Current treatment of hereditary angioedema: An update on clinical studies.

Hereditary angioedema (HAE) is a rare, potentially life-threatening disease that manifests as recurrent episodes of nonpruritic swelling that may affect the extremities, face, genitalia, gastrointestinal tract, and/or larynx. HAE is the result of a deficiency of functional C1-esterase inhibitor (C1-INH), a key regulator of the complement, coagulation, and kallikrein-kinin cascades. In HAE patients, overactivation of the kallikrein-kinin cascade results in excessive release of bradykinin, the mediator of the pain and swelling that is characteristic of HAE. Historically, treatment options for HAE have been limited, but newly approved and emerging therapies, such as C1-INH replacement products, a plasma kallikrein inhibitor, and a bradykinin B₂-receptor antagonist, appear to provide safe and effective relief for a significant proportion of patients with HAE. Because they may have therapeutic and practical advantages over existing HAE therapies, the new agents have the potential to improve the overall management of patients with HAE. This article reviews the results from recent clinical trials of these drugs and considers their role in clinical practice.

[1]  J. Bernstein,et al.  Current and Emerging Management Options for Hereditary Angioedema in the US , 2012, Drugs.

[2]  J. Bernstein,et al.  Efficacy of human C1 esterase inhibitor concentrate compared with placebo in acute hereditary angioedema attacks. , 2009, The Journal of allergy and clinical immunology.

[3]  B. Zuraw,et al.  Update on therapeutic developments for hereditary angioedema. , 2009, Allergy and asthma proceedings.

[4]  B. Zuraw,et al.  Pathogenesis and laboratory diagnosis of hereditary angioedema. , 2009, Allergy and asthma proceedings.

[5]  K. Wyrwich,et al.  Psychometric validation of two patient-reported outcome measures to assess symptom severity and changes in symptoms in hereditary angioedema , 2009, Quality of Life Research.

[6]  T. Craig,et al.  When is prophylaxis for hereditary angioedema necessary? , 2009, Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology.

[7]  William H. Yang,et al.  C1 Esterase Inhibitor Concentrate (C1-INH) for the Treatment of Acute Attacks of Hereditary Angioedema: Interim Results of an Ongoing, Prospective, Open Label Study in North America after 640 attacks (I.M.P.A.C.T.2) , 2009 .

[8]  A. Sheffer,et al.  The spectrum of chronic angioedema. , 2009, Allergy and asthma proceedings.

[9]  T. Craig,et al.  Treatment of hereditary angioedema with plasma-derived C1 inhibitor , 2008, Therapeutics and clinical risk management.

[10]  A. Davis,et al.  New treatments addressing the pathophysiology of hereditary angioedema , 2008, Clinical and molecular allergy : CMA.

[11]  H. Farkas,et al.  Clinical Studies of Recombinant Human C1 Inhibitor (rhC1INH) in Patients with Acute Attacks of Hereditary Angioedema , 2008 .

[12]  A. Reshef,et al.  Icatibant, a New Bradykinin B2 Receptor Antagonist, for Acute Hereditary Angioedema Attacks , 2008 .

[13]  J. Bernstein.  Hereditary angioedema: a current state-of-the-art review, VIII: current status of emerging therapies. , 2008, Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology.

[14]  William H. Yang,et al.  Hereditary angiodema: a current state-of-the-art review, VII: Canadian Hungarian 2007 International Consensus Algorithm for the Diagnosis, Therapy, and Management of Hereditary Angioedema. , 2008, Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology.

[15]  B. Zuraw.  Hereditary angiodema: a current state-of-the-art review, IV: short- and long-term treatment of hereditary angioedema: out with the old and in with the new? , 2008, Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology.

[16]  H. Farkas,et al.  Management of Hereditary Angioedema in Pediatric Patients , 2007, Pediatrics.

[17]  H. Farkas,et al.  Recombinant human C1‐inhibitor in the treatment of acute angioedema attacks , 2007, Transfusion.

[18]  C. Hermans.  Successful management with C1-inhibitor concentrate of hereditary angioedema attacks during two successive pregnancies: a case report , 2007, Archives of Gynecology and Obstetrics.

[19]  M. Frank.  Hereditary angioedema: the clinical syndrome and its management in the United States. , 2006, Immunology and allergy clinics of North America.

[20]  K. Bork,et al.  Missense mutations in the coagulation factor XII (Hageman factor) gene in hereditary angioedema with normal C1 inhibitor. , 2006, Biochemical and biophysical research communications.

[21]  K. Bork,et al.  Hereditary angioedema: new findings concerning symptoms, affected organs, and course. , 2006, The American journal of medicine.

[22]  M. López-Trascasa,et al.  Hereditary angioedema due to C1 inhibitor deficiency: patient registry and approach to the prevalence in Spain. , 2005, Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology.

[23]  M. Gompels,et al.  C1 inhibitor deficiency: consensus document , 2005, Clinical and experimental immunology.

[24]  K. Kragballe,et al.  Hereditary Angioedema. , 2018, The New England journal of medicine.

[25]  J. Lindner,et al.  Safety and efficacy of pasteurized C1 inhibitor concentrate (Berinert® P) in hereditary angioedema: a review , 2003 .

[26]  A. Adinoff.  Treatment of 193 Episodes of Laryngeal Edema with C1 Inhibitor Concentrate in Patients with Hereditary Angioedema , 2002, Pediatrics.