
7,1
0 0 0 0
medicine
due
to
omissions
in
0 0
standard
textbooks.
1 2 3 4 1 2 3 4
To
promote
learning,
anAbody
ESID INGID
deficiencies
should
be
described
in
textbooks
with
100 100 100
the
aid
of
concise
figures:
74,2
75 75
1.
yes,
I
agree
50 50
25 25
12,9 12,9
2.
maybe
0 0 0 0
0 0
1 2 3 4 1 2 3 4
3.
probably
won’t
help
4.
no,
that
won’t
work
IPOPI SPONSOR

32. “Care
Path
for
Immunodeficiency”
in
general
hospitals
Esther
de
Vries
ESID

33. “Care
Path
for
Immunodeficiency”
in
general
hospitals
Results
of
QuesBonnaires
Data
Care
Path
history
&
tests
Online
StaBsBcal
PublicaBons
system
analysis

34. EducaBon
through
distance
learning
strategies
Peter
Vickers
INGID

35. Education through the internet and
other distance learning strategies
DR. PETER VICKERS

36. WHY?
Ò The cheapest and easiest way to communicate
and interact with the highest number of people.
Ò Can easily allow for two-way dialogue.
Ò Can utilise many types of media – oral, written
word, pictures/diagrams, animation.
Ò Proven method of teaching and involving
people in diverse and distant countries/
communities.
Ò Can be tailored to individual needs.

37. WHAT?
Ò Sound/visual lectures, e.g. Immune Deficiency
Foundation
Ò Interactive teaching programmes, e.g. http//
ig.clinicalcommunities.net
Ò Interactive information giving/receiving
Ò Self-help groups
Ò Professional/patient organisations/groups, e.g.
INGID
Ò Educational establishments, e.g. University of
Hertfordshire
Ò In-hospital/medical centre teaching
Ò On-line groups/on-line case studies, e.g. LinkedIn
Ò Skype

38. WHAT TO CONSIDER?
Ò Access to equipment and electricity to run the
equipment.
Ò Technophobia.
Ò Different languages.
Ò Cost.
Ò Time.
Ò Standardisation/credibility of information.
Ò Regular updating.
Ò Enthusiasm.

39. Electronic voting – Question 10
OPINION - DISTANCE LEARNING:
100 100
88,9
1. is the only way
forward for the 75 75
60
dissemination of 50 50
information about 40
immunology and 25 25
PID. 0 0
11,1
0 0
0 0
1 2 3 4 1 2 3 4
2. is a waste of time
ESID INGID
3. could be very
useful as long as it 100 94,4 100
is combined with 75 75
75
face-to-face
teaching 50 50
25
25 25
4. has not totally 5,6
0 0 0 0
convinced me, but 0
1 2 3 4
0
1 2 3 4
I am prepared to
try it.
IPOPI SPONSOR

40. How
to
organize
awareness
with
limited
resources
Lokman
Mohd
Noh
ESID

41. How
to
organize
awareness
when
limited
resources
are
available
LOKMAN
MOHD
NOH
Pediatric
Immunologist
,
previously
University
Science
Malaysia
Many
countries
in
South
East
Asia
would
encounter
limitaBon
of
resources.
Malaysia,
populaBon
28,728,607
(July
2011
est.);
0-­‐14
years:
29.6%
PID
esBmates
that
significantly
affects
health
(based
on
prevalence
of
1:
8,000-­‐
10
,000)1
Malaysia
expected
à
2900
-­‐
3500
PID
paAents
(Singapore
2.7
per
100,000
)Lim
etal
2003
My
PIN
diagnosed
150
PID
up
to
Apr
2012
2
clinical
Immunologist
(
1
per
14mill);
InfecBous
disease
consultant
(19)
in
2009.
Ped
immunologist:
Ped
InfecBous
Diseases
specialist
[1:
10]
TerBary
clinical
immunology
lab
3
(Penang,
KL,
Kota
Bharu)
Hospital
with
terBary
faciliBes
for
PID
clinic
-­‐
3.
1.European
PID
Expert
group.
Chair
Jorgo
Chatzimarkakis
2009

42. Strategy-­‐
collaboraBve
effort
Local
(
NPII-­‐
My
PIN)
InternaAonal
(NACLIS)
NaAonal
clinical
Immunology
symposium
• 1987-­‐1993
1
ped
immunologist
&
1
immunopathologist
[HKL(paBent
care)
&
IMR(Lab)]
• Sponsored
Univ
Sains
Malaysia,
co
host
• 1994-­‐1999
(
1
ped
Imm(LMN)&
1
lab
Immu)
HUSM
Malayan
Society
Allergy
&
Immunology
&
Lab
• Invited
guest
faculty
from
HK
,
UK
&
Australia
• 2007-­‐2011
(2
ped
Immunologists(LMN+AHAL)
).
to
above
sympopsium
yearly
since
2009
AMDI
Penang/UPM
Serdang
• 2007
Nat
PID
iniBaBve
(NPII)
`
interacBve
• Difficult
cases
are
discussed
with
invited
cooperaBon
clinician
&
Immunopath
&
lab
scienBst
Guest
during
a
dedicated
sessions
• 2009
Malaysian
primary
immunodeficiency
network
•
assistance
from
referred
labs
for
special
test
(MyPIN)
replacing
NPII
• AcAvity:
Pediatricians
&
Ped
InfecAous
Dis
Specialist
refers
to
3
centres:
a)
H
Sg
Petani
DIRECTION
:
1. b)
H
USM
Kota
Bharu
(a&
b)
LMN
as
visiAng
• To
get
clinical
immunology
subspecialty
c)
H
K
Lumpur
(AHAL
&
LMN)
credenBalled
&
to
allow
training
g
program
part
local
/Overses
Awareness
creaAon
;
meeAngs
Ped
Soc
• Gezng
criBcal
mass
of
clinical/physician
– Immunological
society
immunologists
&
immunopathologists
– Media
/
books
/proceedings
• Building
terBary
infra
structures
• CollaboraBve
research

43. Total
number
of
PID
cases
from
1986-­‐2011
(1
clin
immunologist)
(2
Clin
Immunologists)
(1.2x (10 x
increase) increase)
Average 2.5/yr 24 case / yr
3 cases/ yr
N=108
N=30
up
to
July
HKL-­‐UPM
HUSM
(2
centres)
HUSM,
UKM-­‐HKL
HSAH, HKL,
(3 centres)

44. PID
occurs
in
South
East
Asia
but
resources
are
limited
1. Create
awareness
(
Local
bodies
with
ESID)
2. Generous
support
(
clinical
&
invesAgaAons)
With
InsAtuAons
,
with
potenAal
for
collaboraAon
3.
Teaching
sBnts
in
countries
with
limited
resources
supporBng
specialty
program
As
areas
of
interest
or
on
its
own.
.

45. PROVOKING
STATEMENT
• PID
occurs
in
S
E
Asia
/ASEAN
region
with
a
populaBon
of
almost
500,000
million.
• The
morbidity
and
mortality
of
PID
is
high
especially
with
delayed
recogniBon.
• Coupled
with
limited
resources
the
situaBon
could
be
catastrophic
for
the
PID
paBent.
• The
ESID
group
has
led
the
way
by
having
iniBated
this
workshop,
more
could
be
done
to
assist
in
providing
improved
care
for
their
PID
paBents

46. Electronic
voAng
–
QuesAon
11
100 100 100
ESID
/
INGID
/
90
75 75
IPOPI
can
help
to
50 50
change
PID
25 25
10
0
awareness
0
1 2
0
1 2
and
care
in
ESID INGID
countries
like
Malaysia:
100 93,8 100 100
75 75
1.
yes
50 50
25 25
6,3
0
0 0
2.
no
1 2 1 2
IPOPI SPONSOR

47. Everyone

48. Let’s
work
together
to
define
suitable
signals
for
awareness
• Discussion!

49. Esther
de
Vries
Anna
Sediva
e.d.vries@jbz.nl
anna.sediva@lfmotol.cuni.cz
esid@estherdevries.nl
www.estherdevries.nl
Thank
you
for
your
a?enAon!