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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.

Venetoclax-based salvage therapy as a bridge to transplant is feasible and effective in patients with relapsed/refractory AML

Originally Published: 01/17/2025
Key Points VEN plus HMA induces superior response rates in patients with R/R AML than conventional salvage therapy. The VEN plus HMA combination allows for a safe and effective bridging to allo-HCT. Abstract The B-cell lymphoma 2 inhibitor venetoclax (VEN) in combination with hypomethylating agents has been approved for first-line treatment of patients with acute myeloid leukemia (AML) ineligible for intensive treatment. VEN-containing treatment strategies may also be effective in relapsed/refractory (R/R) AML; however, comparative studies with conventional therapies for fit patients as a...

Drug in clinical trials for breast cancer could also treat some blood cancers

Originally Published: 01/16/2025
Two new studies led by researchers at Washington University School of Medicine in St. Louis have identified a possible way to block the progression of several forms of blood cancer using a drug already in clinical trials against breast cancer. The studies — both conducted in patient samples and animal models — found that inhibiting a protein called RSK1 reduces inflammation and stops the progression of blood cancers called myeloproliferative neoplasms (MPNs) as well as an aggressive form of acute myeloid leukemia (AML). With the RSK1 inhibitor already in clinical testing, the path to...

FDA Approves Axatilimab in 9 Mg and 22 Mg Vial Sizes for GVHD

Originally Published: 01/16/2025
Axatilimab-csfr (Niktimvo), a CSF-1R–targeting agent that reduces the drivers of inflammation and fibrosis, has been approved by the FDA in 9 mg and 22 mg vial sizes to treat adult and pediatric patients with graft-versus-host disease (GVHD), according to a news release from the drug’s developers, Incyte and Syndax Pharmacueticals.1 Furthermore, the developers expect that the product will be available for order in the United States in early February 2025. The FDA approved axatilimab for patients who weigh at least 40 kg with chronic GVHD after progression on at least 2 prior lines of...

How I Treat Higher-Risk MDS with Alain Mina, Rami S. Komrokji

Originally Published: 01/14/2025
Myelodysplastic syndromes/neoplasms (MDS) are a widely heterogenous group of myeloid malignancies characterized by morphologic dysplasia, a defective hematopoiesis, and recurrent genetic abnormalities. The original and revised International Prognostic Scoring Systems (IPSS) have been used to risk-stratify patients with MDS to guide treatment strategies. In higher-risk MDS, the therapeutic approach is geared toward delaying leukemic transformation and prolonging survival. For over a decade, the hypomethylating agents azacitidine and decitabine have been the standard of care and, when feasible...

Iron accelerates MDS progression

Originally Published: 01/09/2025
In this issue of Blood, Antypiuk et al, utilizing a murine model, demonstrate that iron overload (IOL), accumulated via a mutation in the iron importer ferroportin, accelerates the myelodysplastic syndrome (MDS) phenotype, leading to exacerbation of marrow failure, inferior leukemia-free survival, and inferior overall survival (OS).1 Conversely, blocking iron uptake with vamifeport (VIT) reverses these effects, resulting in improved erythropoiesis, superior leukemia-free survival, and superior OS (see figure). Finally, the addition of luspatercept to VIT had an additive beneficial effect on...

COMMANDS: Long-Term Analysis, Changes in RBC Transfusion Burden, Hemoglobin Levels and ELEMENTS Trial

Originally Published: 12/25/2024
Panelists discuss how long-term analysis of the ELEMENTS trial reveals significant improvements in red blood cell (RBC) transfusion burden and hemoglobin levels among study participants. The link goes to a video presentation.

Early Findings on Ziftomenib With Chemotherapy in AML From KOMET-007 Trial

Originally Published: 12/23/2024
Ziftomenib plus standard cytarabine and daunorubicin chemotherapy was well tolerated with consistent safety and continued to show “robust clinical activity” in patients newly diagnosed with NPM1-mutated (NPM1-m) or KMT2A-rearranged (KMT2A-r) acute myeloid leukemia (AML), according to an interim report from the ongoing KOMET-007 study. The phase Ia findings were presented by Amer Zeidan, MBBS, MHS, of Yale University, at the 66th American Society of Hematology Annual Meeting & Exposition in San Diego, California. The interim analysis covered 34 patients,15 with high-risk NPM1-m AML and 19...

State of the Art Treatment in Myelodysplastic Syndromes

Originally Published: 12/20/2024
Presenter: Mikkael SekeresProfessor of Medicine, Chief, Division of Hematology, Sylvester Comprehensive Cancer Center, University of Miami Miller School of MedicineMiami, FL, USA Release Date: December 20, 2024 Congress: ASH 2024 Area: Hematology Description: Mikkael A. Sekeres, MD, MS discusses the state of the art treatment in MDS at the 2024 ASH Annual Meeting. Video plays at link.

Allogeneic Hematopoietic Cell Transplantation for the Treatment of Severe Aplastic Anemia: Evidence-Based Guidelines From the American Society for Transplantation and Cellular Therapy

Originally Published: 12/04/2024
Allogeneic hematopoietic cell transplantation (HCT) is a potentially curative treatment for severe aplastic anemia (SAA). Existing guidance about HCT in SAA is primarily derived from expert reviews, registry data and societal guidelines; however, transplant-specific guidelines for SAA are lacking. A panel of SAA experts, both pediatric and adult transplant physicians, developed consensus recommendations using Grading of Recommendation, Assessment, Development, and Evaluation (GRADE) methodology employing a GRADE guideline development tool. The panel agrees with previous recommendations for...

Therapy for acute myeloid leukemia in older and unfit adults

Originally Published: 12/04/2024
TITLE Azacitidine and venetoclax in previously untreated acute myeloid leukemia. AUTHORS DiNardo CD, Jonas BA, Pullarkat V, et al. JOURNAL The New England Journal of Medicine. 2020;383:617-629. doi: 10.1056/NEJMoa2012971. The therapy of acute myeloid leukemia (AML) has experienced a renaissance in the past 5 years with the approval of nine new targeted agents and over 12 regimens.1 Historically, therapy for AML was the domain of healthy, young, ‘fit’ individuals.2 In 1973 the “7+3” regimen achieved an unprecedented 63% remission rate.2 However, this intense regimen confines patients to the...