Treatment Of Hereditary Angioedema Attacks Research Articles

Treatment of Hereditary Angioedema Attacks with Icatibant: A Comparison of Observational Data with Clinical Trial Data

Treatment of Hereditary Angioedema Attacks with Icatibant: A Comparison of Observational Data with Clinical Trial Data

Benefits of Early Administration with Icatibant for the Treatment of Hereditary Angioedema Attacks

Benefits of Early Administration with Icatibant for the Treatment of Hereditary Angioedema Attacks

Per-Attack Reporting of Prodromal Symptoms Concurrent with C1-Inhibitor Treatment of Hereditary Angioedema Attacks

Prodromal symptoms commonly precede hereditary angioedema (HAE) attacks. There is continuing interest in evaluating prodromes as treatment indicators, but a paucity of relevant data. This study was designed to prospectively identify prodomal characteristics in patients voluntarily reporting such information around the time of seeking treatment for an acute HAE attack. Twenty-eight patients with HAE were enrolled in this survey, which was conducted in the context of an open-label study of treatment of HAE attacks with plasma-derived C1-inhibitor concentrate. At the time of treatment, patients were encouraged to answer survey questions about prodromal symptoms preceding that particular HAE attack. Twenty-one patients provided prodromal information for 253 treated HAE attacks. Seventy-one percent of patients (15/21) reported prodromes. Three patients accounted for approximately 80% of the attacks and 89% of the reported prodromal symptoms. Prodromes were experienced before 67.6% (171/253) of attacks, with a mean of 1.4 prodromes per attack. Fatigue was the most frequent prodrome (42% of attacks), followed by nausea (26%), and flu-like symptoms (22%). The median duration of a prodrome before an attack was 12 h (range, 0.33-24 h). Despite many limitations in the study design, these findings confirm that prodromes are frequently associated with HAE attacks in many patients and occur sufficiently early to allow time for treatment initiation. The frequency of "false positive" prodromal symptoms remains undetermined, and the authors captured data only on attacks severe enough to warrant treatment. Additional well-designed prospective studies are clearly needed to continue investigating the potential clinical relevance of prodromes.

Home therapy with intravenous human C1‐inhibitor in children and adolescents with hereditary angioedema

C1-esterase inhibitor (C1-INH) replacement therapy is the treatment of choice for acute edema attacks in patients with hereditary angioedema (HAE). Our retrospective, observational study assessed the efficacy and safety of home therapy with a human plasma-derived C1-INH concentrate (pC1-INH) in 20 pediatric patients with HAE who had previously been treated with physician-based therapy. While on home therapy, 15 patients received on-demand treatment and five received individual replacement treatment (IRT). The switch to home therapy did not involve a significant increase in the dose of pC1-INH administered, but there was a significant increase in dosing frequency. Although only two patients were affected, the frequency of laryngeal attacks appeared to decrease on home therapy. All attacks, including laryngeal edema, were treated successfully during home therapy with pC1-INH. The mean annual number of days hospitalized was reduced from 3.8 during physician-based therapy to 0.11 during home therapy. No side effects or injection site complications were reported. The median time from onset of attack to administration of pC1-INH was reduced from 67.5 minutes during physician-based therapy to 15 minutes after switching to home therapy. The corresponding median time to initial symptom relief for all types of attack was reduced from 60 to 40 minutes. As in adults, home therapy with pC1-INH is effective and safe in the treatment of HAE attacks in pediatric patients; a larger, randomized study should ideally confirm our findings before this approach can be considered the standard of care for pediatric patients.

Icatibant in the Treatment of Acute Attacks of Hereditary Angioedema (HAE) in Adults

Introduction: Hereditary Angioedema (HAE) is a potentially life-threatening condition consisting of recurrent episodes of limb, abdominal, genital, facial or laryngeal swelling. Unregulated bradykinin activity is known to be the main driving mechanism for the characteristic edema flares in HAE. Thus, a bradykinin receptor antagonist is a sensible treatment for acute attacks of HAE. Methods: A Pubmed literature search was conducted from 2005-present using the search terms icatibant, HOE 140, angioedema, and hereditary angioedema singly and in combination. Non-English articles were excluded. Results: Overall, there have been a total of 116 patients (314 attacks) treated with icatibant. Of these attacks, the majority reported the onset of symptom relief within two hours and the resolution of symptoms within 12–24 hours. Thirty patients (9.6%) had recurrent symptoms; five were treated with rescue Berinert, seven were treated with an additional dose of icatibant, and the remainder 18 were given either C1-INH concentrate, anti-emetics, or opiates. The most reported adverse effect from icatibant was a local reaction (swelling, burning, erythema, and itching). Icatibant use has also been reported with ACE-inhibitor induced, acquired, and HAE type III angioedema as well as in one case as prophylaxis. Conclusion: The numerable new treatments for HAE now gaining favor, including icatibant, provide an exciting opportunity for both patients and providers to take swift control of this disease process.

Pasteurized C1 inhibitor concentrate in hereditary angioedema: pharmacology, safety, efficacy and future directions

Hereditary angioedema (HAE) is a relatively rare genetic disorder that is most commonly caused by a deficiency of C1 inhibitor. It is estimated that HAE affects at least one in 10,000 to one in 50,000 of the worldwide population, with relapsing swelling of the skin and abdominal pain attacks being the most common clinical symptoms. Most seriously, laryngeal edema associated with HAE may lead to death. Replacement therapy with intravenous pasteurized C1 inhibitor concentrate is the recommended treatment for acute attacks of HAE, resulting in a rapid resolution of symptoms. Pasteurized C1 inhibitor concentrates can also be used for prophylaxis of HAE, and are currently also being assessed for home therapy in this setting. Future advances may improve disease burden and mortality associated with HAE.

Inherited C1 inhibitor deficiency.

The paper reports our experience of a 15-year follow-up of 179 patients with hereditary angioedema (HAE). The disease is transmitted as an autosomal dominant trait and two variants have been described: type I characterized by functional and antigenic C1-inhibitor (C1-INH) deficiency and type II with normal or upper normal C1-INH antigenic levels but no functional activity. The clinical picture is characterized by self-limiting bouts of swelling in the subcutaneous and/or mucous tissues. Data about frequency, localization and possible triggering factors of HAE attacks are reported. Analysis of genomic DNA from our patients using a C1-INH cDNA probe demonstrated the presence of restriction fragment length polymorphisms tightly linked with the disease in a minority of them. Data concerning prophylaxis and treatment of HAE attacks are also shown.