Practical Application Corner – Journal Club.

Extended follow-up of pegcetacoplan and new oral factor D inhibitors confirms near-complete hemolysis control with improved quality of life.

Long-term data showing sustained endogenous FVIII expression and reduced annualized bleed rates; establishes gene therapy as durable disease modification.

First telomerase inhibitor to significantly improve transfusion independence in ESA-refractory MDS — now a frontline option before luspatercept failure.

Prospective cohort demonstrating that targeted anti-inflammatory therapy in high-risk CHIP carriers reduces progression to AML/MDS. Bridges genomics, inflammation, and prevention.

Landmark long-term follow-up showing durable transfusion independence and reversal of vaso-occlusive crises with exa-cel and related CRISPR therapies — functional cures achieved.