Advancing the Treatment Landscape for Patients With Myeloproliferative Neoplasms

Despite recent advancements in treatments for patients with myeloproliferative neoplasms, a term encompassing several rare cancers, many challenges persist when it comes to these hematologic malignancies, primarily due to lack of uniform treatment plans for each of the classical subgroups.

These findings were presented in a landscape analysis by the Association of Community Cancer Centers (ACCC) at the 2023 National Oncology Conference from October 4-6, 2023, in Austin, Texas.

In 2008, the World Health Organization (WHO) reclassified myeloproliferative disease from a blood disorder to clonal hematopoietic stem cell malignancies, and in 2016, the WHO identified 4 classic types of myeloproliferative neoplasms: chronic myeloid leukemia (BCR-ABL1 positive), polycythemia vera (BCR-ABL1 negative), essential thrombocythemia (BCR-ABL1 negative), and primary myelofibrosis (ABCR-ABL1 negative).

Patients with myeloproliferative neoplasms report debilitating symptoms, including fevers, night sweats, fatigue, sleep disturbances, weight loss, bone pain, itchy skin, headaches, difficulty concentrating, anxiety, and depressive symptoms.

Although these patients are considered to have favorable life expectancy, with 60% of patients living up to 15 years after diagnosis, about 84% of these patients have reported a reduced quality of life directly due to these symptoms. Moreover, many low-risk patients are given a “watch-and-wait” treatment plan and receive no drug therapy despite experiencing a moderate to high symptom burden.

Gaps in care coordination also exist, and current standard-of-care pharmacological treatments are unable to fully relieve symptom burden. For example, approximately 84% of patients with myeloproliferative neoplasms reported reduced quality of life due to the effects of their current pharmacological interventions.

Moreover, patient and physicians perceptions regarding treatment are often misaligned, with patients reporting that their most important treatment goal was to slow or delay disease progression, whereas physicians reported the most important goal was symptom improvement and prevention of vascular or thrombotic events. Furthermore, an average of 30% of patients with myeloproliferative neoplasms did not believe their physician had a treatment plan, and an average of 35% of patients believed their physician was not providing updates on new treatments.

The researchers also identified health literacy as an important factor in understanding and managing symptom burdens, shared decision-making, and proper disease management. Additionally, a 2016 online survey of 904 adults found significant employment disruptions were common, with at least 1 employment change reported among 65.5% of patients with myelofibrosis, 48% of patients with polycythemia vera, and 38.8% of patients with essential thrombocythemia. Furthermore, respondents reported reduced working hours, medical disability leave, early retirement, dropping to part-time hours, and switching to a lower-paying job due to their disease.

For these reasons, myeloproliferative neoplasms have a significant disease burden and impact on patients, commonly due to anxieties about disease advancement and disruptive symptoms that affect a person’s quality of life.

Furthermore, lack of uniform treatment, risk diagnostic assessments, and care coordination can result in late diagnosis and rapid potential disease progression, which may also lead to a lack of transparency and patients feeling excluded in decision-making of their care.

Moreover, a lack of awareness surrounding available care guidelines, standardized symptom alleviation, and disease progression control plans are stressors that add to this disease burden, so further knowledge on the management of myeloproliferative neoplasms and related symptoms is necessary to improve patient outcomes.

Reference

Advancing care for patients with myeloproliferative neoplasms. Association of Community Cancer Centers. Accessed October 17, 2023. https://www.accc-cancer.org/home/learn/cancer-types/hematologic-malignancies/advancing-care-for-patients-with-myeloproliferative-neoplasms.