jurpsy @ 01-12-2013 20:50:39 (http://www.ErikErik.com / Pharming)
DIFFERENT FORMS OF HAE PROPHYLAXIS
Murat Bas, Ulrich Straßen
Technical University Munich, Munich, Germany
Several forms for prophylactic treatment of HAE are possible. We have used in our angioedema center three forms of prophylaxis: 1. Short term prophylaxis before surgery, 2. Long term prophylaxis and 3. On demand prophylaxis. We used for the three prophylaxis forms following drugs: Icatibant in short term prophylaxis and on demand prophylaxis, C1 inhibitor concentrates in all prophylaxis forms. All prophylaxis treatment forms were successful.
Patients with short term prophylaxis were treated with icatibant or C1 INH 30-60 minutes pre surgery, 2 hours post-surgery and 12 h post-surgery. In long surgeries with more than 2 h or large blood-loss an additional application of icatibant or C1 INH was given. Long term prophylaxis was possible with Ruconest, Berinert and Cynrize. We have used all three C1 INH options in long term prophylaxis. Patients get 2 times in the week the C1 INH concentrates for 12 weeks. Our results demonstrate here a significant reduction of angioedema attacks.
jurpsy @ 01-12-2013 20:45:19 (http://www.ErikErik.com / Pharming)
Background: Hereditary Angioedema (HAE) is a rare disease with estimated prevalence of 1:10000 to 1:50000 individuals. According to this incidence, Latin America should have up to 57,000 HAE patients. In order to evaluate LA situation regarding HAE diagnosis and therapy, we developed a registry of confirmed cases. Methods: The 1st Latin American Meeting on HAE took place on June 2nd, 2012 and the delegates representing LA countries collected HAE data. Clinical data and access to therapy was evaluated. Results: The following countries participated: Panama, Paraguay, Uruguay, Chile, Peru, El Salvador, Costa Rica, Colombia, Mexico, Brazil and Argentina. The last 4 countries presented data from 20, 34, 276 and 227 cases, respectively. Only 15 HAE patients were identified in the other countries. Difficult access to therapy was detected and fresh frozen plasma is still the therapeutic resource to treat the patients. Conclusions: Deficient identification of HAE patients as well as knowledge about the disease was identified. The access to laboratorial diagnosis and therapy is restricted in allcountries, with more profound problems regarding drugs and assistance for HAE attacks.The authors propose a plan to action including registry of identified cases, educative programs, laboratorial support with reference centers, and access to prophylactic and attack treatment.
jurpsy @ 01-12-2013 20:40:19 (http://www.ErikErik.com / Pharming)
RUCONEST IN ROUTINE CLINICAL PRACTICE: UK EXPERIENCE
A.L. Manson, J. Dempster, S. Grigoriadou, M.S. Buckland, Hilary J. Longhurst
Department of Immunology, Barts Health NHS Trust, London E1 2ES, UK
Conestat alfa (Ruconest) is a recombinant human C1 inhibitor protein (C1INH) that is extracted from the milk of transgenic rabbits. It was licensed in Europe for the treatment of acute attacks of hereditary angiodema (HAE) in 2010. Here we describe our experiences treating the first 11 adult patients who have received Ruconest outside a clinical trial setting in the UK. Nine patients had HAE type 1, one had HAE type 2 and one had acquired C1INH deficiency.
We have considered the prescription of Ruconest in preference to plasma-derived C1INH (pdC1INH), which is comparatively well established in routine clinical practice, in several clinical situations:- when patients prefer to avoid blood products, for heavier patients, when the licensed dose of Ruconest is more cost effective and for patients showing a sub-optimal response to 20 U/Kg of pdC1INH.
The majority of the patients self administered the drug at home and reported that having fewer vials to prepare was a benefit. Eight patients were treated with 4200 and three with 2100 units of Ruconest. Ruconest appeared effective and was well-tolerated by all patients, and six of the patients chose to continue to use it as their preferred treatment for acute attacks. Some patients reported a more rapid time to initial response and complete resolution of their symptoms following treatment with Ruconest compared to their usual pdC1INH. However, three patients experienced recurrent swellings following treatment with Ruconest:- Two patients with very frequent attacks (occurring more than twice weekly) treated with 4200 and 2100 units reported recurrences after 11 hours and 2 days respectively - these were treated successfully with pdC1INH; The third patient, who has acquired C1INH deficiency associated with mild C1INH resistance, had a recurrence at 7 hours after Ruconest that subsequently resolved with tranexamic acid.
Overall, Ruconest was effective and well-tolerated; it has the potential to improve outcomes in HAE