Dr. Rampal on Challenges With Targeting High-Risk Mutations in Myelofibrosis


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Raajit K. Rampal, MD, PhD, discusses challenges with targeting high-risk mutations in patients with myelofibrosis.

Raajit K. Rampal, MD, PhD, a hematologic oncologist at Memorial Sloan Kettering Cancer Center, discusses challenges with targeting high-risk mutations in patients with myelofibrosis.

One challenge in the field of myelofibrosis is that oftentimes, drugs are not available for the targets identified, says Rampal. IDHis prevalent in patients who have more advanced disease, as well as those whose disease transforms to acute myeloid leukemia (AML) from myelofibrosis. Two inhibitors for IDH1/2 mutations have been approved by the FDA. One clinical trial is examining the combination of ruxolitinib (Jakafi) and enasidenib (Idhifa); that is an MPN Research Consortium trial, Rampal adds.

The field will have to wait and see what happens but there are already data showing that targeting IDH2 in patients who transformed to AML from myeloproliferative neoplasm can have efficacy. Effective drugs are not yet available for the other high-risk mutations, such as RAS, says Rampal. Splicing factor mutations are being investigated in clinical trials; targeting these mutations might be the next big focus with regard to research, as they appear to be associated with poor prognosis, concludes Rampal.

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