2024
Phase 3 randomized double-blind study evaluating selinexor, an XPO1 inhibitor, plus ruxolitinib in JAKi-naïve myelofibrosis.
Mascarenhas J, Maher K, Rampal R, Bose P, Podoltsev N, Hong J, Wang X, Kye S, Harrison C. Phase 3 randomized double-blind study evaluating selinexor, an XPO1 inhibitor, plus ruxolitinib in JAKi-naïve myelofibrosis. Journal Of Clinical Oncology 2024, 42: tps6594-tps6594. DOI: 10.1200/jco.2024.42.16_suppl.tps6594.Peer-Reviewed Original ResearchXPO1 inhibitorsIntent-to-treat populationCo-primary study endpointsBaseline platelet countTreatment-related AEsPlacebo-controlled studyStem cell transplantationPhase 1 portionPhase 3 trialYears of ageNausea prophylaxisOral selinexorIntermediate-1Intermediate-2Anemia responseMF patientsTreatment discontinuationDouble-blindCell transplantationMyeloproliferative neoplasmsSecondary endpointsPlatelet countSpleen volumeRuxolitinib treatmentIWG-MRT
2019
Phase 1b Study of the Epichaperome Inhibitor PU-H71 Administered Orally with Ruxolitinib Continuation for the Treatment of Patients with Myelofibrosis
Pemmaraju N, Gundabolu K, Pettit K, Talpaz M, Podoltsev N, Schiller G, Eisenberg P, Youssoufian H, Duggan S, Wallner B, Verstovsek S. Phase 1b Study of the Epichaperome Inhibitor PU-H71 Administered Orally with Ruxolitinib Continuation for the Treatment of Patients with Myelofibrosis. Blood 2019, 134: 4178. DOI: 10.1182/blood-2019-130310.Peer-Reviewed Original ResearchPhase 1b studyJazz PharmaceuticalsExpansion cohortPU-H71Daiichi SankyoMyeloproliferative Neoplasm Symptom Assessment Form Total Symptom ScoreInternational Working Group-Myeloproliferative Neoplasms ResearchPeripheral blood mononuclear cellsAdvisory CommitteeDose-expansion cohortsHigh-risk myelofibrosisPhase 2 doseSerum cytokine profilesBaseline platelet countDose-ranging studySpeakers bureauVentricular ejection fractionAST/ALTDose-escalation designTotal symptom scoreBlood mononuclear cellsDisease-related symptomsTreatment of patientsBone marrow histologyPost-PV myelofibrosis