C1 Esterase Inhibitor (Human) for the Treatment of Acute Hereditary Angioedema

2011 ◽  
Vol 4 ◽  
pp. CMBD.S4090 ◽  
Author(s):  
Solange Oliveira Rodrigues Valle ◽  
Alfeu Tavares França ◽  
Regis A. Campos ◽  
Anete Sevciovic Grumach
Keyword(s):  
Quality Of Life ◽  
Rare Disease ◽  
Hereditary Angioedema ◽  
C1 Esterase Inhibitor ◽  
Genetic Deficiency ◽  
Life Threatening ◽  
Patients Experience ◽  
Tremendous Impact ◽  
Esterase Inhibitor

Hereditary angioedema (HAE) is a relatively rare disease characterized by acute episodes of swelling. These swellings can be disfiguring, painful and life-threatening. Since the symptoms occur in different areas and most patients experience a delay in their diagnosis, resulting in unnecessary suffering and dangerous situations. HAE can have a tremendous impact on the quality of life. The major genetic deficiency in this disorder is either an absent or nonfunctional C1INH which regulates the complement, fibrinolitic, kalikrein and plasmin pathways.

Successful Long-Term Prophylactic Treatment With Subcutaneous C1 Esterase Inhibitor in a Patient With Hereditary Angioedema

2019 ◽  
Vol 33 (6) ◽  
pp. 907-911
Author(s):  
Janina Hahn ◽  
Melanie Nordmann-Kleiner ◽  
Susanne Trainotti ◽  
Thomas K. Hoffmann ◽  
Jens Greve
Keyword(s):  
Quality Of Life ◽  
Hereditary Angioedema ◽  
Prophylactic Treatment ◽  
Upper Airway ◽  
Venous Access ◽  
Current Standard ◽  
C1 Esterase Inhibitor ◽  
Esterase Inhibitor

Background: Hereditary angioedema (HAE) patients suffer from recurrent swellings. Current standard therapy consists of C1 esterase inhibitor (C1-INH) and bradykinin receptor B2 antagonists. Severe courses require prophylactic treatment. For such patients, it has been demonstrated that the intravenous (IV) administration of C1-INH [C1-INH(IV)] is safe and effective. A new prophylactic option is subcutaneous (SC) treatment with C1-INH. Methods and Case: We present the case of an HAE patient placed on prophylactic C1-INH(IV) therapy due to frequent attacks when managed with on-demand therapy. An implanted port allowed the periodical and safe application of medication until the device was explanted due to an infection. Due to the poor venous access, repeated IV application failed. Therefore, we began a SC treatment with 1500 IU C1-INH [C1-INH(SC)] as long-term prophylaxis and analyzed the clinical course over 16 months. Results: Under the SC prophylaxis, the number of attacks were reduced to 1/month in comparison to 4.33/month with no prophylactic treatment and 1.83/month with C1-INH(IV). No severe attacks and no attack within the upper airway occurred over the 16 months of C1-INH(SC) treatment. As a result, quality of life improved, as measured by the Angioedema quality of life questionaire (AE-QoL). Conclusion: Self-administered SC prophylactic use of C1-INH over a period of 16 months seems to be a well tolerated and efficient. The patient’s quality of life improved, and by learning self-application, the patient gained independence.

scholarly journals Hereditary Angioedema: a Challenging Diagnosis for the Gastroenterologist

2016 ◽  
Vol 1 (3) ◽  
pp. 287-292
Author(s):  
Gabriella Gábos ◽  
Dumitru Moldovan ◽  
Daniela Dobru
Keyword(s):  
Quality Of Life ◽  
Differential Diagnosis ◽  
Hereditary Angioedema ◽  
Genetic Disorder ◽  
Laryngeal Edema ◽  
Cutaneous Manifestations ◽  
C1 Esterase Inhibitor ◽  
High Degree ◽  
Esterase Inhibitor

Abstract Hereditary angioedema (HAE) caused by a deficiency of C1 esterase inhibitor enzyme (C1-INH) is a very rare, autosomal dominantly inherited genetic disorder, characterized by recurrent peripheral angioedema, painful abdominal attacks and episodes of laryngeal edema. Abdominal attacks are frequent symptoms in adult HAE patients, occurring in more than 90% of the cases. Angioedema in the bowel or abdomen can occur in the absence of cutaneous manifestations and may be easily misdiagnosed unless the clinician has a high degree of awareness to include HAE in the differential diagnosis. Misdiagnosis is associated with inadequate treatments, including unnecessary surgical procedures. Any patient who presents recurrent episodes of swelling should be evaluated for HAE caused by C1-INH deficiency. New therapies could save lives and dramatically improve their quality of life.

Etiology and predictors of cluster attacks of hereditary angioedema that recur despite pharmaceutical treatment

2021 ◽  
Vol 42 (4) ◽  
pp. 317-324
Author(s):  
Felix. A. Johnson ◽  
Magdalena Wirth ◽  
Zhaojun Zhu ◽  
Janina Hahn ◽  
Jens Greve ◽  
...  
Keyword(s):  
Hereditary Angioedema ◽  
Upper Airway ◽  
Poor Quality ◽  
Prophylactic Therapy ◽  
On Demand ◽  
C1 Esterase Inhibitor ◽  
Physical Stimuli ◽  
Inhibitor Treatment ◽  
Esterase Inhibitor

Introduction: Hereditary angioedema (HAE) is a disease that leads to recurrent swelling of the skin and mucous membranes, including the upper airway tract. Apart from being deadly, these attacks can be debilitating, which leads to a poor quality of life in patients. Clinicians are occasionally confronted with patients who have recurrent attacks despite treatment with C1 esterase inhibitor concentrate or β2-receptor antagonists. The goal of this study was to investigate repeated attacks that occur 48 hours to 7 days (“cluster attacks”) after treatment, to determine why they occur and the factors that may be associated with them, and thus to prevent their occurrence. Methods: We conducted a multicenter mixed retrospective-prospective study with data acquired from all documented attacks in our patients with collective (n = 132) between 2015 and 2018. Results: Eighty-five percent (n = 132) of our total patient collective (N = 156) agreed to participate in the study. Nine percent of these patients (n = 12) had cluster attacks, with a total of 48 cluster attacks. The data procured from the patients were mixed retrospective‐prospective. Approximately 72% of all the cluster attacks were caused by exogenous stimuli (41% due to psychological stress, 29% due to physical stimuli, and 2% due to menstruation). Cluster attacks occurred in 7% of the patients who received prophylactic therapy in comparison with 12.5% of patients who received on-demand therapy. Cluster attacks comprised 48.4% of all the attacks that patients with cluster-attacks (n= 9) experienced. In addition, the patients who were underdosing their C1 esterase inhibitor treatment had cluster attacks more often. A lower “time to repeated attack” was seen in the patients who received on-demand therapy compared with those who received prophylactic therapy. Discussion: The percentage of the patients who had attacks as a result of exogenous triggers was higher in the cluster-attack group (70.5%) compared with the general HAE population (30‐42%). Repeated attacks, therefore, were strongly associated with external triggers. The patients who received prophylactic treatment and who experienced cluster attacks were highly likely to have been underdosing, which may explain the repeated attacks despite treatment. In the patients prone to cluster attacks, prophylaxis should be considered.

scholarly journals Hereditary angioedema: an update on causes, manifestations and treatment

2019 ◽  
Vol 80 (7) ◽  
pp. 391-398 ◽  
Author(s):  
Hilary J Longhurst ◽  
Konrad Bork
Keyword(s):  
Quality Of Life ◽  
Hereditary Angioedema ◽  
Genetic Disorder ◽  
Treatment Options ◽  
Correct Diagnosis ◽  
Self Administration ◽  
Diagnosis And Management ◽  
Life Threatening ◽  
Short And Long Term

Hereditary angioedema is a rare genetic disorder caused by deficiency of C1 esterase inhibitor (C1-INH) and characterized by recurrent episodes of severe swelling that affect the limbs, face, intestinal tract and airway. Since laryngeal oedema can be life-threatening as a result of asphyxiation, correct diagnosis and management of hereditary angioedema is vital. Hereditary angioedema attacks are mediated by bradykinin, the production of which is regulated by C1-INH. Hereditary angioedema therapy relies on treatment of acute attacks, and short- and long-term prophylaxis. Acute treatment options include C1-INH concentrate, icatibant and ecallantide. Self-administration of treatment is recommended and is associated with increased quality of life of patients with hereditary angioedema. Advances in diagnosis and management have improved the outcomes and quality of life of patients with hereditary angioedema.

scholarly journals Hereditary angioedema: experience of substitution therapy with a C1 esterase inhibitor in the Sverdlovsk region

2019 ◽  
Vol 16 (3) ◽  
pp. 61-66
Author(s):  
E K Beltyukov ◽  
S S Vedenskaya ◽  
I S Skorokhodov ◽  
V V Naumova ◽  
M V Beltyukova ◽  
...  
Keyword(s):  
Side Effects ◽  
Pregnant Women ◽  
Receptor Antagonist ◽  
Rare Disease ◽  
Hereditary Angioedema ◽  
Substitution Therapy ◽  
National Guidelines ◽  
Ministry Of Health ◽  
C1 Esterase Inhibitor ◽  
Esterase Inhibitor

Hereditary angioedema (HAO) is rare disease, however, it's lifethreatening localization can be fatal. Antifibrinolytics and attenuated androgens used for the prevention of HAO attacks have side effects, which limit their use. The bradykinin B2 receptor antagonist, Icatybant is an effective but shortacting dmedication. The human C1 esterase inhibitor (berinert) is safe and effective for the prevention and relief of angioedema of lifethreatening localization, including in pregnant women. The management of patients with HAO is regulated by national guidelines, and the use of a human C1 esterase inhibitor is recommended by instruction of the drug and by the Ministry of Health of the Sverdlovsk region. Patients with HAO should be provided with patient’s passport and fill a diary of symptoms monitoring of HAO.

НАСЛЕДСТВЕННЫЙ АНГИООТЕК: ОРГАНИЗАЦИЯ ОКАЗАНИЯ ПОМОЩИ БОЛЬНЫМ И ОПЫТ ЗАМЕСТИТЕЛЬНОЙ ТЕРАПИИ ИНГИБИТОРОМ С1 ЭСТЕРАЗЫ В СВЕРДЛОВСКОЙ ОБЛАСТИ

2019 ◽  
pp. 61-66
Author(s):  
E.K. Beltyukov ◽  
S.S. Vedenskaya ◽  
I.S. Skorokhodov ◽  
V.V. Naumova ◽  
M.V. Beltyukova ◽  
...  
Keyword(s):  
Side Effects ◽  
Pregnant Women ◽  
Receptor Antagonist ◽  
Rare Disease ◽  
Hereditary Angioedema ◽  
National Guidelines ◽  
Ministry Of Health ◽  
Bradykinin B2 Receptor ◽  
C1 Esterase Inhibitor ◽  
Esterase Inhibitor

Наследственный ангиоотек (НАО) встречается редко, тем не менее его жизнеугрожающая локализация может привести к летальному исходу. Применяемые для профилактики атак НАО антифибринолитики и аттенуированные андрогены обладают побочными действиями, что ограничивает их использование. Антагонист Р2рецепторов брадикинина икатибант является эффективным, но короткодействующим препаратом. Ингибитор С1 эстеразы человека (Беринерт) является безопасным и эффективным для профилактики и купирования ангиоотеков жизнеугрожающей локализации, в том числе у беременных женщин. Ведение больных НАО регламентировано национальными рекомендациями, а применение ингибитора С1 эстеразы человека инструкцией на препарат и письмом Минздрава Свердловской области. Больные с НАО должны при себе иметь паспорт пациента и дневник контроля симптомов НАО.Hereditary angioedema (HAO) is rare disease, however, its lifethreatening localization can be fatal. Antifibrinolytics and attenuated androgens used for the prevention of HAO attacks have side effects, which limit their use. The bradykinin B2 receptor antagonist, Icatybant is an effective but shortacting dmedication. The human C1 esterase inhibitor (berinert) is safe and effective for the prevention and relief of angioedema of lifethreatening localization, including in pregnant women. The management of patients with HAO is regulated by national guidelines, and the use of a human C1 esterase inhibitor is recommended by instruction of the drug and by the Ministry of Health of the Sverdlovsk region. Patients with HAO should be provided with patients passport and fill a diary of symptoms monitoring of HAO.

scholarly journals Successful use of lanadelumab in an elderly patient with type II hereditary angioedema

2021 ◽  
Author(s):  
Tasha S. Hellu ◽  
Samuel L. Weiss ◽  
Derek M. Smith
Keyword(s):  
Hereditary Angioedema ◽  
Genetic Disorder ◽  
Poor Quality ◽  
Type I ◽  
Type Ii ◽  
C1 Esterase Inhibitor ◽  
Submucosal Edema ◽  
Esterase Inhibitor

Hereditary angioedema (HAE) is a rare genetic disorder characterized by recurring episodes of subcutaneous and/or submucosal edema without urticaria due to an excess of bradykinin (1, 2). HAE is classified into 2 main types (1). Type I HAE is caused by deficiency of C1 esterase inhibitor, accounting for 85% of cases (1). Type II HAE occurs in only 15% of cases and is marked by normal to elevated levels of C1 esterase inhibitor but with a reduction in activity (1). An angioedema attack can range in severity depending on the location and degree of edema (2). Furthermore, patients with HAE are often diagnosed with anxiety and depression secondary to their poor quality of life (3). Thus, long-term prophylaxis of attacks can be crucial to reduce the physical and psychological implications. For long-term prophylaxis, lanadelumab, a subcutaneously delivered monoclonal antibody inhibitor of plasma kallikrein, has been proven to decrease the frequency of HAE attacks without significant side effects (4). However, data is limited, specifically regarding patients with type II HAE and patients >/= 65 years (4).

scholarly journals Hereditary angioedema and pregnancy: successful management of recurrent and frequent attacks of angioedema with C1-inhibitor concentrate, danazol and tranexamic acid – a case report

2009 ◽  
Vol 2 (3) ◽  
pp. 123-125 ◽  
Author(s):  
D S Milingos ◽  
P Madhuvrata ◽  
J Dean ◽  
A Shetty ◽  
D M Campbell
Keyword(s):  
Case Report ◽  
Pregnant Woman ◽  
Tranexamic Acid ◽  
Hereditary Angioedema ◽  
C1 Inhibitor ◽  
Successful Management ◽  
C1 Esterase Inhibitor ◽  
Threatening Condition ◽  
Life Threatening ◽  
Esterase Inhibitor

Hereditary angioedema (HAE) is a rare but potentially life-threatening condition caused by deficiency of C1 esterase inhibitor. It is characterized by subcutaneous swelling in any part of the skin, gastrointestinal and respiratory tracts. We present the case of a pregnant woman with known HAE that deteriorated during pregnancy with frequent attacks that were managed successfully with danazol, tranexamic acid and regular intravenous administration of C1 esterase inhibitor.

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