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2015

PATIENT

REGISTRY

ANNUAL DATA REPORT

MISSION OF THE

CYSTIC FIBROSIS FOUNDATION

The mission of the Cystic Fibrosis Foundation is to cure cystic brosis 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.

SOURCE OF DATA

Cystic brosis patients under care at CF

Foundation-accredited care centers in the United

States, who consented to have their data entered.

SUGGESTED CITATION

Cystic Fibrosis Foundation Patient Registry

2015 Annual Data Report

Bethesda, Maryland

©2016 Cystic Fibrosis Foundation

PHOTOGRAPHY BY

Cade Martin and Gregory Miller

SPECIAL ACKNOWLEDGEMENTS

Those who contributed to the maintenance of

PortCF, analysis of data and creation of this report:

Bruce Marshall

Alexander Elbert

Kristofer Petren

Samar Rizvi

Aliza Fink

Josh Ostrenga

Ase Sewall

Deena Loefer

Annual Data Report 2015 Cystic Fibrosis Foundation Patient Registry 1

August 2016

Dear Friends and Colleagues:

It is a pleasure to share the 2015 Patient Registry Annual Data Report with you. e impact of the Cystic

Fibrosis Foundation Patient Registry continues to grow and inform many important initiatives, including:

quality improvement, clinical trial design, “real world" observational research, and safety and eectiveness

studies of newly approved therapies.

I call your attention to a recent publication, “Cystic Fibrosis Foundation Patient Registry: Design and

Methods of a National Observational Disease Registry," 1 which describes the history of the CF Foundation

Patient Registry from its inception in the 1960"s to the present, the patient population, and the methods

for collection, security, and processing of the data. Of note, the journal editor commented: “e article

this month by Knapp and colleagues features one of the most fully developed disease registries in all

of medicine. e current publication oers a benchmark and roadmap for the development of other observational patient registries." I hope that all of you are proud of your contributions to the tremendous success of the registry.

It would not be possible without the vital contributions of many, most notably the individuals with CF

and their families who generously agree to share their data and the Registry coordinators and care team

members who collect and enter the data. e audit studies conrm th e high degree of completeness

and accuracy of the Registry data. We are deeply grateful to all who have helped make the Registry an

indispensable tool in our shared endeavors to help those with CF enjoy the best health and quality of life.

is year"s report includes an update to the survival section and information on the uptake of CFTR modulators (over 5,500 people with CF in the registry were prescribed a CFTR modulator in 2015). In addition, we continue to see the year-over-year incremental improvements in key metrics such as pulmonary function and nutritional status that bode well for the future. We hope you nd this report interesting and that it sparks discussions among members of the CF community. As always we are open to your comments and suggestions for improvement.

is is a truly exciting time in CF, with advances in health care delivery and new therapeutics that have

transformative potential. Together, we will track these and other important developments in the Registry.

ank you all for your hard work throughout the year and your commitment to the CF Foundation"s mission.

Bruce C. Marshall, M.D.

Senior Vice President of Clinical Aairs

Cystic Fibrosis Foundation

Cystic Fibrosis Foundation Patient Registry Annual Data Report 20152

TABLE OF CONTENTS

SUMMARY OF THE CYSTIC FIBROSIS FOUNDATION PATIENT REGISTRY 4

DEMOGRAPHICS

6 Characteristics of Adults with CF 18 Years and Older 8

Health Insurance Information

10

DIAGNOSIS

11 Characteristics of Diagnoses among Individuals with CF 11

Diagnostic Tests

14

Sweat Chloride Testing

14

Genotyping

15

CFTR GENE MUTATIONS

16

GUIDELINES: CARE, SCREENING AND PREVENTION

21

Patient Care Guidelines

21

Infant Care Guidelines

26

MICROBIOLOGY

29

Pseudomonas aeruginosa 31

Staphylococcus aureus 32

Burkholderia cepacia Complex 33

Nontuberculous Mycobacteria

33

NUTRITION

35

Infant Feeding

40

PULMONARY FUNCTION

41

Variation in FEV

1

Outcomes by Mutation Class

43
FEV 1

AND BMI OUTCOMES

44

PULMONARY EXACERBATIONS

46

THERAPIES

48

Gastrointestinal (GI) Therapies

48

Pulmonary Therapies

50

Medications Recommended for Chronic Use

53
Medications with Insufcient Evidence to Recommend for or Against Chronic Use 54

Medications Not Recommended for Chronic Use

54

Medication Use in Young Children

55

Airway Clearance Techniques

56

CFTR Modulator Therapies

57

Ivacaftor 57

Ivacaftor / Lumacaftor 58

Annual Data Report 2015 Cystic Fibrosis Foundation Patient Registry 3

COMPLICATIONS

60

CF Complications by Age, 2015

62

Cystic Fibrosis-Related Diabetes (CFRD)

66

TRANSPLANTATION

69

Lung Transplantation

69

SURVIVAL

71

Survival Metrics

72

Median Predicted Survival 72

Mortality Rate 73

Life Expectancy 73

Median Age at Death 75

Causes of Death

75

REFERENCES

76

CF FOUNDATION PATIENT REGISTRY QUESTIONNAIRE

78

APPENDIX

87
Box-and-Whisker Charts to Show Center-Level and Population-Level Variation 87
Using Combined Data Charts to Display Selected Attributes, by Age 88
Cystic Fibrosis Foundation Patient Registry Annual Data Report 20154 Summary of the Cystic Fibrosis Foundation Patient Registry, 2000-2015

Demographics20002005201020142015

People with CF (n)22,20123,08226,36628,68028,983

Newly diagnosed individuals (n)

A

9729381,112923853

Detected by newborn screening (%)8.118.654.963.159.6

Mean age at diagnosis (years)3.13.23.53.83.8

Median age at diagnosis (months)66544

Mean age (years)16.917.819.220.520.9

Median age (years)14.815.817.218.218.6

Adults 18 years (%)38.742.747.550.751.6

Race (not mutually exclusive)

White (%)95.495.194.393.993.8

African American (%)3.93.94.34.64.6

Other race (%)1.42.12.83.13.3

Hispanic (any race) (%)5.36.37.28.28.5

Males (%)52.952.051.751.551.6

Mortality

Total deaths (n)422358420467448

Annual mortality rate (per 100)1.91.51.61.61.5

Predicted median survival (years)33.337.939.040.041.7

95% condence interval (years)31.0-35.134.7-40.836.4-41.638.2-42.138.5-44.0

Median age at death (years)26.226.527.529.430.1

GI/Nutrition

BMI percentile, individuals 2 to 19 years (median)40.345.650.253.454.2 Percent weight < 10th CDC percentile25.219.215.212.812.4 Percent height < 5th CDC percentile16.214.011.310.49.9 BMI, individuals 20 to 40 years (median)21.021.522.122.322.4 Pancreatic enzyme replacement therapy (%)96.194.687.487.487.1 Supplemental feeding - tube (%)8.810.011.211.511.7 Supplemental feeding - oral only (%)27.937.440.944.443.9

Pulmonary

FVC % predicted (mean)

B

81.584.486.987.687.8

FEV 1 % predicted (mean) B

70.673.675.676.276.4

FEV 1 /FVC ratio (mean)73.674.774.874.674.5

Respiratory Microbiology

P. aeruginosa (PA) (%)

C

58.856.551.447.647.5

Multidrug-resistant PA (%)

D

3.78.79.18.69.2

B. cepacia complex (%)3.23.12.52.52.6

S. aureus (SA) (%)

E

49.863.667.070.070.6

Methicillin-sensitive S. aureus (MSSA) (%)45.351.750.453.254.0 Methicillin-resistant S. aureus (MRSA) (%)6.117.425.825.826.0

S. maltophilia (%)6.912.513.913.313.6

Mycobacterial species (%)

F --10.112.211.9

Table continues on the next page

Annual Data Report 2015 Cystic Fibrosis Foundation Patient Registry 5 Summary of the Cystic Fibrosis Foundation Patient Registry, 2000-2015 continued Health Care Utilization and Pulmonary Exacerbations G

20002005201020142015

Outpatient visits to CF centers reported per year (mean)5.44.24.74.54.4 Treated with IV antibiotics for a pulmonary exacerbation (%)-34.634.334.934.9 Number of pulmonary exacerbations per year (mean)-0.60.60.70.7 Number of days of treatment for pulmonary exacerbation per year (mean) H -30.630.531.631.1 Number of days of home IV treatment for exacerbations per year (mean) H -13.511.711.610.8 Number of days of hospitalization for pulmonary exacerbation per ye ar (mean) H -17.118.820.020.3

Pulmonary Therapies

I

Dornase alfa ( 6 years) (%)60.171.681.886.186.9

Inhaled tobramycin (PA+ and 6 years) (%)

J

65.169.070.669.870.2

Inhaled aztreonam (PA+ and 6 years) (%)--22.542.542.7

Azithromycin (PA+ and 6 years) (%)

K --69.367.566.6

Hypertonic saline ( 6 years) (%)--52.0 65.768.6

Ivacaftor ( 6 years with G551D mutation) (%)---89.390.4 Ivacaftor/Lumacaftor ( 12 years and F508del Homozygous) (%) 41.3

Oxygen (%)

L --10.811.311.1

Non-invasive ventilation (%)--2.32.92.9

Transplants

Lung (all procedures) (n)168154193207216

Liver (n)2115171615

Kidney (n)047138

Lost to Follow Up

M Lost to follow up (per 100 individuals)2.54.12.82.82.5 A We anticipate that additional 2015 diagnoses will be entered into the Registry in 2016. B Pulmonary function data throughout this report reect the use of GLI equations 2 for both children and adults. C Includes PA and multidrug-resistant PA, found in any culture during the year. D Dened as resistant to all antibiotics tested in two or more classes. E

Includes MSSA and MRSA and reects the prevalence of S. aureus among individuals who had a bacterial culture during the year.

e percentages for MSSA and MRSA individually are greater than the total S. aureus percentage because MSSA and MRSA are not

mutually exclusive. F

Percentage of individuals with one or more mycobacterial species isolated out of those individuals who had a mycobacterial culture

during the year. is includes M. tuberculosis as well as nontuberculous mycobacteria (NTM) species. G Dened as a period of treatment with intravenous (IV) antibiotics in the hospital and/or at home. Hquotesdbs_dbs23.pdfusesText_29

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