Gallia G. Levy

TL;DR: Empicizumab prophylaxis was associated with a significantly lower rate of bleeding events than no proPHylaxis among participants with hemophilia A with inhibitors and resulted in a bleeding rate that was significantly lower by 79% than the rate with previous bypassing‐agent prophylum.

TL;DR: Empicizumab prophylaxis administered subcutaneously once weekly or every 2 weeks led to a significantly lower bleeding rate than no proPHylaxis among persons with hemophilia A without inhibitors; more than half of the participants who received prophYLaxis had no treated bleeding events.

TL;DR: Pictilisib was safely administered with a dose-proportional pharmacokinetic profile, on-target pharmacodynamic activity at dose levels ≥100 mg and signs of antitumor activity.

TL;DR: Emicizumab given once every 4 weeks showed clinically meaningful bleed control while being well tolerated, and could improve patient care by decreasing treatment burden and increasing adherence to effective prophylaxis, potentially decreasing the development of secondary complications for people with haemophilia A.

TL;DR: No difference was found in median progression-free survival between the pictilisib and placebo groups, and there was no difference when patients were analysed according to presence or absence.