Advances in Targeted and Immune-Based Therapies for Myeloid Neoplasms

Myeloid neoplasms, including acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), and myeloproliferative neoplasms (MPN), represent a diverse group of hematologic malignancies defined by complex molecular and clinical heterogeneity. Despite advances in supportive care, these diseases carry a poor prognosis, a high risk of relapse, and significant therapeutic challenges. Recent years have witnessed transformative changes in the landscape of myeloid neoplasm therapy, driven by breakthroughs in genomic profiling that have unraveled critical disease-driving mutations and pathways. The advent of targeted therapeutics, such as FLT3 and IDH1/2 inhibitors and anti-apoptotic pathway disruptors like BCL-2 and MCL-1 inhibitors, has begun to shift survival outcomes, particularly in AML. Parallel progress in immunotherapy, notably with checkpoint inhibitors, bispecific antibodies, antibody–drug conjugates, and cellular therapies, is broadening the armamentarium. Important questions remain regarding optimal combinations, mechanisms that underlie resistance, and the clinical translation of laboratory discoveries.

The aim of this Research Topic is to highlight and synthesize recent advances and future prospects in targeted and immune-based therapies for myeloid neoplasms. By drawing together clinical, translational, and mechanistic research, this collection seeks to illuminate how emerging therapeutic approaches, used alone or in combination, are reshaping the treatment of AML, MDS, and MPN. The focus is on showcasing new agents and regimens, evolving biomarkers for response and resistance, and strategies for integrating personalized treatment into standard-of-care algorithms. Special emphasis is placed on research that bridges laboratory findings with clinical impact, with the goal of improving survival, reducing relapse, and enhancing quality of life for patients with myeloid neoplasms.

This Research Topic is confined to novel targeted agents and immune-based interventions in the management of myeloid neoplasms, with particular attention to clinically relevant and translational studies in AML, MDS, and MPN. To gather further insights into this rapidly evolving field, we welcome articles addressing, but not limited to, the following themes:

• Clinical application and real-world outcomes of targeted therapies
• Development and evaluation of immune-based approaches (for example, checkpoint inhibitors, bispecific antibodies, and cellular therapies)
• Combination regimens that integrate targeted, epigenetic, or immunotherapeutic strategies
• Biomarkers that predict therapeutic response, resistance, or minimal residual disease
• Safety, tolerability, and quality-of-life considerations in emerging treatments
• Translational studies that refine patient selection and sequencing of therapies

We invite submission of original research, reviews, and mini-reviews related to the themes listed above.

Article types and fees

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Brief Research Report
• Case Report
• Clinical Trial
• Data Report
• Editorial
• FAIR² Data
• General Commentary
• Methods
• Mini Review
• ... View all formats

Articles that are accepted for publication by our external editors following rigorous peer review incur a publishing fee charged to Authors, institutions, or funders.

Article types

This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

• Brief Research Report
• Case Report
• Clinical Trial
• Data Report
• Editorial
• FAIR² Data
• General Commentary
• Methods
• Mini Review
• Opinion
• Original Research
• Perspective
• Review
• Study Protocol
• Systematic Review

Keywords: AML, MDS, MPN, targeted therapy, antibody-based therapies

Important note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.