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TP53 mutations are common in relapsed/refractory (R/R) disease and confer a dismal prognosis in hematological malignancies. APR-246 is the first compound targeting mutant p53 to be used in a clinical trial. Eight patients with R/R non-M3 Acute Myeloid Leukemia (AML) were included in the trial. S. Deneberg, et al., published their results in Blood Cancer Journal in July 2016. The trial was conducted in accordance to the Declaration of Helsinki and ICH-GCP guidelines and was registered at ClinicalTrials.gov (NCT00900614).
These data provide a basis for the further exploration of the use of APR-246 in hematological indications, preferentially in combination with standard chemotherapy.
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