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News and Treatment Updates

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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.

Germline genetic variation impacts clonal hematopoiesis landscape and progression to malignancy

Originally Published: 07/15/2025
Abstract With age, clonal expansions occur pervasively across normal tissues yet only in rare instances lead to cancer, despite being driven by well-established cancer drivers. Characterization of the factors that influence clonal progression is needed to inform interventional approaches. Germline genetic variation influences cancer risk and shapes tumor mutational profile, but its influence on the mutational landscape of normal tissues is not well known. Here we studied the impact of germline genetic variation on clonal hematopoiesis (CH) in 731,835 individuals. We identified 22 new CH-...

Hematopoietic stem cell transplantation is effective in achieving long-term survival for post-aplastic anemia myeloid neoplasms: the EBMT Severe Aplastic Anemia Report

Originally Published: 07/03/2025
Abstract Aplastic anemia (AA) transformation into myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) is associated with a dismal prognosis. Hematopoietic stem cell transplant offers the sole possibility of cure, but data on long-term survival are scarce. We retrospectively analyzed 270 patients transplanted for MDS, AML, or an isolated cytogenetic abnormality after a diagnosis of AA or paroxysmal nocturnal hemoglobinuria reported to the EBMT. The median age at transplantation was 39 years. The 5-year overall survival (OS) rate was 64%, unaffected by chromosome 7 abnormalities,...

SRSF2-mutated MDS: bortezomib STAT?

Originally Published: 07/03/2025
In this issue of Blood, Takashima et al show that reduced STAT1 abundance in SRSF2-mutant myelodysplastic syndrome (MDS) cells confers protection against interferon (IFN)-driven cell suppression relative to normal wild-type (WT) cells.1 Importantly, the authors demonstrate that treatment with the proteasome inhibitor bortezomib in vitro increases STAT1 abundance and sensitizes SRSF2-mutant cells to IFN. These findings provide potential rationale for using bortezomib therapy in SRSF2-mutated MDS, which is characterized by poor outcomes. It has been several years since the initial...

Study supports frontline use of imetelstat for low-risk MDS patients ineligible for ESAs

Originally Published: 06/30/2025
A post-hoc analysis of pooled data from the IMerge trial found that patients with low-risk myelodysplastic syndromes (MDS) who were ineligible for erythropoiesis-stimulating agents (ESAs) due to elevated baseline serum erythropoietin derived clinical benefit from frontline treatment with imetelstat. Rami Komrokji, MD, of the H. Lee Moffitt Cancer Center in Tampa, Florida, presented the results at the 2025 EHA Congress. Researchers used data from the phase 2, phase 3, and QTc substudy results from IMerge, which treated adults with imetelstat 7.1 mg/kg or placebo, both administered as a two-...

BEXMAB Study: Dr. Amer Zeidan Remarks on the Results’ Meaning for MDS Care

Originally Published: 06/27/2025
Amer Zeidan, MBBS, MHS, is professor of internal medicine at Yale School of Medicine in New Haven, Connecticut. He is also the chief of the Division of Hematologic Malignancies at Yale Cancer Center, New Haven. Dr. Zeidan spoke with Blood Cancers Today about the phase 1/2 BEXMAB study, which was presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, recently held in Chicago, Illinois, and will be presented at the European Hematology Association (EHA) 2025 Congress in Milan, Italy. The BEXMAB study evaluates the combination of bexmarilimab, a macrophage Clever-1...

Treatment patterns and blood count control in 10,112 patients with polycythemia vera

Originally Published: 06/26/2025
ABSTRACT Background Elevated blood counts in polycythemia vera (PV) are associated with increased thrombotic risk, which contributes to morbidity and mortality. Research design and methods This retrospective study describes treatment patterns and blood count control in patients with PV managed at community oncology practices (January 2014–February 2023; Integra Precision Q database). Results Of a total 10,112 patients, most received phlebotomy (68.1%) or hydroxyurea (HU; 28.2%) as initial treatment, with median follow-up of 32.1 (IQR, 13.5–58.5) months and 31.5 (IQR, 16.8–54.9) months,...

Integrated Immune Landscape Analysis of RNA Splicing Factor-Mutant AML and Higher risk MDS Treated with Azacitidine ± Durvalumab

Originally Published: 06/21/2025
Abstract Background: RNA splicing factor (SF) mutations are associated with adverse outcomes in patients with acute myeloid leukemia (AML) and higher-risk myelodysplastic syndromes/neoplasms (HR-MDS). Preclinical data suggest that aberrant RNA splicing can lead to the generation of neoantigens, which renders these tumors more susceptible to immune checkpoint inhibitors. However, dedicated studies on immune checkpoint inhibitors in AML and MDS patients with SF mutations are limited. Objectives: To characterize the immune and epigenetic landscape of AML and MDS patients with SF mutations....

Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis After Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation

Originally Published: 06/17/2025
Abstract Purpose Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment for advanced hematologic malignancies. HSCT using human leukocyte antigen (HLA)-mismatched donors is historically associated with inferior survival. Patients from underrepresented racial and ethnic groups more frequently rely on HLA-mismatched donors. We hypothesized that post-transplant cyclophosphamide (PTCy) based graft versus host disease (GVHD) prophylaxis would improve outcomes for HSCT recipients using peripheral blood stem cells (PBSCs) from HLA-mismatched unrelated donors (MMUDs) by...

Cyclosporin/Cyclophosphamide Is a Potential New SOC in GVHD Management

Originally Published: 06/13/2025
Cyclophosphamide plus cyclosporin, a calcineurin inhibitor, significantly improved graft-vs-host disease (GVHD)–free relapse-free survival (RFS) compared with standard prophylaxis in patients with aggressive blood cancers who are undergoing stem cell transplant from a matched related blood stem cell donor with reduced-intensity or myeloablative conditioning, according to results from the phase 3 BM12 CAST trial (ACTRN12618000505202) presented during the European Hematology Association 2025 Congress.1 Findings, which were initially presented in a press briefing during the meeting and...

Iptacopan Yields Meaningful Hemoglobin Improvements in Pretreated PNH Group

Originally Published: 06/13/2025
Data from APPULSE-PNH may support oral iptacopan as a potentially practice-changing option in patients with paroxysmal nocturnal hemoglobinuria. "Results from APPULSE-PNH and previous trials establish oral iptacopan monotherapy as a potentially practice-changing treatment capable of inducing improvements in Hb to normal or near normal levels by providing comprehensive hemolysis control,” according to study author Austin Kulasekararaj, MBBS, MD, MRCP, FRCPath. "Results from APPULSE-PNH and previous trials establish oral iptacopan monotherapy as a potentially practice-changing treatment...