News and Treatment Updates

Here's where you'll find a regularly updated, broad range of articles written by the AAMDSIF team, allied health organizations and news organizations. By staying well-informed, patients and families are practicing a form of self-support that will help them be more effective self-advocates when engaging with health care providers.

Cyclophosphamide and Cyclosporin for GVHD Prevention

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Curtis et al. (July 17 issue)1 report evidence of longer graft-versus-host disease (GVHD)–free, relapse-free survival with post-transplantation cyclophosphamide–based prophylaxis than with cyclosporin–methotrexate among patients receiving a stem-cell transplant from a matched…

Azacitidine as therapy for VEXAS syndrome

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How should patients with VEXAS (an acronym for vacuoles [in myeloid and erythroid precursors], E1 enzyme, X-linked, autoinflammatory, somatic) syndrome be treated? Until now, we have only had case reports and small series to guide us. In this issue of Blood, Jachiet and…

A 30-gene classifier distinguishes low-risk MDS HSPCs from healthy HSPCs

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Highlights • Single-cell RNA-sequencing performed on 16 lower-risk myelodysplastic syndrome (LR-MDS) bone marrow-derived mononuclear cells (BMMCs) • A 30-gene signature distinguishes LR-MDS hematopoietic stem and progenitor cells (HSPCs) from Healthy HSPCs • Vesicular trafficking…

Finding His Way Through: Evan’s Story of PNH

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Meet Evan. In a recent conversation with the Aplastic Anemia and MDS International Foundation, he shared his journey from paroxysmal nocturnal hemoglobinuria (PNH) diagnosis through treatment and survival. Evan, please tell us a little about yourself. I’m 31 years old. I’m based…

Germline and somatic genetic landscape of pediatric myelodysplastic syndromes

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Abstract Pediatric myelodysplastic syndromes (MDS) represent a rare group of clonal hematopoietic stem cell disorders accounting for approximately 5% of pediatric hematologic malignancies. They are characterized by ineffective hematopoiesis, cytopenia, and dysplastic changes in…

A clinical guide to TP53 mutations in myeloid neoplasms

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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…

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