Efficacy of emicizumab in a pediatric patient with type 3 von Willebrand disease and alloantibodies
Angela C Weyand, Veronica H Flood, Jordan A Shavit, Steven W Pipe, Angela C Weyand, Veronica H Flood, Jordan A Shavit, Steven W Pipe
Abstract
Type 3 von Willebrand disease with alloantibodies is a rare clinical entity with few treatment options.
Emicizumab prophylaxis in such patients may result in improved hemarthrosis control, lower cost, and enhanced quality of life.
Conflict of interest statement
Conflict-of-interest disclosure: A.C.W. has received consulting fees from Bayer, Shire, Aptevo, and Kedrion Biopharma and research funding from Shire and NovoNordisk. J.A.S. has received consulting fees from Shire, Bayer, CSL Behring, Sanofi, Spark Therapeutics, and NovoNordisk. S.W.P. has served as a consultant to Apcintex, Bayer, Biomarin, Bioverativ, Catalyst Biosciences, CSL-Behring, HEMA Biologics, Freeline, NovoNordisk, Pfizer, Roche/Genentech, Sanofi, Shire, Spark Therapeutics, and uniQure. V.H.F. declares no competing financial interests.
Source: PubMed