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The evolution of epigenetic therapy in myelodysplastic syndromes and acute myeloid leukemia

Author(s): 
Jesus D Gonzalez-Lugo, Samarpana Chakraborty, Amit Verma, Aditi Shastri
Primary Author: 
Gonzalez-Lugo J
Journal Title: 
Seminars in Hematology
Original Publication Date: 
Jan 2021

Mutations in the group of epigenetic modifiers are the largest group of mutated genes in

Bone Marrow Disease(s): 

Myelodysplastic syndromes with ring sideroblasts (MDS-RS) and MDS/myeloproliferative neoplasm with RS and thrombocytosis (MDS/MPN-RS-T) - "2021 update on diagnosis, risk-stratification, and management"

Author(s): 
Mrinal M Patnaik, Ayalew Tefferi
Primary Author: 
Patnaik M
Journal Title: 
American Journal of Hematology
Original Publication Date: 
Jan 2021

Disease overview: Ring sideroblasts (RS) are erythroid precursors with abnormal perinuclear mitochondrial iron accumulation. Two myeloid neoplasms defined by the presence of RS, include

Bone Marrow Disease(s): 

Eprenetapopt (APR-246) and Azacitidine in TP53-Mutant Myelodysplastic Syndromes

Author(s): 
Sallman DA, DeZern AE, Garcia-Manero G, Steensma DP, Roboz GJ, Sekeres MA, Cluzeau T, Sweet KL, McLemore A, McGraw KL, Puskas J, Zhang L, Yao J, Mo Q, Nardelli L, Al Ali NH, Padron E, Korbel G, Attar EC, Kantarjian HM
Primary Author: 
Sallman DA
Journal Title: 
Journal of clinical oncology: official journal of the American Society Clinical Oncology
Original Publication Date: 
Jan 2021

Purpose: Approximately 20% of patients with TP53-mutant

Bone Marrow Disease(s): 

Imetelstat Achieves Meaningful and Durable Transfusion Independence in High Transfusion-Burden Patients With Lower-Risk Myelodysplastic Syndromes in a Phase II Study

Author(s): 
Steensma DP, Fenaux P, Van Eygen K, Raza A, Santini V, Germing U, Font P, Diez-Campelo M, Thepot S, Vellenga E, Patnaik MM, Jang JH, Varsos H, Bussolari J, Rose E, Sherman L, Sun L, Wan Y, Dougherty S, Huang F, Feller F, Rizo A, Platzbecker U
Primary Author: 
Steensma DP
Journal Title: 
Journal of clinical oncology: official journal of the American Society Clinical Oncology
Original Publication Date: 
Jan 2020

Purpose: Patients with lower-risk (LR)

Bone Marrow Disease(s): 

Implication of PIGA genotype on erythrocytes phenotype in Paroxysmal Nocturnal Hemoglobinuria

Author(s): 
Gurnari C, Pagliuca S, Patel BJ, Awada H, Kongkiatkamon S, Terkawi L, Zawit M, Corey S, Lichtin AE, Carraway HE, Wahida A, Visconte V, Maciejewski JP
Primary Author: 
Gurnari C
Journal Title: 
Leukemia
Original Publication Date: 
Jan 2021
Bone Marrow Disease(s): 

Randomized trials with checkpoint inhibitors in acute myeloid leukaemia and myelodysplastic syndromes: What have we learned so far and where are we heading?

Author(s): 
Bewersdorf JP, Zeidan AM
Primary Author: 
Bewersdorf JP
Journal Title: 
Best practice & research. Clinical haematology
Original Publication Date: 
Dec 2020

The treatment of acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS) has seen dramatic advances with the approval of multiple novel agents in recent years. However, unlike solid malignancies, immune checkpoint inhibitors have yet to garner regulatory approval in AML and MDS with recent randomized clinical trials yielding only underwhelming results. Novel targets have been explored in early phase clinical trials with impressive results leading to ongoing subsequent controlled trials.

Bone Marrow Disease(s):