| Summary: | Background: Patients with the rare blood cancer myelofibrosis often have symptoms (eg, tiredness, weakness, shortness of breath) that have a negative impact on their quality of life and daily activities.1-3 Some of these symptoms may be due to anemia, which is common in myelofibrosis and is often treated with RBC transfusions.4 However, the time, costs, and potential medical complications associated with transfusions can further decrease quality of life for these patients.4-6 Objective: To characterize the relationship between transfusions and patient quality of life in myelofibrosis, we performed an analysis of patient-reported outcomes based on transfusions received in the phase 3 SIMPLIFY-1 and SIMPLIFY-2 clinical trials. Methods: The analysis included 432 patients treated with either the Janus kinase 1 and 2 (Jak1/Jak2)/activin A receptor type 1 inhibitor momelotinib or the Jak1/Jak2 inhibitor ruxolitinib in SIMPLIFY-1 (these patients had not been previously treated with a Jak inhibitor); as well as 156 patients treated with either momelotinib or the best available therapy (which was ruxolitinib in 88.5% of patients) in SIMPLIFY-2 (these patients had been previously treated with a Jak inhibitor) for 24 weeks.7,8 Patients were classified as transfusion independent (TI) if they had no transfusions and all blood hemoglobin levels were ≥8 g/dL in the previous 12 weeks; and transfusion dependent (TD) if they had ≥4 units transfused or a hemoglobin level <8 g/dL. Results: The results of the SF-36v2® Health Survey, a quality- of-life survey,9 at baseline were available for 503 of the 588 patients in SIMPLIFY-1 and SIMPLIFY-2 clinical trials. Compared with the general population average score of 50, average baseline scores were lower in all quality- of-life areas assessed (average score range, 39.0-46.3) in the SIMPLIFY-1 and SIMPLIFY-2 patients. Furthermore, at both baseline and week 24, average scores were lower for TD patients than for those who were TI. Among the 150 patients who were TD at baseline, 75 were still TD, 40 became TI, 21 became TR, and 14 did not have results available at week 24. The group who became TI at week 24 had greater improvement (positive average score change vs baseline score) in most quality-of-life areas assessed, compared with those who remained TD. Conclusions: In these myelofibrosis clinical trials, patient quality of life was worse on average compared with the general population. Patients who were TD had worse quality of life than those who were TI, highlighting the fact that transfusions further negatively impact quality of life for patients with myelofibrosis. Patients who were TD at baseline but became TI at week 24 had greater improvements in quality of life than those who remained TD, demonstrating the potential benefits of helping patients to need fewer transfusions by using other treatment options for anemia.
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