Myeloproliferative Neoplasms: Treatment

Myeloproliferative Neoplasms: overview

This algorithm covers treatment of the classic myeloproliferative neoplasms, branching first by diagnosis. In polycythemia vera, all patients receive phlebotomy to a hematocrit below 45% plus low-dose aspirin. High-risk patients add cytoreduction with hydroxyurea or ropeginterferon, and refractory disease is treated with ruxolitinib (RESPONSE). In the site's RESPONSE summary, ruxolitinib was superior to standard therapy after hydroxyurea failure or intolerance, achieving the composite endpoint of hematocrit control and spleen reduction in 21% vs 1%, with fewer thromboembolic events. In essential thrombocythemia, low-risk patients receive low-dose aspirin with observation, and high-risk patients receive hydroxyurea for cytoreduction.

For myelofibrosis, the chart lists JAK inhibition with ruxolitinib or fedratinib (COMFORT), pacritinib for thrombocytopenic patients (PERSIST-2), and momelotinib for anemic patients (MOMENTUM), which improved symptoms, spleen volume and transfusion independence versus danazol in JAK inhibitor-exposed anemic patients. Allogeneic transplant is labeled as the only curative option.

Trials behind this algorithm

• PROUD-PV and CONTINUATION-PV Ropeg vs HU in PV Gisslinger H et al, Lancet Haematol, 2020, PMID: 32014125
• RESPONSE Vannucchi AM et al, NEJM, 2015; PMID: 25629741
• PT1 Harrison CN et al, NEJM, 2005; PMID: 16000354
• COMFORT-I Verstovsek S et al, NEJM, 2012; PMID: 22375971
• PERSIST-2 trial, Pacritinib in 2nd-L MF Mascarenhas J et al, JAMA Oncol, 2018, PMID:29522138
• MOMENTUM Verstovsek S et al, Lancet, 2023; PMID: 36709073

Background reading: MPN and MDS summary board review notes.