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An agenda to advance research in myelodysplastic syndromes: a TOP 10 priority list from the first international workshop in MDS

Author(s): 
Stahl M, Abdel-Wahab O, Wei AH, Savona MR, Xu ML, Xie Z, Taylor J, Starczynowski D, Sanz GF, Sallman DA, Santini V, Roboz GJ, Patnaik MM, Padron E, Odenike O, Nazha A, Nimer SD, Majeti R, Little RF, Gore S, List AF, Kutchroo V, Komrokji RS, Kim TK, Kim N
Primary Author: 
Stahl M
Journal Title: 
Blood Advances
Original Publication Date: 
Jun 2023
Bone Marrow Disease(s): 

Nationwide study of eculizumab in paroxysmal nocturnal hemoglobinuria: Evaluation of treatment indications and outcomes

Author(s): 
Schaap CCM, Heubel-Moenen FCJI, Nur E, Bartels M, van der Heijden OWH, de Jonge E, Preijers FWMB, Blijlevens NMA, Langemeijer SMC; Dutch PNH Working Group
Primary Author: 
Schaap CCM
Journal Title: 
European Journal of Haematology
Original Publication Date: 
Jun 2023

Eculizumab is an effective treatment for

Bone Marrow Disease(s): 

Treatment of eculizumab refractory paroxysmal nocturnal hemoglobinuria: A systematic review about current treatment options and future direction

Author(s): 
Syed S, Khan R, Khurram F, Khan FH, Safi D, Safi SUD
Primary Author: 
Syed S
Journal Title: 
SAGE Open Medicine
Original Publication Date: 
Jun 2023

Objectives: C5 inhibitors such as eculizumab and ravulizumab are the first-line treatment in the management of

Bone Marrow Disease(s): 

Pegcetacoplan controls hemolysis in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria

Author(s): 
Wong RSM, Navarro-Cabrera JR, Comia NS, Goh YT, Idrobo H, Kongkabpan D, Gómez-Almaguer D, Al-Adhami M, Ajayi T, Alvarenga P, Savage J, Deschatelets P, Francois C, Grossi F, Dumagay T
Primary Author: 
Wong RSM
Journal Title: 
Blood Advances
Original Publication Date: 
Jun 2023

Bone Marrow Disease(s): 

Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations

Author(s): 
Bodó I, Amine I, Boban A, Bumbea H, Kulagin A, Lukina E, Piekarska A, Zupan IP, Sokol J, Windyga J, Cermak J
Primary Author: 
Bodó I
Journal Title: 
Advances in Therapy
Original Publication Date: 
Jun 2023

Introduction: Hemolysis in

Bone Marrow Disease(s): 

Conditional survival and standardized mortality ratios of patients with severe aplastic anemia surviving at least one year after hematopoietic cell transplantation or immunosuppressive therapy

Author(s): 
Nakamura R, Patel BA, Kim S, Wong FL, Armenian SH, Groarke EM, Kessler DA, Hebert KM, Heim M, Eapen M, Young NS
Primary Author: 
Nakamura R
Journal Title: 
Haematologica
Original Publication Date: 
Jun 2023

Immunosuppressive treatment (IST) and hematopoietic cell transplant (HCT) are standard therapies for severe

Bone Marrow Disease(s): 

Alternative donor BMT with posttransplant cyclophosphamide as initial therapy for acquired severe aplastic anemia

Author(s): 
DeZern AE, Zahurak M, Symons HJ, Cooke KR, Huff CA, Jain T, Swinnen LJ, Imus PH, Wagner-Johnston ND, Ambinder RF, Levis M, Luznik L, Bolaños-Meade J, Fuchs EJ, Jones RJ, Brodsky RA
Primary Author: 
DeZern AE
Journal Title: 
Blood
Original Publication Date: 
Jun 2023

Severe

Bone Marrow Disease(s):