Unfortunately a lot of the event was in German and the translation sometimes left a lot to be desired. Information contained within this report is my understanding of what was sometimes explained in very medical terms and and every best effort has been made to ensure accuracy.

Dr Karin Schmid-Scherzer, Wilhelminenspital, Wien,

The Austrian Alpha1 Scene

In Austria they receive Prolastin, an augmentation therapy used to treat Alpha1. It is only used in Austria to treat patients with a FEV1 >35% and <60%. 0.1% of the Austrian population had A1AD.

Prof. Dr. Robert Stockley, ADAPT, Queen Elizabeth Hospital, Birmingham, UK,

The Influence of EXACTLE for replacement therapy

He stated that FEV1/FVC measurements were the best to indicate lung function for those suffering from bottom lung emphysema. Whereas Gas Transfer measurements were best to indicate lung function for the top of lung emphysema.

Two thirds of Alpha?s suffer with lower lung problems and one third with top lung problems.

EXACTLE stands for EXAcerbations CT scan as Lung Endpoints. The trial used 60mg Prolastin per kg body weight.

In year 1 they noted that there was not much change although subsequent years showed much better results. The trial was shown to be good for lower lung emphysema.

They used 4 methods to analyse the data each giving slightly different results. He explained to be an accepted treatment trial data results must be within certain pre determined margins. Not all 4 methods produced results within these margins although those that were outside were not by a lot. Unfortunately, this means that the trial does not prove efficacy.

Therefore, before any augmentation therapy can be approved within the UK we will need Phase IV trials.

There are 3 companies currently producing Alpha1 augmentation therapies:-

Talecris ? Prolastin
Baxter ? Aralast
Behring ? Zemaira

Both Kamada and Talecris are trialing inhaled therapies at the moment. Both will start Phase II trials in 2009 with Phase III being planned for 2010. I understand both Phase?s will include the UK.

Dr. Till Voigtlander, Orphanet, Austria,

The activities of Orphanet Austria

www.orpha.net is a web site which acts as a portal for rare diseases. Alpha1 is also featured on this site along with over 5,000 other rare diseases. The web site developers are interested in receiving feedback on the functionality of the site or further information to be included on the site i.e. Patient Associations.

Dr. Klaus Kenn/Ursula Krutt-Bockemuhl, Klinikum Berchtesgadener
Land/LOT e.V.,

Travelling with Oxygen

It was advised that travelling to Altitudes of >1200/1500m (3950 ? 4900ft) is probably not recommended.

It was also recommended that when travelling you should take O2 certificates with you confirming your O2 requirement.

John Walsh of the Alpha-1 Foundation, USA, advised that passengers are now authorized to use any FAA approved portable oxygen concentrator
during any commercial flight that lands or takes off in the USA.

Dr. Klaus Pittschieler, Zentralkrankenhaus Bozen,

Neonatal Screening within Europe

Antioxidants are good for the liver, for example Vitamin A. Also it is advised to not be overweight and avoid alcohol.

Dr. Laura Fregonese, Leids Universitair Medisch Centrum,

PAAIR Project

AIR ? Alpha1 International Registry ? the International Registry for Alpha1, a clinical and scientific network.

PAAIR ? Patient Association and Alpha1 International Registry ? this is a pilot project financed by the EU to test the concept of networks of reference for rare diseases. They are currently producing a ?patients' experience and expectations? questionnaire.

Laura indicated that the REPAIR trial was not really positive although it did look good in parts.

Dr. George Lang, Universitatsklinik Wien,

The Transplant Centre

We were given various statistics relating to transplants in Austria.

Living Donor Transplants for Lung are only being completed for
children in Austria.

Living Donor Transplant for Kidney ? we were advised that a living
donor transplantee gains an extra 2 ½ years when compared against
cadaver donors.

Living Donor Transplants for Liver ? the right wing of the donor liver
is transplanted into the recipient.

Best wishes
Karen, Dundee