Patient preferences for the treatment of paroxysmal nocturnal hemoglobinuria: Interim results of a patient survey of ravulizumab (ALXN1210) and eculizumab
Peipert J, Kulasekararaj A, Gonzalez-Fernandez F, Yount S, Martens C, Sparling A, Webster K, Cella D, Rottinghaus S, Donato B, Tomazos I, Gaya Valls A, Mitchell L, Hill A, Wells R, Piatek C, Kuriakose P, Murakhovskaya I, Khaled S, and Kaiser K. Patient preferences for the treatment of paroxysmal nocturnal hemoglobinuria: Interim results of a patient survey of ravulizumab (ALXN1210) and eculizumab. J Manag Care Spec Pharm 2019; 25:S37.
J Manag Care Spec Pharm
BACKGROUND: Eculizumab inhibits complement-mediated hemolysis and thrombosis and improves quality of life (QoL) in patients with paroxysmal nocturnal hemoglobinuria (PNH). However, eculizumab has a treatment burden associated with every 2-week dosing. Ravulizumab, a new C5 inhibitor for PNH administered every 8 weeks, was shown to be non-inferior to eculizumab in 2 phase 3 trials. In the presence of multiple treatment options, patient preference should be considered when selecting a treatment plan. OBJECTIVE: To evaluate patient treatment preference for eculizumab or ravulizumab in clinical sub-study ALXN1210-PNH-302s. METHODS: ALXN1210-PNH-302 sub-study aims to enroll at least 95 PNH patients. Patients were enrolled from the ALXN1210-PNH-302 extension study, had received at least 2 doses of ravulizumab and provided informed consent. All patients were on stable eculizumab therapy prior to entering the trial. Patient treatment preference was evaluated at one time point by using an 11-item PNH specific patient preference questionnaire (PNH-PPQ). RESULTS: To date, 52 completed PNH-PPQs have been analyzed. Mean age was 50 years (range: 28-78) and sex was equally distributed (~50%). Mean time since diagnosis was 15 years (range: 2-48) and the mean number of days between the last randomized study treatment and the survey was 278. Overall, 94% of patients (n = 49) preferred ravulizumab vs. 6% (n = 3; P < 0.001) who preferred eculizumab (n = 1) or had no preference (n = 2). When asked for drug preference, ravulizumab was preferred based on frequency of infusions (98%), ability to plan activities (98%), convenience of receiving treatment (92%), overall QoL (88%), and effectiveness of medication until the next infusion (79%). When asked for the most important characteristic for treatment preference, the most common choice was frequency of infusions (42%). Moderate to large effect sizes were observed for factors differentiating ravulizumab and eculizumab, including the frequency of infusions disrupting everyday life (-1.43, P < 0.001), feeling fatigued after infusions (-0.61, P < 0.001), and being able to enjoy life while receiving treatment (0.96, P < 0.001). CONCLUSIONS: This interim analysis provides insights into PNH patient treatment preferences, and indicates that the vast majority of patients surveyed preferred ravulizumab due to reduced infusion frequency, ability to plan activities, effectiveness of medication, and convenience of treatment compared to eculizumab.