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Journal Articles

Molecular gene panel analysis in aplastic anaemia: Diagnostic utility and clinical implications

Francesco Versino, Roochi Trikha, Joanna Large, Guy Hannah, Steve Best, Shreyans Gandhi, Luke Ficinski, Paul Inocencio, Ana Olivera, Jianne Miran, Kar Lok Kong, Austin Kulasekararaj

Blood (2025) 146 (Supplement 1): 1711.

https://doi.org/10.1182/blood-2025-1711

Published: 2025

Journal Articles

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

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

Blood (2025) 146 (Supplement 1): 1114.

https://doi.org/10.1182/blood-2025-1114

Published: 2025

Journal Articles

Patient experience sub-study of a randomised UK multicentre Phase 2 trial of ASTX727 versus hydroxycarbamide/best supportive care in myelodysplastic (MDS)/myeloproliferative (MPN) overlap syndromes (AMMO)

Ashleigh Ward, Sally Taylor, Grant Punnett, Ramsay Devaraj, Chhaya Sankhalpara, Rebecca Record, Kiran Batta, Jennifer Byrne, Catherine Cargo, Onima Chowdhury, Dominic Culligan, Nicholas Cunningham, Steve Knapper, Austin Kulasekararaj, Srinivasan Narayanan, Manoj Raghavan, Rob Sellar, Daniel Wiseman, Janelle Yorke

Blood (2025) 146 (Supplement 1): 8164.

https://doi.org/10.1182/blood-2025-8164

Published: 2025

Journal Articles

The CSMD1 gene is involved in hematopoietic stem cell HSC pruning in patients with acquired immune-mediated aplastic anemia

Antonio Risitano, Deniz Ece Kaya, Simona Iacobelli, Riley Cook, Giorgio Napolitani, Sila Gerlevik, Mohammad M Karimi, Nogayhan Seymen, Flore Sicre-de-Fontbrune, Morag Griffin, Camilla Frieri, Carlo Dufour, Regis Peffault De Latour, Ghulam Mufti, Austin Kulasekararaj

Blood (2025) 146 (Supplement 1): 4982.

https://doi.org/10.1182/blood-2025-4982

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

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

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

Blood (2025) 146 (Supplement 1): 4979.

https://doi.org/10.1182/blood-2025-4979

Published: 2025

Journal Articles

The 2-year efficacy and safety of iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria with a history of aplastic anemia on concomitant immunosuppressive therapy who entered the roll-over extension program

Regis Peffault De Latour, Carlos de Castro, Anna Gaya, Morag Griffin, Bing Han, Austin Kulasekararaj, Jaroslaw Maciejewski, Bhumika Patel, Alexander Röth, Phillip Scheinberg, Hubert Schrezenmeier, Yasutaka Ueda, Tim Costigan, Marion Dahlke, Philippe Ferber, Rajeshri Karki, Luca Monaco, Silvia Sanz, Antonio Risitano

Blood (2025) 146 (Supplement 1): 4978.

https://doi.org/10.1182/blood-2025-4978

Published: 2025

Journal Articles

DDX41-related myeloid neoplasms: Insights and treatment landscape in 250 patients managed at a large tertiary centre

Francesco Versino, Shreyas Hanmantgad, Pramila Krishnamurthy, Victoria Potter, Roochi Trikha, Mili Shah, Varun Mehra, Lynn Quek, Guy Hannah, Henry Wood, Shreyans Gandhi, Joanna Large, Clair Engelbrecht, Katie Snape, Guilherme Sapinho, Steve Best, Kar Lok Kong, Austin Kulasekararaj

Blood (2025) 146 (Supplement 1): 5262.

https://doi.org/10.1182/blood-2025-5262

Published: 2025

Journal Articles

Allogeneic hematopoietic cell transplantation in dyskeratosis congenita: Short and long- term outcome of 183 consecutive patients transplanted at EBMT centers

Blood (2025) 146 (Supplement 1): 2490.

https://doi.org/10.1182/blood-2025-2490

Published: 2025

Journal Articles

6pLOH as a marker of immune-mediated bone marrow failure: Insights from a large single center experience

Francesco Versino, Roochi Trikha, Joanna Large, Henry Wood, Azim Mohamedali, Kar Lok Kong, Shreyans Gandhi, Austin Kulasekararaj

Blood (2025) 146 (Supplement 1): 3194.

https://doi.org/10.1182/blood-2025-3194

Published: 2025

Journal Articles

Thymoglobuline and grafalon show comparable transplant outcomes in patients with aplastic anemia undergoing allogeneic stem cell transplantation: A multicenter EBMT saawp study of 1603 patients

Blood (2025) 146 (Supplement 1): 3192.

https://doi.org/10.1182/blood-2025-3192

Published: 2025

Journal Articles

Excellent long-term outcomes after alemtuzumab-based hematopoietic cell transplantation (HCT) for severe aplastic anemia (SAA) and inherited bone marrow failure syndromes (IBMF): A 25-year British society of blood and marrow transplantation and cellular therapy (BSBMTCT) retrospective analysis of 1754 patients from the United Kingdom (UK)

Austin Kulasekararaj, Kanchan Rao, Rachel Pearce, Julia Lee, Marie Wilson, Anjum Khan, Rachel Protheroe, Pramila Krishnamurthy, R.F. Wynn, Persis Amrolia, Victoria Potter

Blood (2025) 146 (Supplement 1): 984.

https://doi.org/10.1182/blood-2025-984

Published: 2025

Journal Articles

First-in-human evaluation of IL2RG blockade in patients with severe aplastic anemia that is refractory to or relapsed on immunosuppressive therapy

Blood (2025) 146 (Supplement 1): 27.

https://doi.org/10.1182/blood-2025-27

Published: 2025

Journal Articles

Deep immune signature of immune-mediated aplastic anemia patients shows distinct subsets of regulatory T cells associated with response to treatment: Results from the phase 3, randomized EBMT race clinical trial

Blood (2025) 146 (Supplement 1): 26.

https://doi.org/10.1182/blood-2025-26

Published: 2025

Journal Articles

The global PNH patient registry: real-world data on PNH symptom burden and quality of life

David Araten, Austin Kulasekararaj, Jun-ichi Nishimura, Regis Peffault De Latour, Jeff Szer, Ilene Weitz, Azalea Candelaria, Aliza Fink, Alice Houk, Elizabeth Kottke, Brad Wong

Blood (2025) 146 (Supplement 1): 2697.

https://doi.org/10.1182/blood-2025-2697

Published: 2025

Journal Articles

Paroxysmal nocturnal hemoglobinuria in pediatric patients: A UK experience

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

Blood (2025) 146 (Supplement 1): 4662.

https://doi.org/10.1182/blood-2025-4662

Published: 2025

Journal Articles

Diagnostic conformity to who criteria and use of cytoreductive therapy in classical myeloproliferative neoplasms: Experience from a resource-limited country

Sayanthan Sivakumaran, Ahalyaa Sivashangar, Visaka Ratnamalala, Sanjeewa Pathirage, Manu Wimalachandra, Austin Kulasekararaj, Lallindra Gooneratne

Blood (2025) 146 (Supplement 1): 7324.

https://doi.org/10.1182/blood-2025-7324

Published: 2025

Journal Articles

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

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

Blood (2025) 146 (Supplement 1): 4974.

https://doi.org/10.1182/blood-2025-4974

Published: 2025

Journal Articles

Improved outcomes following allogeneic hematopoietic stem cell transplantation in patients with DDX41-mutated myeloid neoplasms: A prospective analysis of survival, GVHD, and transplant-related mortality

Shreyas Hanmantgad, Alan Mitchell, Kayleigh Mccloskey, James Aries, Francesco Versino, Roochi Trikha, Shreyans Gandhi, Madson Correia De Farias, Maria Cuadrado, Mili Shah, Varun Mehra, Pramila Krishnamurthy, Michelle Kenyon, Victoria Potter, Austin Kulasekararaj

Blood (2025) 146 (Supplement 1): 6083.

https://doi.org/10.1182/blood-2025-6083

Published: 2025

Journal Articles

17p loss in TP53-non-mutated myeloid neoplasms confers survival comparable to TP53-mutated disease

Blood (2025) 146 (Supplement 1): 3858.

https://doi.org/10.1182/blood-2025-3858

Published: 2025

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Molecular gene panel analysis in aplastic anaemia: Diagnostic utility and clinical implications

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

Patient experience sub-study of a randomised UK multicentre Phase 2 trial of ASTX727 versus hydroxycarbamide/best supportive care in myelodysplastic (MDS)/myeloproliferative (MPN) overlap syndromes (AMMO)

The CSMD1 gene is involved in hematopoietic stem cell HSC pruning in patients with acquired immune-mediated aplastic anemia

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

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

The 2-year efficacy and safety of iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria with a history of aplastic anemia on concomitant immunosuppressive therapy who entered the roll-over extension program

DDX41-related myeloid neoplasms: Insights and treatment landscape in 250 patients managed at a large tertiary centre

Allogeneic hematopoietic cell transplantation in dyskeratosis congenita: Short and long- term outcome of 183 consecutive patients transplanted at EBMT centers

6pLOH as a marker of immune-mediated bone marrow failure: Insights from a large single center experience

Thymoglobuline and grafalon show comparable transplant outcomes in patients with aplastic anemia undergoing allogeneic stem cell transplantation: A multicenter EBMT saawp study of 1603 patients

First-in-human evaluation of IL2RG blockade in patients with severe aplastic anemia that is refractory to or relapsed on immunosuppressive therapy

Deep immune signature of immune-mediated aplastic anemia patients shows distinct subsets of regulatory T cells associated with response to treatment: Results from the phase 3, randomized EBMT race clinical trial

The global PNH patient registry: real-world data on PNH symptom burden and quality of life

Paroxysmal nocturnal hemoglobinuria in pediatric patients: A UK experience

Diagnostic conformity to who criteria and use of cytoreductive therapy in classical myeloproliferative neoplasms: Experience from a resource-limited country

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

Improved outcomes following allogeneic hematopoietic stem cell transplantation in patients with DDX41-mutated myeloid neoplasms: A prospective analysis of survival, GVHD, and transplant-related mortality

17p loss in TP53-non-mutated myeloid neoplasms confers survival comparable to TP53-mutated disease

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