# ANIL MEHTA Slides.ppt by hands2urself

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```									                    Registries need a mission statement

Leonardo Da Vinci

‘measure what is measurable’

But then (use the registry)

‘make measurable what is not measurable’
Why do we need a Registry?

My answer is centred around a frequent question:

How long will my CF child live?

Median or middle value or 50% or half way
Sorting data small to large; boys & girls

Discussion on what it means
Country
CF child 1
CF child 2
3         Suppose we only have
4       5 new CF patients in 1 year
5             in a country

Categorise by age
Unknown number
of
CF patients

…………..

1Yr 2   3   4 etc etc

Getting older

A parent asks: How long will my child live?    Survival and CF
Problem 1   5
?????? – numbers vary per annum

REGISTER
1

2

? number                 3

4
5

1   2   3   4   5   6

Problem 2             ??? CF
Deaths
We want to do this for Europe but we cannot – need for a register

Measure median survival

Median (Middle, 50%) Survival = the same patients tracked over time

1988
50% have died

1970            20 years               1990

1988 - 1970 = 18 as median (middle) 50% survival
250 CF babies/children every year in the UK

Eur Respir J. 2007 29:522-6.
Males                Females
1989

1979
**

Born in 1960-79

** Debate point 1: Three quarter survival should be the new measure

Median Survival is no longer a good measure of outcome today
Median survival for a child born today…:

high-risk ‘severe’ genotype lowest age 36.3yr

low-risk ‘mild’ genotype lowest age 50yr

US data Chest 2006; 130: 1441-7

Parents ask: when will my CF baby die?
Deaths : concept of median age at death

CF Deaths

A new question:
Of those (few) who die,
when are half of them dead
Parents ask: when will my CF baby die?

Median survival for a child born today…:

high-risk ‘severe’ genotype 36.3yr

low-risk ‘mild’ genotype 50yr

50% value of the small number who die      Median
high-risk genotype 24.2yr                  Age
low-risk genotype 37.6yr                   Death
McKone et al. Chest 2006; 130: 1441-7
Median age at Death (20-30):

High Death Group

High Death Risk
+             High Death Group

Severe CF

Mild
CF

Median survival (30-50) is not the same as median age at death
When will my CF baby die?

It depends…
› When / where was my CF baby born?
› What is the condition of my baby now (genes:
CF + LUCK M6PR, TGFbeta etc)?
› What treatment do I have access to?
› Does my carer know about CF?
› Does my carer team work as a team?
Answer: probably well into the 50’s…but
› With huge variability
Update on gene modifiers in cystic fibrosis.
Collaco JM, Cutting GR.
Curr Opin Pulm Med. 2008 Nov;14(6):559-66. Review.
Europe as a whole is diverse
International Registry:
How many/where/deaths

Has to be cross sectional

International Clinical
Database: how sick; new
drugs; clinical trials
What will we need to make it work?

New skill Mix

Margaret Fraser…………………..………………….someone who knows data

Gita Mehta…………………………someone who knows project management

Milan Macek…………………someone who has a network through genetics

David Sheppard………………………………………….manage the resources

Who has taken up the job?

Hanne Oleson /Laura Viviani…. ……………………………..to carry the torch forward when we finish in June

Martin Stern……………………………………………….who knows clinical data quality and defines everything
Who do I really have to thank?

The CF patients
The CF Centre staff across Europe
The CFF for linking up with the ECFS

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