Myelodysplastic Syndrome

The most used agents include hydroxyurea, interferon, hypomethylating agents, and JAK inhibitors, although none of them are disease-modifying. Allogeneic hematopoietic stem cell transplant remains the only potentially curative approach and should be considered in all eligible patients. Actionable mutations (CSF3R, NRAS/KRAS, and KIT) have also been identified, supporting the development of new agents targeting the involved pathways.

Despite these gains, the absolute survival benefit remains modest, prolonged cytopenias are universal, and outcomes in TP53-mutated or younger adverse-risk patients are still poor, raising legitimate questions about cost-effectiveness and generalizability. Nonetheless, CPX-351 stands as the first clinically validated example of ratiometric nanomedicine in oncology, proving that reformulating established drugs can yield meaningful progress where novel agents have often failed.

P1/2, N=36, Active, not recruiting, Groupe Francophone des Myelodysplasies | Recruiting --> Active, not recruiting

P2, N=70, Recruiting, H. Lee Moffitt Cancer Center and Research Institute | Suspended --> Recruiting

P2, N=57, Completed, Sidney Kimmel Cancer Center at Thomas Jefferson University | Active, not recruiting --> Completed

P1, N=34, Active, not recruiting, City of Hope Medical Center | Trial completion date: Aug 2025 --> Jul 2026

This study suggests that ddPCR may be a transformative tool for MRD monitoring with the superior sensitivity and longer lead time for predicting relapse than conventional methods in AML/MDS patients after allo-HSCT. Its outstanding performance supports its integration into routine post-transplant monitoring to enable early intervention and improve outcomes.

P2, N=57, Recruiting, Groupe Francophone des Myelodysplasies | Not yet recruiting --> Recruiting

We propose that such a low-intensity regimen may be suitable for older patients with AML, who are unfit for intensive chemotherapy. We also present data indicating that PRI5202 induces myeloid differentiation in blasts from patients with myelodysplastic syndrome (MDS), and we propose to further investigate PRI5202 as a differentiation therapy for patients suffering from MDS.

dexamethasone, ara-C, cisplatin (DHAP) was the most common salvage regimen (47.8%)...BCNU, etoposide, ara-C, melphalan (BEAM) was the most common conditioning regimen 91.9%...t-MN significantly contributes to NRM post-auto-HCT. Age at transplant is the primary risk factor, highlighting the need for vigilant monitoring and risk mitigation strategies.

P2, N=70, Suspended, St. Jude Children's Research Hospital | Recruiting --> Suspended

We uncovered 31 potential key genes showing differential early responses to DAC treatment in TP53-mutant versus wild-type cells, which may be associated with resistance development. This study revealed the potential molecular mechanisms of TP53 gene locus mutation in DAC-treated MDS.