Journal
BEST PRACTICE & RESEARCH CLINICAL HAEMATOLOGY
Volume 33, Issue 4, Pages -Publisher
ELSEVIER SCI LTD
DOI: 10.1016/j.beha.2020.101222
Keywords
Acute myeloid leukaemia; Myelodysplastic syndrome; Checkpoint inhibitor; Clinical trial; Biomarkers
Categories
Funding
- NCI's Cancer Clinical Investigator Team Leadership Award (CCITLA)
- Dennis Cooper Hematology Young Investigator Award
- National Cancer Institute of the National Institutes of Health [P30 CA016359]
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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 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 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.
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