Emerging treatments

Tyrosine kinase inhibitor (TKI) plus interferon

A multicenter, randomized phase 3 trial to evaluate the efficacy and tolerability of nilotinib (a second-generation TKI) plus peginterferon combination therapy as first-line treatment for patients with chronic myeloid leukemia (CML) in chronic phase is ongoing.[93]​ Interim results suggest that the combination is associated with a higher rate of molecular responses, but impaired tolerability.[94]

Olverembatinib

An investigational third-generation TKI. Olverembatinib has been granted orphan-drug designation by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of CML in patients resistant to first- and second-generation TKIs, including those harboring the T315I mutation. In an open-label multicenter phase 1/2 trial, olverembatinib demonstrated significant activity in adults with TKI-resistant chronic-phase CML and accelerated-phase CML.[95]​ Phase 3 trials are recruiting.[96][97]

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