The use of plasma-derived C1 inhibitor in the treatment of hereditary angioedema

C1-inhibitor (C1-INH) deficiency is the genetic defect underlying hereditary angioedema (HAE). Subjects with HAE suffer from recurrent angioedema that may result in death when it affects the larynx, severe abdominal pain when it affects the gastrointestinal mucosa and disfiguration when it affects the skin. The use of plasma-derived C1-INH concentrates to revert angioedema in HAE patients started in the 1970s. Since that time, three different preparations arrived onto the market, two of them are still present. Controlled studies and a large clinical experience indicate that C1-INH concentrate should be considered the treatment of choice for disabling angioedema attacks at any site. Efficacy has also been shown in preventing angioedema induced by invasive medical manoeuvres. Limited experience with repeated weekly infusions indicates that C1-INH can be used for long-term prophylaxis in selected patients. The safety profile is excellent and there are no reports of transmission of viral infections with the preparations available at present. C1-INH is licensed only in a limited number of countries. Clinical trials are ongoing at present to expand registration.

[1]  M. Frank 8. Hereditary angioedema. , 2008, The Journal of allergy and clinical immunology.

[2]  P. Gál,et al.  C1 Inhibitor Serpin Domain Structure Reveals the Likely Mechanism of Heparin Potentiation and Conformational Disease* , 2007, Journal of Biological Chemistry.

[3]  M. Cicardi,et al.  The deficiency of C1 inhibitor and its treatment. , 2007, Immunobiology.

[4]  H. Schuitemaker,et al.  Viral safety of C1-inhibitor NF. , 2007, Biologicals : journal of the International Association of Biological Standardization.

[5]  A. Leunig,et al.  [Quincke's edema: diagnosis and management of 102 patients with sudden upper airway obstruction]. , 2007, Laryngo- rhino- otologie.

[6]  T. Craig,et al.  Fresh frozen plasma for the treatment of hereditary angioedema. , 2007, Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology.

[7]  H. Farkas,et al.  C1-inhibitor (C1-INH) autoantibodies in hereditary angioedema. Strong correlation with the severity of disease in C1-INH concentrate naïve patients. , 2007, Molecular immunology.

[8]  Susan L. Stramer,et al.  Emerging Infectious Diseases That Threaten the Blood Supply , 2006, Seminars in Hematology.

[9]  K. Khair,et al.  C1-inhibitor concentrate home therapy for hereditary angioedema: a viable, effective treatment option , 2006, Clinical and experimental immunology.

[10]  D. Churchill,et al.  Pregnancy and C1 esterase inhibitor deficiency: a successful outcome , 2006, Archives of Gynecology and Obstetrics.

[11]  M. Levi,et al.  Self-administration of C1-inhibitor concentrate in patients with hereditary or acquired angioedema caused by C1-inhibitor deficiency. , 2006, The Journal of allergy and clinical immunology.

[12]  P. Tsang,et al.  Hereditary angioedema managed with low-dose danazol and C1 esterase inhibitor concentrate: a case report. , 2006, Journal of obstetrics and gynaecology Canada : JOGC = Journal d'obstetrique et gynecologie du Canada : JOGC.

[13]  Andrea Zanichelli,et al.  C1 inhibitor: molecular and clinical aspects , 2005, Springer Seminars in Immunopathology.

[14]  K. Bork,et al.  Treatment with C1 inhibitor concentrate in abdominal pain attacks of patients with hereditary angioedema , 2005, Transfusion.

[15]  G. Ambrus,et al.  A True Autoactivating Enzyme , 2005, Journal of Biological Chemistry.

[16]  H. Longhurst Emergency treatment of acute attacks in hereditary angioedema due to C1 inhibitor deficiency: what is the evidence? , 2005, International journal of clinical practice.

[17]  D. Lomas,et al.  Molecular mousetraps and the serpinopathies. , 2005, Biochemical Society transactions.

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

[19]  S. Thomas,et al.  Fresh frozen plasma for acute exacerbations of hereditary angioedema. , 2004, The American journal of emergency medicine.

[20]  William H. Yang,et al.  Canadian 2003 International Consensus Algorithm For the Diagnosis, Therapy, and Management of Hereditary Angioedema. , 2004, The Journal of allergy and clinical immunology.

[21]  A. Tordai,et al.  Hereditary and acquired angioedema: Problems and progress: Proceedings of the third C1 esterase inhibitor deficiency workshop and beyond , 2004, Journal of Allergy and Clinical Immunology.

[22]  Tsutomu Kobayashi,et al.  [A case of lung cancer with hereditary angioedema treated effectively by chemo-radiotherapy with C1 esterase inhibitor concentrate and danazol]. , 2004, Nihon Kokyuki Gakkai zasshi = the journal of the Japanese Respiratory Society.

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

[24]  G. M. Orfila,et al.  Profilaxis y tratamiento del angioedema hereditario y adquirido en el HUB; utilización del inhibidor de la C1-esterasa , 2003 .

[25]  A. Davis,et al.  Complement Regulatory Protein C1 Inhibitor Binds to Selectins and Interferes with Endothelial-Leukocyte Adhesion 1 , 2003, The Journal of Immunology.

[26]  M. Cicardi,et al.  Recent advances in the use of C1 inhibitor as a therapeutic agent. , 2003, Molecular immunology.

[27]  R. Jódar Massanés,et al.  [Profhylaxis and treatment of hereditary and acquired angioedema at HUB; use of the C1-esterase inhibitor]. , 2003, Farmacia hospitalaria : organo oficial de expresion cientifica de la Sociedad Espanola de Farmacia Hospitalaria.

[28]  H. Farkas,et al.  Clinical management of hereditary angio‐oedema in children , 2002, Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology.

[29]  M. Abinun,et al.  Current management of hereditary angio-oedema (C'1 esterase inhibitor deficiency). , 2002, Journal of clinical pathology.

[30]  W. Tremaine,et al.  Hereditary angioedema: a broad review for clinicians. , 2001, Archives of internal medicine.

[31]  C. Hack,et al.  In vitro interaction of C1-inhibitor with thrombin , 2001, Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis.

[32]  K. Bork,et al.  Treatment of 193 episodes of laryngeal edema with C1 inhibitor concentrate in patients with hereditary angioedema. , 2001, Archives of internal medicine.

[33]  C1 esterase inhibitor: new preparation. A major advance in emergency treatment of hereditary angioneurotic oedema. , 2001, Prescrire international.

[34]  T. Standl,et al.  Die perioperative Behandlung von Patienten mit hereditärem Angioödem (HAE) am Beispiel eines Jugendlichen mit Osteosynthese einer Oberschenkelfraktur , 2000 .

[35]  S. Thiel,et al.  Proteolytic Activities of Two Types of Mannose-Binding Lectin-Associated Serine Protease , 2000, The Journal of Immunology.

[36]  J. Myers,et al.  Successful extubation with use of C1 esterase inhibitor concentrate in a patient with hereditary angioedema. , 2000, Mayo Clinic proceedings.

[37]  J. Alvarez Successful use of C1 esterase inhibitor protein in a patient with hereditary angioneurotic edema requiring coronary artery bypass surgery. , 2000, The Journal of thoracic and cardiovascular surgery.

[38]  B. Eifrig,et al.  [Perioperative treatment of a patient with hereditary angioedema (HAE) in a case of a young patient with osteosynthesis from a femoral fracture]. , 2000, Anasthesiologie, Intensivmedizin, Notfallmedizin, Schmerztherapie : AINS.

[39]  A. Gaucher,et al.  Parturition and angioneurotic oedema. , 1999, International journal of obstetric anesthesia.

[40]  J. Karsh,et al.  C1-esterase inhibitor transfusions in patients with hereditary angioedema. , 1998, Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology.

[41]  F. Rosen,et al.  A randomized, controlled trial to study the efficacy and safety of C1 inhibitor concentrate in treating hereditary angioedema , 1998, Transfusion.

[42]  J. Bissler,et al.  Antibody to C1-inhibitor in a patient receiving C1-inhibitor infusions for treatment of hereditary angioneurotic edema with systemic lupus erythematosus reacts with a normal allotype of residue 458 of C1-inhibitor. , 1996, The Journal of laboratory and clinical medicine.

[43]  F. Rosen,et al.  Treatment of hereditary angioedema with a vapor-heated C1 inhibitor concentrate. , 1996, The New England journal of medicine.

[44]  A. Holdcroft,et al.  Hereditary angioneurotic oedema: current management in pregnancy , 1995, Anaesthesia.

[45]  P. Mannucci,et al.  Reduction in transmission of hepatitis C after the introduction of a heat‐treatment step in the production of C1‐inhibitor concentrate , 1995, Transfusion.

[46]  Maves Kk,et al.  Tonsillectomy in a patient with hereditary angioedema after prophylaxis with C1 inhibitor concentrate. , 1994 .

[47]  K. Aulak,et al.  Functional analysis of the serpin domain of C1 inhibitor. , 1994, Journal of immunology.

[48]  T. Burnouf,et al.  Large-scale preparation of highly purified human C1-inhibitor for therapeutic use. , 1994, Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis.

[49]  J. Weiner,et al.  C1‐esterase inhibitor concentrate prevents upper airway obstruction in hereditary angio‐oedema , 1994, The Medical journal of Australia.

[50]  J. Weiler,et al.  Tonsillectomy in a patient with hereditary angioedema after prophylaxis with C1 inhibitor concentrate. , 1994, Annals of allergy.

[51]  P. Spaeth,et al.  Hereditary angioedema: uncomplicated maxillofacial surgery using short-term C1 inhibitor replacement therapy. , 1993, International archives of allergy and immunology.

[52]  M. Cicardi,et al.  Hereditary and Acquired C1‐Inhibitor Deficiency: Biological and Clinical Characteristics in 235 Patients , 1992, Medicine.

[53]  J. Beechem,et al.  Mechanism of serpin action: evidence that C1 inhibitor functions as a suicide substrate. , 1991, Biochemistry.

[54]  X. Qian [Treatment of hereditary angioedema]. , 1990, Zhonghua er bi yan hou ke za zhi.

[55]  B. Wüthrich [Hereditary angioedema--substitution therapy with C1-inhibitor concentrate]. , 1989, Der Hautarzt; Zeitschrift fur Dermatologie, Venerologie, und verwandte Gebiete.

[56]  A. Szczeklik,et al.  [New possibilities of treating acute angioedema caused by C1-inhibitor deficiency]. , 1989, Polski tygodnik lekarski.

[57]  K. Bork,et al.  Long-term prophylaxis with C1-inhibitor (C1 INH) concentrate in patients with recurrent angioedema caused by hereditary and acquired C1-inhibitor deficiency. , 1989, The Journal of allergy and clinical immunology.

[58]  M. Wickerhauser,et al.  Heat Stability of Lyophilized C1 Inactivator Concentrates 1 , 1987, Vox sanguinis.

[59]  B. Bennett,et al.  Plasminogen activator in normal subjects after exercise and venous occlusion: t-PA circulates as complexes with C1-inhibitor and PAI-1. , 1987, Blood.

[60]  G. Lagrue,et al.  Oedème angioneurotique héréditaire. Traitement d'une crise digestive pseudo-chirurgicale par le C1-inhibiteur concentré. , 1987 .

[61]  K. Rahbar,et al.  [Hereditary angioneurotic edema. Treatment of a pseudosurgical digestive attack with C1 inhibitor concentrate]. , 1987, Presse medicale.

[62]  R. Huber,et al.  Human C1 inhibitor: primary structure, cDNA cloning, and chromosomal localization. , 1986, Biochemistry.

[63]  A. Kaplan,et al.  Distribution of plasma kallikrein between C-1 inactivator and alpha 2-macroglobulin in plasma utilizing a new assay for alpha 2-macroglobulin-kallikrein complexes. , 1985, The Journal of biological chemistry.

[64]  R. Colman,et al.  The regulation of human factor XIIa by plasma proteinase inhibitors. , 1985, The Journal of biological chemistry.

[65]  M. Greaves,et al.  Hereditary Angio-Oedema: Treatment with Cl Esterase Inhibitor Concentrate , 1984, Journal of the Royal Society of Medicine.

[66]  R. Colman,et al.  Inactivation of factor XII active fragment in normal plasma. Predominant role of C-1-inhibitor. , 1984, The Journal of clinical investigation.

[67]  F. Rosen,et al.  Behavior in vivo of normal and dysfunctional C1 inhibitor in normal subjects and patients with hereditary angioneurotic edema. , 1983, The Journal of clinical investigation.

[68]  M. Cicardi,et al.  C1 INH Concentrate in the Therapy of Hereditary Angioedema , 1983, Allergy.

[69]  F van der Graaf,et al.  Inactivation of kallikrein in human plasma. , 1983, The Journal of clinical investigation.

[70]  M. Cicardi,et al.  Hereditary Angioedema: An Appraisal of 104 Cases , 1982, The American journal of the medical sciences.

[71]  R. Colman,et al.  Contribution of plasma protease inhibitors to the inactivation of kallikrein in plasma. , 1982, The Journal of clinical investigation.

[72]  R. Ziccardi Activation of the early components of the classical complement pathway under physiologic conditions. , 1981, Journal of immunology.

[73]  J. Minta The role of sialic acid in the functional activity and the hepatic clearance of C1-INH. , 1981, Journal of immunology.

[74]  M. Cicardi,et al.  C1-inhibitor concentrate for treatment of hereditary angioedema. , 1980, The New England journal of medicine.

[75]  M. Cicardi,et al.  Treatment of acute attacks of hereditary angioedema with C1-inhibitor concentrate. , 1980, Annals of allergy.

[76]  J. Gelfand,et al.  Replacement therapy in hereditary angioedema: successful treatment of acute episodes of angioedema with partly purified C1 inhibitor. , 1980, The New England journal of medicine.

[77]  Robert B Sim,et al.  C1 inhibitor-dependent dissociation of human complement component C1 bound to immune complexes. , 1979, The Biochemical journal.

[78]  M. Cicardi,et al.  [Treatment of hereditary angioedema with a plasmatic concentrate of CU esterase inhibitor]. , 1976, Bollettino della Societa italiana di cardiologia.

[79]  N. Cooper,et al.  Studies on human plasma C1 inactivator-enzyme interactions. I. Mechanisms of interaction with C1s, plasmin, and trypsin. , 1975, The Journal of clinical investigation.

[80]  P. J. Hoek,et al.  Contributions to the Optimal Use of Human Blood , 1974, Vox sanguinis.

[81]  Vogelaar Ef,et al.  Contributions to the Optimal Use of Human Blood , 1974, Vox sanguinis.

[82]  O. Ratnoff,et al.  The inhibitory properties of plasma against activated plasma thromboplastin antecedent (factor XIa) in hereditary angioneurotic edema. , 1972, The Journal of laboratory and clinical medicine.

[83]  H. Gewurz,et al.  Replacement therapy in hereditary angioedema. Successful treatment of two patients with fresh frozen plasma. , 1969, Lancet.

[84]  R. R. Evans,et al.  A BIOCHEMICAL ABNORMALITY IN HEREDIATRY ANGIONEUROTIC EDEMA: ABSENCE OF SERUM INHIBITOR OF C' 1-ESTERASE. , 1963, The American journal of medicine.

[85]  J. Pensky,et al.  Partial purification of a serum inhibitor of C'1-esterase. , 1961, The Journal of biological chemistry.

[86]  I. Lepow,et al.  Assay and Properties of Serum Inhibitor of C'1-Esterase∗ , 1959, Proceedings of the Society for Experimental Biology and Medicine. Society for Experimental Biology and Medicine.

[87]  Margaret,et al.  Distribution of Plasma Kallikrein between C i Inactivator and cy 2-Macroglobulin in Plasma Utilizing a New Assay for a 2-Macroglobulin-Kallikrein Complexes * , 2022 .