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Terminal complement inhibition and control of hemolysis in patients with paroxysmal nocturnal hemoglobinuria who switched from high-dose eculizumab to ravulizumab: a phase IV, single-arm clinical trial

Author(s): 
Griffin M, Gandhi S, Kelly RJ, Munir T, Trikha R, Hicks E, Jain D, Ogawa M, Yu J, Muus P, Kulasekararaj AG
Primary Author: 
Griffin M
Journal Title: 
Haematologica
Original Publication Date: 
Nov 2024
Bone Marrow Disease(s): 

Thrombosis in Paroxysmal Nocturnal Hemoglobinuria (PNH): From Pathogenesis to Treatment

Author(s): 
Kokoris S, Polyviou A, Evangelidis P, Grouzi E, Valsami S, Tragiannidis K, Gialeraki A, Tsakiris DA, Gavriilaki E
Primary Author: 
Kokoris S
Journal Title: 
International Journal of Molecular Sciences
Original Publication Date: 
Nov 2024

Bone Marrow Disease(s): 

Abatacept for acute graft-versus-host disease prophylaxis after unrelated donor hematopoietic cell transplantation

Key Points
This study illustrates the survival benefits of abatacept combined with CNI/MTX in patients with hematologic malignancies undergoing HCT.

The addition of abatacept may provide an approach for alternative donor pool expansion when HLA-identical sibling donors are unavailable.

Efficacy and Safety of Inhibitors of the Proximal Pathway of the Complement System in Paroxysmal Nocturnal Hemoglobinuria: A Meta-analysis of Randomized Controlled Trials

Author(s): 
Sobral MVS, Moulaz IR, Soares VG, Pirolla RDC, Bendaham LCAR, Faria HS, Peres CAP, Al-Sharif L, Bressa RC
Primary Author: 
Sobral MVS
Journal Title: 
American Journal of Therapeutics
Original Publication Date: 
Oct 2024
Bone Marrow Disease(s): 

Safety and efficacy of danicopan in patients with paroxysmal nocturnal hemoglobinuria: a systematic review and meta-analysis

Author(s): 
Muvaffak E, Mokresh ME, Varda A, Lakmoush M, Ilter MK
Primary Author: 
Muvaffak E
Journal Title: 
Expert Review of Hematology
Original Publication Date: 
Oct 2024

Introduction: 

Bone Marrow Disease(s): 

Cost-effectiveness of iptacopan for paroxysmal nocturnal hemoglobinuria

Author(s): 
Ito S, Chetlapalli K, Wang D, Potnis KC, Richmond R, Krumholz H, Lee AI, Cuker A, Goshua G
Primary Author: 
Ito S
Journal Title: 
Blood
Original Publication Date: 
Oct 2024

Iptacopan, a novel oral Factor B inhibitor, recently obtained FDA approval for treating

Bone Marrow Disease(s):