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Review
. 2023 Oct;62(9):973-980.
doi: 10.1177/00099228231155703. Epub 2023 Mar 12.

A Comprehensive Management Approach in Pediatric and Adolescent Patients With Hereditary Angioedema

Raffi Tachdjian  1   2 , Allen P Kaplan  3
Affiliations
Review

A Comprehensive Management Approach in Pediatric and Adolescent Patients With Hereditary Angioedema

Raffi Tachdjian et al. Clin Pediatr (Phila). 2023 Oct.

Abstract

Hereditary angioedema (HAE) is a rare autosomal-dominant disorder; most cases are characterized by low plasma levels of C1 esterase inhibitor (C1-INH). Clinical manifestations of HAE due to C1-INH deficiency include unpredictable, acute, recurrent episodes of nonpruritic swelling that can affect the face, trunk, limbs, and the respiratory, gastrointestinal, and genitourinary tracts. Attacks can be disfiguring, disabling, painful, and even life-threatening if laryngeal swelling occurs. Symptoms of HAE generally manifest in childhood. Effective medications are available and approved to treat HAE in children. However, evidence informing use of these medications in pediatric clinical practice is limited. Hereditary angioedema management plans are critical to optimize outcomes and should address on-demand treatment for acute attacks and plans to prevent potentially fatal laryngeal attacks. The plan should also comprise a holistic approach to address nonclinical aspects of HAE, including quality of life (QoL) and psychological issues. This article provides an overview of HAE management principles that health care providers can apply to treat pediatric patients to improve their QoL.

Keywords: hereditary angioedema; on-demand therapy; prophylaxis; quality of life.

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Conflict of interest statement

The authors declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Raffi Tachdjian- Speaker or advisory board: Biocryst, Takeda, CSL Behring, Pharming; Research contracts: Biocryst, Takeda, CSL Behring, Pharming, Kalvista, Pharvaris, Ionis.

Figures

Figure 1.
Figure 1.
HAE workup algorithm. Abbreviations: HAE, hereditary angioedema; C1-INH, C1 esterase inhibitor; C4, complement protein 4.
See this image and copyright information in PMC

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