Treatment of Hereditary C1 Inhibitor Deficiency in Patients Under 18 Years
Clinical Scenario
This protocol addresses the management of hereditary C1 inhibitor deficiency in pediatric patients — those under 18 years of age. Indications for first-line treatment are the same in children as in adults, but age-related regulatory differences affect which agents are appropriate for a given patient.
Treatment Approach
For on-demand treatment of acute attacks in this age group, plasma-derived C1 inhibitor is the treatment of choice, with select alternative agents available depending on the child's age. Anabolic androgens are not used in children. The full protocol specifies agent selection, age-based applicability, and the complete clinical algorithm.
References
DOI: 10.1016/j.jaip.2020.08.046
- Indications for the use of first-line HAE medications are the same in children as in adults, although regulatory differences affect the use of some medications depending on the child's age.
- The record of safety and efficacy data for pdC1INH in pediatric studies has made this the treatment of choice for on-demand use in the United States.
- Ecallantide is approved in the United States for children >12 years of age.
- Although icatibant is not currently approved for children <18 years of age in the United States, an open-label study in children (aged 2-17 years) demonstrated that it provided rapid relief and was well tolerated.
- The US HAEA MAB does not recommend the use of anabolic androgens for children due to the multiple concerns surrounding side effects and potential impact on growth, bone and, sexual development.
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