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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