MPN clinical trials
17 MPN trials (Malignant Hematology) summarized for fellows and board review, newest first within each disease. Each summary covers design, results, toxicity and what changed in practice.
MF/ET (13)
- SENTRY trial JCO 2026In JAK inhibitor-na ve myelofibrosis, selinexor + ruxolitinib met the SVR35 co-primary endpoint (~50% vs 28%) but did NOT meet the symptom (AbsTSS) co-primary. An...
- TRANSFORM-1 trial Blood 2026Adding navitoclax to ruxolitinib doubled the rate of ≥35% spleen volume reduction versus ruxolitinib alone but did NOT improve symptom burden (TSS). The regimen...
- MANIFEST-2 Nat Med 2025Adding pelabresib (a BET inhibitor) to ruxolitinib significantly improved spleen volume reduction (SVR35 65.9% vs 35.2%) in first-line myelofibrosis - roughly...
- SURPASS-ET Lancet Haematol 2025Ropeginterferon alfa-2b significantly outperformed anagrelide as second-line therapy for high-risk, HU-intolerant/refractory ET with leukocytosis, with 43% achieving...
- MOMENTUM Lancet 2023Momelotinib significantly improved symptom burden, spleen volume, and transfusion independence compared with danazol in JAK-inhibitor-exposed, anemic MF patients. The...
- PERSIST-2 trial, Pacritinib in 2nd-L MF JAMA Oncol 2018Despite truncation by a clinical hold that compromised the week-24 analysis, PERSIST-2 demonstrated superior spleen and symptom benefit of pacritinib over best...
- JAKARTA-2 Lancet Haematol 2017Fedratinib 400 mg achieved a 55% spleen response rate in post-ruxolitinib patients, meeting its primary endpoint and demonstrating clinically meaningful activity in a...
- PERSIST-1 trial, Pacritinib in MF Lancet Haematol 2017Pacritinib produced a significant improvement in spleen volume reduction over best available therapy and may be a treatment option for myelofibrosis, including...
- SIMPLIFY-1 JCO 2017Momelotinib was noninferior to ruxolitinib for spleen volume response but failed to demonstrate noninferiority for symptom response, suggesting inferior symptomatic...
- JAKARTA JAMA Oncol 2015Fedratinib 400 mg significantly improved spleen volume and symptom burden in treatment-na ve intermediate/high-risk MF versus placebo, with results comparable to...
- COMFORT-I NEJM 2012Ruxolitinib produced highly significant spleen volume reduction and symptom improvement in intermediate-2/high-risk myelofibrosis, with a preliminary signal of...
- COMFORT-II NEJM 2012Ruxolitinib produced marked, durable reductions in splenomegaly versus BAT in intermediate-2/high-risk myelofibrosis, with improved quality of life and manageable...
- PT1 NEJM 2005Hydroxyurea + low-dose aspirin is superior to anagrelide + low-dose aspirin for high-risk ET, with significantly lower rates of arterial thrombosis, serious...
PV (4)
- MAJIC-PV JCO 2023MAJIC-PV demonstrated ruxolitinib's superiority over BAT for CR, duration of CR, and event-free survival in HC-INT/RES PV. Critically, it was the first trial to...
- PROUD-PV and CONTINUATION-PV Ropeg vs HU in PV Lancet Haematol 2020Ropeginterferon alfa-2b achieved durable hematologic and molecular responses and was non-inferior, then superior over time, to hydroxyurea, offering a safe long-term...
- RESPONSE-2 Lancet Oncol 2016Ruxolitinib achieved superior hematocrit control versus BAT in PV patients without splenomegaly who failed hydroxyurea, meeting the primary endpoint with a 3.3-fold...
- RESPONSE NEJM 2015Ruxolitinib was superior to standard therapy in PV patients with hydroxyurea failure or intolerance, achieving the composite primary endpoint of hematocrit control...