Perceived and experienced impacts of anemia and transfusion dependence in myelofibrosis: a mixed-methods study

Abstract Background Myelofibrosis (MF) is a rare myeloproliferative neoplasm characterized by bone marrow fibrosis, anemia, splenomegaly, and debilitating symptoms. Anemia, present in approximately 35% of patients at diagnosis, often worsens over time and is associated with poor quality of life and worsened survival outcomes. Red blood cell transfusions help to manage anemia but are burdensome and may lead to iron overload. Existing literature focuses on clinical outcomes, and little is known about patient-perceived and -experienced impacts of transfusion and anemia. Methods This mixed-methods study across 6 countries included qualitative interviews and an online survey of symptomatic (Myelofibrosis Symptom Assessment Form v4.0 Total Symptom Score ≥ 10) adults with MF with prior Janus kinase inhibitor experience, including both transfusion-dependent (TD) and transfusion-independent (TI) patients. Twenty participants (5 TD, 15 TI) completed interviews to inform an online survey fielded among 155 (76 TD, 51 TI, 28 transfusion-naive) participants. Questions assessed perceived and experienced burdens and impacts of anemia and transfusions. Results Concept elicitation interviews ( N = 20) identified safety (85%), convenience (85%), and travel requirements (60%) as top concerns associated with transfusions; 40% noted symptom relief from transfusions, and 90% valued treatments to control anemia and reduce transfusion need. Across online survey participants ( N = 155), fatigue (85.2%), weakness (72.9%), and headaches (47.7%) were the most frequently reported symptoms, with negative impacts to daily activities (73%), physical activities (56%), ability to think (54%), and emotional health (53%). Transfusion-related inconveniences included time spent at the clinic (81%), travel time (40%), and scheduling issues (32%). The most burdensome impacts of transfusions were to daily activities (55.5%), emotional health (52.3%), and family responsibilities (35.5%). Among TD participants, 52.2% rated reducing their needed transfusion frequency as “extremely important,” although some acknowledged benefits associated with symptom relief. Conclusions This study underscores the significant symptom burden of anemia and the broad impacts of transfusion dependence in MF. Qualitative and quantitative findings reveal emotional, physical, and logistical challenges associated with transfusions and patients’ strong desire to minimize their frequency. Results support the development of patient-centered therapies that improve anemia and reduce transfusions, addressing key medical needs in MF management.