Prolastin Campaign - Mar 2008 | Details about Prolastin | Patient Comments |
Announcement of the Results of the ADAPT EXACTLE Prolastin Trial
Mon 9 June - Birmingham
On Mon 9 June Professor Stockley announced in the UK that the EXACTLE trial of Prolastin had shown Prolastin to be effective in slowing the progression of emphysema. Prolastin is Talecris' replacement therapy.
A key figure in medical research is that the level of accuracy of the results of a drug trial needs to allow for an error of less that 5% of the results to be wrong (5 in a 100) and the figure in EXACTLE is calculated to be 0.049 (49 in 1000) using one of the methods of analysis.
Professor Stockley's presented in as clear a form as possible to patients, the science of the Prolastin trial. The meeting took place in the Malmaison Hotel in Birmingham, UK and was attended by patients on the trial, Dr Alice Wood and Dr Richard Carter, Dr Anita Pye, three reps from Talecris who manufacture Prolastin including Amy McGrath of Global Marketing.
Professor Stockley gave some history of ADAPT including the point that its foundation had the objective of collecting results from a wide variety of respiratory tests, blood tests, CT scans etc. These results should be accessible and reusable for research into the future. NHLBI had done a research project years ago but access to the data had been limited.
Other previous Prolastin studies include:
a. NHLBI Study, reported in AJRCCM 1998
b. Danish-German Study, reported in ERJ in 1997
c. Danish-Dutch study, reported in AJRCCM - monthly infusion
d. German, reported in Chest, 2001
e. UK Phase 2 Study, reported in AJRCCM 2002
Summary of the EXACTLE Trial
a) The rate of decline in Prolastin patients became very clearly slower after about 12 months, when the levels of AAT started to have a real affect on the lungs.
b) hospitalisation of Prolastin patients appeared to be 1/3 of that of the placebo patients
c) The comparative graphs over the 24 - 30 months of the trial showed that there was a difference in decline between those on the placebo and those on Prolastin.
d) Most decrease in lung deterioration took place in the lower lung
e) the trial involved patient groups in UK, Denmark and Sweden.
f) A1R (the alpha-1 register in Europe) has a total constituency of about 4000 patients now
g) EXACTLE stands for EXacerbation And CT Scan as Lung Endpoint
h) There was a lot of discussion about how to evaluate the results so eventually 4 methods of analysis were used in parallel. All four methods showed a decreased rate of lung deterioration, although only one method reflected the conventional level of clinical significance.
The Next Steps
a) The Research Paper is a joint document between UK, Denmark and Sweden
b) The Research Paper is written and complete
c) Publication of the Research Paper is the next step and 3 journals are being considered.
d) Once publication has occurred it will come to the notice of the wider respiratory community and in particular to the notice of NICE - the National Institute for Clinical Excellence, the UK’s licensing body.
e) NICE can fast-track a new treatment in 12-months, particularly if there are no other options. However this is not to be assumed.
f) It is hoped that there will be a second trial of inhaled Prolastin starting in about 18 months time.
Talecris
a) Talecris will be proactive with NICE in working towards licensing in the UK.
b) This study supports the decision to allow the drug to be prescribed in the USA. It was pointed out that the basis of the US license 20 years ago was the fact that Prolastin infusions caused the serum levels in the blood to rise and it was assumed that the presence of a normal level would be efficacious. However, EXACTLE was the first clinical trial that had proved efficacy.
The Role of the Patient Group: alpha-1 awareness
Professor Stockley emphasized the importance of the patient group and the need for us to be involved in lobbying.
It was reported that the patient group had had a mini-campaign recently to write to MPs and MEPs about Prolastin and the interest and response was quite encouraging.
This information is now in the public domain and there is every reason for patients to inform their consultants and GPs of a) the results of the Prolastin trial, that b) a journal publication is hopefully imminent and that c) NICE will be subsequently contacted about licensing
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