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Consensus proposal for revised International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes

Author(s): 
Zeidan AM, Platzbecker U, Bewersdorf JP, Stahl M, Adès L, Borate U, Bowen D, Buckstein R, Brunner A, Carraway HE, Daver N, Díez-Campelo M, de Witte T, DeZern AE, Efficace F, Garcia-Manero G, Garcia JS, Germing U, Giagounidis A, Griffiths EA, Hasserjian RP
Primary Author: 
Zeidan AM
Journal Title: 
Blood
Original Publication Date: 
Apr 2023

Bone Marrow Disease(s): 

Transplant 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: 
Apr 2023

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

Bone Marrow Disease(s): 

Therapeutic Targets in Myelodysplastic Neoplasms: Beyond Hypomethylating Agents

Author(s): 
Pophali P, Desai SR, Shastri A
Primary Author: 
Pophali P
Journal Title: 
Current Hematologic Malignancy Reports
Original Publication Date: 
Apr 2023

Purpose of review: To discuss novel targeted therapies under investigation for treatment of myelodysplastic neoplasms (MDS).

Bone Marrow Disease(s): 

VEXAS syndrome: a new paradigm for adult-onset monogenic autoinflammatory diseases

Author(s): 
Vitale A, Caggiano V, Bimonte A, Caroni F, Tosi GM, Fabbiani A, Renieri A, Bocchia M, Frediani B, Fabiani C, Cantarini L
Primary Author: 
Vitale A
Journal Title: 
Internal and Emergency Medicine
Original Publication Date: 
Apr 2023

VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic) syndrome is a recently described pathological entity. It is an acquired monogenic autoinflammatory disease caused by somatic mutations of the UBA1 gene in blood cells precursors; the gene encodes one of the two E1 enzyme isoforms that initiates ubiquitylation in cell's cytoplasm. VEXAS syndrome leads to systemic inflammation, with all organs and tissues potentially involved.

Bone Marrow Disease(s): 

Alternative donor BMT with post-transplant cyclophosphamide as initial therapy for acquired severe aplastic anemia

Author(s): 
DeZern A, Zahurak ML, Symons HJ, Cooke KR, Huff CA, Jain T, Swinnen LJ, Imus PH, Wagner-Johnston ND, Ambinder RF, Levis MJ, Luznik L, Bolaños-Meade J, Fuchs EJ, Jones RJ, Brodsky RA
Primary Author: 
DeZern A
Journal Title: 
Blood
Original Publication Date: 
Apr 2023

Severe

Bone Marrow Disease(s): 

What causes aplastic anaemia?

Author(s): 
Gale RP, Hinterberger W, Young NS, Gennery AR, Dvorak CC, Hebert KM, Heim M, Broglie L, Eapen M
Primary Author: 
Gale RP
Journal Title: 
Leukemia
Original Publication Date: 
Apr 2023

Aplastic anaemia has diverse aetiologies including: (1) direct damage to haematopoietic stem or progenitor cells such as from chemicals, drugs and ionising radiations; (2) an abnormal bone marrow micro-environment, (3) immune-mediated mechanism(s); or (4) combination of these. Aetiologies may differ between persons with similar phenotypes and even genotypes [1] (References in the Supplementary).

Bone Marrow Disease(s):