Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia
Key claim: This randomized phase II/III trial evaluates whether adding lenalidomide or vorinostat to azacitidine improves outcomes compared to azacitidine alone in patients with higher-risk myelodysplastic syndromes or chronic myelomonocytic leukemia.
Abstract
This randomized phase II/III trial studies how well azacitidine works with or without lenalidomide or vorinostat in treating patients with higher-risk myelodysplastic syndromes or chronic myelomonocytic leukemia. Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells, stopping them from dividing, or by stopping them from spreading. Lenalidomide may stop the growth of cancer cells by stopping blood flow to the cancer. Vorinostat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. It is not yet known whether azacitidine is more effective with or without lenalidomide or vorinostat in treating myelodysplastic syndromes or chronic myelomonocytic leukemia.
Why this matters
Most prospective CMML evidence to date comes from early-phase, single-arm trials of novel agents; a randomized phase II/III comparison of azacitidine-based combinations is rare and directly informs whether established add-ons (lenalidomide, vorinostat) shift outcomes versus HMA monotherapy in this subtype. Results will help calibrate the comparative benchmark against which the many emerging CMML-specific HMA combinations (venetoclax, ERK-, LSD1-, CSF-1R-, PLK1-directed) are evaluated.