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Real-world Effectiveness of Azacitidine in Treatment-Naive Patients With Higher-risk Myelodysplastic Syndromes

Author(s): 
Rajakumaraswamy N, Gandhi M, Wei AH, Sallman DA, Daver NG, Mo S, Iqbal S, Karalliyadda R, Chen M, Wang Y, Vyas P
Primary Author: 
Rajakumaraswamy N
Journal Title: 
Clinical Lymphoma, Myeloma & Leukemia
Original Publication Date: 
Apr 2024

Introduction: Azacitidine (AZA) is an approved frontline therapy for higher-risk

Bone Marrow Disease(s): 

Molecular and clinical presentation of UBA1-mutated myelodysplastic syndromes

Author(s): 
Sirenko M, Bernard E, Creignou M, Domenico D, Farina A, Arango Ossa JE, Kosmider O, Hasserjian RP, Jädersten M, Germing U, Sanz GF, van de Loosdrecht AA, Gurnari C, Follo MY, Thol FR, Zamora L, Pinheiro RF, Pellagatti A, Elias HK, Haase DT, Sander B
Primary Author: 
Sirenko M
Journal Title: 
Blood
Original Publication Date: 
Apr 2024

Mutations in UBA1, which are disease-defining for VEXAS syndrome, have been reported in patients diagnosed with

Bone Marrow Disease(s): 

T-cell dysfunctions in myelodysplastic syndromes

Author(s): 
Rodriguez-Sevilla JJ, Colla S
Primary Author: 
Rodriguez-Sevilla JJ, Colla S
Journal Title: 
Blood
Original Publication Date: 
Apr 2024

Escape from immune surveillance is a hallmark of cancer. Immune deregulation caused by intrinsic and extrinsic cellular factors, such as altered T-cell functions, leads to immune exhaustion, loss of immune surveillance, and clonal proliferation of tumoral cells. The T-cell immune system contributes to the pathogenesis, maintenance, and progression of myelodysplastic syndrome (MDS).

Bone Marrow Disease(s): 

Hemolysis events in the Phase 3 PEGASUS study of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuria

Author(s): 
Peffault de Latour R, Griffin M, Kelly RJ, Szer J, de Castro C, Horneff R, Tan L, Yeh MM, Panse JP
Primary Author: 
Peffault de Latour R
Journal Title: 
Blood Advances
Original Publication Date: 
Apr 2024

Patients with

Bone Marrow Disease(s): 

Safety and Efficacy of Pegcetacoplan in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria over 48 Weeks: 307 Open-Label Extension Study

Author(s): 
Patriquin CJ, Bogdanovic A, Griffin M, Kelly RJ, Maciejewski JP, Mulherin B, Peffault de Latour R, Röth A, Selvaratnam V, Szer J, Al-Adhami M, Horneff R, Tan L, Yeh M, Panse J
Primary Author: 
Patriquin CJ
Journal Title: 
Advances in Therapy
Original Publication Date: 
Apr 2024

Introduction: 

Bone Marrow Disease(s):