Director, HEOR Apellis Pharmaceuticals, Inc., Waltham, MA, USA, United States
Background: Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare (1-1.5 cases per million), acquired, life-threatening disease characterized by complement-mediated hemolysis and thrombosis. PNH symptoms can lead to transfusion dependance, along with hospital and emergency room (ER) visits, increasing healthcare resource utilization (HCRU). Although clinical trials have assessed the efficacy of pegcetacoplan (PEG), there is limited information on the use of PEG in a real-world setting.
Objectives: We report preliminary trends in HCRU, in the form of transfusions, all-cause ER visits and hospitalizations, as well as hemoglobin (Hb) for patients in OPERA, a descriptive, observational, exploratory outcomes study, presenting real-world data on PEG treatment for US adults with PNH.
Methods: This study recruited (via pharmacy) US patients ≥18 years of age, diagnosed with PNH and prescribed PEG by a licensed medical professional. Patients consented then enrolled to participate in the study as approved by an institutional review board. OPERA collected information from routine care and did not direct any medical interventions. Patients provided baseline (BL) HCRU through online questionnaires while BL Hb was reported by the site (healthcare provider verified). Patients reported Hb and HCRU over 1 year follow-up during monthly calls (subject to data availability). Hb analysis only included patients who did not report a transfusion in the treatment period and reported both a BL and ≥1 follow-up Hb value. Given disease rarity, a small sample size was expected.
Results: Of 43 patients with PNH enrolled in OPERA who completed at least 1 monthly call, mean age was 43.8 years and 53.5% were female. At baseline, 23.3% of patients were previously treated with eculizumab, 51.2% with ravulizumab and 18.6 % with both. At baseline, 55.8% of patients reported receiving transfusions prior to enrollment, with 48.8% reporting spending ≥3 hours at centers for transfusion care. Over the first 12 months of the study, patients reporting transfusions reduced to 16.3%; the incidence rate per person year for OPERA patients was 0.8 (95% CI 0.6–1.0) for transfusions, 0.3 (95% CI 0.2–0.6) for ER visits and 0.2 (95% CI 0.1–0.5) for hospitalizations, after initiating PEG treatment. For 38 patients with available data, dosed at 1080 mg of PEG twice weekly or every 3 days, over 161.0 person months, mean (range) PEG treatment compliance was 98% (87–100%) since OPERA enrollment. Of the 31 patients who met Hb analysis criteria, mean (SD) BL Hb was 8.7 (2.0) g/dL and latest Hb was 11.5 (1.8) g/dL, with a median (IQR) follow-up period of 6.0 (3.0) months.
Conclusions: This real-world study of US adults with PNH receiving PEG indicates low HCRU with a positive trend in Hb, after treatment.
This abstract has been accepted and presented at the EHA 2023 Annual Congress.
