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A clinical guide to TP53 mutations in myeloid neoplasms

TP53 mutations are found in 10-15% of myeloid neoplasms and are one of its most important prognostic factors. Emerging data show that TP53 mutational allele status is a key determinant of clinical outcomes, with multi-hit TP53 mutant myeloid neoplasms having a very poor prognosis. Significant differences exist among the methods used in clinical and research settings to assess TP53 mutational status, leading to variability in reported patient characteristics, response to therapy, and survival.

Clinical Study on Targeted Drug Expands Treatment Options for Myelodysplastic Syndrome

Their research led to the 2022 approval of olutasidenib for certain patients with IDH1-mutant AML, which occurs in about 10% of AML. After that success, Dr. Watts and his team began to focus closely on testing the drug in MDS, a related condition that often progresses to AML.

IDH-1 mutations also occur in MDS, at a frequency of 3 to 5%.

Strong Responses
In the current study, the researchers tested olutasidenib in 22 MDS patients with IDH1-mutant tumors. All patients were classified with intermediate to high-risk disease, with 86% being high or very high risk.