Promotional content developed and funded by Novartis Pharmaceuticals UK Ltd
for UK healthcare professionals only

Prescribing information Adverse event reporting information 

INGRAIN-MF
Incorporating genetics, risk and associated burden into MF management

MODULE 1: The burden of myelofibrosis

Understanding myeloproliferative neoplasms

Myeloproliferative neoplasms (MPNs) are disorders that affect haematopoietic stem cells and are characterised, in the chronic phase, by an overproduction of differentiated haematopoietic cells.1 WHO classifies MPNs based on the presence or absence of the BCR-ABL1 fusion gene on the Philadelphia chromosome.2,3

The BCR-ABL1 negative disorders include:1

  • myelofibrosis (MF)
  • polycythaemia vera (PV)
  • essential thrombocythaemia (ET)

Although complete understanding of the molecular basis of these disorders is currently lacking, hyperactivation of the JAK-STAT signalling pathway due to mutations in various associated genes is clearly a central part of the pathogenesis.4

JAK: Janus-associated kinase; STAT: signal transducers and activators of transcription; WHO: World Health Organization

Global incidence of MF:5

Characterised by:6

bone marrow fibrosis and abnormal cell haematopoiesis

Global incidence of PV:5

Characterised by:7

elevated red blood cells

Global incidence of ET:5

Characterised by:8

elevated platelet count and enlarged megakaryocytes

MF is associated with poor overall survival, and risk of transformation to acute myeloid leukaemia9-11

The disease burden faced by patients with MF can be divided into four categories:10

  • enlarged spleen and associated complications
  • abnormal blood counts including anaemia, thrombocytopenia
  • systemic symptoms
  • decreased life expectancy

5-year survival rates for MPNs compared with other haematological cancers:9,12-16

MF:

PV:

ET:

31.9%

Acute myeloid leukaemia

61.1%

Myeloma

72.0%

Acute lymphocytic leukaemia

74.3%

Non-Hodgkin lymphoma

88.5%

Chronic lymphocytic leukaemia

Even in low-risk patients with MF, survival is reduced compared with the general population (matched demographics).10

  • 5 years after diagnosis: 40% greater mortality
  • 10 years after diagnosis: 60% greater mortality

Survival rates decrease with increasing age (HR: 1.06 per year), and are also lower for black race (HR: 1.44), male sex (HR: 1.32) and for patients with more than one lifetime malignancy (HR: 1.12).9

Transformation to secondary acute myeloid leukaemia (or blast phase MPN) occurs in 10%-20% of patients with primary MF within 10 years. The leukaemic transformation rate is lower with PV and ET.11

HR: hazard ratio

Continue to next section: Presentation and diagnosis of myelofibrosis 
Continue  Homepage  Previous Download certificate

Adverse events should be reported. Reporting forms and information can be found at www.mhra.gov.uk/yellowcard.
Adverse events should also be reported to Novartis online through the pharmacovigilance intake (PVI) tool at www.novartis.com/report or alternatively email medinfo.uk@novartis.com or call 01276 698370

References
  1. Nangalia J, Green A R. Myeloproliferative neoplasms: from origins to outcomes. Blood 2017;130(23):2475-2483
  2. Arber D A, Orazi A et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood 2016;127(20):2391-2405
  3. Vardiman J W, Thiele J et al. The 2008 revision of the World Health Organization (WHO) classification of myeloid neoplasms and acute leukemia: rationale and important changes. Blood 2009;114(5):937-951
  4. Venugopal S, Mascarenhas J. Novel therapeutics in myeloproliferative neoplasms. J Hematol Oncol 2020;13(1):162
  5. Titmarsh G J, Duncombe A S et al. How common are myeloproliferative neoplasms? A systematic review and meta-analysis. Am J Hematol 2014;89(6):581-587
  6. MPN Research Foundation. Primary myelofibrosis (PMF). Available at: mpnresearchfoundation.org. Accessed April 2025
  7. MPN Research Foundation. Polycythemia vera (PV). Available at: mpnresearchfoundation.org. Accessed April 2025
  8. Pathology Outlines. Bone marrow - neoplastic myeloid. Essential thrombocythemia. Available at: pathologyoutlines.com. Accessed April 2025
  9. Brunner A M, Hobbs G et al. A population-based analysis of second malignancies among patients with myeloproliferative neoplasms in the SEER database. Leuk Lymphoma 2016;57(5):1197-1200
  10. Cervantes F, Dupriez B et al. New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment. Blood 2009;113(13):2895-2901
  11. Iurlo A, Cattaneo D, Gianelli U. Blast transformation in myeloproliferative neoplasms: risk factors, biological findings, and targeted therapeutic options. Int J Mol Sci 2019;20(8)
  12. National Cancer Institute. SEER Cancer stat facts: chronic lymphocytic leukemia. Available at: seer.cancer.gov. Accessed April 2025
  13. National Cancer Institute. SEER Cancer stat facts: acute myeloid leukemia. Available at: seer.cancer.gov. Accessed April 2025
  14. National Cancer Institute. SEER Cancer stat facts: acute lymphocytic leukemia. Available at: seer.cancer.gov. Accessed April 2025
  15. National Cancer Institute. SEER Cancer stat facts: non-Hodgkin lymphoma. Available at: seer.cancer.gov. Accessed April 2025
  16. National Cancer Institute. SEER Cancer stat facts: myeloma. Available at: seer.cancer.gov. Accessed April 2025