Table
3: Assessment on pediatric use for the treatment of HAE-C1-INH.
Active ingredient/trade
name |
Risks and adverse
events |
Human pdC1-INH
concentrates |
|
Berinert |
Pediatric data for Berinert indicate that is efficacy and safety of 20U/kg
in pediatric patients are comparable with that for adults |
Cinryze |
Pediatric data for Cinryze indicate that its efficacy, safety and
tolerability in pediatric patients are comparable with that for adults. The
recommended dose (1000 U) is the same for all types of attacks and for all
body weigths. The paucity of data on body
weight-based dosing, as is otherwise common in pediatric, is seen as
critical. No adverse effects in adults and adolescents from high doses have
become known to date. |
Recombinat
human C1-INH concentrate |
|
Ruconest |
Until data on children
and adolescents are published and approval of the drug is granted, Ruconest should only be used when specifically warranted. |
Kallikrein and
bradikinin receptor antagonist |
|
Icatibanr/Firazyr |
On a preliminary basis,
no recommendation favoring use in children and adolescents can be made, given
the lack studies in pediatric patients. |
Ecallantide/Kalbitor |
The currently available
data on the treatment of pediatric patients is insufficient. Moreover, no European
approval is available. |
Attenuated androgens |
|
Danazol/Danatrol |
Given their adverse
reaction profile, at present, androgens should not be used for |
Danazol/Danokrin |
The long-term propylaxis of pediatric HAE-C1-INH. Neither can a
recommendation |
Danazol/Danokrine |
be given for short-term
prophylaxis, despite the fact that danazol is
approved in |
Stanozolol/Winstrol |
Switzerland for
children < 12 years of age |
Antifibrinolitics |
|
Tranexamic
acid/Cylokapron |
The use of Ԑ-aminocaproic
acid cannot be recommended, as it is approved in some countries by regulatory
authorities for the treatment of HAE. Due to the doubtful efficacy of tranexamic acid, it should be avoided for short-term and
long-term prophylaxis in pediatric patient. |