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Clinical and prognostic significance of small paroxysmal nocturnal hemoglobinuria clones in myelodysplastic syndrome and aplastic anemia

Author(s): 
Fattizzo B, Ireland R, Dunlop A, Yallop D, Kassam S, Large J, Gandhi S, Muus P, Manogaran C, Sanchez K, Consonni D, Barcellini W, Mufti GJ, Marsh JCW, Kulasekararaj AG
Primary Author: 
Fattizzo B
Journal Title: 
Leukemia
Original Publication Date: 
Mar 2021

In this large single-centre study, we report high prevalence (25%) of, small (<10%) and very small (<1%),

Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria

Author(s): 
Hillmen P, Szer J, Weitz I, Röth A, Höchsmann B, Panse J, Usuki K, Griffin M, Kiladjian JJ, de Castro C, Nishimori H, Tan L, Hamdani M, Deschatelets P, Francois C, Grossi F, Ajayi T, Risitano A, de la Tour RP
Primary Author: 
Hillman P
Journal Title: 
The New England Journal of Medicine
Original Publication Date: 
Mar 2021

Background: 

Bone Marrow Disease(s): 

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

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.