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Patient

Registry
Annual Data Report

2013

Robert J. Beall, PhD
President and CEO

Since its inception in 1966, the Cystic Fibrosis
Foundation’s Patient Registry has been central to our
efforts to ensure all people with CF receive the highestquality, specialized care and most effective treatments.
As the Registry has expanded to capture and analyze a
broader range of data, it has become an indispensable
tool in many important initiatives – from quality
improvement in Foundation-accredited care centers to
the design of clinical trials studying a wide array of CF
therapies.
The steady gains in the health of people with CF
highlighted in this year’s Registry report reflect the hard
work and dedication of many, especially those living
with CF and their families. I thank our community for its
contributions to this crucial endeavor and look forward
to working with you to achieve our shared mission of
ending this disease.

To the CF Community and Friends,
We are pleased to present the Cystic Fibrosis Foundation’s 2013 Patient Registry
Annual Data Report.
For close to 50 years, the Foundation has collected information on the health of people
with cystic fibrosis who receive care at Foundation-accredited care centers. We then share
the information with the wider CF community, highlighting trends in key health outcomes
to help people with CF and their families, clinicians and researchers work together to raise
the quality of care.

Preston W.
Campbell, III, MD

Thanks to steady progress in CF care and treatment, the face of the disease has changed
dramatically over the last few decades. We are now on the cusp of an important milestone,
when more than half of those with CF in the United States will be 18 years and older, and
we anticipate continued growth in the adult CF population in the years to come.
Positive trends shown in this year’s report include:
• Continued improvements in pulmonary function and nutritional status
• Increase in the number of new CF diagnoses through newborn screening
• Decrease in lung infections from Pseudomonas aeruginosa and MRSA

Bruce C.
Marshall, MD

The data also show that much work remains in order for all people with CF to be better
able to lead healthy and fulfilling lives. Many people with CF still require hospitalization for
treatment of exacerbations and, as the CF population ages, many now face other health
problems like CF-related diabetes and depression, which add to the daily demands of
living with this disease. We remain committed to addressing these challenges.
This year, we have used more graphics to summarize the data and convey more fully the
impact that CF has on those living with the disease. We hope this report encourages
people with CF to partner with their care center teams and take an active role in shaping
an individualized care plan to stay healthy and thrive.

The Mission of the Cystic Fibrosis Foundation
The mission of the Cystic Fibrosis Foundation is to cure cystic fibrosis and to
provide all people with the disease the opportunity to lead full, productive lives by
funding research and drug development, promoting individualized treatment, and
ensuring access to high-quality, specialized care.

We are deeply grateful to all who have contributed to this report, especially people with
CF and their families who so generously agree to share their information.
Thank you for your continued commitment to our mission to cure CF.
Sincerely,

Preston W. Campbell, III, MD
Executive Vice President
Medical Affairs

Bruce C. Marshall, MD
Senior Vice President
Clinical Affairs
1

Highlights of CF Foundation Patient Registry Data

Table of Contents

From the analysis of the CF Foundation Patient Registry data in 2013

About the CF Foundation Care Model

4

• Care Center Network 4
• CF Foundation Clinical Care Practice Guidelines 4
More than

28,000

people with CF were seen at a
CF Foundation-accredited care
center and consented to have their
data or their child’s data entered into
the Registry. Almost 50 percent of
those followed in the Registry were
age 18 years or older.

66%

of new CF diagnoses
were made in the
first year of life.

40.7

years in 2013.

prevalence is declining and the
prevalence of MRSA has stabilized.
Nontuberculous mycobacteria (NTM)
infections are of concern.

people with CF
were 40 years or older.

Registry data shows
continuous
improvement in

nutrition
and
pulmonary
health outcomes in
children and adults.

is an extremely valuable diagnostic
test, yet fewer people currently have
a sweat test recorded in the Registry
than in the past. This is especially
common among those with two
copies of the F508del mutation.

2

• About the CF Foundation Patient Registry 5
• CF Foundation Resources and Assistance Programs 5

Overview of the Health of People with CF
and the Standards of Care in the
CF Foundation Care Center Network

6

46%

are working full time
or part time and

22%

were students.

• Diagnosis of CF 8
• Sweat Test Data 9
• CF Genetics 10
• CF Clinical Care Practice Guidelines: Care, Screening and Prevention 12
• Microbiology 13
• Growth and Nutrition 14
• Lung Health 16

Of adults with CF,

Sweat testing

• Quality Improvement Initiative 4

• Demographics 6

Pseudomonas
2,697

The median predicted age of
survival has increased from
33.4 years in 2003 to

• Complications 19

97%

of people with CF have had
their mutations
identified through genetic
testing.

• Transplantation 21
• Survival 22

Resources 23

3

About the CF Foundation Care Model
Care Center Network

About the Cystic Fibrosis Foundation Patient Registry

The CF Foundation accredits and funds a
nationwide network of more than 120 care centers.
Multidisciplinary teams of health care professionals
at the care centers work together to provide expert,
age-appropriate care tailored to meet the unique
needs of individuals living with CF.
Each center undergoes an assessment by the
CF Foundation’s Care Center Committee before it

receives accreditation and funding. Accredited centers
are reevaluated annually to ensure that people with
CF receive effective and consistent levels of care and
state-of-the-art treatments.

Each year, information on the health status of children and adults with CF who
receive care at CF Foundation-accredited care centers is entered into the Registry.
This information provides critical data to help care teams and researchers identify new
health trends, recognize the most effective treatments, design CF clinical trials and
develop clinical care practice guidelines.

The CF Foundation’s care center network has been
widely recognized as a national model for care of a
chronic disease and for driving improvements in care.
Uses of the
Cystic Fibrosis Foundation Patient Registry

The Multidisciplinary Team

respiratory
therapist
social
program
worker
coordinator

physician
dietitian

Required Team Members

FRAMEWORK FOR
CLINICAL TRIALS

Track progress
in curing CF and
the impact of
treatments

Test promising
new therapies

ψ

RT
nurse

DISEASE
SURVEILLANCE

physical
research
therapist
coordinator
psychologist
pharmacist

Recommended Team Members

POST-MARKETING
SURVEILLANCE
STUDIES

QUALITY
IMPROVEMENT

COMPARATIVE
EFFECTIVENESS
RESEARCH

Ensure safety
and effectiveness
of approved
products

Provide
all patients
with
high-quality care

Promote
evidence-based
clinical
decision making

CF Foundation Clinical Care Practice Guidelines
The CF Foundation provides accredited care centers with clinical care practice
guidelines, which are updated regularly based on the latest research, care and
treatments. The Foundation brings together committees of subject-matter experts,
including physicians, nurses, respiratory therapists and dietitians, along with adults
with CF and CF parents, to develop care recommendations on each topic.

Quality Improvement Initiative

CF Foundation Resources and Assistance Programs
The CF Foundation offers a variety of resources and programs to help
people with CF obtain essential CF care and treatments. Please refer
to the appendix in the back of this report for information on patient
assistance resources.

In 2013,

25%

of people
with CF
participated in a
patient assistance
program through
CFF or another
source.

Through its quality improvement initiative, the CF Foundation works closely with
care centers to ensure all people with CF receive the highest quality of care. The
quality improvement initiative is aimed at identifying best practices for CF care and
treatment, and providing training and tools to implement improvements across the
care center network. People with CF and their families are important partners in this
process.
4

5

Overview of the Health of People with CF and the
Standards of Care in the CF Foundation Care Center Network
Demographics

Of adults with CF,

As people with CF are living longer and pursuing more
opportunities, there are more college graduates in the
CF population. There are also more adults who are
working full time or part time. Since the late 1990s, the
number of people with CF with college degrees has
more than doubled.

46%
work

full time or
part time
and

22%

Records of 28,103 people with CF were included in the Registry in 2013.

are students.

People with CF are living longer and healthier lives than ever before. Today, nearly
half of all people with CF in the United States are adults. We project that survival will
continue to improve over the next decade and beyond.

Characteristics of Adults 18 Years and Older with CF in 2013

Number of People with CF Included in the Registry in Each State

Education

Marital Status

Employment
Retired
1.6%

618
125

236

74

118

407

183

592
180

649

113

1,743
1,063

54
392
189

1,047

373

678

262

261

138

Single
54.0%

Unemployed
8.2%

Full Time
34.3%
Student
22.0%

Widowed
0.2%

883

Disabled
17.6%

Homemaker
4.3%

College
Graduate
29.2%

570
667

402

Some
College
33.7%

Married/
Living
Together
40.9%

38

667

717

Less Than
High School
6.8%
Masters/
Doctoral-Level
Degree
6.4%

99

538
68

1,523

552
366

284
385

1,659

246

2,332

914

High School
Diploma
23.8%

Part
Time
12.0%

Separated/
Divorced
4.9%

381

261
217

444

Number of people with CF

782

0 – 99
1,744

319
100 – 199

Number of Children and Adults with CF, 1986–2013

200 – 499

1,434

65

Number of Children and Adults with CF, 1986–2013  

500 – 999

30,000

≥1,000

25,000

Distribution of Race/Ethnicity among People with CF

People with CF age
18 years or older

18

African American
4.3%
White
86.8%

Number of Patients

16

Adults 18 Years and Older
Children Under 18 Years

20,000
15,000
10,000
5,000

or older

0
86 87 88 89 90 91 92 93 94 95 96 97 98 99 00 01 02 03 04 05 06 07 08 09 10 11 12 13
Year

Hispanic 7.9%
Other 1%

1986

29.2%
6

2013

49.7%
7

Diagnosis of CF

Sweat Test Data

Nationwide newborn screening for CF has been in place since 2010. As a result, more
people with CF are now diagnosed in infancy, often before symptoms of the disease
appear. While a growing proportion of people with CF are diagnosed by newborn
screening, some are not diagnosed until adolescence or adulthood. Early diagnosis
allows for earlier treatment, which may lead to better lung function and nutritional
outcomes later in life.

The CF Foundation guidelines for diagnosis of cystic fibrosis recommend that
a sweat chloride test be part of the diagnostic evaluation for CF. Despite this
recommendation, there is a decrease in the number of individuals with a sweat
chloride value reported in the Registry among individuals who are newly diagnosed
and especially among those with two copies of the F508del mutation. This decrease
in reported sweat tests may be due to an increased reliance on genetic testing to
determine a definitive diagnosis of CF.

60%

In 2013,
of new
diagnoses were detected
by newborn screening.

While newborn screening provides opportunities for early intervention, it also
increases the risk of misdiagnosing symptomless infants. In these cases, infants
may receive unneeded CF treatments that can be potentially harmful. Visiting a CF
Foundation-accredited care center to receive a complete diagnostic evaluation is
extremely important for people who are newly diagnosed. A complete diagnostic
evaluation includes a sweat test, genetic test and clinical evaluation.

Percent of Patients with Sweat Chloride Values Reported by Year of Diagnosis, 1986-2013

Percent of Patients with a Sweat Chloride Reported by Year of Diagnosis, 1988–2013
100

Age at Diagnosis for all People with CF in the Registry, 2013

Age at Diagnosis for All People with CF in the Registry, 2013  
16 Years and Older, 6.8%
Prenatal, 2.3%

Percent of Patients

90

80

70

Under 1 Month, 29.0%
2-15 Years Old, 20.8%

Under
1 Year,
65.7%

60
1988

1993

1998

1 to 3 Months, 13.2%
F508del Homozygotes

4 to 6 Months, 12.2%

Year

2003

2008

2013

Other

7 to 11 Months, 9.0%
1 Year Old, 6.6%

Percent of New Diagnoses Detected by Newborn Screening, 1990-2013

Percent of
newly diagnosed
people with CF
with sweat values
recorded.

1993

2003

2013

97 82 75

100
80

Percent

60
40
20
0
90 91 92 93 94 95 96 97 98 99 00 01 02 03 04 05 06 07 08 09 10 11 12 13
Year

8

9

CF Genetics

Prevalence of the 25 Most Common CFTR Mutations in 2013

In people with CF, mutations in both copies of the cystic fibrosis transmembrane
conductance regulator (CFTR) gene disrupt normal production of the CFTR protein.
Different mutations cause CFTR to malfunction in different ways. In some people with CF,
little to no CFTR is produced. In others, the defective protein is produced, but cannot
move to the surface of the cell where it is needed to regulate the transfer of chloride
and water in and out of cells. In others, CFTR is produced and moves to the surface
of the cell but the gate that controls chloride movement does not open properly. The
malfunctioning CFTR leads to an accumulation of unusually thick and sticky mucus in the
lungs, pancreas and other organs.
Today, as new therapies are developed to target problems caused by specific
CF mutations, it is extremely important for each person with CF to know his or
her mutations. There are more than 1,500 known CFTR mutations, many of which
researchers have categorized into different groups. People with two mutations in
classes I, II and III typically exhibit more severe pulmonary disease and pancreatic
insufficiency as compared to people with at least one mutation in classes IV and V.
CFTR Mutation Classes
The chart below shows just one of the ways that researchers classify CFTR mutations. Individuals with CF
can consult with a genetic counselor or other member of their care team to learn more about their specific
mutations. To find more information on CFTR and mutation classes, visit www.cftr2.org.
Cl Cl -

Cl -

Cl Cl -

Cl -

Cl -

EXAMPLES

DESCRIPTION

X

X

X

NUMBER OF PEOPLE

PERCENT OF PEOPLE WITH
ONE OR MORE COPY OF
THE MUTATION

F508del

23,478

86.4

G542X

1,252

4.6

G551D

1,182

4.4

R117H

767

2.8

N1303K

672

2.5

W1282X

625

2.3

R553X

493

1.8

621+1G->T

437

1.6

1717-1G->A

425

1.6

3849+10kbC->T

411

1.5

2789+5G->A

369

1.4

3120+1G->A

267

1.0

I507del

220

0.8

D1152H

196

0.7

R1162X

193

0.7

3659delC

189

0.7

1898+1G->A

187

0.7

G85E

178

0.7

R560T

165

0.6

R347P

158

0.6

2184insA

151

0.6

R334W

145

0.5

A455E

142

0.5

Q493X

129

0.5

2184delA

116

0.4

MUTATION

Cl -

Cl -

Cl -

XX

Normal

Class I

Class II

Class III

Class IV

Class V

CFTR is created,
reaches cell
surface and
functions properly,
allowing transfer
of chloride and water.

No
functional
CFTR
created.

CFTR protein
is created,
but misfolded,
keeping it from
reaching the
cell surface.

CFTR protein
is created and
reaches cell
surface, but
does not
function properly.

The opening
in the CFTR
protein ion
channel
is faulty.

CFTR
is created
in insufficient
quantities.

G542X
W1282X
R553X

F508del
N1303K
I507del

G551D
S549N
V520F

R117H
D1152H
R347P

3849+10kbC->T
2789+5G->A
A455E

Homozygotes (two copies) - 46.5%
Heterozygotes (one copy) - 39.9%

97%
of people

with CF
had their
mutations
identified
through
genetic
testing.

Adapted from: http://www.umd.be/CFTR/W_CFTR/gene.html

10

11

CF Clinical Care Practice Guidelines: Care, Screening and Prevention

Microbiology

CF clinical care practice guidelines are developed by expert multidisciplinary committees, based
on published evidence and clinical experience. Guidelines are intended to inform care centers
about CF care and treatment best practices and to be adapted by care center teams to the
needs, preferences and values of the individual with CF and his or her family.

Pulmonary infections are a serious and chronic problem for many living with CF. People
with CF are at greater risk of getting lung infections because the thick and sticky mucus
that accumulates in their lungs allows germs to thrive and multiply. The prevalence of
the bacteria, Pseudomonas aeruginosa or Pseudomonas has been gradually decreasing
over time in people with CF. Prevalence of nontuberculous mycobacteria (NTM)
infections is increasing in the general population. This is of concern because people
with CF are at a higher risk of developing NTM infections which require long periods of
treatment with multiple antibiotics.

Current CF Foundation clinical care practice guidelines recommend that individuals ages 6 and
older visit their care center at least four times, receive four microbiological cultures and perform
two pulmonary function tests (PFTs) per year. There are other annual guidelines for CF care,
some of which are listed below.

To help reduce the spread of germs, the infection prevention and control guidelines
for CF were created. These CF Foundation guidelines provide recommendations for
people with CF, their families and CF health care professionals to help reduce the
spread of germs in the clinic and hospital setting, and at home, school or work.

4
clinic visits

4
cultures

2
PFTs

The majority of people with CF followed in the Registry receive care as recommended by
the Guidelines; however, adults with CF do not receive guideline-recommended care to the
same extent as children. There are various possible reasons for this difference. Teens and
adults may be busy with school or jobs, and some may have milder disease.

Care, Screening and Prevention Guidelines for People with CF
2013
PERCENT OF PEOPLE WITH CF WHO HAD

4 or more clinic visits

UNDER
18 YEARS

18 YEARS
AND OLDER

ALL

74

56

65

ELIGIBILITY CRITERIA
FOR RECOMMENDATION

Prevalence of Respiratory Microorganisms in People with CF, 1988–2013

Prevalence
of Respiratory Microorganisms, 1988–2013
80
S. aureus
S.
aureus
60
Percent of Patients

CF Foundation Annual Clinical Care Practice Guidelines

P. aeruginosa
P.
aeruginosa
MRSA

40

MDR-PA
MDR-PA
H. influenzae
Influenzae
S. maltophilia
maltophilia

20

A. xylosoxidans
Achromobacter
All

B.cepacia
B.
cepacia
complex

0
4 or more sputum/throat cultures

63

40

52

All

2 or more lung function tests (PFTs)

91

87

89

If 6 years of age or older and physically able

An influenza vaccine (flu shot)

96

93

95

If 6 months of age or older

Fat-soluble vitamin blood levels measured
(Vitamins A, D & E)

90

80

85

All

An oral glucose tolerance test (OGTT)

50

29

37

If 10 years of age or older

A blood test to measure liver enzymes

84

76

80

All

12

88 89 90 91 92 93 94 95 96 97 98 99 00 01 02 03 04 05 06 07 08 09 10 11 12 13
Year

Change in percent of people with CF with a positive
culture of Pseudomonas over the last 10 years
down

8%

16%

Percent of
people with CF
tested between
2010 and 2013
who had
NTM infections

13

Growth and Nutrition
Children and teens with CF need adequate nutrition to grow and
prosper, and it’s important for adults to maintain proper nutrition
to stay healthy. Because of the thick secretions that build up in the
pancreas and prevent the release of digestive enzymes, people with
CF have difficulty absorbing vital vitamins and nutrients from food,
which leads to poor growth and malnutrition. To address this, most
people with CF take pancreatic enzyme supplements.

BMI
Body mass index (BMI)
is based on a person’s
weight and height. BMI is
calculated by dividing body weight
in kilograms (kg) by the person’s
height in meters squared (m2).

Median BMI Value by Age, In Adults 20 years and Older, 1988-2013

Median BMI Value by Age, 1986–2013  
23

22

BMI

In recent decades, nutritional outcomes have improved markedly for
both children and adults. Body mass index (BMI) and BMI percentile
are two important measures for assessing growth, nutrition and
gastrointestinal function in people with CF.

Better nutrition is associated with better lung function in people with CF. This
association suggests that maintaining a healthy weight is important not only to
nutrition and growth, but to lung function as well. This association is especially
important for infants whose length and weight can be measured, but who cannot
perform lung function tests.

Despite the gains in BMI, height and weight percentiles among
people with CF remain below the 50th percentile, suggesting room
for improvement.

21

20

Median Nutritional Outcome Percentiles
for Patients 2 to 19 Years, 1986–2013  

Median Nutritional Outcome Percentiles for Children and Adolescents Ages 2 to 19 years, 1986-2013

19
1988

1993

1998

2003

2008

2013

Years

60
20 Year Olds

25 Year Olds

30 Year Olds

35 Year Olds

Percentile

50
40
30

BMI Percentile

20
10

BMI

Weight

Height

0
86

89

92

95

98
Year

87%
of people with CF take
pancreatic enzyme
supplements.

14

01

04

07

10

13

BMI percentile
matches a child’s BMI to other
children in the United States
of the same age and gender.
A BMI of the 50th percentile
means half of the children of the
same age and gender are larger
and half are smaller.

Percentage of people with CF
with a BMI percentile less than 10.
1993

2013

20% 6%
Percentage of people with CF
with a BMI less than 18.
1993
2013

20% 7%

15

Lung Health
Median FEV1 Percent Predicted in 1993 and 2013

Pulmonary function in individuals with CF has improved over time, but the
pattern of decreasing pulmonary function beginning in adolescence persists.
Research shows that people with CF of all ages, including infants, have some
lung damage — even when FEV1 percent predicted is within the normal
range. This damage to the lungs is primarily the result of mucus buildup and
lung infections.
To keep their lungs healthy, people with CF must take a number of drugs.
Most pulmonary medications recommended by CF care providers are widely
used by people with CF. However, medications alone cannot keep CF lungs
healthy, and people with CF also use various airway clearance techniques
(ACTs), including exercise, to help move mucus out of the lungs.
Despite notable improvements in pulmonary function and nutritional status
over the past two decades, a significant proportion of people with CF in the
Registry are still treated with IV antibiotics for pulmonary exacerbations.

FEV1
Percent
Predicted
FEV1 is a measure of lung
function. It is the forced
exhaled volume of air
in the first second of an
exhaled breath. It is shown
as a percent predicted,
based on the FEV1 of
healthy, non-smoking
people of the same age,
height and gender.

85

95

62

83

1993

2013

1993

2013

For 10 year olds

For 18 year olds

Median FEV1 Percent Predicted in 18 Year Olds, 1988-2013  
Median 100
FEV1 Percent Predicted in 18-Year-Olds, 1988-2013
1988

0
100

Median FEV1 Percent Predicted for People with CF by Age, 1988-2013

90  

25
75

80  
70  
60  
50  
40  
30  

6  Year  Olds  

1998  
11  Year  Olds  

Years  

2003  

16  Year  Olds  

2008  
21  Year  Olds  

2013  
26  Year  Olds  

Percent of Patients
Percent of Patients

100

stneiof
taPPatients
fo tnecreP
Percent
Percent
of Patients

FEVP1ercent  
Percent
Predicted
FEV1  
Predicted  

100  

1993  

75

75
100 24.1

37.3

24.1
24.1 38.6

50
75

38.6

50
50

50

38.6 38.6
25 38.6
50
37.3

75
25

25

0
25

0

37.3

24.1 1988
37.3 37.3

1000

Median

1 Percent
FEV1988

0

1988 1988

16

1998 14.2

23.4

9.6
2003

5.5
2008

5.5
Median
Predicted
18 Olds,
Year9.6Olds,
1988-2013  
27.8
Median
FEV1 FEV
Percent
Predicted
in 18 in
Year
1988-2013  
1 Percent

Median FEV1 Precent Predicted by Age, 1986-2013  

1988  

24.1

1993

14.2

23.4

34.8
14.214.2

23.4
42.1
23.4 34.5

34.8

34.5

34.834.8

34.5

51.0

42.1

34.8

51.0

42.1

51.0
51.01998

1993
23.4
42.1 42.1

1993
Predicted
1993 1993

9.69.6

5.5
5.5
27.8

7.2
7.2
21.0

27.8
27.8

21.0
21.0

FEV1 Severe (<40%)

56.8
33.7
33.7

66.6
66.6

34.5 34.5

14.2

in

1998
18
Year
19981998

21.0
7.2

33.7

33.7

51.0

7.2
2013

71.9
71.9

71.9

56.8

33.7

56.8
56.8
2003

9.6

66.6
66.6

27.8

2008

5.5

2003
2008
Olds,
1988-2013  
2003
2003

2008
2008

FEV1 Normal/Mild (≥70%)
FEV1 Normal/Mild (≥70%)
toto
69%)
FEV1Moderate
Moderate(40%
(40%
69%)
FEV1
FEV1Severe
Severe(<40%)
(<40%)
FEV1
Severe
(<40%)

56.8

66.6

FEV1Normal/Mild
Moderate (40%
to 69%)
(>
_70%)

FEV1
Moderate
(40%
Severe
(<40%)
FEV1
Moderate
(40%toto69%)
69%)
FEV1
Normal/Mild
(≥70%)
FEV1 Normal/Mild (≥70%)

71.9
71.9

21.0
2013

7.2
2013
2013
2013

17

Complications

Lung Health
Percentage (%) of People Prescribed CF Medications
CRITERIA FOR
PRESCRIBING MEDICATION

2012

2013

Dornase alfa
(Pulmozyme®)

84

85

• At least 6 years old

Hypertonic saline

61

63

• At least 6 years old

Tobramycin for inhalation
solution (such as TOBI®)

66

63

• At least 6 years old
• P. aeruginosa in cultures

42

• At least 6 years old
• P. aeruginosa in cultures

Aztreonam for inhalation
solution (such as Cayston®)

Azithromycin
(such as Zithromax®)

Ivacaftor (Kalydeco™)

39
71
78

• At least 6 years old
• P. aeruginosa in cultures
• Weight over 25 kg (55 lbs)
• FEV1 over 30% predicted

69

• At least 6 years old
• A G551D gene mutation

87

Detecting complications early and managing them properly is crucial for the health
and well-being of those with CF. Complications of CF include cystic fibrosis-related
diabetes (CFRD), liver disease, bone disease, distal intestinal obstructive syndrome
(DIOS), gastroesophageal reflux disease (GERD) and depression.

Prevalence of Common Complications by Age in 2013

Prevalence of Common Complications by Age in 2013  
50

35%
of people
with CF were
treated with
IV antibiotics
for pulmonary
exacerbations
in 2013.

40
Percent of Patients

RECOMMENDED CHRONIC
MEDICATIONS FOR LUNG HEALTH

CF is often associated with complications other than lung disease and impaired
nutritional status. These complications can be a direct result of the disease or a
result of treatments for CF.

30

20

10

0

Primary Airway Clearance Technique by People with CF in 2013  

<6

Primary Airway Clearance Technique Used by People with CF in 2013

6 to 10

Arthritis/Arthropathy

11 to 17

18 to 24
Age (Years)

Bone Disease

25 to 34

CFRD

35 to 44

Depression

Pulmonary
exacerbation
Exercise
4.9%
3.0%
3.0%None

71.7%

HFCWO
(vest)

8.9% PEP/OscPEP

10.5%

Postural Drainage

A pulmonary exacerbation
refers to a period of
time when IV antibiotics
are administered either
during a hospitalization
or via home IV therapy in
response to worsening
signs and symptoms of
lung disease.

35%
of people
with CF age
18 years and
older have
CFRD.

1.0%
1.0%Forced Expiration

18

19

Complications

Transplantation

4,657

Cystic Fibrosis-Related Diabetes (CFRD)
CFRD is a form of diabetes that is unique to people with CF. People
with CFRD have to undergo more treatments than those without the
complication and most commonly use insulin. It is one of the most
widespread complications of the disease, especially among adults
with CF. As the number of adults with CF has increased, so has the
prevalence of CFRD. CFRD is also more common in people with
mutations in classes I – III.

In 2013,

Lung transplantation remains an option for some people with CF who
have severe lung disease. However, lung transplantation has its own
risks and requires life-long, post-transplant care.

245

people with CF
received a
lung transplant.

The Registry also collects data on people with CF who have had other
types of transplants, and there were 135 people with CF followed who
received a kidney, heart or liver transplant in 2013 or in a prior year.

The median
age of
recipients
was
years.

people with CF
used insulin
in 2013.

Research shows that early diagnosis and treatment of CFRD leads to
better nutrition and pulmonary function. The CF care guidelines for
CFRD recommend that people with CF ages 10 and older should be
tested annually for CFRD via the oral glucose tolerance test (OGTT).

31

Number of Patients Receiving a Lung Transplant, 1990–2013

Number of Patients Receiving a Lung Transplant, 1990–2013
250

Prevalence of CFRD, 1988–2013
Number of Patients

200

Percent of Patients with CFRD

40
35
30

150

100

25
20

50

15
10

0
90

5

91

92

93

94

95

96

97

98

99

00

01 02
Year

03

04

05

06

07

08

09

10

11

12

13

0
1988

1993

1998

2003

2008

2013

Year
Patients 10 to 17 Years

20

Patients 18 Years and Older

21

Appendix of Resources

Survival
People with CF are living longer than ever before with the median predicted
survival age continuing to increase.

Median
Predicted
Survival Age

Median Predicted Survival Age, 1989–2013 (in 5 year bands)

Median Predicted Survival (Years)

Median  Predicted  Survival  Age,  1989-­‐2013    In  5  Year  Bands  

The median predicted
survival age is the age to
which half of the current
Registry population
would be expected to
survive, given their ages
in 2013 and assuming that
mortality rates do not
change. Median predicted
survival age is calculated
using a method called life
table analysis.

40

36

32

28

24
1989–1993
1989 - 1993

1994–1998
1994
- 1998

1999–2003
1999
- 2003

2004–2008
2004
- 2008

2009–2013
2009
- 2013

Year

40.7
years

Median
predicted
survival age
in 2013.

About the CF Foundation Care Model

Sweat Test Data

Care Center Network

The Sweat Test
www.cff.org/aboutcf/testing/sweattest/

Care Center Network
www.cff.org/treatments/CareCenterNetwork/

CF Genetics

Partnering for Care: CF Experts Talk About Managing Life
with CF
www.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/
PartneringCare

About CF Genetics
www.cff.org/AboutCF/Testing/Genetics

CF Foundation Clinical Care Practice Guidelines

Clinical and Functional Translation of CFTR (CFTR2)
www.cftr2.org/

CF Care Guidelines
www.cff.org/treatments/CFCareGuidelines
Quality Improvement Initiative
Improve Your CF Care
www.cff.org/LivingWithCF/QualityImprovement/
ImproveYourCare/
Quality Improvement
www.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/
PartneringCare/#Partnering_for_Improvement
www.cff.org/LivingWithCF/QualityImprovement

Genetics-Related CF Webcasts
www.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/
Genetics/
Types of CF Mutations
www.cff.org/AboutCF/Testing/Genetics/CFMutations/
Microbiology
Additional Clinical Initiatives: Burkholderia cepacia,
Methicillin-resistant Staphylococcus aureus (MRSA) and
Nontuberculous Mycobacteria (NTM)
www.cff.org/research/DrugDevelopmentPipeline/
AdditionalClinicalInitiatives

Overview of the Health of People with CF and
the Standards of Care in the CF Foundation
Care Center Network

Centers for Disease Control and Prevention (CDC)
Information on Handwashing
www.cdc.gov/handwashing

Demographics

Centers for Disease Control and Preventions (CDC)
Information on Vaccines
www.cdc.gov/vaccines

CF Healthcare Coverage and Advocacy and Building Life
Skills to Manage CF
www.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/
PartneringCare
Cystic Fibrosis Patient Assistance Foundation
www.cfpaf.org
Information for Adults with CF
www.cff.org/Adults
Patient Assistance Resource Center
www.cff.org/LivingWithCF/AssistanceResources
Scholarships and Financial Aid for People with CF
www.cff.org/LivingWithCF/Scholarships/

The CF Foundation is committed to promoting individualized treatment
and ensuring access to high-quality, specialized care for people with CF.
The findings from 2013 Registry data provide much insight into the current
health of the CF population and also provide clinicians with a road map for
improving care. As new treatments and therapies emerge, the Registry will
remain a rich resource for researchers, clinicians, people with CF and families
as they partner to improve care, treatment and research.

CF Mutation Analysis Program
www.cff.org/LivingWithCF/AssistanceResources/MAP

Diagnosis of CF
CF Care Guidelines – Age Specific Care
www.cff.org/treatments/CFCareGuidelines/
AgeSpecificCare
CF Infant Care: First Year of Life
www.cff.org/LivingWithCF/StayingHealthy/CFInfantCare

CF Care Guidelines – Infection Prevention and Control
www.cff.org/treatments/CFCareGuidelines/
InfectionControl
Get Germ Smart
www.cff.org/LivingWithCF/StayingHealthy/GermSmart/
Information about Burkholderia cepacia
www.cff.org/LivingWithCF/StayingHealthy/Germs/
Bcepacia
Information about Allergic Bronchopulmonary
Aspergillosis
www.cff.org/LivingWithCF/StayingHealthy/Germs/ABPA/
Methicillin-resistant Staphylococcus aureus (MRSA) and
Cystic Fibrosis
www.cff.org/LivingWithCF/StayingHealthy/Germs/MRSA/
Webcasts on Germs and Infection Control
www.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/
Germs

Testing for Cystic Fibrosis
www.cff.org/AboutCF/Testing
Webcast – CF Infant Care: First Year of Life
www.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/
AgeSpecific/#Infant_Care

22

23

Growth and Nutrition

Complications

Centers for Disease Control and Prevention (CDC) Clinical
Growth Charts
www.cdc.gov/growthcharts/clinical_charts.htm

CF and Depression and Anxiety
www.cff.org/Adults/Other/DepressionAnxiety

CF Care Guidelines – Nutrition/Gastrointestinal (GI)
www.cff.org/treatments/CFCareGuidelines/Nutrition
Food and Recipes for People with CF
www.cff.org/LivingWithCF/StayingHealthy/Diet/
FoodIdeasRecipes/
Information about Body Mass Index
www.cdc.gov/healthyweight/assessing/bmi
Nutrition/Gastrointestinal (GI) Webcasts
www.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/
Nutrition
Staying Healthy with CF
www.cff.org/LivingWithCF/StayingHealthy
World Health Organization (WHO) Growth Standards and
WHO Growth Charts
www.cdc.gov/growthcharts/who_charts.htm
Lung Health
About Clinical Trials
www.cff.org/research/ClinicalResearch
Centers for Disease Control and Prevention (CDC)
Information about How to Quit Smoking
www.cdc.gov/tobacco/quit_smoking/index.htm
CF Care Guidelines - Respiratory
www.cff.org/treatments/CFCareGuidelines/Respiratory

Cystic Fibrosis-Related Diabetes (CFRD)
www.cff.org/LivingWithCF/StayingHealthy/Diet/
Diabetes/
Nutrition and Bone Health
www.cff.org/UploadedFiles/treatments/Therapies/
Nutrition/BoneHealth/Nutrition-Bone-Health-andCystic-Fibrosis.pdf
Transplantation
Clinical Trials Relevant to Lung Transplantation
www.clinicaltrials.gov/ct2/search using the term “lung
transplant”
Information on Lung Transplantation
www.cff.org/treatments/LungTransplantation
transplantliving.org
optn.transplant.hrsa.gov/data
Organ Donation
www.organdonor.gov
Webcasts on Lung Transplantation
www.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/
Respiratory
Survival
CF Life-Expectancy
www.cff.org/AboutCF/Faqs/#CF-life-expectancy

Drug Development Pipeline
www.cff.org/research/DrugDevelopmentPipeline
Find a Clinical Trial
www.cff.org/research/ClinicalResearch/Find
Global Lung Initiative (GLI) Reference Equations for
Spirometry
lungfunction.org/files/GLI-2012_Reference_values.pdf
Information about CF and Exercise
www.cff.org/Adults/DailyLife/Exercise
Risks of Smoking for People with CF
www.cff.org/LivingWithCF/StayingHealthy/LungHealth/
Smoking/
Sign up for Clinical Trial Alerts
www.cff.org/research/ClinicalResearch/Find/
ClinicalTrialAlerts
Therapies for Cystic Fibrosis
www.cff.org/treatments/Therapies
Webcasts on CF Research
www.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/
Research
Webcasts on Respiratory/Lungs
www.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/
Respiratory

24

SOURCE OF DATA
Cystic fibrosis patients under care at CF Foundation-accredited
care centers in the United States, who consented to have their
data entered in 2013.
SUGGESTED CITATION
Cystic Fibrosis Foundation Patient Registry
2013 Annual Data Report
Bethesda, Maryland
©2014 Cystic Fibrosis Foundation
COVER PHOTO BY
Cade Martin Photography

If you have any questions about CF care, please
talk with your CF care center or contact the
Foundation at 1-800 FIGHT CF or info@cff.org.

CYSTIC FIBROSIS FOUNDATION
6931 Arlington Road
Bethesda, MD 20814
1.800.FIGHT.CF
www.cff.org
info@cff.org