Pediatric hereditary angioedema

Hereditary angioedema (HAE) is a lifelong illness characterized by recurrent swelling of the skin, intestinal tract, and, ominously, the upper airway. It is caused by inadequate activity of the protein C1‐inhibitor, with dysfunction in the kallikrein/bradykinin pathway underlying the clinical symptoms. In addition to the physical symptoms, patients experience significant decrements in vocational and school achievement as well as in overall quality of life. Symptoms often begin in childhood and occur by age 20 in most patients, but life‐threatening attacks are uncommon in the pediatric population. The availability of new therapies has transformed the management of HAE.

[1]  R. Tachdjian,et al.  Use of Ecallantide in Pediatric Hereditary Angioedema , 2013, Pediatrics.

[2]  W. Lumry,et al.  Nanofiltered C1-esterase inhibitor for the acute management and prevention of hereditary angioedema attacks due to C1-inhibitor deficiency in children. , 2013, The Journal of pediatrics.

[3]  R. Ree,et al.  Immunosafety of Recombinant Human C1-Inhibitor in Hereditary Angioedema: Evaluation of IgE Antibodies , 2013, Clinical Drug Investigation.

[4]  H. Farkas,et al.  Treatment of attacks with plasma-derived C1-inhibitor concentrate in pediatric hereditary angioedema patients. , 2013, The Journal of allergy and clinical immunology.

[5]  J. Bernstein,et al.  Efficacy and safety of ecallantide in treatment of recurrent attacks of hereditary angioedema: open-label continuation study. , 2013, Allergy and asthma proceedings.

[6]  D. Vasconcelos,et al.  Hereditary angioedema: first report of the Brazilian registry and challenges , 2013, Journal of the European Academy of Dermatology and Venereology : JEADV.

[7]  T. Machnig,et al.  C1‐INH concentrate for treatment of acute hereditary angioedema: a pediatric cohort from the I.M.P.A.C.T. studies , 2013, Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology.

[8]  H. Farkas,et al.  Prophylactic therapy in children with hereditary angioedema. , 2013, The Journal of allergy and clinical immunology.

[9]  L. Lundquist,et al.  Icatibant for the Treatment of Hereditary Angioedema , 2013, The Annals of pharmacotherapy.

[10]  R. Gower.  HAE update: determining optimal patient specific therapy. , 2013, Allergy and asthma proceedings.

[11]  W. Aberer,et al.  International consensus on hereditary and acquired angioedema. , 2012, Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology.

[12]  H. Farkas,et al.  Short‐term prophylaxis in hereditary angioedema due to deficiency of the C1‐inhibitor – a long‐term survey , 2012, Allergy.

[13]  R. Lockey,et al.  WAO Guideline for the Management of Hereditary Angioedema , 2012, The World Allergy Organization journal.

[14]  T. Machnig,et al.  Per-Attack Reporting of Prodromal Symptoms Concurrent with C1-Inhibitor Treatment of Hereditary Angioedema Attacks , 2012, Advances in Therapy.

[15]  K. Bork,et al.  Fatal laryngeal attacks and mortality in hereditary angioedema due to C1-INH deficiency. , 2012, The Journal of allergy and clinical immunology.

[16]  B. Zuraw,et al.  Safety and efficacy of prophylactic nanofiltered C1-inhibitor in hereditary angioedema. , 2012, The American journal of medicine.

[17]  T. Machnig,et al.  C1-inhibitor therapy for hereditary angioedema attacks: prospective patient assessments of health-related quality of life. , 2012, Allergy and asthma proceedings.

[18]  M. Abuzakouk,et al.  Danazol-Induced Hepatocellular Carcinoma in a Patient with Hereditary Angioedema , 2012, Journal of Gastrointestinal Cancer.

[19]  M. Triggiani,et al.  Efficacy and safety of recombinant human C1‐inhibitor for the treatment of attacks of hereditary angioedema: European open‐label extension study , 2012, Clinical and experimental allergy : journal of the British Society for Allergy and Clinical Immunology.

[20]  D. Meyer‐Olson,et al.  Hereditary angioedema (HAE) in children and adolescents—a consensus on therapeutic strategies , 2012, European Journal of Pediatrics.

[21]  Jay L. Martello,et al.  Ecallantide for treatment of acute attacks of hereditary angioedema. , 2012, American journal of health-system pharmacy : AJHP : official journal of the American Society of Health-System Pharmacists.

[22]  M. Triggiani,et al.  Evidence‐based recommendations for the therapeutic management of angioedema owing to hereditary C1 inhibitor deficiency: consensus report of an International Working Group , 2012, Allergy.

[23]  T. Machnig,et al.  C1 esterase inhibitor concentrate in 1085 Hereditary Angioedema attacks – final results of the I.M.P.A.C.T.2 study , 2011, Allergy.

[24]  C. Town,et al.  Randomized placebo-controlled trial of the bradykinin B₂ receptor antagonist icatibant for the treatment of acute attacks of hereditary angioedema: the FAST-3 trial. , 2011, Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology.

[25]  B. Chipps.  Nanofiltered C1 Inhibitor Concentrate for Treatment of Hereditary Angioedema , 2011, Pediatrics.

[26]  J. Jacobs.  Recurrent attacks of hereditary angioedema: a case of delayed diagnosis. , 2011, Allergy and asthma proceedings.

[27]  C. Fagerberg,et al.  Mutational spectrum and phenotypes in Danish families with hereditary angioedema because of C1 inhibitor deficiency , 2011, Allergy.

[28]  M. López-Trascasa,et al.  A new case of homozygous C1-inhibitor deficiency suggests a role for Arg378 in the control of kinin pathway activation. , 2010, The Journal of allergy and clinical immunology.

[29]  J. Nuijens,et al.  Recombinant human C1-inhibitor for the treatment of acute angioedema attacks in patients with hereditary angioedema. , 2010, The Journal of allergy and clinical immunology.

[30]  B. Zuraw.  The Pathophysiology of Hereditary Angioedema , 2010, The World Allergy Organization journal.

[31]  W. Lumry,et al.  The humanistic burden of hereditary angioedema: Impact on health-related quality of life, productivity, and depression. , 2010, Allergy and asthma proceedings.

[32]  William H. Yang,et al.  Icatibant, a new bradykinin-receptor antagonist, in hereditary angioedema. , 2010, The New England journal of medicine.

[33]  P. Keith,et al.  2010 International consensus algorithm for the diagnosis, therapy and management of hereditary angioedema , 2010, Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology.

[34]  H. Farkas.  Pediatric hereditary angioedema due to C1-inhibitor deficiency. , 2010, Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology.

[35]  W. Lumry,et al.  EDEMA4: a phase 3, double-blind study of subcutaneous ecallantide treatment for acute attacks of hereditary angioedema. , 2010, Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology.

[36]  H. Farkas,et al.  Baseline level of functional C1-inhibitor correlates with disease severity scores in hereditary angioedema. , 2010, Clinical immunology.

[37]  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.

[38]  M. Pedrosa,et al.  Usefulness of abdominal ultrasonography in the follow-up of patients with hereditary C1-inhibitor deficiency. , 2009, Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology.

[39]  S. Büyüköztürk,et al.  A Turkish family with a novel mutation in the promoter region of the C1 inhibitor gene. , 2009, The Journal of allergy and clinical immunology.

[40]  B. Zuraw.  Clinical practice. Hereditary angioedema. , 2008, The New England journal of medicine.

[41]  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.

[42]  A. Sheffer,et al.  Hereditary angioedema: a current state-of-the-art review, V: attenuated androgens for the treatment of hereditary angioedema. , 2008, Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology.

[43]  M. Tarzi,et al.  An evaluation of tests used for the diagnosis and monitoring of C1 inhibitor deficiency: normal serum C4 does not exclude hereditary angio‐oedema , 2007, Clinical and experimental immunology.

[44]  D. Cleri,et al.  Hereditary angioedema presenting in late middle age after angiotensin-converting enzyme inhibitor treatment. , 2007, Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology.

[45]  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.

[46]  M. López-Trascasa,et al.  First case of homozygous C1 inhibitor deficiency. , 2006, The Journal of allergy and clinical immunology.

[47]  J. Hagan,et al.  Acute pancreatitis due to hereditary angioedema. , 2006, Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology.

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

[49]  J. Laurent,et al.  Œdème angioneurotique héréditaire : à propos d'une observation chez un enfant de trois ans , 2005 .

[50]  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.

[51]  B. Zuraw.  Current and future therapy for hereditary angioedema. , 2005, Clinical immunology.

[52]  M. Guillot,et al.  [Hereditary angioneurotic edema: a case report in a 3-year-old child]. , 2005, Archives de pediatrie : organe officiel de la Societe francaise de pediatrie.

[53]  A. Davis.  The pathophysiology of hereditary angioedema. , 2005, Clinical immunology.

[54]  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.

[55]  S. Choquet,et al.  Frequent de novo mutations and exon deletions in the C1inhibitor gene of patients with angioedema. , 2000, The Journal of allergy and clinical immunology.

[56]  H. Bürgi,et al.  Type I hereditary angio‐oedema. Variability of clinical presentation and course within two large kindreds , 1997, Journal of internal medicine.

[57]  T. Mollnes,et al.  C1 Inhibitor and Diagnosis of Hereditary Angioedema in Newborns , 1994, Pediatric Research.

[58]  A. Castaldo,et al.  Hereditary angioedema: danazol therapy in a 5-year-old child. , 1993, American journal of diseases of children.

[59]  A. Davis.  C1 inhibitor and hereditary angioneurotic edema. , 1988, Annual review of immunology.

[60]  K. Wolff,et al.  Hereditary angio‐oedema: treatment with danazol , 1979, The British journal of dermatology.

[61]  D. Alling,et al.  Treatment of hereditary angioedema with danazol. Reversal of clinical and biochemical abnormalities. , 1976, The New England journal of medicine.

[62]  D. Alling,et al.  Treatment of hereditary angioedema with danazol. Reversal of clinical and biochemical abnormalities. , 1976, The New England journal of medicine.

[63]  A. Johnston,et al.  Hereditary angioneurotic oedema. , 1973, Lancet.

[64]  A. Sheffer,et al.  Tranexamic acid therapy in hereditary angioneurotic edema. , 1972, The New England journal of medicine.

[65]  F. Rosen,et al.  Hereditary angioneurotic edema: a clinical survey. , 1966, Pediatrics.

[66]  F. Rosen,et al.  Hereditary Angioneurotic Edema: Two Genetic Variants , 1965, Science.

[67]  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.

[68]  E. Becker,et al.  Hereditary angioneurotic edema: II. Deficiency of inhibitor for serum globulin permeability factor and/or plasma kallikrein , 1962 .

[69]  W. Osler.  HEREDITARY ANGIO‐NEUROTIC ŒDEMA , 1888 .