Friday 12 October 2012

CF Registry Clinical Data Manager - Day two

Elaine Gunn, CF Registry Clinical Data Manager, presents her highlights of day 2 at the NACFC
Day two was the first full day of meetings at conference. There were several parallel workshops during the morning and I chose to attend W2 - Emerging approaches to CF therapy. The highlights of this workshop, and most relevant to me in terms of the Registry, were:
a) The presentation about predicting response to inhaled Mannitol treatment in CF patients. After the worldwide trials of Mannitol, of which we in the UK were heavily involved, the data was assessed to see whether an early response to the drug could be maintained. The data showed that if someone responded well in terms of improved FEV1 and reduction in exacerbations at six weeks then this response would be maintained at six months. Likewise if the patient did not respond at six weeks to Mannitol then there would be no response at six months
b) The presentation about the Phase 2 trials of the investigational CFTR corrector VX-809 (lumacaftor) administered in combination with ivacaftor (better known as Kalydeco) for patients with the genetic mutation F508del. Several different doses were used and the data showed that 600mg of VX-809 in conjunction with 250 mg of ivacaftor gave the best response in patients with two copies of F508del, showing a significant improvement in FEV1. The next step for this was to move to Phase 3 studies – a really exciting proposal for the future for patients with F508del gene mutations.
This brought me to lunchtime where I had a chance to visit the poster presentations in the exhibit hall. I looked out the posters with reference to the Registry including looking at some work on renal clearance from the Nottingham group using Registry data and gender differences in the UK from the Brompton group using Registry data. It is always exciting to see the UK CF Registry being referenced.
The afternoon took me to a symposium on updates to infection control guidelines and then onto the first plenary session where the 3000+ delegates all sat engrossed in the topic of reversing the basic defect – a vision for the future (I will leave this bit of the blog to Dr Janet Allen, our Director of Research!).
Finally at 6pm the welcome reception provided a chance for networking and renewal of acquaintances from previous conferences, and discussion around the common themes of our work.
And so to bed with the alarm set for 0600 for day 3!

No comments:

Post a Comment