Hereditary angioedema in women

Bouillet, Laurence

doi:10.1186/1710-1492-6-17

Cited by 62 publications

(47 citation statements)

References 51 publications

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“…The clinical presentation of HAE, including age of symptom onset, anatomical location, frequency, and severity [6,7,8], are diverse, and about 50% of patients can experience potentially fatal laryngeal attacks [9,10]. Examples of attack triggers are stress, hormonal changes, surgical or dental procedures, infection, or hormonal therapy for women such as oral contraceptives or hormone replacement therapy [11,12,13,14]. …”

Section: Introductionmentioning

confidence: 99%

Preventing Hereditary Angioedema Attacks in Children Using Cinryze®: Interim Efficacy and Safety Phase 3 Findings

Aygören‐Pürsün

Soteres

Moldovan

et al. 2017

Int Arch Allergy Immunol

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Background: Hereditary angioedema (HAE) is a rare genetic disease causing unpredictable and potentially life-threatening subcutaneous and submucosal edematous attacks. Cinryze® (Shire ViroPharma Inc., Lexington, MA, USA), a nanofiltered C1 inhibitor (C1-INH), is approved in Europe for the treatment, preprocedure prevention, and routine prophylaxis of HAE attacks, and for the routine prophylaxis of attacks in the USA. This phase 3 study assessed the safety and efficacy of 2 C1-INH doses in preventing attacks in children aged 6-11 years. Methods: A randomized single-blind crossover study was initiated in March 2014. Results for the first 6 patients completing the study are reported here. After a 12-week qualifying observation period, patients were randomly assigned to 1 of 2 C1-INH doses, 500 or 1,000 U, every 3-4 days for 12 weeks and crossed over to the alternative dose for a second 12-week period. The primary efficacy endpoint was the number of angioedema attacks per month. Results: Six females with HAE type I and a median age of 10.5 years received 2 doses of C1-INH (500 and 1,000 U). The mean (SD) difference in the number of monthly angioedema attacks between the baseline observation period and the treatment period was -1.89 (1.31) with 500 U and -1.89 (1.11) with 1,000 U. During the treatment periods, cumulative attack severity, cumulative daily severity, and the number of attacks needing acute treatment were lower. No serious adverse events or study drug discontinuations occurred. Conclusions: Interim findings from this study indicate that routine prevention with intravenous administration of C1-INH is efficacious, safe, and well tolerated in children ≥6 years of age.

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Section: Introductionmentioning

confidence: 99%

Preventing Hereditary Angioedema Attacks in Children Using Cinryze®: Interim Efficacy and Safety Phase 3 Findings

Aygören‐Pürsün

Soteres

Moldovan

et al. 2017

Int Arch Allergy Immunol

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“…It is characterized by normal C1-INH levels and activity 30. This group includes diseases that vary in their etiopathogenesis; coagulation disorders and links to the endocrine system have been identified in this group 9,31,32…”

Section: Classificationmentioning

confidence: 99%

Brazilian guidelines for the diagnosis and treatment of hereditary angioedema

Giavina‐Bianchi

França

Grumach

et al. 2011

Clinics

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Hereditary angioedema is an autosomal dominant disease characterized by edema attacks with multiple organ involvement. It is caused by a quantitative or functional deficiency of the C1 inhibitor, which is a member of the serine protease inhibitor family. Hereditary angioedema is unknown to many health professionals and is therefore an underdiagnosed disease.The causes of death from hereditary angioedema include laryngeal edema with asphyxia. The estimated mortality rate in patients in whom the disease goes undetected and who are therefore incorrectly treated is 25-40%. In addition to edema of the glottis, hereditary angioedema often results in edema of the gastrointestinal tract, which can be incapacitating. Patients with hereditary angioedema may undergo unnecessary surgical interventions because the digestive tract can be the primary or only organ system involved, thus mimicking acute surgical abdomen. It is estimated that patients with hereditary angioedema experience some degree of disability 20-100 days per year.The Experts in Clinical Immunology and Allergy of the “Associação Brasileira de Alergia e Imunopatologia - ASBAI” developed these guidelines for the diagnosis, therapy, and management of hereditary angioedema.

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“…Acne resulting from attenuated androgen therapy also can be a significant source of anxiety among patients. Because of the risk of causing teratogenic effects in an unborn fetus, in particular masculinization, nonhormonal contraception or progestin-only birth control pills are recommended in fertile female patients taking attenuated androgens[79]. Many female patients also report menstrual irregularities such as menometrorrhagia or amenorrhea while receiving attenuated androgen therapy[60,75].…”

Section: Experience With Attenuated Androgen Therapy In the Long-termmentioning

confidence: 99%

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

Gower¹,

Busse²,

Aygören‐Pürsün³

et al. 2011

World Allergy Organization Journal

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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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Hereditary angioedema in women

Cited by 62 publications

References 51 publications

Preventing Hereditary Angioedema Attacks in Children Using Cinryze®: Interim Efficacy and Safety Phase 3 Findings

Preventing Hereditary Angioedema Attacks in Children Using Cinryze®: Interim Efficacy and Safety Phase 3 Findings

Brazilian guidelines for the diagnosis and treatment of hereditary angioedema

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

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