HAE international home therapy consensus document

Longhurst, Hilary; Farkas, Henriette; Craig, Timothy J.; Aygören‐Pürsün, Emel; Bethune, Claire; Björkander, Janne; Bork, Konrad; Bouillet, Laurence; Boysen, Henrik Balle; Bygum, Anette; Caballero, Teresa; Cicardi, Marco; Dempster, John; Gompels, Mark; Gooi, Jimmy; Grigoriadou, Sofia; Huffer, Ursula; Kreuz, W.; Levi, Marcel; Long, Janet; Martinez‐Saguer, Inmaculada; Raguet, Michel; Reshef, Avner; Bowen, Tom; Zuraw, Bruce L.

doi:10.1186/1710-1492-6-22

Cited by 157 publications

(207 citation statements)

References 40 publications

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“…Ecallantide, SDP, and FFP are not recommended for self‐therapy because of a small risk of anaphylaxis; however, in‐home therapy by a nurse trained in the treatment of anaphylaxis is an option for ecallantide 22, 90, 111, 112, 113.…”

Section: Resultsmentioning

confidence: 99%

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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

174

308

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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.

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Section: Resultsmentioning

confidence: 99%

“…In addition, even at a young age, pediatric patients can be taught to safely administer intravenous and subcutaneous therapy as is obvious from data from hemophilic patients 90.…”

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

174

308

View full text Add to dashboard Cite

show abstract

“…Early C1-INH treatment during an attack shortens its duration and reduces its severity more effectively. [76][77][78][79][80] In addition, this might facilitate prophylactic treatment in patients with very frequent angioedema attacks. [81][82][83][84][85] In life-threatening bradykinin-mediated edema of the pharynx or larynx, if the threat of asphyxion is present, conicotomy is necessary.…”

Section: Characteristic Features Of Bradykinin-mediatedmentioning

confidence: 99%

Bradykinin-mediated angioedema

Obtułowicz¹

2016

Polish Archives of Internal Medicine

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“…103 Home therapy refers to treatment given by the patient (self-administration) or a trained caregiver outside of a healthcare facility. On-demand home therapy with C1-esterase inhibitor has proven successful in selected patients and offers patients the possibility of earlier treatment and better symptom control [103][104][105][106] ; however, this use is not currently approved in the United States. For prophylaxis, oral attenuated androgens or IV C1-esterase inhibitor are at the forefront.…”

Section: Clinical Experience Discussionmentioning

confidence: 99%

Hereditary Angioedema Caused By C1-Esterase Inhibitor Deficiency: A Literature-Based Analysis and Clinical Commentary on Prophylaxis Treatment Strategies

Gower

Busse

Aygören‐Pürsün

et al. 2011

World Allergy Organization Journal

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Abstract:Hereditary angioedema (HAE) caused by C1-esterase inhibitor deficiency is an autosomal-dominant disease resulting from a mutation in the C1-inhibitor gene. HAE is characterized by recurrent attacks of intense, massive, localized subcutaneous edema involving the extremities, genitalia, face, or trunk, or submucosal edema of upper airway or bowels. These symptoms may be disabling, have a dramatic impact on quality of life, and can be life-threatening when affecting the upper airways. Because the manifestations and severity of HAE are highly variable and unpredictable, patients need individualized care to reduce the burden of HAE on daily life. Although effective therapy for the treatment of HAE attacks has been available in many countries for more than 30 years, until recently, there were no agents approved in the United States to treat HAE acutely. Therefore, prophylactic therapy is an integral part of HAE treatment in the United States and for selected patients worldwide. Routine long-term prophylaxis with either attenuated androgens or C1-esterase inhibitor has been shown to reduce the frequency and severity of HAE attacks. Therapy with attenuated androgens, a mainstay of treatment in the past, has been marked by concern about potential adverse effects. C1-esterase inhibitor works directly on the complement and contact plasma cascades to reduce bradykinin release, which is the primary pathologic mechanism in HAE. Different approaches to long-term prophylactic therapy can be used to successfully manage HAE when tailored to meet the needs of the individual patient.

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HAE international home therapy consensus document

Cited by 157 publications

References 40 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

Bradykinin-mediated angioedema

Hereditary Angioedema Caused By C1-Esterase Inhibitor Deficiency: A Literature-Based Analysis and Clinical Commentary on Prophylaxis Treatment Strategies

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