In this open-label single arm phase 2 study, approximately 20 patients with MF demonstrating suboptimal response to ruxolitinib monotherapy will be enrolled. Patients will continue to receive ruxolitinib at a stable dose and ropeginterferon alfa 2b will be added to the regimen.
Willing and able to provide informed consent
Age ≥18 years
Diagnosis of Overt Myelofibrosis (primary, post-ET, or post-PV) per World Health Organization (WHO) 2022 diagnostic criteria
Intermediate-1, Intermediate-2, or high-risk disease by Dynamic International Prognostic Scoring System (DIPSS)
Prior or current use of interferon alfa (IFNα) preparations for MPN
Patients currently on other investigational therapy (ies)
Contraindications or hypersensitivity to IFNα preparations
History of organ and haematopoietic stem cell transplantation
Single group
Ropeginterferon alfa-2b (BESREMi®)
DrugRopeginterferon alfa 2b is administered subcutaneously once every 2 weeks. The dosing will be 250mcg at Week 0, 350mcg at Week 2, 500mcg at Week 4, and 500mcg every 2 weeks thereafter
Adverse events
Enumeration and description of adverse events (AEs), including determination of dose limiting toxicities (DLTs), serious adverse events (SAEs), and other AEs
Spleen volume change at 24 weeks
Proportion of patients who experience a spleen length reduction by palpation of ≥30% OR spleen volume reduction (SVR) of ≥35% by MRI or CT by 24 weeks of treatment
Symptom response at 24 weeks
Proportion of patients who describe a ≥50% reduction in symptom burden by the myelofibrosis symptom assessment form (MFSAF) by 24 weeks of treatment. The MFSAF has a scale of 0-70 where a higher score indicates worse outcome.
Rate of molecular responses
Changes in allelic burden of JAK2V617F, MPL, CALR or other driver mutations will be assessed. A molecular responses is assessed using the International Working Group of Myeloproliferative Neoplasm Research and Treatment Criteria.
Rate of morphologic response
Changes in bone marrow morphology (cellularity, erythroid, myeloid, megakaryocyte, reticulin and fibrosis) during treatment. Response is assessed using the International Working Group of Myeloproliferative Neoplasm Research and Treatment Criteria.
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