Global assessment of patient and caregiver unmet needs in myeloproliferative neoplasms (MPNs): Findings from the 2024 international assessment

Abstract Background Despite therapeutic advances, patients with myeloproliferative neoplasms (MPNs) continue to face significant unmet needs, particularly in clinical trial awareness, symptom monitoring, and alignment of care with personal treatment goals. To better understand these challenges, MPN Research Foundation conducted a global Unmet Needs Assessment in late 2024 to capture the perspectives of patients and caregivers. The insights gathered through this effort help elevate patient voices and inform future research and advocacy. Methods From October 14 to November 11, 2024, 676 individuals from 31 countries responded to a structured online survey, with 627 providing complete responses. Participants included patients (87%), caregivers (6%), and advocacy representatives, researchers, and healthcare professionals (7%). The survey explored diagnostic experiences, treatment access, symptom burden, clinical trial awareness, and treatment priorities. Respondents were drawn from the MPN Research Foundation's global network. Results Nearly half of respondents (47%) received care at academic medical centers, while others were treated at community hospitals or a mix of both, reflecting varied care settings across the MPN community. Only 10% of respondents reported participating in a clinical trial, and 71% said their healthcare provider had never discussed trial options with them. Among those who did enroll, just over half (52%) learned about the opportunity from their provider, while others discovered trials through advocacy organizations (25%) or online platforms such as ClinicalTrials.gov (11%). These findings suggest a communication gap that may limit patient access to research opportunities. Fatigue (71%), pruritus (55%), and cognitive difficulties (42%) were among the most frequently reported symptoms. However, awareness and use of the MPN 10 symptom tracking tool remained low: 72% had never heard of it, 85% had never used it, and 87% reported that their provider had not introduced it. This indicates a gap in symptom monitoring that could be addressed through improved provider engagement and patient education. Participant responses revealed clear treatment priorities. The most frequently reported goals were slowing disease progression (80%), normalizing blood counts (54%), improving quality of life (52%), and increasing energy (48%). Yet only 54% of patients indicated that their individual treatment goals were considered in clinical decision-making, suggesting a potential misalignment between patient priorities and care planning. Conclusions The 2024 MPN Unmet Needs Assessment highlights persistent gaps in patient-centered care, particularly in the areas of trial communication, symptom tracking, and goal-aligned treatment. Participant responses suggest that key priorities include slowing disease progression and improving quality of life, but these goals are not always reflected in care discussions. Based on these findings, greater emphasis on proactive provider engagement, increased use of tools like the MPN 10, and incorporation of shared decision-making practices may help better align care with the priorities reported by patients and caregivers. These insights can guide future research, education, and improvements in care delivery across the MPN landscape. To build on this work, the MPN Research Foundation plans to conduct a follow-up Unmet Needs Assessment in 2026. Collecting this information on a serial basis will allow us to track progress over time, identify emerging challenges, and evaluate the impact of interventions aimed at improving patient outcomes. Study Sponsor Statement: This assessment was conducted with support from the following sponsors: Sobi, MorphoSys, Incyte, Karyopharm Therapeutics, PharmaEssentia, Bristol Myers Squibb, and Sumitomo Pharma.