2013 patient registry - cf foundation · 1 to the cf community and friends, we are pleased to...

Patient Registry

2013Annual Data Report

1

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.

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

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.

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

Preston W. Campbell, III, MD

Bruce C. Marshall, MD

Robert J. Beall, PhDPresident and CEO

The Mission of the Cystic Fibrosis FoundationThe 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.

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 highest-quality, 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.

3 2

Highlights of CF Foundation Patient Registry Data

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

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.

2,697 people with CF

were 40 years or older.

Sweat testing 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.

66% of new CF diagnoses

were made in the first year of life.

Pseudomonas prevalence is declining and the

prevalence of MRSA has stabilized. Nontuberculous mycobacteria (NTM)

infections are of concern.

Of adults with CF,

46% were working full time

or part time and

22% were students.

The median predicted age of survival has increased from

33.4 years in 2003 to

40.7 years in 2013.

Registry data shows continuous

improvement in

nutrition and

pulmonary health outcomes in children and adults.

97% of people with CF have had

their mutations identified through genetic

testing.

About the CF Foundation Care Model 4

• Care Center Network 4

• CF Foundation Clinical Care Practice Guidelines 4

• Quality Improvement Initiative 4

• 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

• Demographics 6

• 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

• Complications 19

• Transplantation 21

• Survival 22

Resources 23

Table of Contents

5 4

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

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.

About the CF Foundation Care Model

Care Center Network

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.

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.

nurse physician respiratory therapist

Required Team Members Recommended Team Members

dietitian

social worker

program coordinator

physical therapist

research coordinator

psychologist pharmacist

The Multidisciplinary Team

RT ψ

About the Cystic Fibrosis Foundation Patient Registry 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.

Uses of the Cystic Fibrosis Foundation Patient Registry

DISEASE SURVEILLANCE

FRAMEWORK FOR CLINICAL TRIALS

POST-MARKETING SURVEILLANCE

STUDIES

QUALITY IMPROVEMENT

COMPARATIVE EFFECTIVENESS

RESEARCH

Track progress in curing CF and

the impact of treatments

Test promising new therapies

Ensure safety and effectiveness

of approved products

Provide all patients

with high-quality care

Promote evidence-based

clinical decision making

In 2013,

25% of people with CF

participated in a patient assistance program through

CFF or another source.

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.

7 6

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

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

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.

1 8

2013

49.7%

People with CF age 18 years or older

or older

1986

29.2%

0 – 99

100 – 199

200 – 499

500 – 999

≥1,000

618

407

2,332

402

189

180

125

138

373

1,744

552

54

65

16

261

366

246

113

74

319

261

717

392

592

649

1,047 6781,523

1,063

1,659

262

570

667

217 444 782

1,434

381

883

667

1,743

236

38

68538

385284

99

914

183

118

Number of people with CF

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

Number of Children and Adults with CF, 1986–2013

0

5,000

10,000

15,000

20,000

25,000

30,000

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

Num

ber

of

Pat

ient

s Year

Adults 18 Years and Older Children Under 18 Years

Number of Children and Adults with CF, 1986–2013

Of adults with CF,

46%work

full time or part time

and

22% are students.

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.

Characteristics of Adults 18 Years and Older with CF in 2013

Marital Status

Widowed0.2%

Separated/Divorced

4.9%

Married/ Living

Together 40.9%

Single54.0%

Unemployed8.2%

Homemaker4.3%

Employment

Retired1.6%

Disabled17.6%

Student22.0%

Full Time34.3%

Part Time 12.0%

Education

Less ThanHigh School

6.8%

Masters/Doctoral-Level

Degree6.4%

High SchoolDiploma23.8%

SomeCollege33.7%

CollegeGraduate

29.2%

Distribution of Race/Ethnicity among People with CF

African American 4.3%

Hispanic 7.9%

Other 1%

White86.8%

9 8

Diagnosis of CF 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.

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.

In 2013, 60% of new diagnoses were detected

by newborn screening.

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

Prenatal, 2.3%

Under 1 Month, 29.0%

1 to 3 Months, 13.2%



1 Year Old, 6.6%

2-15 Years Old, 20.8%

16 Years and Older, 6.8%

Under 1 Year, 65.7%

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

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

Sweat Test Data 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.

97 82 75Percent of

newly diagnosed people with CF

with sweat values recorded.

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

1993 2003 2013

60

70

80

90

100

1988 1993 1998 2003 2008 2013

Per

cent

of

Pat

ient

s

Year

F508del Homozygotes Other

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

0

20

40

60

80

100

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

Per

cent

Year

11 10

Homozygotes (two copies) - 46.5%

Heterozygotes (one copy) - 39.9%

97%of people with CF had their mutations identified through genetic testing.

Prevalence of the 25 Most Common CFTR Mutations in 2013

MUTATION NUMBER OF PEOPLEPERCENT 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

CF Genetics 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

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

Cl -

Cl -

Cl -

Cl -

Cl -

Cl -

XX

X X

Cl -

Cl -

X

Cl -

Cl -

Class INo

functional CFTR

created.

G542XW1282XR553X

Class IICFTR protein

is created, but misfolded,keeping it from

reaching thecell surface.

F508delN1303KI507del

Class IIICFTR protein is created and reaches cell surface, but

does not function properly.

G551DS549NV520F

Class IVThe opening in the CFTR protein ion

channel is faulty.

R117HD1152HR347P

Class VCFTR

is createdin insufficient

quantities.

3849+10kbC->T2789+5G->A

A455E

NormalCFTR is created,

reaches cell surface and

functions properly, allowing transfer

of chloride and water.

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.

EX

AM

PLE

S

D

ESC

RIP

TIO

N

13 12

CF Clinical Care Practice Guidelines: Care, Screening and Prevention

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.

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.

4 clinic visits

4 cultures

2 PFTs

CF Foundation Annual Clinical Care Practice Guidelines

Care, Screening and Prevention Guidelines for People with CF

PERCENT OF PEOPLE WITH CF WHO HAD

2013ELIGIBILITY CRITERIA

FOR RECOMMENDATIONUNDER 18 YEARS

18 YEARS AND OLDER

ALL

4 or more clinic visits 74 56 65 All

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 to perform the test

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 and doesn’t have CFRD

A blood test to measure liver enzymes 84 76 80 All

Microbiology 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.

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.

S. aureus

P. aeruginosa

MRSA

MDR-PA

H. influenzae

S. maltophilia

A. xylosoxidans

B.cepacia complex

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

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.

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

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

down

8%

0

20

40

60

80

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

Per

cent

of

Pat

ient

s

Year

S. aureus

P. aeruginosa

MRSA

MDR-PA

H. Influenzae

S. maltophilia

Achromobacter

B. cepacia complex

Prevalence of Respiratory Microorganisms, 1988–2013

16%

15 14

1993

20%2013

6%

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

1993

20%2013

7%

Percentage of people with CF with a BMI less than 18.

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

19

20

21

22

23

1988 1993 1998 2003 2008 2013

BM

I

Years

20 Year Olds 25 Year Olds 30 Year Olds 35 Year Olds

Median BMI Value by Age, 1986–2013

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.

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.

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.

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

BMI Percentile 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.

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

0

10

20

30

40

50

60

86 89 92 95 98 01 04 07 10 13

Per

cent

ile

Year

BMI Weight Height

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

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).

87% of people with CF take pancreatic enzyme supplements.

17 16

37.3 42.1 51.0

56.8 66.6

71.9

38.6 34.5

34.8 33.7

27.8 21.0 24.1 23.4

14.2 9.6 5.5 7.2

0

25

50

75

100

1988 1993 1998 2003 2008 2013

Per

cent

of

Pat

ient

s

0

25

50

75

100

1988 1993 1998 2003 2008 2013

Percent of Patients

FEV1 Severe (<40%)

FEV1 Moderate (40% to 69%)

FEV1 Normal/Mild (≥70%)

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

37.3

38.6

24.1

42.1

34.5

23.4

51.0

34.8

14.2

56.8

33.7

9.6

66.6

27.8

5.5

71.9

21.0

7.237.3

42.1 51.0

56.8 66.6

71.9

38.6 34.5

34.8 33.7

27.8 21.0 24.1 23.4

14.2 9.6 5.5 7.2

0

25

50

75

100

1988 1993 1998 2003 2008 2013

Per

cent

of

Pat

ient

s

FEV1 Severe (<40%)




37.3 42.1

51.0 56.8

66.6 71.9

38.6 34.5

34.8 33.7

27.8 21.0 24.1 23.4

14.2 9.6 5.5 7.2

0

25

50

75

100

1988 1993 1998 2003 2008 2013

Per

cent

of

Pat

ient

s

FEV1 Severe (<40%)




37.3 42.1

51.0 56.8

66.6 71.9

38.6 34.5

34.8 33.7

27.8 21.0 24.1 23.4

14.2 9.6 5.5 7.2

0

25

50

75

100

1988 1993 1998 2003 2008 2013

Per

cent

of

Pat

ient

s

FEV1 Severe (<40%)




Normal/Mild (>_70%)

Moderate (40% to 69%)

Severe (<40%)

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

For 10 year olds For 18 year olds 1993 2013 1993 2013

Median FEV1 Percent Predicted in 1993 and 2013

85 95 62 83

Lung Health

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.

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

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.

30

40

50

60

70

80

90

100

1988 1993 1998 2003 2008 2013

FEV1

Percent Predicted

Years

6 Year Olds 11 Year Olds 16 Year Olds 21 Year Olds 26 Year Olds

Median FEV1 Precent Predicted by Age, 1986-2013

FEV

1 P

erce

nt P

red

icte

d

19 18

Complications

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.

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.

35%

of people with CF age 18 years and older have

CFRD.

Prevalence of Common Complications by Age in 2013

0

10

20

30

40

50

<6 6 to 10 11 to 17 18 to 24 25 to 34 35 to 44

Per

cent

of

Pat

ient

s

Age (Years)

Arthritis/Arthropathy Bone Disease CFRD Depression

Prevalence of Common Complications by Age in 2013

3.0%

8.9%

10.5%

1.0%

71.7%

4.9%

Primary Airway Clearance Technique by People with CF in 2013

Percentage (%) of People Prescribed CF Medications

RECOMMENDED CHRONIC

MEDICATIONS FOR LUNG HEALTH2012 2013

CRITERIA FOR

PRESCRIBING MEDICATION

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

Aztreonam for inhalation solution (such as Cayston®) 39 42 • At least 6 years old

• P. aeruginosa in cultures

Azithromycin (such as Zithromax®) 71 69

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

Ivacaftor (Kalydeco®) 78 87 • At least 6 years old • A G551D gene mutation

HFCWO (vest)

3.0% None

PEP/OscPEP

Postural Drainage

1.0% Forced Expiration

Exercise

Primary Airway Clearance Technique Used by People with CF in 2013

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

Pulmonary exacerbation

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.

Lung Health

21 20

4,657

people with CF used insulin

in 2013.

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.

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).

Prevalence of CFRD, 1988–2013

Transplantation 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.

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.

Number of Patients Receiving a Lung Transplant, 1990–2013

0

50

100

150

200

250

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

Num

ber

of

Pat

ient

s

Year

Number of Patients Receiving a Lung Transplant, 1990–2013

In 2013,

245 people with CF received a lung transplant.

The median age of recipients was years.

Complications

31

0

5

10

15

20

25

30

35

40

1988 1993 1998 2003 2008 2013

Per

cent

of

Pat

ient

s w

ith

CFR

D

Year

Patients 10 to 17 Years Patients 18 Years and Older

23 22

Survival

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

40.7years

Median predicted

survival age in 2013.

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

Median Predicted Survival Age

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.

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.

Appendix of Resources

About the CF Foundation Care Model Care Center Network

Care Center Networkwww.cff.org/treatments/CareCenterNetwork/

Partnering for Care: CF Experts Talk About Managing Life with CF

www.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/PartneringCare

CF Foundation Clinical Care Practice Guidelines

CF Care Guidelineswww.cff.org/treatments/CFCareGuidelines

Quality Improvement Initiative

Improve Your CF Carewww.cff.org/LivingWithCF/QualityImprovement/

ImproveYourCare/

Quality Improvement www.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/

PartneringCare/#Partnering_for_Improvementwww.cff.org/LivingWithCF/QualityImprovement

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

CF Healthcare Coverage and Advocacy and Building Life Skills to Manage CF

www.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/PartneringCare

Cystic Fibrosis Patient Assistance Foundationwww.cfpaf.org

Information for Adults with CFwww.cff.org/Adults

Patient Assistance Resource Centerwww.cff.org/LivingWithCF/AssistanceResources

Scholarships and Financial Aid for People with CFwww.cff.org/LivingWithCF/Scholarships/

Diagnosis of CF

CF Care Guidelines – Age Specific Carewww.cff.org/treatments/CFCareGuidelines/

AgeSpecificCare

CF Infant Care: First Year of Lifewww.cff.org/LivingWithCF/StayingHealthy/CFInfantCare

Testing for Cystic Fibrosiswww.cff.org/AboutCF/Testing

Webcast – CF Infant Care: First Year of Lifewww.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/

AgeSpecific/#Infant_Care

Sweat Test Data

The Sweat Testwww.cff.org/aboutcf/testing/sweattest/

CF Genetics

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

CF Mutation Analysis Programwww.cff.org/LivingWithCF/AssistanceResources/MAP

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

Genetics-Related CF Webcastswww.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/

Genetics/

Types of CF Mutationswww.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

Centers for Disease Control and Prevention (CDC) Information on Handwashing

www.cdc.gov/handwashing

Centers for Disease Control and Preventions (CDC) Information on Vaccines

www.cdc.gov/vaccines

CF Care Guidelines – Infection Prevention and Controlwww.cff.org/treatments/CFCareGuidelines/

InfectionControl

Get Germ Smartwww.cff.org/LivingWithCF/StayingHealthy/GermSmart/

Information about Burkholderia cepaciawww.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 Controlwww.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/

Germs

24

28

32

36

40

1989 - 1993 1994 - 1998 1999 - 2003 2004 - 2008 2009 - 2013

Med

ian

Pre

dic

ted

Sur

viva

l (Y

ears

)

Year

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

1989–1993 1994–1998 1999–2003 2004–2008 2009–2013

24

Growth and Nutrition

Centers for Disease Control and Prevention (CDC) Clinical Growth Charts

www.cdc.gov/growthcharts/clinical_charts.htm

CF Care Guidelines – Nutrition/Gastrointestinal (GI)www.cff.org/treatments/CFCareGuidelines/Nutrition

Food and Recipes for People with CFwww.cff.org/LivingWithCF/StayingHealthy/Diet/

FoodIdeasRecipes/

Information about Body Mass Index www.cdc.gov/healthyweight/assessing/bmi

Nutrition/Gastrointestinal (GI) Webcastswww.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/

Nutrition

Staying Healthy with CFwww.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 Trialswww.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 - Respiratorywww.cff.org/treatments/CFCareGuidelines/Respiratory

Drug Development Pipelinewww.cff.org/research/DrugDevelopmentPipeline

Find a Clinical Trialwww.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 Exercisewww.cff.org/Adults/DailyLife/Exercise

Risks of Smoking for People with CFwww.cff.org/LivingWithCF/StayingHealthy/LungHealth/

Smoking/

Sign up for Clinical Trial Alertswww.cff.org/research/ClinicalResearch/Find/

ClinicalTrialAlerts

Therapies for Cystic Fibrosiswww.cff.org/treatments/Therapies

Webcasts on CF Researchwww.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/

Research

Webcasts on Respiratory/Lungswww.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/

Respiratory

Complications

CF and Depression and Anxietywww.cff.org/Adults/Other/DepressionAnxiety

Cystic Fibrosis-Related Diabetes (CFRD)www.cff.org/LivingWithCF/StayingHealthy/Diet/

Diabetes/

Nutrition and Bone Healthwww.cff.org/UploadedFiles/treatments/Therapies/

Nutrition/BoneHealth/Nutrition-Bone-Health-and-Cystic-Fibrosis.pdf

Transplantation

Clinical Trials Relevant to Lung Transplantationwww.clinicaltrials.gov/ct2/search using the term “lung

transplant”

Information on Lung Transplantationwww.cff.org/treatments/LungTransplantationtransplantliving.orgoptn.transplant.hrsa.gov/data

Organ Donationwww.organdonor.gov

Webcasts on Lung Transplantationwww.cff.org/LivingWithCF/Webcasts/ArchivedWebcasts/

Respiratory

Survival

CF Life-Expectancywww.cff.org/AboutCF/Faqs/#CF-life-expectancy

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 [email protected].

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

CYSTIC FIBROSIS FOUNDATION

6931 Arlington Road

Bethesda, MD 20814

1.800.FIGHT.CF

www.cff.org

[email protected]

2013 patient registry - cf foundation · 1 to the cf community and friends, we are pleased to...

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