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2. Misdiagnosis trends in patients with hereditary angioedema from the real-world clinical setting

Author

Aberer, W., Grumach, A., Bygum, A., Blanchard Delaunay, C., Bouillet, L., Coppere, B., Fain, O., Goichot, B., Gompel, A., Guez, S., Jeandel, P., Kanny, G., Launay, D., Maillard, H., Martin, L., Masseau, A., Ollivier, Y., Sobel, A., Arnolds, J., Aygören-Pürsün, E., Baş, M., Bauer, A., Bork, K., Martinez, I., Maurer, M., Papadopoulou-Alataki, E., Psarros, F., Graif, Y., Kivity, S., Reshef, A., Toubi, E., Arcoleo, F., Cicardi, M., Manconi, P., Marone, G., Montinaro, V., Baeza, M.L., Caballero, T., Cabañas, R., Guilarte, M., Hernandez de Rojas, D., Hernando de Larramendi, C., Lleonart, R., Lobera, T., Sáenz de San Pedro, B., Bjorkander, J., Helbert, M., Longhurst, H.J., Zanichelli, Andrea, Longhurst, Hilary J., Maurer, Marcus, Bouillet, Laurence, Aberer, Werner, Fabien, Vincent, Andresen, Irmgard, and Caballero, Teresa

Published
2016
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5. International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency

Author

Farkas, H., Martinez‐Saguer, I., Bork, K., Bowen, T., Craig, T., Frank, M., Germenis, A. E., Grumach, A. S., Luczay, A., Varga, L., Zanichelli, A., Aberer, Werner, Andrejevic, Sladjana, Aygoeren‐Pürsün, Emel, Banerji, Alena, Bara, Noemi‐Anna, Bas, Murat, Bernstein, Jonathan, Betschel, Stephen, Björkander, Janne, Boccon‐Gibod, Isabelle, Bouillet, Laurence, Bova, Maria, Boysen, Henrik Halle, Branco‐Ferreira, Manuel, Bygum, Anette, Caballero, Teresa, Cancian, Mauro, Castaldo, Anthony, Christiansen, Sandra, Cicardi, Marco, Drouet, Christian, Fabiani, Jose, Gompels, Mark, Gonzalez‐Quevedo, Maria Teresa, Gooi, Jimmy, Gower, Richard, Gökmen, Nihal Mete, Grivcheva‐Panovska, Vesna, Guilarte, Mar, Gülbahar, Okan, Hack, Erik, Hakl, Roman, Harmat, György, Jeseňák, Miloš, Jolles, Stephen, Kaplan, Allen, Katelaris, Connie, Kosnik, Mitja, Kőhalmi, Kinga Viktória, Leibovich, Iris, Levi, Marcel, Li, Henry, Longhurst, Hilary J., Lumry, William, Magerl, Markus, Malbran, Alejandro, Martin, Ludovic, Maurer, Marcus, Mihály, Enikő, Moldovan, Dumitru, Murdjeva, Mariana, Nagy, Imola Beatrix, Nielsen, Erik W., Nieto, Sandra, Nordenfelt, Patrik, Obtulowitzc, Kristine, Pedrosa, Maria, Porębski, Grzegorz, Prior, Nieves, Reshef, Avner, Riedl, Marc A., Rosenkranz, Bernd, Schmid‐Grendelmeier, Peter, Péter, Spath, Speletas, Matthaios, Staevska, Maria, Stobiecki, Marcin, Triggiani, Massimo, Veszeli, Nóra, Wuillemin, Walter, Xiang, Zhi Yu, Yamamoto, Beverley, and Zuraw, Bruce

Published
2017
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9. WAO/EAACI.

Author

Maurer, M., Magerl, M., Ansotegui, I., Aygören-Pürsün, E., Betschel, S., Bork, K., Bowen, T., Balle Boysen, H., Farkas, H., Grumach, A. S., Hide, M., Katelaris, C., Lockey, R., Longhurst, H., Lumry, W. R., Martinez-Saguer, I., Moldovan, D., Nast, A., Pawankar, R., and Potter, P.

Published
2023

16. Hereditäres Angioödem durch C1-Inhibitor-Mangel 1 1Bei diesem Beitrag handelt es sich um einen genehmigten Nachdruck des Springer Verlags. Veröffentlicht unter: Bork K et al. Hereditäres Angioödem durch C1-Inhibitor-Mangel. Allergo J, 2012; 21 (2): 109–118.

Author

BORK, K., primary, MAURER, M., additional, BAS, M., additional, HARTMANN, K., additional, BIEDERMANN, T., additional, KREUZ, W., additional, AYGÖREN-PÜRSÜN, E., additional, MARTINEZ-SAGUER, I., additional, OTT, H., additional, and WEDI, B., additional

Published
2015
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22. Prevention of Hereditary Angioedema Attacks with a Subcutaneous C1 Inhibitor

Author

Longhurst, H, Cicardi, M, Craig, T, Bork, K, Grattan, C, Baker, J, Li, HH, Reshef, A, Bonner, J, Bernstein, JA, Anderson, J, Lumry, WR, Farkas, H, Katelaris, CH, Sussman, GL, Jacobs, J, Riedl, M, Manning, ME, Hebert, J, Keith, PK, Kivity, S, Neri, S, Levy, DS, Baeza, ML, Nathan, R, Schwartz, LB, Caballero, T, Yang, W, Crisan, I, Hernandez, MD, Hussain, I, Tarzi, M, Ritchie, B, Kralickova, P, Guilarte, M, Rehman, SM, Banerji, A, Gower, RG, Bensen-Kennedy, D, Edelman, J, Feuersenger, H, Lawo, JP, Machnig, T, Pawaskar, D, Pragst, I, Zuraw, BL, COMPACT Investigators, Institut Català de la Salut, [Longhurst H] Barts Health NHS Trust, London, United Kingdom. [Cicardi M] Ospedale Luigi Sacco–U.O. Medicina Generale, Milan, Italy. [Craig T] Department of Medicine and Pediatrics, Penn State Hershey Allergy, Asthma, and Immunology, Hershey, United States. [Bork K] Johannes Gutenberg University Mainz, Mainz, Germany. [Grattan C] St. John’s Institute of Dermatology, Guy’s Hospital, London, United Kingdom. [Baker J] Baker Allergy, Asthma and Dermatology Research Center, Portland, United States. [Guilarte M] Hospital Universitari Vall d'Hebron, Barcelona, Spain., Vall d'Hebron Barcelona Hospital Campus, and Hospital Universitari Vall d'Hebron

Subjects
Male, aminoácidos, péptidos y proteínas::péptidos::serpinas::proteínas inactivadoras del complemento C1::proteína inhibidora del complemento C1 [COMPUESTOS QUÍMICOS Y DROGAS], 0301 basic medicine, Otros calificadores::Otros calificadores::/prevención & control [Otros calificadores], Enzims proteolítics - Inhibidors, Self Administration, Severity of Illness Index, law.invention, C1-inhibitor, Subcutaneous injection, 0302 clinical medicine, Randomized controlled trial, law, Cross-Over Studies, Hereditary Angioedema Types I and II, biology, Edema - Prevenció, General Medicine, Cardiovascular Diseases::Vascular Diseases::Angioedema::Angioedemas, Hereditary [DISEASES], Anesthesia, Hereditary angioedema, Other subheadings::Other subheadings::/administration & dosage [Other subheadings], Female, Amino Acids, Peptides, and Proteins::Peptides::Serpins::Complement C1 Inactivator Proteins::Complement C1 Inhibitor Protein [CHEMICALS AND DRUGS], medicine.symptom, Complement C1 Inhibitor Protein, enfermedades cardiovasculares::enfermedades vasculares::angioedema::angioedemas hereditarios [ENFERMEDADES], Adult, Risk, medicine.medical_specialty, Injections, Subcutaneous, Aminoácidos, Péptidos y Proteínas::Péptidos::Serpinas::Proteínas Inactivadoras del Complemento 1::Proteína Inhibidora del Complemento C1 [COMPUESTOS QUÍMICOS Y DROGAS], Placebo, Other subheadings::Other subheadings::/prevention & control [Other subheadings], 03 medical and health sciences, Double-Blind Method, Internal medicine, medicine, Humans, Therapeutics::Drug Therapy::Drug Administration Routes::Injections::Injections, Subcutaneous [ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES AND EQUIPMENT], Otros calificadores::Otros calificadores::/administración & dosificación [Otros calificadores], Dose-Response Relationship, Drug, Angioedema, business.industry, Terapéutica::Tratamiento Farmacológico::Vías de Administración de Medicamentos::Inyecciones::Inyecciones Subcutáneas [TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS], Injeccions hipodèrmiques, medicine.disease, Crossover study, Clinical trial, terapéutica::farmacoterapia::vías de administración de medicamentos::inyecciones::inyecciones subcutáneas [TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS], 030104 developmental biology, 030228 respiratory system, biology.protein, business
Abstract

Prevenció; Atac d'angioedema; Inhibidor C1 Prevención; Ataque de angioedema; Inhibidor C1 Prevention; Angioedema attack; C1 inhibitor BACKGROUND: Hereditary angioedema is a disabling, potentially fatal condition caused by deficiency (type I) or dysfunction (type II) of the C1 inhibitor protein. In a phase 2 trial, the use of CSL830, a nanofiltered C1 inhibitor preparation that is suitable for subcutaneous injection, resulted in functional levels of C1 inhibitor activity that would be expected to provide effective prophylaxis of attacks. METHODS: We conducted an international, prospective, multicenter, randomized, double-blind, placebo-controlled, dose-ranging, phase 3 trial to evaluate the efficacy and safety of self-administered subcutaneous CSL830 in patients with type I or type II hereditary angioedema who had had four or more attacks in a consecutive 2-month period within 3 months before screening. We randomly assigned the patients to one of four treatment sequences in a crossover design, each involving two 16-week treatment periods: either 40 IU or 60 IU of CSL830 per kilogram of body weight twice weekly followed by placebo, or vice versa. The primary efficacy end point was the number of attacks of angioedema. Secondary efficacy end points were the proportion of patients who had a response (≥50% reduction in the number of attacks with CSL830 as compared with placebo) and the number of times that rescue medication was used. RESULTS: Of the 90 patients who underwent randomization, 79 completed the trial. Both doses of CSL830, as compared with placebo, reduced the rate of attacks of hereditary angioedema (mean difference with 40 IU, -2.42 attacks per month; 95% confidence interval [CI], -3.38 to -1.46; and mean difference with 60 IU, -3.51 attacks per month; 95% CI, -4.21 to -2.81; P

Published
2017

23. Long-term safety of icatibant treatment of patients with angioedema in real-world clinical practice

Author

Zanichelli, A., Maurer, M., Aberer, W., Caballero, T., Longhurst, H. J., Bouillet, L., Fabien, V., Andresen, I., Grumach, A., Bygum, A., Blanchard Delaunay, C., Boccon-Gibod, I., Coppere, B., Du Thanh, A., Dzviga, C., Fain, O., Goichot, B., Gompel, A., Guez, S., Jeandel, P., Kanny, G., Launay, D., Maillard, H., Martin, L., Masseau, A., Ollivier, Y., Sobel, A., Aygören-Pürsün, E., Bas, M., Bauer, A., Bork, K., Greve, J., Magerl, M., Martinez Saguer, I., Strassen, U., Papadopoulou-Alataki, E., Psarros, F., Graif, Y., Kivity, S., Reshef, A., Toubi, E., Arcoleo, F., Bova, M., Cicardi, M., Manconi, P., Marone, G., Montinaro, V., Triggiani, M., Baeza, L., Cabañas, R., Gala Ortiz, G., Guilarte, M., Hernandez, D., Hernando de Larramendi, C., Lleonart, R., Lobera, T., Marques, L., Saenz de San Pedro, B., Björkander, J., Bethune, C., Garcez, T., Zanichelli, A., Maurer, M., Aberer, W., Caballero, T., Longhurst, H. J., Bouillet, L., Fabien, V., Andresen, I., Grumach, A., Bygum, A., Blanchard Delaunay, C., Boccon-Gibod, I., Coppere, B., Du Thanh, A., Dzviga, C., Fain, O., Goichot, B., Gompel, A., Guez, S., Jeandel, P., Kanny, G., Launay, D., Maillard, H., Martin, L., Masseau, A., Ollivier, Y., Sobel, A., Aygören-Pürsün, E., Bas, M., Bauer, A., Bork, K., Greve, J., Magerl, M., Martinez Saguer, I., Strassen, U., Papadopoulou-Alataki, E., Psarros, F., Graif, Y., Kivity, S., Reshef, A., Toubi, E., Arcoleo, F., Bova, M., Cicardi, M., Manconi, P., Marone, G., Montinaro, V., Triggiani, M., Baeza, L., Cabañas, R., Gala Ortiz, G., Guilarte, M., Hernandez, D., Hernando de Larramendi, C., Lleonart, R., Lobera, T., Marques, L., Saenz de San Pedro, B., Björkander, J., Bethune, C., and Garcez, T.

Subjects
safety, 0301 basic medicine, real-world, medicine.medical_specialty, Immunology, Brief Communication, Off-label use, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Icatibant, Internal medicine, angioedema, icatibant, Immunology and Allergy, Medicine, Reflux esophagitis, Adverse effect, Pregnancy, Angioedema, business.industry, real‐world, medicine.disease, 030104 developmental biology, 030228 respiratory system, chemistry, Anesthesia, Observational study, Long term safety, medicine.symptom, business
Abstract

The Icatibant Outcome Survey (IOS) is an observational study monitoring safety and effectiveness of icatibant in the real‐world setting. We analyzed safety data from 3025 icatibant‐treated attacks in 557 patients (enrolled between July 2009 and February 2015). Icatibant was generally well tolerated. Excluding off‐label use and pregnancy, 438 patients (78.6%) did not report adverse events (AEs). The remaining 119 (21.4%) patients reported 341 AEs, primarily gastrointestinal disorders (19.6%). Of these, 43 AEs in 17 patients (3.1%) were related to icatibant. Serious AEs (SAEs) occurred infrequently. A total of 143 SAEs occurred in 59 (10.6%) patients; only three events (drug inefficacy, gastritis, and reflux esophagitis) in two patients were considered related to icatibant. Notably, no SAEs related to icatibant occurred in patients with cardiovascular disease, nor in those using icatibant at a frequency above label guidelines. Additionally, no major differences were noted in AEs occurring in on‐label vs off‐label icatibant users.

Published
2017

24. Icatibant, a new bradykinin-receptor antagonist, in hereditary angioedema

Author

Cicardi, M., Banerji, A., Bracho, F., Malbran, A., Rosenkranz, B., Riedi, M., Bork, K., Lumry, W., Aberer, W., Bier, H., Bas, M., Greve, J., Hoffmann, T.K., Farkas, H., Reshef, A., Ritchie, B., Yang, W., Grabbe, J., Kivity, S., Kreuz, W., Levy, R.J., Luger, T., Obtulowicz, K., Schmid-Grendelmeier, P., Bull, C., Sitkauskiene, B., Smith, W.B., Toubi, E., Werner, S., Anne, S., Bjorkander, J., Bouillet, L., Cillari, E., Hurewitz, D., Jacobson, K.W., Katelaris. C.H., Maurer, M., Merk, H., Bernstein, J.A., Feighery, C., Floccard, B., Gleich, G., Hebert, J., Kaatz, M.Keith, P., Kirkpatrick, C.H., Langton, D., Martin, L., Pichler, C., Resnick, D., Wombolt, D., Romero, D.S. Fernandez, Zanichelli, A.Arcoleo, F., Knolle, J., Kravec, I., Dong, L., Zimmermann, J., Rosen. K., and Fan, W.-T

Subjects
Tranexamic acid -- Health aspects, Tranexamic acid -- Comparative analysis, Angioneurotic edema -- Genetic aspects, Angioneurotic edema -- Drug therapy, Bradykinin -- Health aspects, Bradykinin -- Comparative analysis
Abstract

A study was conducted to evaluate the efficacy and safety of icatibant, a new bradykinin-receptor antagonist in the treatment of hereditary angioedema. Results indicated that in the case of such patients, icatibant was found to be quite effective and having positive outcomes as compared to placebo or tranexamic acid.

Published
2010

30. Autorenverzeichnis

Author

Domschke, W., primary, Berger, M., additional, Hohenberger, W., additional, Meinertz, T., additional, Vogelmeier, C., additional, Domschke, W., additional, Sauerbruch, T., additional, Kramer, H.J., additional, Müller, S.C., additional, Serve, H., additional, Weber, M.M., additional, Göke, B., additional, Kalden, J.R., additional, Manger, B., additional, Rascher, W., additional, Appenrodt, B., additional, Atta, J., additional, Auernhammer, C., additional, Autenrieth, I.B., additional, Avenhaus, W., additional, Backmund, M., additional, Bausewein, C., additional, Behr, J., additional, Behrens, A., additional, Berner, R., additional, Berr, F., additional, Blank, N., additional, Blind, E., additional, Bockhorn, M., additional, Bokemeyer, D., additional, Böhm, M., additional, Bönner, G., additional, Borasio, G.D., additional, Bork, K., additional, Braun, J., additional, Bruch, H.-P., additional, Brümmendorf, T.H., additional, Brunnberg, U., additional, Brüwer, M., additional, Büchler, M.W., additional, Detter, C., additional, Diederich, S., additional, Diehm, C., additional, Distler, J., additional, Domagk, D., additional, Dörner, T., additional, Dörr, H.-G., additional, Dralle, H., additional, Dreyling, M., additional, Ehlebracht-König, I., additional, Ell, C., additional, Enzensberger, W., additional, Epple, H.-J., additional, Fassnacht, M., additional, Feußner, H., additional, Fiegel, P., additional, Fisang, C., additional, Filipas, D., additional, Fischbach, W., additional, Flamme, C.H., additional, Floege, J., additional, Fölsch, U.R., additional, Fottner, C., additional, Frank, W., additional, Frey, N., additional, Friese, K., additional, Frilling, A., additional, Fröhner, M., additional, Frühmorgen, P., additional, Galle, P.R., additional, Geidel, S., additional, Genth, E., additional, Gingelmaier, A., additional, Goebel, F.-D., additional, Gökbuget, N., additional, Göke, R., additional, Grabitz, K., additional, Grünke, M., additional, Hahner, S., additional, Handrick, W., additional, Hasslacher, C., additional, Heidbreder, E., additional, Heindel, W., additional, Heinemann, V., additional, Heitmann, J., additional, Heiß, H.W., additional, Hof, H., additional, Hering, L., additional, Hiller, E., additional, Hirner, A., additional, Hofmann, W.-K., additional, Holler, E., additional, Hölscher, A.H., additional, Holtmann, G., additional, Hölzen, J., additional, Honegger, J., additional, Hörle, S., additional, Hörmann, K., additional, Hörmann, R., additional, Hornke, I., additional, Huber, R.M., additional, Hübner, J., additional, Hummel, R., additional, Irmscher, S., additional, Jelinek, T., additional, Jonas, S., additional, Jost, E., additional, Jung, H.H., additional, Kahaly, G.J., additional, Karaus, M., additional, Katsoulis, S., additional, Katus, H., additional, Kessler, H.P., additional, Kiehne, K., additional, Kiess, W., additional, Kindermann, M., additional, von Kodolitsch, Y., additional, Köhler, H., additional, Köhler, L., additional, Köhler, M., additional, Kohne, E., additional, Kolb, H.-J., additional, Köninger, J., additional, Koop, K., additional, Köster, R., additional, Kötter, I., additional, Kramer, H.J.J., additional, Kremer, B., additional, Kroll, P., additional, Kuipers, J.G., additional, Lammert, F., additional, Langer, M., additional, Laukötter, M., additional, Lehnert, H., additional, Lembcke, B., additional, Lerch, M.M., additional, Liebe, S., additional, Lieber, A., additional, Loddenkemper, R., additional, Löhr, M., additional, Lorenz, H.-M., additional, Lorenz, J., additional, Löscher, T., additional, Luster, M., additional, Lux, G., additional, Mann, K., additional, Mayerle, J., additional, Merle, U., additional, Meyer, H.-J., additional, Möbius, C., additional, Moehler, M., additional, Mönnikes, H., additional, Mössner, J., additional, Müller, S.A., additional, Musholt, T.J., additional, Nattermann, J., additional, Neubrand, M., additional, Neuhaus, P., additional, Neundörfer, B., additional, Nicolai, T., additional, Nolde, J., additional, Olschewski, H., additional, Ostermeyer, J., additional, Ott, C., additional, Pahernik, S., additional, Pankratius, U., additional, Parhofer, K.G., additional, Passlick, B., additional, Pech, O., additional, Pfaffenbach, B., additional, Pfeiffer, T., additional, Pilatz, A., additional, Pohle, T., additional, Pohl-Koppe, A., additional, Ponto, Katharina A., additional, Prange, H., additional, Pruß, A., additional, Rädle, J., additional, Rauch, B., additional, Raue, F., additional, Reichel, C., additional, Reindl, C., additional, Reißfelder, C., additional, Rendl, Dipl.-Phys. J., additional, Rietschel, E., additional, Rijcken, E., additional, Roos, R., additional, Rudofsky†, G., additional, Samtleben, W., additional, Sandmann, W., additional, Sauter, G., additional, Schaal, K.P., additional, Schaefer, J.R., additional, Schäfer-Graf, U., additional, Schepp, W., additional, Schlemmer, M., additional, Schliep, S., additional, Schmidt, H., additional, Schmied, B., additional, Schmiegel, W., additional, Schießl, A., additional, Schmid, A., additional, Schneider, A., additional, Schneider, T., additional, Schölmerich, J., additional, Scholz, H., additional, Schönermarck, U., additional, Schopohl, J., additional, Schrezenmeier, H., additional, Schulze-Koops, H., additional, Schuppan, D., additional, Schuster, V., additional, Schüßler, G., additional, Schwandner, O., additional, Schwarz, T.F., additional, Secknus, R., additional, Senninger, N., additional, Sezer, O., additional, Simmen, B.R., additional, Spengler, U., additional, Stabenow-Lohbauer, U., additional, Stebler, R., additional, Steven, D., additional, Sticherling, M., additional, Strauch, U., additional, Stremmel, C., additional, Stremmel, W., additional, Stuck, B.A., additional, Stürz, H., additional, Taube, C., additional, Thulesius, O., additional, Thurau, K., additional, Thüroff, J.W., additional, Tomiak, C., additional, Uhl, W., additional, Vallböhmer, D., additional, Vogel, T., additional, von den Driesch, P., additional, Wagenlehner, F.M.E., additional, Wagner, A., additional, Wagner, U., additional, Weber, K., additional, Weidner, W., additional, Weinke, T., additional, Weis-Müller, B.T., additional, Weiß, M., additional, Willems, S., additional, Wintergerst, U., additional, Wirth, M., additional, Wolkersdörfer, G.W., additional, and Zeitz, M., additional

Published
2011
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43. Long-Term Outcomes with Subcutaneous C1-Inhibitor Replacement Therapy for Prevention of Hereditary Angioedema Attacks

Author

Zuraw, B, Katelaris, C, Sussman, G, Keith, PK, Yang, W, Hebert, J, Hanzlikova, J, Staubach-Renz, P, Martinez-Saguer, I, Magerl, M, Aygoren-Pursun, E, Farkas, H, Reshef, A, Kivity, S, Neri, S, Crisan, I, Caballero, T, Baeza, ML, Hernandez, MD, Li, H, Lumry, W, Bernstein, JA, Hussain, I, Anderson, J, Schwartz, LB, Jacobs, J, Manning, M, Levy, D, Riedl, M, Christiansen, S, Zuraw, BL, Cicardi, M, Craig, T, Longhurst, H, Grattan, C, Bork, K, Kreuz, W, Jacobs, I, Pragst, I, Herget, S, Dalton, S, Clement, C, Mycroft, S, Pawaskar, D, Zhang, Y, Machnig, T, Waldhauser, H, Feuersenger, H, Lawo, JP, Lang, D, Hoernlein, S, Mildenberger, M, Foeller, K, Ellis, F, Wood, D, Walsh, M, Qu, QL, Mueller-Stark, K, Feussner, A, Kalina, U, Ma, X, Rigsby, S, Scheffler, E, Fischer, JF, Kolb, C, Katelaris, CH, Frankum, B, Keat, K, Philip, B, Lee, JA, Urriola, N, Lee, MW, Sussman, GL, Levi, G, Gould, W, Ferrie, P, Rosenberg, E, Waserman, S, O'Quinn, J, Gagnon, R, Vachova, M, Stauerbach-Renz, P, Weber, A, Zimmer, S, Gilfert, T, Lang, B, Escuriola-Ettingshausen, C, Maurer, M, Metz, M, Schoepke, N, Altrichter, S, Hawro, T, Schwabe, D, Graff, J, Behrens, F, Kohm, M, Andarawewa, S, Temesszentandrasi, G, Kohalmi, VK, Kidon, M, Kadar, L, Benor, S, Bonanni, E, Wu, M, Zanichelli, A, Mansi, M, Rizzotto, A, Giardino, F, Fidone, F, Varga, M, Iftene, M, Badiu-Tisa, ID, Cabanas, R, Pedrosa, M, Rivero-Paparoni, D, Gomez-Traseira, C, Alvez, A, Phillips, E, Prieto, A, Zubeldia, J, Ibanez, E, Almero, R, Buckland, M, Grigoriadou, S, Manson, A, Yeatman, N, Laffan, J, Nasr, I, Ghurye, R, Rehman, T, Schaeffer, C, Ghaffari, G, Kelbel, T, Reddy, V, Buyantseva, L, Mende, C, Jose, J, Novchicht, T, Li, HH, Scarupa, M, Economides, A, White, M, Kaliner, M, Ward, C, Shaikh, S, Johnson, T, Kosh, L, Dauphin, P, Baker, J, Persons, S, Newman, A, Noonan, MJ, Lumry, WR, Poarch, KP, Tucker, J, Aguilar, D, Noth, D, Bernstein, J, Bernstein, D, Evans, S, Crawford, M, McGuckin, SD, McCollum, JR, Bradley, B, Wagner, C, Cartwright, A, Bonner, J, Soong, W, Sikora, M, Lemke, M, Luthin, P, Youngblood, B, DeBerry, E, Gilbert, E, Zhao, W, Ward, B, Alvarez, A, Kumar, S, Akl, E, Curl, J, Silva, K, Mostofi, T, Schultz, N, Manning, ME, Davis, A, Nelson, J, Levy, DS, Christiansen, SC, and COMPACT Investigators

Subjects
Hereditary angioedema, HAEGARDA, Long-term, Prophylaxis, Subcutaneous, Safety, C1-esterase inhibitor
Abstract

BACKGROUND: For the prevention of attacks of hereditary angioedema (HAE), the efficacy and safety of subcutaneous human C1-esterase inhibitor (C1-INH [SC]; HAEGARDA, CSL Behring) was established in the 16-week Clinical Study for Optimal Management of Preventing Angioedema with Low-Volume Subcutaneous C1-Inhibitor Replacement Therapy (COMPACT). OBJECTIVE: To assess the long-term safety, occurrence of angioedema attacks, and use of rescue medication with C1-INH(SC). METHODS: Open-label, randomized, parallel-arm extension of COMPACT across 11 countries. Patients with frequent angioedema attacks, either study treatment-naive or who had completed COMPACT, were randomly assigned (1:1) to 40 IU/ kg or 60 IU/kg C1-INH(SC) twice per week, with conditional uptitration to optimize prophylaxis (ClinicalTrials.gov registration no. NCT02316353). RESULTS: A total of 126 patients with a monthly attack rate of 4.3 in 3 months before entry in COMPACT were enrolled and treated for a mean of 1.5 years; 44 patients (34.9%) had more than 2 years of exposure. Mean steady-state C1-INH functional activity increased to 66.6% with 60 IU/kg. Incidence of adverse events was low and similar in both dose groups (11.3 and 8.5 events per patient-year for 40 IU/kg and 60 IU/kg, respectively). For 40 IU/kg and 60 IU/kg, median annualized attack rates were 1.3 and 1.0, respectively, and median rescue medication use was 0.2 and 0.0 times per year, respectively. Of 23 patients receiving 60 IU/kg for more than 2 years, 19 (83%) were attack-free during months 25 to 30 of treatment. CONCLUSIONS: In patients with frequent HAE attacks, long-term replacement therapy with C1-INH(SC) is safe and exhibits a substantial and sustained prophylactic effect, with the vast majority of patients becoming free from debilitating disease symptoms. (C) 2019 The Authors. Published by Elsevier Inc. on behalf of the American Academy of Allergy, Asthma & Immunology.

Published
2019

44. Elderly versus younger patients with hereditary angioedema type I/II: patient characteristics and safety analysis from the Icatibant Outcome Survey

Author

Bygum A, Caballero T, Grumach A, Longhurst H, Bouillet L, Aberer W, Zanichelli A, Botha J, Andresen I, Maurer M, Delaunay C, Boccon-Gibod I, Coppere B, Du Thanh A, Dzviga C, Fain O, Goichot B, Gompel A, Guez S, Jeandel P, Kanny G, Launay D, Maillard H, Martin L, Masseau A, Ollivier Y, Sobel A, Aygoren-Pursun E, Bas M, Bauer M, Bork K, Greve J, Magerl M, Martinez-Saguer I, Strassen U, Papadopoulou-Alataki E, Psarros F, Graff Y, Kivity S, Reshef A, Toubi E, Arcoleo F, Bova M, Cicardi M, Nconi P, Marone G, Montinaro V, Triggiani M, Baeza M, Cabanas R, Guilarte M, Hernandez D, de Larramendi C, Lleonart R, Lobera T, Marques L, Pedro B, Bjorkander J, Bethune C, Garcez T, and IOS Study Grp

Subjects
Hereditary angioedema, Elderly, Icatibant Outcome Survey, Safety
Abstract

Background: Hereditary angioedema with C1 inhibitor deficiency (C1-INH-HAE) is characterized by recurrent swelling in subcutaneous or submucosal tissues. Symptoms often begin by age 5-11 years and worsen during puberty, but attacks can occur at any age and recur throughout life. Disease course in elderly patients is rarely reported. Methods: The Icatibant Outcome Survey (IOS) is an observational study evaluating the safety, tolerability, and efficacy of icatibant. We conducted descriptive analyses in younger (age < 65 years) versus elderly patients (age >= 65 years). Here, we report patient characteristics and safety-related findings. Results: As of February 2018, 872 patients with C1-INH-HAE type I/II were enrolled, of whom 100 (11.5%) were >= 65 years old. Significant differences between elderly versus younger patients, respectively, were noted for median age at symptom onset (17.0 vs 12.0 years), age at diagnosis (41.0 vs 19.4 years), and delay between symptom onset and diagnosis (23.9 vs 4.8 years) (P

Published
2019

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