Blood Cancer — Myeloproliferative (MPN) Clinical Trials

About Blood Cancer — Myeloproliferative (MPN) trials

MPN trial activity centers on the three classic Philadelphia-negative myeloproliferative neoplasms (polycythemia vera, essential thrombocythemia, and primary myelofibrosis), the driver mutation class, and the presence of high-molecular-risk (HMR) features. JAK2 mutations (V617F most common) are present in roughly 95% of polycythemia vera and 50-60% of essential thrombocythemia and myelofibrosis. CALR mutations occur in another 20-25% of ET and MF, and MPL mutations in about 5-10%, defining the three driver classes. Triple-negative MPN (no JAK2, CALR, or MPL mutation) carries worse prognosis. ASXL1 is the most common HMR mutation in MF and gates trials testing HMR-specific strategies. IDH1 and IDH2 mutations are relevant in advanced MF and especially in post-MPN AML transformation, where they overlap with AML mutation-targeted trials. TP53 mutations carry adverse prognosis and often associate with leukemic transformation, defining a high-priority subgroup for combination trials.

Open MPN trials sort by type, line of therapy, and risk category. First-line myelofibrosis trials test new combinations on the JAK inhibitor backbone (ruxolitinib + a novel agent like a BET inhibitor, BCL-XL inhibitor, MDM2 inhibitor, or PIM kinase inhibitor) for symptomatic MF. JAKi-resistant or JAKi-intolerant trials test alternative JAK inhibitors and novel mechanisms for patients who progressed on or didn't tolerate first-line ruxolitinib or fedratinib. Anemia-focused trials enroll MF patients with significant anemia (using momelotinib or novel agents). Pre-allogeneic transplant trials test cytoreduction strategies for fit MF patients heading to transplant (the only curative option). Post-MPN AML trials enroll patients whose MPN transformed to acute leukemia, often combining HMA-based regimens with IDH-targeted or other novel agents. Polycythemia vera and essential thrombocythemia trials are smaller in number and focus on interferon-based strategies (especially ropeginterferon comparisons), novel cytoreductive agents, and prevention of transformation.

Most MPN trials screen on subtype, prior JAKi exposure, and risk category. MPN subtype confirmation (PV, ET, primary MF, post-PV MF, post-ET MF, blast-phase MPN) determines which trials apply. Driver mutation status (JAK2 vs CALR vs MPL vs triple-negative) gates some trials. Risk category by DIPSS, DIPSS-plus, MIPSS70, or MIPSS70-plus appears in many MF trial inclusion criteria. Prior ruxolitinib exposure and discontinuation reason (resistance, intolerance, suboptimal response) define which JAKi-treated trials apply. Prior fedratinib, pacritinib, or momelotinib exposure matters for second-line JAKi trials. Spleen size measured by palpation or imaging and symptom burden by Total Symptom Score (TSS) or MPN-SAF gate many MF trials. Anemia and transfusion dependency status. Allogeneic transplant eligibility for transplant-related trials. Performance status, with most trials accepting ECOG 0-2 because MPN patients are often older.

Biomarkers tested in Blood Cancer — Myeloproliferative (MPN) trials

These are the molecular markers most commonly required or evaluated in Blood Cancer — Myeloproliferative (MPN) eligibility criteria. OncoMatch extracts them from each trial's protocol and matches them against your test results.