Hematology
Paroxysmal Nocturnal Hemoglobinuria
Expert Roundtables Podcast: Continued Progress in the Treatment of Paroxysmal Nocturnal Hemoglobinuria
Over the last 20 years, the availability and use of complement inhibition therapy has greatly improved the outlook for patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare and debilitating disease. Decades ago, complement inhibition was a somewhat theoretical, abstract proposition. Today, it is a therapeutic mainstay in PNH, and, as noted by our experts, there is growing excitement about research looking into its potential across many different disease states.
Lee J, Lee H, Kim S, Suh HS. Efficacy of complement inhibitors for patients with paroxysmal nocturnal hemoglobinuria: a systematic review and meta-analysis. Ther Adv Hematol. 2023;14:20406207231216080. doi:10.1177/20406207231216080
Peffault de Latour R, Hosokawa K, Risitano AM. Hemolytic paroxysmal nocturnal hemoglobinuria: 20 years of medical progress. Semin Hematol. 2022;59(1):38-46. doi:10.1053/j.seminhematol.2022.01.001
Peffault de Latour R, Kulasekararaj A, Scheinberg P, et al. Clinical breakthrough hemolysis (BTH) during monotherapy with the oral factor B inhibitor iptacopan is generally not severe and managed without treatment discontinuation: 48-week data from the phase III APPLY-PNH and APPOINT-PNH trials in paroxysmal nocturnal hemoglobinuria (PNH) [abstract 1338]. Abstract presented at: 65th American Society of Hematology Annual Meeting and Exposition; December 9-12, 2023; San Diego, CA.
Risitano AM, de Castro CM, Han B, et al. Patient-reported improvements in fatigue and health-related quality of life in the phase 3 studies APPLY-PNH and APPOINT-PNH evaluating the use of iptacopan in C5 inhibitor-treated and treatment-naïve patients with paroxysmal nocturnal hemoglobinuria [abstract 487]. Abstract presented at: 65th American Society of Hematology Annual Meeting and Exposition; December 9-12, 2023; San Diego, CA.
Risitano AM, Kulasekararaj A, Röth A, et al. Factor B inhibition with oral iptacopan monotherapy demonstrates sustained long-term efficacy and safety in anti-C5-treated patients (pts) with paroxysmal nocturnal hemoglobinuria (PNH) and persistent anemia: final 48-week results from the multicenter, phase III APPLY-PNH trial [abstract 571]. Abstract presented at: 65th American Society of Hematology Annual Meeting and Exposition; December 9-12, 2023; San Diego, CA.
Waheed A, Shammo J, Dingli D. Paroxysmal nocturnal hemoglobinuria: review of the patient experience and treatment landscape. Blood Rev. 2024;64:101158. doi:10.1016/j.blre.2023.101158
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Paroxysmal Nocturnal Hemoglobinuria
