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Why do we not have more drugs approved for MDS? A critical viewpoint on novel drug development in MDS

Author(s): 
Frumm SM, Shimony S, Stone RM, DeAngelo DJ, Bewersdorf JP, Zeidan AM, Stahl M
Primary Author: 
Frumm SM
Journal Title: 
Blood Advances
Original Publication Date: 
Jul 2023

Approval of new agents to treat higher risk (HR) myelodysplastic syndrome (MDS) has stalled since the approval of DNA methyltransferase inhibitors (DNMTi). In addition, the options for patients with lower risk (LR) MDS who have high transfusion needs and do not harbor ring sideroblasts or 5q- syndrome are limited. Here, we review the current treatment landscape in MDS and identify areas of unmet need, such as treatment after failure of erythropoiesis-stimulating agents or DNMTis, TP53-mutated disease, and MDS with potentially targetable mutations.

Bone Marrow Disease(s): 

Transplantation Referral Patterns for Patients with Newly Diagnosed Higher-Risk Myelodysplastic Syndromes and Acute Myeloid Leukemia at Academic and Community Sites in the Connect® Myeloid Disease Registry: Potential Barriers to Care

Author(s): 
Tomlinson B, de Lima M, Cogle CR, Thompson MA, Grinblatt DL, Pollyea DA, Komrokji RS, Roboz GJ, Savona MR, Sekeres MA, Abedi M, Garcia-Manero G, Kurtin SE, Maciejewski JP, Patel JL, Revicki DA, George TI, Flick ED, Kiselev P, Louis CU, DeGutis IS
Primary Author: 
Tomlinson B
Journal Title: 
Transplantation and Cellular Therapy
Original Publication Date: 
Jul 2023

Hematopoietic stem cell transplantation (HCT) is indicated for patients with higher-risk (HR)

Bone Marrow Disease(s): 

Health-related quality of life and vulnerability among people with myelodysplastic syndromes: a US national study

Author(s): 
Abel GA, Hebert D, Lee C, Rollison D, Gillis N, Komrokji R, Foran JM, Liu JJ, Al Baghdadi T, Deeg J, Gore S, Saber W, Wilson S, Otterstatter M, Thompson J, Borchert C, Padron E, DeZern A, Cella D, Sekeres MA
Primary Author: 
Abel GA
Journal Title: 
Blood Advances
Original Publication Date: 
Jul 2023

Health-related quality of life (HRQoL) and vulnerability are variably affected in patients with

Bone Marrow Disease(s): 

Activity of luspatercept and ESAs combination for treatment of anemia in lower-risk myelodysplastic syndromes

Author(s): 
Komrokji RS, Aguirre LE, Al Ali NH, Chan O, Xie Z, Kuykendall A, Sweet K, Lancet JE, Padron E, Sallman DA
Primary Author: 
Komrokji RS
Journal Title: 
Blood Advances
Original Publication Date: 
Jul 2023
Bone Marrow Disease(s): 

Do anemia treatments improve quality of life and physical function in patients with myelodysplastic syndromes (MDS)? A systematic review

Author(s): 
Mo A, Poynton M, Wood E, Shortt J, Brunskill SJ, Doree C, Sandercock J, Saadah N, Luk E, Stanworth SJ, McQuilten Z
Primary Author: 
Mo A
Journal Title: 
Blood Reviews
Original Publication Date: 
Jul 2023

Anemia is common in

Bone Marrow Disease(s): 

Role of innate immunological/inflammatory pathways in myelodysplastic syndromes and AML: a narrative review

Author(s): 
Vegivinti CTR, Keesari PR, Veeraballi S, Martins Maia CMP, Mehta AK, Lavu RR, Thakur RK, Tella SH, Patel R, Kakumani VK, Pulakurthi YS, Aluri S, Aggarwal RK, Ramachandra N, Zhao R, Sahu S, Shastri A, Verma A
Primary Author: 
Vegivinti CTR
Journal Title: 
Experimental Hematology & Oncology
Original Publication Date: 
Jul 2023

Dysregulation of the innate immune system and inflammatory-related pathways has been implicated in hematopoietic defects in the

Bone Marrow Disease(s): 

Oral therapy for myelodysplastic syndromes/neoplasms and acute myeloid leukemia: a revolution in progress

Author(s): 
Venugopal S, Shallis RM, Zeidan AM
Primary Author: 
Venugopal S
Journal Title: 
Expert Review of Anticancer Therapies
Original Publication Date: 
Jul 2023

Introduction: Patients with myeloid neoplasms such as

Bone Marrow Disease(s):