Brief History in USA & UK Cystic Fibrosis Foundation, USA: 1955 – Foundation was established 1980 – Patient registry established – became a national summary & anonymous 2001 – centres got their own data 2003 – open to centres & public (transparency) Cystic Fibrosis Trust, UK: 2007 – UK joined PORT CF

Brief History in NZ Timeline: 2009 – PORT CF (big ups to CFNZ & CFF USA) 2009 – Standards of Care (minimum requirements) 2010 - IT housed in Christchurch 2011 - Christchurch Earthquake 2012 - CFA NZ support small centres to enter data Annual review & backgrd demographic data

48.3% adults 54% male / 46% female

PORT CF: Demographics 2014 USA UK AUST* NZ Total patients 28,676 10,583 3,235 443 Mean age 20.2 yrs 19 yrs 20 yrs 18.1 yrs % adults 50.7% 57.9%* 49.9% 48.3% Mean FEV1 76.2% 73.5% 85.1% Predicted survival 39.3 yrs 40.1 yrs ??? Australia – 59% children have normal lung function, 15% males & 17% females as adults have normal lung function * Australia registry is 2013 * 16yrs vs 18 yrs

Lung Function trends over time North American Cystic Fibrosis Foundation Patient Registry Annual Report 2012

75% -80% are oral supplements with about 12% gasotrosmy feeds – similar across paeds & adults

< 16 yrs n = 155 Number of individuals employing the technique at least once per year No physiotherapy - in children across the years has increased from 2.3% to 3.9%. No physiotherapy - in adults it is higher at 13.3% but has dropped over the years from 16.1% Using exercise – 13.5% children & 35.5% adults

Number of individuals employing the technique at least once per year No physiotherapy - in children across the years has increased from 2.3% to 3.9%. No physiotherapy - in adults it is higher at 13.3% but has dropped over the years from 16.1% Using exercise – 13.5% children & 35.5% adults > 16 yrs N = 203

PORT CF: Medication use 2014 USA UK AUST* NZ DNase 88.7% 54.5% 41.6% 26% Macrolide 67.3% 52.5% 35.3% 25% H/Saline 66.3% 26.1% Neb Antibx 71.4% (T) 41.8% (Az) 88.6% (49.8%) 12% IHCS 43.8% 28% Some variances here – UK say number with chronic PsA infection, USA has > 6 year age

PORT CF: Medication: Ivacaftor CFF, USA Registry report: Ivacaftor use in those with an eligible mutation: 80%

Survival In 2014 – current life expectancy - 39.3 years CF Foundation Patient Registry Report, USA 2014

Median FEV1 in Paediatric Centres (children >6 years age) UK CF Trust Registry Each bar represents a different centre

Median FEV1 in Adult Centres UK CF Trust Registry Each bar represents a different centre

% with chronic Pseudomonas infection in Paediatric Centres (children >6 years age) UK CF Trust Registry UK mean Chronic infection – whereas our question is ever in the last year Each bar represents a different centre

% with chronic Pseudomonas infection in Adult Centres UK CF Trust Registry UK mean Each bar represents a different centre

Have we made progress? 2011 2015 Persons with CF registered 415 443 Median age in years 15.7 yrs 18.1 yrs PWCF aged > 16 years 49.6% 55.8% Genotype known 87.7% 96.8% Median FEV1 < 16 yrs >16 yrs 80.5% 91.6% 70.7% 85.1% 97.7% 78%

CF Data Network www.cysticfibrosisdata.org

Changes for the future? ABILITY TO INPUT DATA ACCESS TO DATA Questions have been modified Age brackets altered Comparison to other registries