Biblio

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Journal Article
Risitano AM, Röth A, Soret J, Frieri C, de Fontbrune FSicre, Marano L, Alashkar F, Benajiba L, Marotta S, Rozenberg I, et al. Addition of iptacopan, an oral factor B inhibitor, to eculizumab in patients with paroxysmal nocturnal haemoglobinuria and active haemolysis: an open-label, single-arm, phase 2, proof-of-concept trial. Lancet Haematol. 2021.
Kulasekararaj AG, Kuter DJ, Griffin M, Weitz IC, Röth A. Biomarkers and laboratory assessments for monitoring the treatment of patients with paroxysmal nocturnal hemoglobinuria: Differences between terminal and proximal complement inhibition. Blood Rev. 2023:101041.
Röth A, Fryzek J, Jiang X, Reichert H, Patel P, Su J, Arias JMorales, Broome CM. Complement-mediated hemolysis persists year round in patients with cold agglutinin disease. Transfusion. 2021.
Fattizzo B, Röth A, Broome CM, Khan U, Wardęcki M, Cordoba M, Barcellini W. COVID-19 vaccine safety and immunogenicity in patients with cold agglutinin disease receiving concomitant sutimlimab. Am J Hematol. 2024.
Röth A, Ichikawa S, Ito Y, Kim JSeok, Nagy Z, Obara N, Panse J, Schrezenmeier H, Sica S, Soret J, et al. Crovalimab treatment in patients with paroxysmal nocturnal haemoglobinuria: Long-term results from the phase I/II COMPOSER trial. Eur J Haematol. 2023.
Kuter DJ, Piatek C, Röth A, Siddiqui A, Numerof RP, Dummer W. Fostamatinib for warm antibody autoimmune hemolytic anemia: Phase 3, randomized, double-blind, placebo-controlled, global study (FORWARD). Am J Hematol. 2023.
Terriou L, Lee JWook, Forsyth C, Griffin M, Szer J, Röth A, Gustovic P, Metzger J, Patel AS, Patriquin CJ. Long-term effectiveness of eculizumab: Data from the International PNH Registry. Eur J Haematol. 2023.
de Latour RPeffault, Röth A, Kulasekararaj AG, Han B, Scheinberg P, Maciejewski JP, Ueda Y, de Castro CM, Di Bona E, Fu R, et al. Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. 2024;390(11):994-1008.
Wilson K, Rich C, Hakimi Z, Horneff R, Fishman J, Mellor J, Earl L, Taylor Y, Simons A, Conyers J, et al. Pegcetacoplan in paroxysmal nocturnal haemoglobinuria: Its use, its clinical effectiveness, and its influence on health-related quality of life and productivity. Eur J Haematol. 2023.
Hillmen P, Szer J, Weitz I, Röth A, Höchsmann B, Panse J, Usuki K, Griffin M, Kiladjian J-J, de Castro C, et al. Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med. 2021;384(11):1028-1037.
de Latour RPeffault, Szer J, Weitz IC, Röth A, Höchsmann B, Panse J, Usuki K, Griffin M, Kiladjian J-J, de Castro CM, et al. Pegcetacoplan versus eculizumab in patients with paroxysmal nocturnal haemoglobinuria (PEGASUS): 48-week follow-up of a randomised, open-label, phase 3, active-comparator, controlled trial. Lancet Haematol. 2022;9(9):e648-e659.
Höchsmann B, de Latour RPeffault, Hill A, Röth A, Devos T, Patriquin CJ, Chou W-C, Jain D, Zu K, Wu C, et al. Risk factors for thromboembolic events in patients with paroxysmal nocturnal hemoglobinuria (PNH): a nested case-control study in the International PNH Registry. Ann Hematol. 2023.
Patriquin CJ, Bogdanovic A, Griffin M, Kelly RJ, Maciejewski JP, Mulherin B, de Latour RPeffault, Röth A, Selvaratnam V, Szer J, et al. Safety and Efficacy of Pegcetacoplan in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria over 48 Weeks: 307 Open-Label Extension Study. Adv Ther. 2024.
Röth A, Barcellini W, D'Sa S, Miyakawa Y, Broome CM, Michel M, Kuter DJ, Jilma B, Tvedt THenrik And, Weitz IC, et al. Sustained inhibition of complement C1s with sutimlimab over 2 years in patients with cold agglutinin disease. Am J Hematol. 2023.
Röth A, Broome CM, Barcellini W, Jilma B, Hill QA, Cella D, Tvedt THenrik And, Yamaguchi M, Lee M, Shafer F, et al. Sutimlimab provides clinically meaningful improvements in patient-reported outcomes in patients with cold agglutinin disease: Results from the randomised, placebo-controlled, Phase 3 CADENZA study. Eur J Haematol. 2022.