Figure 1.
Potential treatment algorithm for patients with blast-phase MPN.
In the absence of high-quality prospective studies specific to patients with blast-phase MPN, treatment recommendations are extrapolated from studies of patients with AML. At the time of diagnosis, molecular testing should be obtained. In patients who are potential candidates for allo-HCT, this constitutes the only potentially curative therapeutic modality and should therefore be considered. Induction chemotherapy prior to transplant is informed by molecular disease features, with the addition of midostaurin in patients with FLT3 mutations. Due to lower response rates with conventional cytotoxic chemotherapy in patients with TP53 mutations, combination therapy with HMAs plus venetoclax could be considered. Patients in remission after induction chemotherapy should proceed to allo-HCT if eligible. which may also be an option for a selected subset of patients who do not achieve a complete remission with induction therapy. For transplant-ineligible patients and those with R/R disease, clinical trial enrollment, HMAs alone or in combination, IDH1/2 inhibitors, or best supportive care are therapeutic options.