Page 1 | Search Results | Blood | American Society of Hematology

1-20 of 38

Sort by

Sort Order Select

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

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

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

Long-term safety and efficacy of iptacopan in patients with paroxysmal nocturnal hemoglobinuria: 4- and 5-year follow-up of patients from Phase 2 studies who entered the roll-over extension program

Antonio Risitano, Camilla Frieri, Bor-Sheng Ko, Lily Wong Lee Lee, Alexander Röth, Flore Sicre-de-Fontbrune, Eng Soo Yap, Sung-Soo Yoon, Philippe Ferber, Irina Baltcheva, Aurelie Verles, Silvia Sanz, Luca Monaco, Tim Costigan, Regis Peffault De Latour

Blood (2025) 146 (Supplement 1): 3198.

https://doi.org/10.1182/blood-2025-3198

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

Access to innovative treatment for paroxysmal nocturnal hemoglobinuria in low- and middle-income countries

Andrielle Yost, Mercedes Arteaga, Alicia Annamalay, Catherina Scheepers, Mei Ching Ong, Ines Garcia Gonzalez, Antonio Risitano, Katharine Lowndes, Petra Muus, James Michael Wrigglesworth, Bryan Murphy-Eustis, Pat Garcia-Gonzalez

Blood (2025) 146 (Supplement 1): 4387.

https://doi.org/10.1182/blood-2025-4387

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

Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH

Austin Kulasekararaj, Morag Griffin, Caroline Piatek, Jamile Shammo, Jun-ichi Nishimura, Christopher Patriquin, Hubert Schrezenmeier, Wilma Barcellini, Jens Panse, Anna Gaya, Yogesh Patel, Peng Liu, Gleb Filippov, Flore Sicre de Fontbrune, Antonio Risitano, Jong Wook Lee

Blood (2025) 145 (8): 811–822.

https://doi.org/10.1182/blood.2024026299

Published: 2025

Includes: Supplemental data

Journal Articles

Outcomes of hematopoietic stem cell transplantation in 813 pediatric patients with Fanconi anemia

Blood (2024) 144 (12): 1329–1342.

https://doi.org/10.1182/blood.2023022751

Published: 2024

Includes: Supplemental data

Journal Articles

Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT

Blood (2024) 144 (3): 323–333.

https://doi.org/10.1182/blood.2024024173

Published: 2024

Journal Articles

HLA-Haploidentical Stem Cell Transplantation in Children with Inherited Bone Marrow Failure Syndromes: A Retrospective Analysis on Behalf of EBMT Severe Aplastic Anemia Working Party

Blood (2022) 140 (Supplement 1): 5813–5815.

https://doi.org/10.1182/blood-2022-163219

Published: 2022

Journal Articles

Outcome of Haematopoietic Cell Transplantation in 813 Children with Fanconi Anaemia: A Study on Behalf of the EBMT Severe Aplastic Anaemia Working Party and Paediatric Disease Working Party

Blood (2022) 140 (Supplement 1): 657–659.

https://doi.org/10.1182/blood-2022-163140

Published: 2022

Journal Articles

Dose-Exposure-Response Relationships of Biomarkers and Efficacy Measures with Iptacopan, a Complement Factor B Inhibitor, in Patients (pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH) with or without Concomitant Anti-C5 Therapy

Antonio Risitano, Régis Peffault de Latour, Eng Soo Yap, Lily LL Wong, Bor-Sheng Ko, Sung-Soo Yoon, Alexander Roeth, Irina Baltcheva, Julie Milojevic, Niamh Fagan, Kenneth Kulmatycki, Prasanna K Nidamarthy, Guido Junge, Jun Ho Jang

Blood (2022) 140 (Supplement 1): 5803–5804.

https://doi.org/10.1182/blood-2022-160114

Published: 2022

Journal Articles

Current Use of Androgens in Bone Marrow Failure Disorders: A Report from the Severe Aplastic Anemia Working Party (SAAWP) of the European Society of Blood and Marrow Transplantation (EBMT)

Blood (2022) 140 (Supplement 1): 5799–5800.

https://doi.org/10.1182/blood-2022-169086

Published: 2022

Journal Articles

Complement Biomarkers for Tracking Breakthrough Hemolysis in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated with Last-Generation Complement Inhibitors

Luana Marano, Patrizia Ricci, Camilla Frieri, Eleonora Urciuoli, Ada Sanseverino, Fabiana Cacace, Ilenia Manfra, Giovanna De Santis, Selenia Vitiello, Serena Marotta, Antonio Risitano

Blood (2022) 140 (Supplement 1): 5797–5798.

https://doi.org/10.1182/blood-2022-158329

Published: 2022

Journal Articles

Vemircopan (ALXN2050) Monotherapy in Paroxysmal Nocturnal Hemoglobinuria: Interim Data from a Phase 2 Open-Label Proof-of-Concept Study

Peter J. Browett, Austin Kulasekararaj, Rosario Notaro, Masayo Ogawa, Antonio Risitano, Ji Yu, Jong Wook Lee

Blood (2022) 140 (Supplement 1): 717–719.

https://doi.org/10.1182/blood-2022-169301

Published: 2022

Update search

NARROW

Publications

Format

Subjects

Article Type

Topics

Date

Availability

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

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

Long-term safety and efficacy of iptacopan in patients with paroxysmal nocturnal hemoglobinuria: 4- and 5-year follow-up of patients from Phase 2 studies who entered the roll-over extension program

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

Access to innovative treatment for paroxysmal nocturnal hemoglobinuria in low- and middle-income countries

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

Long-term efficacy and safety of danicopan as add-on therapy to ravulizumab or eculizumab in PNH with significant EVH

Outcomes of hematopoietic stem cell transplantation in 813 pediatric patients with Fanconi anemia

Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT

HLA-Haploidentical Stem Cell Transplantation in Children with Inherited Bone Marrow Failure Syndromes: A Retrospective Analysis on Behalf of EBMT Severe Aplastic Anemia Working Party

Outcome of Haematopoietic Cell Transplantation in 813 Children with Fanconi Anaemia: A Study on Behalf of the EBMT Severe Aplastic Anaemia Working Party and Paediatric Disease Working Party

Dose-Exposure-Response Relationships of Biomarkers and Efficacy Measures with Iptacopan, a Complement Factor B Inhibitor, in Patients (pts) with Paroxysmal Nocturnal Hemoglobinuria (PNH) with or without Concomitant Anti-C5 Therapy

Current Use of Androgens in Bone Marrow Failure Disorders: A Report from the Severe Aplastic Anemia Working Party (SAAWP) of the European Society of Blood and Marrow Transplantation (EBMT)

Complement Biomarkers for Tracking Breakthrough Hemolysis in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated with Last-Generation Complement Inhibitors

Vemircopan (ALXN2050) Monotherapy in Paroxysmal Nocturnal Hemoglobinuria: Interim Data from a Phase 2 Open-Label Proof-of-Concept Study

Email alerts

ASH Publications

American Society of Hematology

Update search

Email alerts

ASH Publications

American Society of Hematology

This Feature Is Available To Subscribers Only