Phase II prospective open-label trial of recombinant interleukin-11 in desmopressin-unresponsive von Willebrand disease and mild or moderate haemophilia A

• Published in: Thrombosis and haemostasis Vol. 109; no. 2; p. 248
• Main Authors: Ragni, Margaret V, Novelli, Enrico M, Murshed, Anila, Merricks, Elizabeth P, Kloos, Mark T, Nichols, Timothy C
• Format: Journal Article
• Language: English
• Published: Germany 01.02.2013
• Subjects: Adult; Biomarkers - blood; Deamino Arginine Vasopressin - adverse effects; Deamino Arginine Vasopressin - therapeutic use; Drug Resistance; Factor VIII - metabolism; Female; Hemophilia A - blood; Hemophilia A - diagnosis; Hemophilia A - drug therapy; Hemostatics - adverse effects; Hemostatics - therapeutic use; Humans; Interleukin-11 - adverse effects; Interleukin-11 - therapeutic use; Male; Middle Aged; Pennsylvania; Prospective Studies; Protein Multimerization; Recombinant Proteins - adverse effects; Recombinant Proteins - therapeutic use; Reverse Transcriptase Polymerase Chain Reaction; RNA, Messenger - blood; Severity of Illness Index; Time Factors; Treatment Outcome; von Willebrand Diseases - blood; von Willebrand Diseases - diagnosis; von Willebrand Diseases - drug therapy; von Willebrand Diseases - genetics; von Willebrand Factor - genetics; von Willebrand Factor - metabolism; Young Adult

Desmopressin (DDAVP) is the treatment of choice in those with mild von Willebrand disease (VWD), yet 20% are unresponsive to DDAVP, and among the 80% who respond, the response is transient, as endothelial stores are depleted after three days. We, therefore, conducted a single-center Phase II clinica...