UNDERSTANDING THE BURDEN OF MYELOFIBROSIS: A LITERATURE REVIEW OF EPIDEMIOLOGY AND NATURAL HISTORY TO INFORM VALUE ASSESSMENT
Author(s)
Francesco Passamonti, MD1, Giuseppe A. Palumbo, PhD2, Andrii Danyliv, PhD3, Kavita Rodha, M. Pharm4, Claudia Gorcea-Carson, MD5, Lovneet Saini, M. Pharm6, Greeshma Gopalan, MSc.6, Claire Harrison, MD, PhD7, Patrick. Harrington, MD, PhD7.
1Department of Oncology and Hemato-Oncology, University of Milan, Milan, Italy, 2Dipartimento di Scienze Mediche, Chirurgiche e Tecnologie Avanzate "G.F. Ingrassia", University of Catania, Catania, Italy, 3Novartis, Basel, Switzerland, 4Novartis, Dublin, Ireland, 5Novartis Pharmaceuticals UK Ltd, London, United Kingdom, 6Novartis Healthcare Private Limited, Hyderabad, India, 7Department of Haematology, Guy's and St Thomas' NHS Foundation Trust, London, United Kingdom.
1Department of Oncology and Hemato-Oncology, University of Milan, Milan, Italy, 2Dipartimento di Scienze Mediche, Chirurgiche e Tecnologie Avanzate "G.F. Ingrassia", University of Catania, Catania, Italy, 3Novartis, Basel, Switzerland, 4Novartis, Dublin, Ireland, 5Novartis Pharmaceuticals UK Ltd, London, United Kingdom, 6Novartis Healthcare Private Limited, Hyderabad, India, 7Department of Haematology, Guy's and St Thomas' NHS Foundation Trust, London, United Kingdom.
OBJECTIVES: Myelofibrosis (MF) is a rare myeloproliferative neoplasm associated with substantial morbidity and heterogeneous clinical presentation. A targeted literature review was conducted to synthesize published evidence on the epidemiology and natural history of MF to characterize unmet need and inform decision-making.
METHODS: Embase® and MEDLINE® (including MEDLINE® In-Process) were searched for English-language studies published from 01/01/2016, to 09/03/2026. Epidemiology outcomes (incidence, prevalence) were summarized globally and natural history for the EU-27, where available.
RESULTS: Ninety-nine studies were included (epidemiology, n=53; natural history, n=45; both, n=1). MF incidence varied across geographies, predominantly with primary myelofibrosis (PMF) reported at 0.3-3.13 per 100,000 person-years and increased over time in Sweden (0.34 in 2001 to 0.45 in 2014), Singapore (age-standardized: 0.01 to 0.43 [1993-2017]), and the US (annual percentage change: 1.3%-3.8%) but remained broadly stable in Italy. PMF incidence was higher in males than females (Republic of Korea: 0.384-0.649 vs 0.266-0.442; Sweden: 0.64 vs 0.43 per 100,000). Prevalence patterns suggested growing burden, reaching 9.9 per 100,000 for MF in Germany and rising in the Republic of Korea, where PMF prevalence remained higher in males (0.649 vs 0.442 in 2011; 0.973 vs 0.763 in 2013). MF presents with a substantial complication burden, with splenomegaly reported in up to 87.7% in Italy and major bleeding ranging from 6.5% in Germany to 28.0% in France across overall cohorts. Thrombotic events were reported in 5.4%-24.0% in MF and 8.0%-30.4% in PMF, while infections occurred in 45%-52% of selected cohorts.
CONCLUSIONS: Variability across studies warrants standardization of epidemiologic and natural history reporting to inform health-system decision-making. Overall, the evidence suggests a growing prevalent population and substantial complication burden. MF patients may thus benefit from therapies that provide relief of these manifestations and support disease modification, potentially translating into improved survival.
METHODS: Embase® and MEDLINE® (including MEDLINE® In-Process) were searched for English-language studies published from 01/01/2016, to 09/03/2026. Epidemiology outcomes (incidence, prevalence) were summarized globally and natural history for the EU-27, where available.
RESULTS: Ninety-nine studies were included (epidemiology, n=53; natural history, n=45; both, n=1). MF incidence varied across geographies, predominantly with primary myelofibrosis (PMF) reported at 0.3-3.13 per 100,000 person-years and increased over time in Sweden (0.34 in 2001 to 0.45 in 2014), Singapore (age-standardized: 0.01 to 0.43 [1993-2017]), and the US (annual percentage change: 1.3%-3.8%) but remained broadly stable in Italy. PMF incidence was higher in males than females (Republic of Korea: 0.384-0.649 vs 0.266-0.442; Sweden: 0.64 vs 0.43 per 100,000). Prevalence patterns suggested growing burden, reaching 9.9 per 100,000 for MF in Germany and rising in the Republic of Korea, where PMF prevalence remained higher in males (0.649 vs 0.442 in 2011; 0.973 vs 0.763 in 2013). MF presents with a substantial complication burden, with splenomegaly reported in up to 87.7% in Italy and major bleeding ranging from 6.5% in Germany to 28.0% in France across overall cohorts. Thrombotic events were reported in 5.4%-24.0% in MF and 8.0%-30.4% in PMF, while infections occurred in 45%-52% of selected cohorts.
CONCLUSIONS: Variability across studies warrants standardization of epidemiologic and natural history reporting to inform health-system decision-making. Overall, the evidence suggests a growing prevalent population and substantial complication burden. MF patients may thus benefit from therapies that provide relief of these manifestations and support disease modification, potentially translating into improved survival.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EPH21
Topic
Epidemiology & Public Health, Study Approaches
Topic Subcategory
Public Health
Disease
Oncology, Rare & Orphan Diseases