Original Article
Clinical Characteristics and Safety of Plasma-Derived C1-Inhibitor Therapy in Children and Adolescents with Hereditary Angioedema—A Long-Term Survey

https://doi.org/10.1016/j.jaip.2020.02.043Get rights and content
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Background

Plasma-derived C1-inhibitor (pdC1-INH) is a first-line therapy for hereditary angioedema (HAE) with C1-inhibitor deficiency (C1-INH-HAE) in pediatric patients.

Objective

We intended to study the clinical characteristics and safety of treatment with pdC1-INH in this population.

Methods

In the prospective, long-term survey, real-world data on pdC1-INH (Berinert, CSL Behring) use in pediatric patients, diagnosed and followed up at our Angioedema Reference Center, were analyzed for the period from 1986 to 2018.

Results

A total of 70 pediatric patients (31 boys and 39 girls) experienced a total of 3009 HAE attacks. The most common location of HAE attacks was subcutaneous. HAE attacks of any location were more frequent in girls versus boys, except for genital edema. Among the 70 patients, 37 received pdC1-INH for 456 HAE attacks, or as prophylaxis (69 vials). On average, 14.2 vials were administered per patient. The distribution of pdC1-INH use in the different age groups was as follows: no use (0-1 years), 0.11 vials/year (1-3 years), 0.7 vials/year (3-6 years), 1.26 vials/year (6-12 years), and 1.28 vials/year (12-18 years). No systemic allergic reactions, viral transmission, development of anti-C1-INH antibodies, or thromboembolic events occurred in relation to treatment with this drug.

Conclusion

We confirmed that the clinical manifestations and the use of pdC1-INH are different in the various age groups of pediatric patients with C1-INH-HAE. Our long-term survey shows that the use of pdC1-INH is safe in this patient population.

Key words

Hereditary angioedema
Pediatric patients
Clinical symptoms
Safety
Plasma-derived C1-inhibitor
Inherited risk factors for thromboembolism
Anti-C1-inhibitor antibodies

Abbreviations used

C1-INH
C1-inhibitor
C1-INH-HAE
Hereditary angioedema with C1-inhibitor deficiency
HAE
Hereditary angioedema
ITP
Intermittent prophylaxis
LA
Lupus anticoagulant
pdC1-INH
Plasma-derived C1-inhibitor concentrate
STP
Short-term prophylaxis

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No funding was received for this work.

Conflicts of interest: H. Farkas has received honoraria and travel grants from CSL Behring, Shire, Swedish Orphan Biovitrum, and Pharming; and/or served as a consultant for these companies and has participated in clinical trials/registries for BioCryst, CSL Behring, Pharming, and Shire. K. V. Kőhalmi has received honoraria and travel grants from CSL Behring and Shire and has participated in clinical trials of BioCryst, CSL Behring, Pharming, and Shire. L. Varga has received travel grants from CSL Behring and Shire. The rest of the authors declare that they have no relevant conflicts of interest.

These authors contributed equally to this work.