RT Journal Article
T1 Oral Plasma Kallikrein Inhibitor for Prophylaxis in Hereditary Angioedema.
A1 Aygören-Pürsün, Emel
A1 Bygum, Anette
A1 Grivcheva-Panovska, Vesna
A1 Magerl, Markus
A1 Graff, Jochen
A1 Steiner, Urs C
A1 Fain, Olivier
A1 Huissoon, Aarnoud
A1 Kinaciyan, Tamar
A1 Farkas, Henriette
A1 Lleonart, Ramon
A1 Longhurst, Hilary J
A1 Rae, William
A1 Triggiani, Massimo
A1 Aberer, Werner
A1 Cancian, Mauro
A1 Zanichelli, Andrea
A1 Smith, William B
A1 Baeza, Maria L
A1 Du-Thanh, Aurelie
A1 Gompels, Mark
A1 Gonzalez-Quevedo, Teresa
A1 Greve, Jens
A1 Guilarte, Mar
A1 Katelaris, Constance
A1 Dobo, Sylvia
A1 Cornpropst, Melanie
A1 Clemons, Desiree
A1 Fang, Lei
A1 Collis, Phil
A1 Sheridan, William
A1 Maurer, Marcus
A1 Cicardi, Marco
AB Hereditary angioedema is a life-threatening illness caused by mutations in the gene encoding C1 inhibitor (also called C1 esterase inhibitor) that lead to overactivation of the kallikrein-bradykinin cascade. BCX7353 is a potent oral small-molecule inhibitor of plasma kallikrein with a pharmacokinetic and pharmacodynamic profile that may help prevent angioedema attacks. In this international, three-part, dose-ranging, placebo-controlled trial, we evaluated four doses of BCX7353 (62.5 mg, 125 mg, 250 mg, and 350 mg once daily) for the prevention of angioedema attacks over a 28-day period. Patients with type I or II hereditary angioedema with a history of at least two angioedema attacks per month were randomly assigned to BCX7353 or placebo. The primary efficacy end point was the number of confirmed angioedema attacks. Key secondary end points included angioedema attacks according to anatomical location and quality of life. A total of 77 patients underwent randomization, 75 received BCX7353 or placebo, and 72 completed the trial. The rate of confirmed angioedema attacks was significantly lower among patients who received BCX7353 at daily doses of 125 mg or more than among those who received placebo, with a 73.8% difference at 125 mg (P Once-daily oral administration of BCX7353 at a dose of 125 mg or more resulted in a significantly lower rate of attacks of hereditary angioedema than placebo. Mild gastrointestinal symptoms were the principal side effect. (Funded by BioCryst Pharmaceuticals; APeX-1 ClinicalTrials.gov number, NCT02870972 .).
YR 2018
FD 2018
LK http://hdl.handle.net/10668/12755
UL http://hdl.handle.net/10668/12755
LA en
DS RISalud
RD Apr 17, 2025