Experienced or Perceived Burdens and Associated Quality of Life Impacts of Anemia and Transfusion Dependence in Myelofibrosis: A Patient Self-Report Survey Analysis

Background: Myelofibrosis (MF) is associated with debilitating symptoms, which often negatively impact patients' health-related quality of life (QOL) and functioning. While Janus kinase (JAK) inhibitors are the standard of care in MF, some can exacerbate anemia, a hallmark feature of MF that increases in severity with disease progression. Anemia management in MF often requires red blood cell transfusions, which are an independent predictor of poor survival, inversely correlated with QOL, and may lead to iron overload.

Previously, qualitative concept elicitation interviews demonstrated substantial negative experiences with and perceptions of anemia and transfusions in 20 participants with MF who were transfusion dependent (TD) or transfusion independent (TI) (Cardellino A, et al. EHA 2024. Poster P1686). Here we expand on these findings by quantitatively evaluating the reported burden and associated impact of transfusion dependence on QOL and highlighting the importance of avoiding transfusion dependence in a larger population of participants with MF.

Methods: This was a multinational, cross-sectional, online survey study. The questions and response options were derived from a targeted literature review and qualitative concept elicitation interviews. Eligible participants had symptomatic MF and prior treatment with a JAK inhibitor. TD, TI, transfusion naive (TN), anemic, and nonanemic participants were included. Participants without anemia or transfusion dependence reported on their anticipated or perceived anemia symptoms and transfusion burdens. TD was defined as ≥2 transfusions (if MF diagnosed ≤3 months prior) or ≥1 transfusion every 3 months since diagnosis (if MF diagnosed >3 months prior), TN as never receiving a transfusion, and TI as not meeting criteria for TD or TN. Anemia was defined by TD status or if participants self-reported that their healthcare provider had diagnosed them as anemic.

Results: Between October 2023 and February 2024, 155 participants from the US (n=80), UK (n=18), Germany (n=18), Italy (n=18), Spain (n=11), and Poland (n=10) completed the online survey. The mean age was 59 years, and 56% were male; 49% were TD, 33% TI, 18% TN, 88% anemic, and 12% nonanemic. Most participants with or without an anemia diagnosis experienced anemia-related symptoms (eg, fatigue, weakness) and reported negative impacts on daily activities (73%), physical activities (56%), ability to think (54%), and emotional health (53%). Improving anemia as a treatment outcome was reported as “extremely” or “quite a bit” important in participants with (81%) and without (63%) anemia.

Regardless of transfusion status, approximately 60% reported frequent transfusions as being “extremely” or “quite a bit” bothersome, most commonly impacting daily activities (56%) and emotional health (52%). Most TD participants felt it was “extremely” important to reduce impacts to emotional health (56%), sleep (56%), health insurance (55%), and daily activities (51%).

Overall, 59% reported frequent transfusions to be “extremely” or “quite a bit” inconvenient. The most common inconveniences reported were time spent at the clinic (81%), travel time (40%), and scheduling issues (32%). In the TD group, 62% spent >3 hours at the transfusion clinic, and 78% spent >20 minutes traveling each way to the transfusion clinic. The risk of side effects and the need for frequent transfusions being an indicator of worsening or progression of MF were common concerns among all participants.

Participants placed a high level of importance on reducing the frequency of transfusions, with 80% stating that reducing transfusions to once every 16 weeks was “extremely” or “quite a bit” important. Positive impacts anticipated by TD participants if they experienced reduced transfusion frequency included improvement in daily activities (67%), emotional health (57%), social life (45%), and physical activities (45%). Among these positive impacts, daily activities (55%) and family responsibilities (33%) were ranked as most important. Overall, 74% rated never needing transfusions as “extremely” or “quite a bit” important (TD, 78%; TI, 67%; TN, 79%).

Conclusion: Participants with MF view the need for transfusions as a substantial burden to be avoided. They highly value treatments that can mitigate anemia symptoms as well as reduce the frequency of or avoid the need for transfusions.

LeBlanc:Genentech: Consultancy, Honoraria; Apellis: Consultancy; Astellas: Consultancy, Honoraria; Rigel: Consultancy, Honoraria, Speakers Bureau; AstraZeneca: Consultancy, Honoraria; Gilead: Consultancy; Incyte: Honoraria, Speakers Bureau; GSK: Consultancy, Honoraria, Research Funding; Jazz Pharmaceuticals: Research Funding; Novartis: Consultancy; BMS/Celgene: Consultancy, Honoraria, Research Funding, Speakers Bureau; Pfizer: Consultancy, Honoraria; Menarini/Stemline: Consultancy; Lilly: Consultancy, Honoraria; Agios/Servier: Consultancy, Honoraria, Speakers Bureau; AbbVie: Consultancy, Honoraria, Research Funding, Speakers Bureau; Dosentrx: Current holder of stock options in a privately-held company; ThymeCare: Current holder of stock options in a privately-held company. Collacott:Gsk plc: Consultancy. García Gutiérrez:CTA: Honoraria; BMS: Consultancy, Honoraria, Research Funding, Speakers Bureau; Novartis, Incyte: Speakers Bureau; Novartis, Incyte, GSK, Pfizer: Consultancy, Honoraria, Research Funding, Speakers Bureau; GSK: Consultancy; Novartis BMS Pfizer Incyte GSK: Consultancy. Weinshenker:GSK plc: Consultancy. Fein Revell:MPN Research Foundation, GSK plc: Consultancy; Patient Power.info; The Patient Story; MPN Research Foundation; MorphSYS Biopharma; GSK plc; cancer.net: Honoraria; GSK plc; MPN Research Foundation: Other: Travel support. O'Neill:GSK plc: Current Employment, Current equity holder in publicly-traded company. Cardellino:GSK plc: Current Employment, Current equity holder in publicly-traded company. Zhang:GSK: Current Employment, Current equity holder in publicly-traded company. Patnaik:GSK: Current Employment, Current equity holder in publicly-traded company, Other: support for attending meetings. Ross:GSK plc: Consultancy.