Model schematics. The base-case Markov model (A) consists of 3 health states, including transfusion required, transfusion avoidant, and death. Patients with residual anemia despite complement inhibition enter a transfusion-required state or transfusion-avoidant state in the very first cycle (ie, by the end of 1 month), reflecting their response (or lack thereof) as in the clinical trial. Efficacy (lack of transition from transfusion avoidant to transfusion required and vice versa) is informed by trial results. Patients could remain in these states throughout their lifetime or move to death at any point, due to either transfusion-associated mortality or background mortality. A scenario Markov model (B) consists of 4 health states, including transfusion required, transfusion avoidant (with and without anemia), and death: patients transition across 3 health states as informed by a transition probability matrix derived from adjusted and aggregated individual-level patient data from APPLY-PNH.