A Phase II Study of the Efficacy and Pharmacogenomics of Cladribine-Based Salvage Chemotherapy in Patients with Relapse/Refractory and Secondary Acute Myeloid Leukemia (AML) and High Risk Myelodysplastic Syndrome (MDS)

The optimal treatment regimen for relapsed/refractory AML is unknown. Although several chemotherapy options are available, there is no universally accepted regimen to date. One such regimen is Cladribine, Cytarabine, Mitoxantrone, and G-CSF (CLAG-M) that has been frequently used at our center. However, it is uncertain to predict which patients are likely to respond to CLAG-M or experience treatment related toxicities. In patients with newly diagnosed AML, studies have demonstrated that achievement of minimal residual disease (MRD) negative complete remission (no evidence of disease in your blood and/or bone marrow, using a sensitive test) is associated with a better overall survival. However, this has not been clearly studied in patients with relapsedrefractory AML. Through this study, we aim to demonstrate the influence of achieving minimal residual disease negative complete response on survival of patients with relapsed/refractory AML treated with CLAG-M. In addition to the conventionally used predictive factors, we aim to incorporate pharmacogenomics (how genes contribute to side effects experienced by patients and/or how well the AML responds to treatment) to assess the efficacy and toxicity of therapy.