Hereditary Angioedema

Research on hereditary angioedema (HAE), an autosomal dominant disorder caused by C1 esterase inhibitor deficiency leading to recurrent episodes of severe swelling. Studies evaluate attack resolution time, symptom relief, and prevention of attacks.

Quick Answer

What it is

Research on hereditary angioedema (HAE), an autosomal dominant disorder caused by C1 esterase inhibitor deficiency leading to recurrent episodes of severe swelling. Studies evaluate attack resolution time, symptom relief, and prevention of attacks.

Key findings

  • Grade A: Time to Symptom Relief (Icatibant (Firazyr))
  • Grade A: Primary Symptom Relief Onset (Icatibant (Firazyr))
  • Grade A: Cutaneous Attack Resolution (Icatibant (Firazyr))

Safety

  • Black box warning.
ℹ️ Quick Facts

Quick Facts: Hereditary Angioedema

  • Supplements Studied:2
2 supps · 9 outcomes

Detailed Outcomes

A
Time to Symptom Relief
FAST-3: 2.0 hours to 50%+ symptom reduction vs 19.8 hours placebo (P<0.001). FAST-2: 2.0 hours vs 12.0 hours tranexamic acid (P<0.001).
large↓Improves
A
Primary Symptom Relief Onset
FAST-3: Median onset of primary symptom relief 1.5 hours vs 18.5 hours placebo (P<0.001).
large↓Improves
A
Cutaneous Attack Resolution
Rapid and stable relief from cutaneous attacks demonstrated in Phase III trials.
large↑Improves
A
Abdominal Attack Resolution
Significant reduction in abdominal attack symptoms with rapid onset of action.
large↑Improves
A
Laryngeal Attack Resolution
Effective for potentially life-threatening laryngeal attacks, though emergency medical care still recommended.
large↑Improves
A
Symptom Improvement at 4 Hours
EDEMA3 & EDEMA4 Phase 3 (n=168): Treatment outcome score at 4h significantly greater with ecallantide vs placebo (53.4 vs 8.1, P=0.003 in EDEMA4). Mean symptom complex severity score change -0.8 vs -0.4 placebo (P=0.01). Led to FDA approval.
large↑Improves
A
Time to Symptom Relief
EDEMA3+4 pooled analysis: Benefit apparent within 2 hours of dosing. Maintained through 24 hours. Need for rescue intervention: 14% ecallantide vs 36% placebo within 24h. Earlier treatment associated with better outcomes.
moderate↑Worsens
A
Anaphylaxis Risk
SAFETY CONCERN: Anaphylaxis in 3.9% of patients (10/255) in clinical trials. Most within first hour after injection. Black box warning. Must be administered by healthcare professional. Have anaphylaxis treatment available. Self-administration not recommended.
small↑Worsens
B
Laryngeal Attack Efficacy
Subset analysis of laryngeal attacks: Significant improvement in treatment outcome scores. Potential life-saving for airway-threatening attacks. Limited patient numbers in trials - use with close monitoring for severe attacks.
moderate↑Improves

Research Citations (40)

Safety, efficacy, and pharmacokinetics of icatibant treatment in Japanese pediatric patients with hereditary angioedema: A phase 3, open-label study.
(2023)
PMID: 37381768
Three-Day Icatibant on Top of Standard Care in Patients With Coronavirus Disease 2019 Pneumonia: A Randomized, Open-Label, Phase 2, Proof-of-Concept Trial.
(2023)
PMID: 36610464
Is Icatibant Safe for the Treatment of Hereditary Angioedema During Pregnancy?
(2022)
PMID: 36044174
A multicenter, open-label, randomized, proof-of-concept phase II clinical trial to assess the efficacy and safety of icatibant in patients infected with SARS-CoV-2 (COVID-19) and admitted to hospital units without invasive mechanical ventilation: study protocol (ICAT-COVID).
(2022)
PMID: 35413921
Short review of SEC, a potential dexamethasone-sparing regimen for glioblastoma: Spironolactone, ecallantide, clotrimazole.
(2021)
PMID: 33450263
Immunotargeting and therapy of cancer by advanced multivalence antibody scaffolds.
(2020)
PMID: 32434407
Efficacy, pharmacokinetics, and safety of icatibant for the treatment of Japanese patients with an acute attack of hereditary angioedema: A phase 3 open-label study.
(2020)
PMID: 31672405
Icatibant, another piece of the therapeutic puzzle regarding hemodynamic side effects of angiotensin-converting enzyme inhibitors.
(2019)
PMID: 31462265
Effect of icatibant on angiotensin-converting enzyme inhibitor-induced angioedema: A meta-analysis of randomized controlled trials.
(2019)
PMID: 31290163
Self-administration of icatibant in acute attacks of Type I hereditary angioedema: A case report and review of hereditary angioedema.
(2019)
PMID: 31579975