Recombinant Human-C1 Inhibitor Is Effective and Safe for Repeat Hereditary Angioedema Attacks

Li, H. Henry; Moldovan, Dumitru; Bernstein, Jonathan A.; Reshef, Avner; Porębski, Grzegorz; Stobiecki, Marcin; Baker, James; Levy, Robyn J.; Relan, Anurag; Riedl, Marc A.

doi:10.1016/j.jaip.2014.12.013

Cited by 33 publications

(36 citation statements)

References 22 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…rhC1‐INH is licensed by the FDA and EMA for the acute treatment of C1‐INH‐HAE for the patients aged 13 and older 96, 109. An open‐label treatment study with rhC1‐INH in a pediatric population (2–13 years) is ongoing.…”

Section: Resultsmentioning

confidence: 99%

International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency

Farkas

Martinez‐Saguer²,

Bork

et al. 2016

Allergy

177

308

View full text Add to dashboard Cite

BackgroundThe consensus documents published to date on hereditary angioedema with C1 inhibitor deficiency (C1‐INH‐HAE) have focused on adult patients. Many of the previous recommendations have not been adapted to pediatric patients. We intended to produce consensus recommendations for the diagnosis and management of pediatric patients with C1‐INH‐HAE.MethodsDuring an expert panel meeting that took place during the 9th C1 Inhibitor Deficiency Workshop in Budapest, 2015 (www.haenet.hu), pediatric data were presented and discussed and a consensus was developed by voting.ResultsThe symptoms of C1‐INH‐HAE often present in childhood. Differential diagnosis can be difficult as abdominal pain is common in pediatric C1‐INH‐HAE, but also commonly occurs in the general pediatric population. The early onset of symptoms may predict a more severe subsequent course of the disease. Before the age of 1 year, C1‐INH levels may be lower than in adults; therefore, it is advisable to confirm the diagnosis after the age of one year. All neonates/infants with an affected C1‐INH‐HAE family member should be screened for C1‐INH deficiency. Pediatric patients should always carry a C1‐INH‐HAE information card and medicine for emergency use. The regulatory approval status of the drugs for prophylaxis and for acute treatment is different in each country. Plasma‐derived C1‐INH, recombinant C1‐INH, and ecallantide are the only agents licensed for the acute treatment of pediatric patients. Clinical trials are underway with additional drugs. It is recommended to follow up patients in an HAE comprehensive care center.ConclusionsThe pediatric‐focused international consensus for the diagnosis and management of C1‐INH‐HAE patients was created.

show abstract

Section: Resultsmentioning

confidence: 99%

International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency

Farkas

Martinez‐Saguer²,

Bork

et al. 2016

Allergy

177

308

View full text Add to dashboard Cite

show abstract

“…The efficacy was maintained with treatment of subsequent attacks in the Open Label Extension phases [67][68][69][70]. No relapse or rebound episodes were reported for Ruconest, suggesting that its mechanism of action and PD properties, rather than its relatively short half-life, lead to sustained plasma activity.…”

Section: Parametersmentioning

confidence: 92%

“…The efficacy of Ruconest for the treatment of C1-INH-HAE patients experiencing acute angioedema attacks has been demonstrated in several studies, including three double-blind, placebo-controlled efficacy studies where patients were treated for a single C1-INH-HAE attack [33,35] and five open-label studies where patients could be treated for multiple attacks (Table 4) [67][68][69][70]74].…”

Section: Attack Treatmentmentioning

confidence: 99%

“…No relapse or rebound episodes were reported for Ruconest, suggesting that its mechanism of action and PD properties, rather than its relatively short half-life, lead to sustained plasma activity. In the largest of these studies (n = 224 attacks treated), 96% of attacks were treated with a single dose of Ruconest 50 U/kg (max 4200 U) [68]. Furthermore, no increase in the administration of an additional treatment dose was reported for subsequent attacks.…”

Section: Parametersmentioning

confidence: 99%

See 1 more Smart Citation

Recombinant Replacement Therapy for Hereditary Angioedema Due to C1 Inhibitor Deficiency

2015

Self Cite

View full text Add to dashboard Cite

Hereditary angioedema is a rare genetic condition transmitted as an autosomal dominant trait and characterized most commonly by the production of either inadequate or nonfunctioning C1 esterase inhibitor (C1-INH), a blood protein that regulates proteases in the complement, fibrinolytic and contact systems. Patients with hereditary angioedema suffer from episodic, unpredictable manifestations of edema affecting multiple anatomical locations, including the GI tract, facial tissue, the upper airway, oropharynx, urogenital region and/or the arms and legs. A rational approach to treatment is replacement of C1-INH protein, to normalize the levels of C1-INH activity and halt the progression of the biochemical activation processes underlying the edema formation. Ruconest is a highly purified recombinant human C1-INH. This article will focus on the results of ten clinical studies demonstrating the efficacy and safety of Ruconest(®) (Pharming Group NV, Leiden, the Netherlands), which is now approved for use in Europe, Israel and the USA.

show abstract

“…No relationship between anti-C1-INH antibody presence and rhC1-INH efficacy was observed. The open-label extension study evaluating the efficacy and safety of rhC1INH (50 IU/kg) for the treatment of multiple HAE attacks detected no discontinuation due to adverse events, no thrombotic or anaphylactic events, and no generation of neutralizing anti-C1INH antibodies [34]. Being rhC1INH purified from milk of transgenic rabbits, Hack et al conducted a retrospective analysis of the frequency and clinical relevance of pre-exposure and potentially newly induced, Immunoglobulin E (IgE) antibodies against rabbit and other animal allergens including cow's milk by the Immuno-CAP Specific IgE blood test system.…”

Section: Immunogenicitymentioning

confidence: 99%

The safety of treatments for angioedema with hereditary C1 inhibitor deficiency

Zanichelli

Andreoli

et al. 2015

Expert Opinion on Drug Safety

View full text Add to dashboard Cite

show abstract

Recombinant Human-C1 Inhibitor Is Effective and Safe for Repeat Hereditary Angioedema Attacks

Abstract: A single 50-IU/kg dose rhC1INH was effective for improving symptoms of an HAE attack with sustained efficacy for treatment of subsequent attacks. rhC1INH had a positive safety profile throughout the study. This study supports repeated use of rhC1INH over time in patients with HAE attacks.

Cited by 33 publications

References 22 publications

International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency

International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency

Recombinant Replacement Therapy for Hereditary Angioedema Due to C1 Inhibitor Deficiency

The safety of treatments for angioedema with hereditary C1 inhibitor deficiency

Contact Info

Product

Resources

About