OBJECTIVES: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder characterized by complement mediated hemolysis. Patients often experience anemia, fatigue and require blood transfusions. With several treatments approved (including complement 5 inhibitors [C5i]: infusions and iptacopan: oral monotherapy, factor-B inhibitor), health state utilities are important parameters in economic models that aid in decision making in health technology assessments. This analysis aimed to derive health state utility values for a cost-effectiveness model of iptacopan in PNH.

METHODS: EQ-5D-5L data were collected from pivotal trials of iptacopan in adults with PNH, APPOINT-PNH and APPLY-PNH. Responses were mapped to EQ-5D-3L, then to a utility index based on UK tariffs. A mixed linear model for repeated measures was fit to derive utility values for three health states 1) no transfusion and no anemia, 2) no transfusion and anemia and 3) transfusion. Data from both trials were pooled for model fitting to enhance sample size and precision of model coefficients. Covariates included in the final model were health state, treatment, baseline utility, follow-up visit, and study (APPLY-PNH vs APPOINT-PNH). Pooled and treatment-specific health state utilities are presented.

RESULTS: The pooled and treatment-specific (mean, standard error [SE]) utilities for health states are: no transfusion and no anemia (pooled: 0.878 [0.004]; iptacopan: 0.879 [0.004]; C5i: 0.775 [0.056]), no transfusion and anemia (pooled: 0.785 [0.009]; iptacopan: 0.822 [0.008]; C5i: 0.743 [0.015]), and transfusion (pooled: 0.733 [0.015]; iptacopan: 0.791 [0.015]; C5i: 0.695 [0.021]).

CONCLUSIONS: This analysis showed that patients achieving no transfusion and no anemia had the highest utility values compared to other health states. Treatment-specific utilities indicated higher values for iptacopan than for C5 inhibitors, for all health states. Treatments which allow hemoglobin normalization and transfusion avoidance for a majority of patients will contribute to improvement of patient outcomes.