Long-Term Use of Pegcetacoplan in Patients with PNH
Christopher Patriquin, MD, Hematologist at Toronto General Hospital, discusses data from the long-term extension study of pegcetacoplan in adult patients with paroxysmal nocturnal hemoglobinuria (PNH). These data were recently presented at the American Society of Hematology Meeting & Exposition (ASH 2022).
PNH is a rare, life-threatening blood disorder characterized by hemolysis. Persistently low hemoglobin levels can result in severe fatigue and difficulty breathing. Additionally, individuals with PNH are susceptible to thrombotic events and many have some degree of bone marrow dysfunction. In May 2021, the U.S. Food and Drug Administration (FDA) approved pegcetacoplan to treat PNH.
As Dr. Patriquin explains, this open-label extension study enrolled adults with PNH who completed one of the parent clinical studies for pegcetacoplan. Patients were either continued on their current pegcetacoplan dose (1080 mg subcutaneous twice weekly or every 3 days [PEGASUS and PRINCE studies]) or switched patients to 1080 mg subcutaneous pegcetacoplan treatment (PHAROAH, PADDOCK, PALOMINO studies). Efficacy was evaluated with mean hematologic values (hemoglobin [Hb], lactate dehydrogenase [LDH]), Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score, and transfusion avoidance (i.e., patients [%] who did not require transfusion during the 48 weeks). At ASH 2022, 48-week data from this study was presented.
A total of 137 of 145 patients who completed a previous pegcetacoplan trial chose to enter the extension study; 107 of these patients had received 48 weeks of treatment in the extension study at the time of data cutoff. At baseline, mean Hb was 11.6 g/dL. At week 48, Hb was also 11.6 g/dL, and 35.1% of patients had normal Hb per sex-specified norms. Baseline mean LDH was 284.2 U/L. At week 48, mean LDH was 274.2 U/L, and 73.2% of patients had normalized LDH levels. Baseline mean FACIT-fatigue was 42.8. At week 48, mean FACIT-fatigue was 42.4. Transfusion avoidance was achieved in 83.2% of patients through Week 48. Overall, these results suggest that pegcetacoplan treatment sustains robust improvements in Hb, LDH, and fatigue in patients with PNH, and reduces the need for transfusions.
The most common treatment-emergent AE was hemolysis, reported in 16.8% of patients. All serious hemolytic events were resolved.
To learn more about PNH and other rare blood disorders, visit checkrare.com/diseases/hematologic-disorders/.
