Topotecan Hydrochloride and Carboplatin With or Without Veliparib in Treating Advanced Myeloproliferative Disorders and Acute Myeloid Leukemia or Chronic Myelomonocytic Leukemia
Key claim: A phase II trial is evaluating whether adding the PARP inhibitor veliparib to topotecan hydrochloride and carboplatin improves outcomes in patients with advanced myeloproliferative disorders, AML, or CMML compared to chemotherapy alone.
Abstract
This phase II trial studies how well topotecan hydrochloride and carboplatin with or without veliparib work in treating patients with myeloproliferative disorders that have spread to other places in the body and usually cannot be cured or controlled with treatment (advanced), and acute myeloid leukemia or chronic myelomonocytic leukemia. Drugs used in chemotherapy, such as topotecan hydrochloride and carboplatin, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Veliparib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving topotecan hydrochloride, carboplatin, and veliparib may work better in treating patients with myeloproliferative disorders and acute myeloid leukemia or chronic myelomonocytic leukemia compared to topotecan hydrochloride and carboplatin alone.
Why this matters
Advanced/blast-phase MPNs and CMML have dismal outcomes with no standard therapy, and chemotherapy backbones are typically borrowed from AML paradigms. This trial introduces PARP inhibition (veliparib) layered onto topotecan/carboplatin as a mechanistically novel approach exploiting DNA-damage repair dependencies, and its cross-subtype enrollment (advanced MPNs, AML, CMML) reflects the convergent biology of transformed myeloid disease.