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Risk-Adapted, Individualized Treatment Strategies of Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML)

Author(s): 
Jan Philipp Bewersdorf, Amer M Zeidan
Primary Author: 
Philipp J
Journal Title: 
Cancers
Original Publication Date: 
Mar 2021

Myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML) are two distinct blood cancers with a variable clinical symptom burden and risk of progression to

Bone Marrow Disease(s): 

Aprea Therapeutics Receives FDA Orphan Drug Designation for Eprenetapopt for the Treatment of Acute Myeloid Leukemia (AML)

BOSTON, April 08, 2021 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein, p53, today announced that the U.S. Food and Drug Administration (FDA) has granted

Blood cancer risk higher than expected in kids with Down syndrome

New York, April 7

The risks of acute myeloid leukaemia (AML)—a type of blood cancer—in children with Down syndrome is stronger than expected, according to a new study.

The study led by researchers from the University of Chicago, Davis Health and San Francisco, examined medical data of more than 3.9 million children born between 1996-2016 in seven US healthcare systems or in Ontario, Canada. 

It showed that 2.8 per cent of children with Down syndrome were diagnosed with leukaemia, compared to 0.05 per cent of other children.

Clinical and molecular predictors of response and survival following venetoclax therapy in relapsed/refractory AML

Key Points
In patients with RR-AML, venetoclax combination therapy resulted in responses in 31% of patients and a median OS of 6.1 months.

NPM1 mutations predicted higher response rates; adverse cytogenetics and mutations in TP53, KRAS/NRAS, and SF3B1 predicted worse OS.

Germline mutations in MDS/AML predisposition disorders

Author(s): 
Lucy A Godley
Primary Author: 
Godley LA
Journal Title: 
Current opinion in hematology
Original Publication Date: 
Mar 2021

Purpose of review: Recognition of hereditary hematopoietic malignancies impacts patient management as well as health surveillance strategies for the patient and relatives who share the causative DNA variant. In this review, barriers to the diagnosis and management of patients are outlined.

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: 
Mar 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): 

Alternative donor transplantation for myelodysplastic syndromes: haploidentical relative and matched unrelated donors

Author(s): 
Grunwald MR, Zhang MJ, Elmariah H, Johnson MH, St Martin A, Bashey A, Battiwalla M, Bredeson CN, Copelan E, Cutler CS, George BR, Gupta V, Kanakry C, Mehta RS, Milano F, Mussetti A, Nakamura R, Nishihori T, Saber W, Solh M, Weisdorf DJ, Eapen M
Primary Author: 
Grunwald MR
Journal Title: 
Blood Advances
Original Publication Date: 
Feb 2021

We compared outcomes in 603 patients with myelodysplastic syndrome (MDS) after HLA-haploidentical relative (n = 176) and HLA-matched unrelated (n = 427) donor hematopoietic cell transplantation (HCT) from 2012 to 2017, using the Center for International Blood and Marrow Transplant Research database. All transplantations used reduced-intensity conditioning regimens.

Bone Marrow Disease(s):