OBJECTIVES: Crovalimab is a novel anti-C5 antibody for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). In the randomized, Phase III COMMODORE 2 (C5 inhibitor-naive) study, crovalimab showed non-inferior efficacy outcomes vs eculizumab. These results were supported by results from the randomized, Phase III COMMODORE 1 (C5 inhibitor-pretreated) study. This research aims to investigate the health status of patients as measured by EQ-5D-5L translated into utility scores for the UK, US and Japan.

METHODS: EQ-5D-5L results from pooled randomized COMMODORE 1 and 2 study arms were mapped to recommended value sets to derive utility scores. These were estimated using a linear mixed-effects model controlling for study treatment (crovalimab coded as default), the presence of a breakthrough hemolysis (BTH) event and baseline utility.

RESULTS: The results of the utility score model for the intercept (95% CI) were 0.86 (0.85, 0.88), 0.89 (0.87, 0.91), and 0.85 (0.84, 0.87) using the UK, US, and Japanese tariffs respectively. Likewise, the utility estimates for study treatment with eculizumab were -0.03 (-0.05, -0.0004), -0.03 (-0.05, -0.003), and -0.03 (-0.05, -0.01), respectively and were statistically significantly higher for patients receiving crovalimab. The respective estimates for the impact of a BTH event were -0.06 (-0.11, 0.02), -0.07 (-0.13, 0.003), and -0.033 (-0.085, 0.034). The variance for BTH was large, likely due to the rare nature of BTH events. The model indicated a statistically relevant impact of baseline utility.

CONCLUSIONS: Results from the utility analysis showed that patients with PNH treated with C5 inhibitors can achieve a quality of life similar to healthy individuals. Patient utility was higher for patients receiving crovalimab vs eculizumab regardless of the value set being used. These findings are consistent with results from COMMODORE 1 and 2 in terms of patient preference and change in FACIT-Fatigue score from baseline, which both favored the crovalimab arm.