This research is being done to see if the drug ruxolitinib is effective in reducing the symptoms caused by low-risk essential thrombocythemia (ET) and polycythemia vera (PV). - This research study involves the study drug Ruxolitinib.
Name: Ruxolitinib
Description: Percentage of patients with >50% change in Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score (MPN-SAF TSS), which has use in major clinical trials and as symptom assessment in all patients in clinical practice. Modeling has established baseline MPN-SAF TSS cut-off scores (total MPN-SAF TSS >20 or an individual component score >5) at which symptomatic treatment would be significantly beneficial
Measure: Percentage of patients who achieve >50% reduction from baseline to Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score Time: baseline to 12 weeksDescription: Evaluate the efficacy of ruxolitinib in improving hematologic remission rates, as measured by International Working Group (IWG) working criteria ET: Platelet count ≤ 400 x 109/L, WBC count <10 x 109/L, and absence of leukoerythroblastosis PV: hematocrit <45% and WBC count <10 x 109/L
Measure: Proportion of patients achieving complete hematologic rate at week 12 Time: Week 12Description: Evaluate the efficacy of ruxolitinib in improving hematologic remission rates, as measured by International Working Group (IWG) working criteria ET: Platelet count ≤ 400 x 109/L, WBC count <10 x 109/L, and absence of leukoerythroblastosis PV: hematocrit <45% and WBC count <10 x 109/L
Measure: Proportion of patients achieving complete hematologic rate at 24 Weeks Time: Week 24Description: Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score (MPN-SAF TSS) for use in major clinical trials and as symptom assessment in all patients in clinical practice. Modeling has established baseline MPN-SAF TSS cut-off scores (total MPN-SAF TSS >20 or an individual component score >5) at which symptomatic treatment would be significantly beneficial. Scale title includes absence of symptom (0) and worst imaginable symptoms (10) per question, with minimum score 0 and maximum score 100. Higher scores indicate worse outcomes.
Measure: Best MPN-SAF TSS score Time: 12 WeeksDescription: Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score (MPN-SAF TSS) for use in major clinical trials and as symptom assessment in all patients in clinical practice. Modeling has established baseline MPN-SAF TSS cut-off scores (total MPN-SAF TSS >20 or an individual component score >5) at which symptomatic treatment would be significantly beneficial. . Scale title includes absence of symptom (0) and worst imaginable symptoms (10) per question, with minimum score 0 and maximum score 100. Higher scores indicate worse outcomes.
Measure: Best MPN-SAF TSS score Time: 24 WeeksDescription: Spleen volume reduction will be measured as the change in percentage spleen volume from baseline at 12 weeks as measured by CT, or MRI in patients with medical contraindication to CT. Summary statistics will be used to describe changes in spleen volume. Volume will be calculated by a computer-assisted perimeter method based on Sorenson et al. Spleen and liver volume will be obtained by outlining the circumference of the organ and determining the volume using the validated technique of least squares
Measure: Percentage of change in Spleen Volume Time: baseline to 12 weeksDescription: descriptions and grading scales found in the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 will be utilized for AE reporting
Measure: Number of Participants with Treatment Related Adverse Events as Assessed by CTCAE v 5.0 Time: All patients who initiate treatment with study drug up to 60 monthsAllocation: Non-Randomized
Sequential Assignment
There is one SNP
Exclusion Criteria: - Essential thrombocythemia patients who are high risk by IPSET-R criteria (age > 60 with JAK2 V617F mutation and/or history of thrombosis).1 Polycythemia vera patients who are high risk by NCCN guidelines (age > 60 and/or history of thrombosis). --- V617F ---