Randomized trials with checkpoint inhibitors in acute myeloid leukaemia and myelodysplastic syndromes: What have we learned so far and where are we heading?

The treatment of acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS) has seen dramatic advances with the approval of multiple novel agents in recent years. However, unlike solid malignancies, immune checkpoint inhibitors have yet to garner regulatory approval in AML and MDS with recent randomized clinical trials clinical trials: Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study. Understanding Clinical Trials Clinical… yielding only underwhelming results. Novel targets have been explored in early phase clinical trials with impressive results leading to ongoing subsequent controlled trials. However, major challenges in the field remain such as the validation of predictive genetic, molecular, and immunophenotypic biomarkers, optimization of clinical trial clinical trial: A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a… design, and the identification of novel synergistic combination therapies. Herein, we review recent clinical trial data focusing on randomized clinical trials and highlight limitations of the currently available evidence in an effort to suggest options for advancing the field.