Anti-Inhibitor Coagulant Complex Prophylaxis in Hemophilia with Inhibitors

Women leaders in hematology: Inspirations & insights

“…In Dr. DiMichele's own words, "there is too much left to do." [43,44], acquired hemophilia A [45], and other coagulation disorders, such as von Willebrand disease. She has been an active investigator for numerous research studies and Principle Investigator for 2 investigator-initiated, multiinstitutional clinical trials of treatments for individuals with hemophilia A and factor VIII inhibitors: the Rituximab for Inhibitors in Congenital Hemophilia (RICH) study [46] and the ProFEIBA study of anti-inhibitor coagulant complex prophylaxis [44].…”

“…Among a lifelong career that includes numerous clinical research studies dedicated to improvements in this area, Cindy's most widely known accomplishment is arguably the implementation of prophylaxis in hemophilia A patients with inhibitors. This practice that was not routinely employed prior to her investigator-initiated, international, multicenter clinical trial that showed that prophylactic use of activated prothrombin complex concentrates in hemophilia patients with inhibitors significantly reduces bleeding [44]. Results of her study have changed the lives of patients with inhibitors, allowing them to emerge from the shadows to lead more normal and productive lives, similar to their non-inhibitor counterparts who have been on prophylaxis for years.…”

Postępy w leczeniu chorych na hemofilie

“…Profilaktyka krwawień u chorych na hemofilię powikłaną inhibitorem wydaje się jedyną atrakcyjną opcją leczenia w przypadku ciężkiego przebiegu skazy i niepowodzenia bądź przeciwwskazań do zastosowania leczenia eradykującego inhibitor (immunotolerancji). W ostatnich latach dostarczono ważnych dowodów naukowych wskazujących na bezpieczeństwo i skuteczność długoterminowej profilaktyki krwawień z zastosowaniem koncentratu aktywowanych czynników zespołu protrombiny (aPCC, activated prothrombin complex concentrate) u pacjentów z hemofilią powikłaną inhibitorem [8].…”

“…Badanie Pro-FEIBA jest pierwszym prospektywnym, randomizowanym badaniem typu cross--over służącym ocenie skuteczności aPCC (FEIBA ® ) w długoterminowej profilaktyce krwawień [8]. Czas obserwacji w badaniu wynosił 15 miesięcy.…”

Women leaders in hematology: Inspirations & insights

“…Cindy Leissinger: Well known for her pioneering research involving prophylaxis in patients with hemophilia A who have inhibitors [19], Dr. Leissinger has a diversity of interests and expertise in coagulation disorders. She has conducted extensive research in hematology, especially in the area of coagulation, and has made significant contributions to the field.…”