Access barriers and treatment patterns in Philadelphia-negative myeloproliferative neoplasms (Ph- MPN): Insights from a brazilian physician survey

Abstract Introduction: Effective management of Philadelphia-negative myeloproliferative neoplasms (Ph- MPNs)—including polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF)—requires accurate diagnosis and personalized therapy. Understanding real-world clinical practices is essential to identifying gaps in diagnostic and treatment access, particularly in resource-limited settings. Objectives: To conduct a national survey among Brazilian hematologists to assess current practices and potential challenges in the management of patients with Ph- MPNs. Methods: Between March and July 2025, a 31-question anonymous digital survey was distributed to hematologists across Brazil. The questionnaire assessed physician profiles, diagnostic procedures, and therapeutic decision-making regarding Ph-MPNs. Data were collected using the REDCap platform. Results: A total of 128 responses were received over the five-month period. Among respondents, 41% had more than 15 years of experience in hematology, 15% saw over 30 Ph-MPN patients per month, and 81% were from the Southeast region. Regarding practice setting, 21% worked exclusively in public institutions, 21% in private, and 58% in both. Most (69%) reported not having a dedicated Ph- MPN outpatient clinic, and 21% did not have access to a hematopathologist for bone marrow sample review. While nearly all had access to JAK2 mutation testing, 27% did not have access to CALR and MPL testing, and 47% had only in the private settings. Next-generation sequencing (NGS) was unavailable to 60% of respondents. Although karyotyping was widely available, nearly 30% reported not requesting it at diagnosis. Only 12.5% used the MPN-SAF symptom assessment form, and just 22% used the MYSEC score for secondary MF risk stratification. When asked about venous thromboembolism management in Ph- MPNs, responses varied significantly, with most preferring either indefinite anticoagulation (30%) or adapting based on risk stratification (30%). For MF treatment, JAK2 inhibitors were mostly available in private settings (67%). For high-risk MF patients, 79% of physicians considered allogeneic stem cell transplantation as a treatment option from the diagnosis. The most commonly reported challenges in Ph- MPN management were requesting molecular/genetic tests (82%) and accessing newer therapies (71%).Conclusions: This is the first Brazilian survey exploring the management of Ph- MPN patients among physicians. It highlights significant disparities in access to diagnostics and treatment, particularly within the public healthcare system. The data also reveal underutilization of symptom assessment tools and divergences in some clinical decision-making scenarios, such as anticoagulation management. Data is still being collected, with the expectation of more robust numbers for future analyses. This information could help us understand not only our practices as Brazilian physicians, as well as the possible gaps of knowledge and major access issues.