For how we live.
In the phase 3 VERIFY trial, 77% of patients on the hepcidin-mimic drug avoided phlebotomy over weeks 20–32 versus 33% on placebo, and far more kept their haematocrit under control.
The academic MATRix/IELSG43 trial found autologous transplant lifted 3-year progression-free survival to 78% from 51%, but at the cost of more toxicity and more treatment-related deaths.
In the investigator-led Danish DALIAH trial, pegylated interferon-alfa and hydroxyurea produced similar molecular responses by intention-to-treat, but far more patients stopped interferon.
Gilead's phase 3 trial in 539 patients was terminated after the CD47 antibody failed to improve remission or survival and caused more fatal adverse events than azacitidine alone.
In a 187-patient trial, chronic graft-versus-host-disease-free survival at one year was 78% with the new therapy versus 38% with conventional transplant. The FDA approved it 30 June.