Cystic Fibrosis in Europe – Facts and Figures 2014

The European Cystic Fibrosis Society Patient Registry (ECFSPR) is happy to present this report with key information about how cystic fibrosis (CF) affects people with CF and their families throughout Europe.

The ECFSPR collects, measures and compares data of people with CF living in Europe and neighbouring countries who agree to be in the registry. The information is important to better understand CF, encourage new European standards of care and treatment, conduct research, and inform public health-planning.

If you want to know more about the ECFSPR visit our website.

For more in depth information see the full report here.

November 2016

At-A-Glance report 2014

At-A-Glance report 2014

Map of countriesthat contributed 2014 data

to the ECFSPR

CH782

FR6369

DE5003 CZ

599

AT712SI97

NL1378

BE1186

DK488

SK290HU557 RO

44

MD61

UA147

RU2122

LT12

LV38

SW626

GR517

IL666

RS169

MK109

IT4981

ES1538

PT295

UK10581

IE1218

registered patients in 26 countries number of patients on map

35,582

At-A-Glance report 2014

48.2%Children

51.8%Adults

2

3.6MEDIAN AGE ATDIAGNOSIS

months

2

Proportion children-adults

• The proportion children-adults varies between the countries.• For some countries only a few individual centres sent data to the ECFSPR.

Genetics

F508del mutation in EuropeMutation F508del in Europe100

90

80

70

60

50

40

30

20

10

0

Aust

ria

Czec

h Re

publ

ic

Denm

ark

Fran

ce

Gre

ece

Hun

gary

Irel

and

Isra

el

Ital

y

Latv

ia

Lith

uani

a

Mac

edon

ia

Mol

dova

Net

herl

ands

Port

ugal

Rom

ania

Russ

ian

Fede

rati

on

Serb

ia

Slov

ak R

epub

lic

Slov

enia

Spai

n

Swed

en

Swit

zerl

and

Ukr

aine

Uni

ted

King

dom

Euro

pe

%

F508del heterozygote

F508del homozygote

Other mutations

Mutation F508del in Europe100

90

80

70

60

50

40

30

20

10

0

Aust

ria

Czec

h Re

publ

ic

Denm

ark

Fran

ce

Gre

ece

Hun

gary

Irel

and

Isra

el

Ital

y

Latv

ia

Lith

uani

a

Mac

edon

ia

Mol

dova

Net

herl

ands

Port

ugal

Rom

ania

Russ

ian

Fede

rati

on

Serb

ia

Slov

ak R

epub

lic

Slov

enia

Spai

n

Swed

en

Swit

zerl

and

Ukr

aine

Uni

ted

King

dom

Euro

pe

%

F508del heterozygote

F508del homozygote

Other mutations

• F508del is the most common CF-causing mutation in Europe.

• People with CF have two CF-causing mutations, one inherited from the mother and one from the father.

• Homozygous: both mutations are the same.

• Heterozygous: the two mutations are different.

Children and adults with CF in EuropeChildren and adults with CF in Europe100

90

80

70

60

50

40

30

20

10

0

Aust

ria

Belg

ium

Czec

h Re

publ

ic

Denm

ark

Fran

ce

>=18 Years

<18 Years

%

Gre

ece

Hun

gary

Irel

and

Isra

el

Ital

y

Latv

ia

Lith

uani

a

Mac

edon

ia

Mol

dova

Net

herl

ands

Port

ugal

Rom

ania

Russ

ian

Fede

rati

on

Serb

ia

Slov

ak R

epub

lic

Slov

enia

Spai

n

Swed

en

Swit

zerl

and

Ukr

aine

Uni

ted

King

dom

Euro

pe

Children and adults with CF in Europe100

90

80

70

60

50

40

30

20

10

0

Aust

ria

Belg

ium

Czec

h Re

publ

ic

Denm

ark

Fran

ce

>=18 Years

<18 Years

%

Gre

ece

Hun

gary

Irel

and

Isra

el

Ital

y

Latv

ia

Lith

uani

a

Mac

edon

ia

Mol

dova

Net

herl

ands

Port

ugal

Rom

ania

Russ

ian

Fede

rati

on

Serb

ia

Slov

ak R

epub

lic

Slov

enia

Spai

n

Swed

en

Swit

zerl

and

Ukr

aine

Uni

ted

King

dom

Euro

pe

At-A-Glance report 2014 3

18%41%41% F508del homozygote

F508del heterozygote

Other mutations

Belgium is included only in this graph

Median means that50% of the values are above and

50% are below this value.

BMI or body mass index is a measure for nutritional status:

weightheight2

z-score indicates how far a value is from the average (mean) value of the reference population.

BMI

Lung function

Nutrition

• FEV1 is a measure for lung function. It is the maximum amount of air that can be forcefully exhaled in the first second after taking a deep breath.

• FEV1% is a percentage of the average value for healthy people of the same age, gender and height, which is set at 100%.

• In people with CF aged 45 and older there is a higher incidence of mutations causing milder forms of CF. This influences the lung function value for the people with CF in this age group represented.

* In the 2014 report we changed to a new way of calculating lung function; this means that, from now on, ECFSPR results can be easily compared with those of other CF registries around the world.

• A z-score of 0 means that the BMI is the same as the BMI of healthy children of the same gender and age.

• From the age of 8, children with CF have a lower average BMI than healthy children of a similar age.

• Healthy peers have values for BMI between 18 and 25.

• As people with CF get older BMI increases.

• In adults with CF aged 38 and older there is a higher incidence of mutations causing milder forms of CF. This influences the BMI for the adults in this age group represented.

Median FEV1% of predicted in adults

110

100

90

80

70

60

506-9 10-14 15-19 20-24 25-29 30-34 35-39 40-44 45+

Age

FEV1

% of

pre

dict

ed

Healthy peers

People with CF

Median FEV1% of predicted in adults

110

100

90

80

70

60

506-9 10-14 15-19 20-24 25-29 30-34 35-39 40-44 45+

Age

FEV1

% of

pre

dict

ed

Healthy peers

People with CF

Median BMI Z-scorein children

1

0

-12-7 8-11 12-17

Age

BMI Z

-Sco

re

Healthy children

Children with CF

Median BMI in adults23

21

20

22

1918-21 22-27 28-31

Age

BMI

32-37 38+Adults with CF

Median FEV1%*

Median BMI Z-score in children Median BMI in adults

At-A-Glance report 2014 4

90%Children

LUNG FUNCTION

68%Adults

LUNG FUNCTION

90%Children

LUNG FUNCTION

68%Adults

LUNG FUNCTION

4

Pancreatic enzymes

% of people with CF who use pancreatic enzyme supplements*% use of pancreatic enzymes

1009080706050403020100

Austria

Czech Republic

Denmark

France

Hungary

Ireland

Israel

Latvia

Lithuania

Macedonia

Moldova

Netherlands

Portugal

Romania

Russian Federation

Serbia

Slovak Republic

Slovenia

Spain

Sweden

Switzerland

Ukraine

United Kingdom

%

Yes

No

• Enzymes released from the pancreas are vital for the digestion and absorption of fat and vitamins from food. In many people with CF the release of these enzymes is blocked and they must take supplements.

At-A-Glance report 2014

30%

of the people with CF are chronically infected with the Pseudomonas aeruginosa bacteria in their lungs.*

Pseudomonas

30%

85% Pancreatic enzymes 85%

of the people with CF use pancreatic enzyme supplements.*

5

* Missing values are not included nor are data from countries with a high % of missing values.

Infection

% chronic Pseudomonas aeruginosa in children

Macedonia

Moldova

Netherlands

Portugal

Romania

Russian Federation

Serbia

Slovak Republic

Slovenia

Spain

Sweden

Switzerland

Ukraine

United KingdomYes

No

1009080706050403020100

Austria

Denmark

France

Ireland

Israel

%

% chronic Pseudomonas aeruginosa in adults

Russian Federation

Serbia

Slovak Republic

Slovenia

Spain

Sweden

Switzerland

Ukraine

United Kingdom

1009080706050403020100

%

Lithuania

Macedonia

Moldova

Netherlands

Austria

Denmark

France

Ireland

Israel

Portugal

Yes

No

% chronic Pseudomonas aeruginosa in children*

% chronic Pseudomonas aeruginosa in adults*

At-A-Glance report 2014

30%

of the people with CF are chronically infected with the Pseudomonas aeruginosa bacteria in their lungs.*

Pseudomonas

30%

85% Pancreatic enzymes 85%

of the people with CF use pancreatic enzyme supplements.*

* Missing values are not included nor are data from countries with a high % of missing values.

66

Inhalation Treatment

Transplantation

% of people with CF who use hypertonic saline*

Number of people with CF living with a lung transplant

% of people with CF who use inhaled antibiotics*% use of enhaled hypertonic saline

Yes

No

1009080706050403020100

Latvia

Lithuania

Macedonia

Moldova

Netherlands

Portugal

Romania

Russian Federation

Serbia

Slovak Republic

Slovenia

Spain

Sweden

Switzerland

Ukraine

United Kingdom

%

Austria

Czech Republic

France

Ireland

Israel

Number of people with CF living with a lung transplant

120

100

80

60

180

160

140

40

20

05-9 10-14 15-19 20-24 25-29 30-34 35-39 40-44 45+

Age

Num

ber

Males

Females

% use of enhaled antibiotics

Yes

No

1009080706050403020100

Latvia

Lithuania

Macedonia

Moldova

Netherlands

Portugal

Romania

Russian Federation

Serbia

Slovak Republic

Slovenia

Spain

Sweden

Switzerland

Ukraine

United Kingdom

%

Israel

Austria

Czech Republic

France

Hypertonic saline is prescribed to clear thick mucus from the lungs.

Inhaled antibiotics are prescribed to treat CF specific bacteria, such as chronic Pseudomonas aeruginosa.

At-A-Glance report 2014

* In these graphs the missing values are not included nor are data from countries with a high % of missing values.

Patients living with lung

transplantation

FEMALES

5.5%MALES

5.1%

TOTAL

5.3%

Patients living with liver

transplantation

FEMALES

0.4%MALES

0.6%

TOTAL

0.5%

7

At-A-Glance report 2014