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Journal Articles

Serum ferritin changes in iptacopan-treated patients with paroxysmal nocturnal hemoglobinuria

Free
Antonio Risitano, Carlos de Castro, Camilla Frieri, Bing Han, Richard Kelly, Austin Kulasekararaj, Jaroslaw Maciejewski, Vinod Pullarkat, Alexander Röth, Phillip Scheinberg, Flore Sicre-de-Fontbrune, Yasutaka Ueda, Tim Costigan, Marion Dahlke, Philippe Ferber, Tomasz Lawniczek, Stefanie Senger, Regis Peffault De Latour
Journal: Blood
Blood (2025) 146 (Supplement 1): 4974.
https://doi.org/10.1182/blood-2025-4974
Published: 2025
Journal Articles

Factor bb as a biomarker during breakthrough hemolysis in paroxysmal nocturnal hemoglobinuria

Free
Matthew Holt, Daniel Payne, Natalie Fox, Claire McKinley, Morag Griffin, Petra Muus, Talha Munir, Alexandra Pike, Louise Arnold, Sateesh Nagumantry, Darren Newton, Richard Kelly
Journal: Blood
Blood (2025) 146 (Supplement 1): 4973.
https://doi.org/10.1182/blood-2025-4973
Published: 2025
Journal Articles

Real-world use of oral iptacopan monotherapy in paroxysmal nocturnal hemoglobinuria

Free
Matthew Holt, Richard Kelly, Regis Peffault De Latour, Morag Griffin, Shreyans Gandhi, Edouard Forcade, Francesco Versino, Roochi Trikha, Alexandra Pike, Joanna Large, Michael Loschi, Marc Michel, Petra Muus, Talha Munir, Alice Garnier, Simona Pagliuca, Yosr Hicheri, Isabelle Brindel, Sateesh Nagumantry, Lynda Maafa, Louise Arnold, Austin Kulasekararaj, Flore Sicre-de-Fontbrune
Journal: Blood
Blood (2025) 146 (Supplement 1): 1427.
https://doi.org/10.1182/blood-2025-1427
Published: 2025
Journal Articles

Switching between complement inhibitors in patients with PNH: A real-world analysis of strategy, efficacy, and safety.

Free
Morag Griffin, Bruno Fattizzo, Jong Wook Lee, Richard Kelly, Roochi Trikha, Yasutaka Ueda, Jun-ichi Nishimura, Christopher Patriquin, Alexander Röth, Petra Muus, Jens Panse, Miguel Gómez Álvarez, Alexandra Pike, Talha Munir, Shreyans Gandhi, Austin Kulasekararaj
Journal: Blood
Blood (2025) 146 (Supplement 1): 1114.
https://doi.org/10.1182/blood-2025-1114
Published: 2025
Journal Articles

The use of anticoagulation on paroxysmal nocturnal hemoglobinuria.

Free
Louise Arnold, Petra Muus, Talha Munir, Alexandra Pike, Sateesh Nagumantry, Hussnain Javaid, Matthew Holt, Elizabeth Littlewood, Amy Todd, Emily Groom-White, Morag Griffin, Richard Kelly
Journal: Blood
Blood (2025) 146 (Supplement 1): 1111.
https://doi.org/10.1182/blood-2025-1111
Published: 2025
Journal Articles

The effect of breakthrough hemolysis on complement factor h and factor I levels in paroxysmal nocturnal hemoglobinuria

Free
Matthew Holt, Daniel Payne, Natalie Fox, Claire McKinley, Morag Griffin, Petra Muus, Talha Munir, Alexandra Pike, Louise Arnold, Sateesh Nagumantry, Richard Kelly, Darren Newton
Journal: Blood
Blood (2025) 146 (Supplement 1): 3193.
https://doi.org/10.1182/blood-2025-3193
Published: 2025
Journal Articles

Spontaneous remissions in paroxysmal nocturnal hemoglobinuria: True remission or clonal evolution to malignancy?

Free
Caitlin Roger, Matthew Holt, Louise Arnold, Lindsay Mitchell, Sateesh Nagumantry, Katharine Lowndes, Alexandra Pike, Talha Munir, Petra Muus, Morag Griffin, Richard Kelly
Journal: Blood
Blood (2025) 146 (Supplement 1): 29.
https://doi.org/10.1182/blood-2025-29
Published: 2025
Journal Articles

Clonal architecture and dynamics of somatic evolution in aplastic anemia and paroxysmal nocturnal hemoglobinuria

Free
Michael Spencer Chapman, Kudzai Nyamondo, Nicholas Williams, Daniel Leongamornlert, Joanna Baxter, Behnaz Shojaeddin Givi, Matthew Holt, Theodora Foukaneli, Duncan Brian, Chi Wong, Richard Kelly, Darren Newton, Alan Warren, Jyoti Nangalia
Journal: Blood
Blood (2025) 146 (Supplement 1): 25.
https://doi.org/10.1182/blood-2025-25
Published: 2025
Journal Articles

Outcomes of mecom rearranged AML undergoing intensive chemotherapy in the NCRI AML17 and AML19 trials.

Free
Mike Dennis, Jad Othman, Joanna Canham, Ian Thomas, Sean Johnson, Nick Telford, Beth Harrison, Jeyaprakash Ramachandran, James Cavenagh, Claire Arnold, Claus Marcher, Richard Kelly, Mhairi Copland, Alan Burnett, Richard Dillon, Nigel Russell
Journal: Blood
Blood (2025) 146 (Supplement 1): 3435.
https://doi.org/10.1182/blood-2025-3435
Published: 2025
Journal Articles

Paroxysmal nocturnal hemoglobinuria in pediatric patients: A UK experience

Free
Morag Griffin, Malinthi Adikarama, Shreyans Gandhi, Petra Muus, Alexandra Pike, Talha Munir, Matthew Holt, Louise Arnold, Franceso Versino, Joanna Large, Richard Kelly, Austin Kulasekararaj, Roochi Trikha
Journal: Blood
Blood (2025) 146 (Supplement 1): 4662.
https://doi.org/10.1182/blood-2025-4662
Published: 2025
Journal Articles

Oral iptacopan monotherapy demonstrates clinically meaningful hemoglobin increases in patients with paroxysmal nocturnal hemoglobinuria with baseline hemoglobin levels 10 to <12 g/dl on anti-C5 therapy: Subgroup analysis of the appulse-pnh Phase 3b trial

Free
Austin Kulasekararaj, Flore Sicre-de-Fontbrune, Anna Gaya, Ilene Weitz, David Kuter, David Araten, Bhumika Patel, Abhay Singh, Richard Kelly, Eros Di Bona, Michael Loschi, Vinod Pullarkat, Jörg Schubert, Alessandro Maria Vannucchi, Mustafa Yenerel, Regis Peffault De Latour, Navin Mahajan, Luca Monaco, Tao Ding, Tomasz Lawniczek, Philippe Ferber, Marion Dahlke, Antonio Risitano
Journal: Blood
Blood (2025) 146 (Supplement 1): 4981.
https://doi.org/10.1182/blood-2025-4981
Published: 2025
Journal Articles

Apply-PNH: Analysis of complement pathway biomarkers provides evidence for pharmacodynamic response in PNH patients who receive oral iptacopan monotherapy versus continuing anti-C5 therapy

Free
Regis Peffault De Latour, Carlos de Castro, Richard Kelly, Austin Kulasekararaj, Alexander Röth, Phillip Scheinberg, Yasutaka Ueda, Douglas Applegate, Marion Dahlke, Philippe Ferber, Imelda Schuhmann, Antonio Risitano
Journal: Blood
Blood (2025) 146 (Supplement 1): 4979.
https://doi.org/10.1182/blood-2025-4979
Published: 2025
Journal Articles

Real-world analysis of ravulizumab safety and effectiveness in advanced age patients with paroxysmal nocturnal hemoglobinuria: Insights from the international PNH registry

Free
Shreyans Gandhi, Ami Patel, Alix Augustin-Wright, Hubert Schrezenmeier, Philippe Gustovic, Jesse Metzger, Yasutaka Ueda, Richard Kelly
Journal: Blood
Blood (2025) 146 (Supplement 1): 6238.
https://doi.org/10.1182/blood-2025-6238
Published: 2025
Journal Articles

Experience of compassionate-use pegcetacoplan for paroxysmal nocturnal hemoglobinuria

Free
Clinical Trials & Observations
Morag Griffin, Petra Muus, Talha Munir, Sateesh Nagumantry, Alexandra Pike, Louise Arnold, Briony Forrest, Catherine Barnfield, Nicola Houghton, Nora Youngs, Richard Kelly
Journal: Blood
Blood (2023) 141 (1): 116–120.
https://doi.org/10.1182/blood.2022017266
Published: 2023
Journal Articles

A Phase 2, Open-Label Study Evaluating the Safety and Efficacy of Combination Pozelimab and Cemdisiran Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Switch from Eculizumab

Free
Richard Kelly, Nicola Houghton, Talha Munir, Petra Muus, Nora Youngs, Lisa Aurand, Bradley Dain, Rodrigo Pavani, Lorah Perlee, Amal Souttou, Morag Griffin
Journal: Blood
Blood (2022) 140 (Supplement 1): 8174–8175.
https://doi.org/10.1182/blood-2022-166547
Published: 2022
Journal Articles

A Phase 2, Randomized Trial Evaluating the Safety and Efficacy of Pozelimab and Cemdisiran in Patients with Paroxysmal Nocturnal Hemoglobinuria

Free
Jun Ho Jang, Raymond Wong, Rodrigo Pavani, Lisa Aurand, Bradley Dain, Lorah Perlee, Amal Souttou, Jonathan Weyne, Richard Kelly
Journal: Blood
Blood (2022) 140 (Supplement 1): 8172–8173.
https://doi.org/10.1182/blood-2022-163236
Published: 2022
Journal Articles

Pregnancy in Paroxysmal Nocturnal Hemoglobinuria - a Systematic Review

Free
James E. Manning, Etienne Ciantar, Morag Griffin, Richard Kelly
Journal: Blood
Blood (2022) 140 (Supplement 1): 11438–11440.
https://doi.org/10.1182/blood-2022-166545
Published: 2022
Journal Articles

Long-Term Safety and Efficacy of Pegcetacoplan Treatment in Adults with Paroxysmal Nocturnal Hemoglobinuria

Free
Christopher J. Patriquin, Andrija Bogdanovic, Morag Griffin, Richard Kelly, Jaroslaw P. Maciejewski, Brian Mulherin, Régis Peffault de Latour, Alexander Roeth, Veena Selvaratnam, Jeffrey Szer, Jessica Savage, Regina Horneff, Lisa Tan, Michael Yeh, Jens Panse
Journal: Blood
Blood (2022) 140 (Supplement 1): 2921–2923.
https://doi.org/10.1182/blood-2022-163625
Published: 2022
Journal Articles

Treatment Outcomes of Complement Protein C5 Inhibition in 509 Patients with Paroxysmal Nocturnal Hemoglobinuria in the United Kingdom

Free
Richard Kelly, Matthew Holt, Jennifer Vidler, Louise Arnold, Joanna Large, Briony Forrest, Catherine Barnfield, Alexandra Pike, Morag Griffin, Talha Munir, Petra Muus, Sateesh K. Nagumantry, Roochi Trikha, Austin Kulasekararaj, Lindsay Mitchell, Shreyans Ghandi
Journal: Blood
Blood (2022) 140 (Supplement 1): 5792–5794.
https://doi.org/10.1182/blood-2022-167725
Published: 2022
Journal Articles

Terminal Complement Inhibition and Control of Hemolysis in Paroxysmal Nocturnal Hemoglobinuria Following Switching from High-Dose Eculizumab to Ravulizumab: An Interim Analysis

Free
Morag Griffin, Shreyans Gandhi, Eden Hicks, Deepak Jain, Richard Kelly, Talha Munir, Petra Muus, Masayo Ogawa, Roochi Trikha, Ji Yu, Austin Kulasekararaj
Journal: Blood
Blood (2022) 140 (Supplement 1): 2928–2930.
https://doi.org/10.1182/blood-2022-159498
Published: 2022