| Page 3 | Aplastic Anemia and MDS International Foundation (AAMDSIF)

Finding His Way Through: Evan’s Story of PNH

Meet Evan. In a recent conversation with the

Factors affecting treatment decisions for lower-risk myelodysplastic syndromes across practice settings

Please find all content at the link.

Outcomes and Patterns of Relapse of NPM1 Mutated Acute Myeloid Leukemia Treated With Venetoclax Based Therapies

To the editor,
Mutations in nucleophosmin 1 (NPM1-mut) are detected in approximately 30% of newly diagnosed (ND)

Curing the Incurable: TP53 Mutated Myeloid Neoplasms

Abstract

ESCALATing care? Eltrombopag in pediatric aplastic anemia

Author(s):

Glass J, Groarke E

Primary Author:

Glass J

Journal Title:

Blood Advances

Original Publication Date:

Aug 2025

In this issue of Blood Advances, Shimamura et al1 report on a phase 2

Bone Marrow Disease(s):

Aplastic Anemia

The landscape of disease: mapping aplastic anemia

Author(s):

Patrick M Brunner

Primary Author:

Brunner P

Journal Title:

Blood

Original Publication Date:

Aug 2025

The development of antibody-based spatial multiplex assays, and the more recent introduction of spatial transcriptomic and proteomic platforms, now allows the characterization and profiling of human tissue samples at unprecedented depth, opening up a new exciting window for a better understanding of human pathology in situ, in both inflammatory and neoplastic conditions.

Bone Marrow Disease(s):

Aplastic Anemia

IHC Testing Could Serve as Surrogate to NGS for Early Detection of TP53-Mutant MDS/AML

Immunohistochemistry (IHC) could represent a biomarker for the early detection of TP53 mutations and for the prediction of a TP53 allelic state in patients with myelodysplastic neoplasms (MDS) and

A clinical guide to TP53 mutations in myeloid neoplasms

TP53 mutations are found in 10-15% of myeloid neoplasms and are one of its most important prognostic factors. Emerging data show that TP53 mutational allele status is a key determinant of clinical outcomes, with multi-hit TP53 mutant myeloid neoplasms having a very poor prognosis. Significant differences exist among the methods used in clinical and research settings to assess TP53 mutational status, leading to variability in reported patient characteristics, response to therapy, and survival.

Impaired cytotoxic function and exhausted phenotype of natural killer cells in VEXAS syndrome

Key Points
NK cells phenotyping reveals impaired cytotoxicity, chronic activation and exhaustion in VEXAS syndrome.

Decreased circulating NK cells were independently associated with an increased risk of severe infections in VEXAS syndrome.

Clinical Study on Targeted Drug Expands Treatment Options for Myelodysplastic Syndrome

Their research led to the 2022 approval of olutasidenib for certain patients with IDH1-mutant AML, which occurs in about 10% of AML. After that success, Dr. Watts and his team began to focus closely on testing the drug in MDS, a related condition that often progresses to AML.

IDH-1 mutations also occur in MDS, at a frequency of 3 to 5%.

Strong Responses
In the current study, the researchers tested olutasidenib in 22 MDS patients with IDH1-mutant tumors. All patients were classified with intermediate to high-risk disease, with 86% being high or very high risk.