Historically, MPNS are diagnosed in adults 60 years or older. In recent years, however, there has been recognition of essential thrombocythemia (ET), polycythemia vera (PF), and primary myelofibrosis (PMF) in adolescents and young adults, often referred to as AYAs.
A recent article in British Journal of Haematology offers a comprehensive review of the literature about AYAs, authored by a number of international MPN leaders. It highlights differences in how the disease presents itself as well as its increased affects on AYAs’ quality of life.
What we know:
- The AYA population includes ages 15-39, an accepted guideline by the National Comprehensive Cancer Network (NCCN) and others.
- While reports vary regarding the prevalence of AYA MPNs, the range is approximately 10%-20% of MPN cases.
- Out of the classical MPNs, ET is most common, followed by PV, then PMF.
- Incidence of the CALR mutation was found to be higher in AYA patients.
- Similar to older patients, many AYA MPN patients are asymptomatic at the time of diagnosis.
- Patients with ET may be less symptomatic than those with PV. One study suggests 3.9% of ET patients vs 9.2% of PV patients report symptoms.
- One research group reported that splenomegaly (enlarged spleen) is more common in their AYA patients, both ET and PV, compared to their 60+ patients. Splenomegaly is often cited as a risk factor for complications, including disease transformation/progression, hemorrhage, and thrombosis.
- Thrombotic events (blot clots) at or prior to diagnosis in AYA patients is reportedly higher than in older adults, 19.8% vs. 10.7%. Conversely, bleeding appears to be a rare complication in reported AYA patients.
- Incidence of the CALR mutation was found to be higher in AYA patients.
- AYA patients with PMF reportedly had less anemia than their older counterparts in more than one study.
Quality of Life
- 60% of low risk ET and PV patients report adverse effects on QoL
- MPN-10: questionnaire to assess symptoms
- AYA report lower MPN-10 scores but “they endorsed a higher proportion of adverse effects of their disease and therapy on daily life.”
- Lowest mental health score than other patient groups
Generally, the literature review authors believe that higher blood cell parameters seen in younger MPN patients, i.e., higher platelet counts, may at least be partially explained by the fact that they have not yet experienced an age-related decline in hematopoiesis.
Source: https://onlinelibrary.wiley.com/doi/10.1111/bjh.19557
Goulart, H., Masarova, L., Mesa, R., Harrison, C., Kiladjian, J. J., & Pemmaraju, N. (2024). Myeloproliferative neoplasms in the adolescent and young adult population: A comprehensive review of the literature. British journal of haematology, 205(1), 48–60. https://doi.org/10.1111/bjh.19557
Certain medicines listed may not be approved by the US Food and Drug Administration (FDA) for myelofibrosis but are recommended by the National Comprehensive Cancer Network (NCCN). MPN Research Foundation works with NCCN on the creation and updating of its MPN Patient Guidelines.