beeldscherm schreef op 9 november 2017 10:53:
P-10
Results from an interim analysis of a Ruconest treatment registry
in Europe
R. Hakl1
, M. Staevska2
, H. Farkas3
, M. Jesenak4
, K. Hrubiskova5
, L. Bellizzi6
, A.
Relan6
, M. Cicardi7
1
St. Anne’s University Hospital of Brno, Brno, Czech Republic; 2
University
Hospital “Alexandrovska” Medical University of Sofa, Sofa, Bulgaria; 3
Sem-
melweis University of Budapest, Budapest, Hungary; 4
University Hospital
of Martin, Martin, Slovakia; 5
Bratislava University Hospital, Bratislava,
Slovakia; 6
Pharming Technologies BV, Leiden, The Netherlands; 7
Hospital
Luigi Sacco University of Milan, Milan, Italy
Correspondence: L. Bellizzi (L.bellizzi@pharming.com)
Allergy, Asthma & Clinical Immunology 2017, 13(Suppl 2):P-10
Background: Hereditary angioedema (HAE) due to C1 inhibitor
defciency (C1-INH-HAE) is characterized by recurrent episodes of
disabling and painful swelling. Ruconest is a recombinant human C1
inhibitor that is approved for treatment of HAE attacks. A treatment
registry was established in Europe to review the adverse event profle
and efcacy of Ruconest following single and repeated treatment with
Ruconest.
Methods: Patients with C1-INH-HAE were enrolled following a decision
to treat with Ruconest and after providing written informed
consent. Medical history and baseline HAE information was collected
at a screening visit. Treatment decisions were at the discretion of the
health care professionals (HCP) involved in the patients’ care according
to their standards for the management of C1-INH-HAE and in line with
the approved Ruconest summary of product characteristics. Following
treatment with Ruconest, the HCP entered data using a web-based
questionnaire about the attack, response to therapy, and any adverse
events.
Results: As of 28 February 2017, 45 C1-INH-HAE patients (18 male/27
female, ages 22–76 years) were treated with Ruconest in the registry
for 1351 attacks in 7 European countries.
The average age at diagnosis for these patients was 24 years (range
4–68). Prior to entry in the registry, these patients experienced an average
of 30 HAE attacks in the preceding year. Of the treated patients,
28.8% were on maintenance therapy/prophylaxis at enrollment. There
were 653 (48.3%) abdominal, 528 (39%) peripheral, 203 (15%)facial, 21
(1.6%) laryngeal, and 24 (1.8%) urogenital attacks, including 76 attacks
that involved two and one attack that involved three locations.
The mean Ruconest dose provided was 3268 units 43 U/kg (range
18–67 U/kg), patients reported relief within 4 h in 97.8% (1322/1351)
of the attacks. Almost all attacks (1349/1351, 99.8%) were treated with
a single dose of Ruconest. Two attacks treated with an initial dose of
2100 U (33 and 28 U/kg) received a second dose of 2100 U. No hypersensitivity
or thrombotic/thromboembolic events were reported. No
patients had any related serious adverse events.
Conclusions: The Ruconest treatment registry provides real-world
data on the treatment of 1351 HAE attacks that is consistent with previous
reports on the safety and efcacy of Ruconest therapy
1- - 28.8% were on maintenance therapy/prophylaxis at enrollment
2- - patients reported relief within 4 h in 97.8% (1322/1351)
of the attacks. Almost all attacks (1349/1351, 99.8%) were treated with
a single dose of Ruconest.
do we need to say more....Blockbuster !!