ECONOMIC BURDEN IN PATIENTS WITH MYELOFIBROSIS: A SYSTEMATIC LITERATURE REVIEW
Author(s)
Francesca Palandri, MD, PhD1, Jennifer Mary O'sullivan, PhD2, Andrii Danyliv, PhD3, Kavita Rodha, M.Pharm4, Claudia Gorcea-Carson, MD5, Aditi Kataria, M.Pharm6, Nitin Kaushik, M.Pharm6, Priya Sriskandarajah, PhD2, Massimo Breccia, MD7.
1Università di Bologna, Italy, Italy, 2Guy's and St Thomas' NHS Foundation Trust, London, United Kingdom, 3Novartis Pharma AG, Basel, Switzerland, 4Novartis Ireland Ltd, Dublin, Ireland, 5Novartis Pharmaceuticals UK Ltd, London, United Kingdom, 6Novartis Healthcare Pvt. Ltd, Hyderabad, India, 7Sapienza University of Rome, Rome, Italy.
1Università di Bologna, Italy, Italy, 2Guy's and St Thomas' NHS Foundation Trust, London, United Kingdom, 3Novartis Pharma AG, Basel, Switzerland, 4Novartis Ireland Ltd, Dublin, Ireland, 5Novartis Pharmaceuticals UK Ltd, London, United Kingdom, 6Novartis Healthcare Pvt. Ltd, Hyderabad, India, 7Sapienza University of Rome, Rome, Italy.
OBJECTIVES: Myelofibrosis is a rare, chronic myeloproliferative neoplasm, characterized by progressive splenomegaly, marrow fibrosis, and symptoms. This systematic review assessed evidence on the economic burden associated with myelofibrosis.
METHODS: Embase® and MEDLINE® were searched (inception to 11-February-2026) for relevant English-language publications on healthcare resource utilization (HCRU) and costs among myelofibrosis patients.
RESULTS: Twelve studies reported economic burden: six from the United States (US), two each from France and Italy, one from Malaysia, and one multi-country study. Healthcare costs were substantial across countries: $8,360-$16,618 per-patient-per-month (PPPM) in the US, €51,832-€96,399 per-patient-per-year (PPPY) in France, and $5,217 PPPY in Malaysia. Hospitalizations and transfusions were the major cost-drivers (PPPY), contributing €33,528 and €10,400 in France, and $678 and $178 in Malaysia, respectively. Medication costs also contributed substantially, varying by subgroups: $987-$10,065 PPPM in the US, and $4,278 PPPY in Malaysia. HCRU was primarily driven by hospitalizations (9.47 PPPY in Malaysia, 3.8-15.4 PPPY in France and 0.04-0.32 PPPM in the US), outpatient-visits (6.13 PPPY, 6.6-7.6 PPPY, and 1.69-1.88 PPPM, respectively), transfusions (1.61 PPPY in Malaysia and 1.1-10 PPPY in France), and emergency-department visits (0.5-1.4 PPPY in France and 0.1-0.21 PPPM in the US). Patients with cytopenia or transfusion-dependence had higher healthcare costs ($12,826 vs $8,360 PPPM; €96,399 vs €51,832 PPPY) and hospitalization-rates (0.12 vs 0.04 PPPM; 15.4 vs 3.8 PPPY) than those without cytopenia or were transfusion independent, respectively. The Italian BEAT project reported higher financial burden in transfusion-dependent versus non-anemic patients (€6,603 vs €165 PPPY). Myelofibrosis substantially affected work productivity, with 11.7% of patients reporting absenteeism, 21.0% reduced working hours, and 41.4% overall work impairment in the International MPN-landmark survey.
CONCLUSIONS: Myelofibrosis imposes substantial economic and societal burden, through hospitalizations, transfusions, medication use, outpatient care, and productivity loss. High costs and HCRU highlight the need for effective management strategies to reduce morbidity, transfusion needs, and economic burden.
METHODS: Embase® and MEDLINE® were searched (inception to 11-February-2026) for relevant English-language publications on healthcare resource utilization (HCRU) and costs among myelofibrosis patients.
RESULTS: Twelve studies reported economic burden: six from the United States (US), two each from France and Italy, one from Malaysia, and one multi-country study. Healthcare costs were substantial across countries: $8,360-$16,618 per-patient-per-month (PPPM) in the US, €51,832-€96,399 per-patient-per-year (PPPY) in France, and $5,217 PPPY in Malaysia. Hospitalizations and transfusions were the major cost-drivers (PPPY), contributing €33,528 and €10,400 in France, and $678 and $178 in Malaysia, respectively. Medication costs also contributed substantially, varying by subgroups: $987-$10,065 PPPM in the US, and $4,278 PPPY in Malaysia. HCRU was primarily driven by hospitalizations (9.47 PPPY in Malaysia, 3.8-15.4 PPPY in France and 0.04-0.32 PPPM in the US), outpatient-visits (6.13 PPPY, 6.6-7.6 PPPY, and 1.69-1.88 PPPM, respectively), transfusions (1.61 PPPY in Malaysia and 1.1-10 PPPY in France), and emergency-department visits (0.5-1.4 PPPY in France and 0.1-0.21 PPPM in the US). Patients with cytopenia or transfusion-dependence had higher healthcare costs ($12,826 vs $8,360 PPPM; €96,399 vs €51,832 PPPY) and hospitalization-rates (0.12 vs 0.04 PPPM; 15.4 vs 3.8 PPPY) than those without cytopenia or were transfusion independent, respectively. The Italian BEAT project reported higher financial burden in transfusion-dependent versus non-anemic patients (€6,603 vs €165 PPPY). Myelofibrosis substantially affected work productivity, with 11.7% of patients reporting absenteeism, 21.0% reduced working hours, and 41.4% overall work impairment in the International MPN-landmark survey.
CONCLUSIONS: Myelofibrosis imposes substantial economic and societal burden, through hospitalizations, transfusions, medication use, outpatient care, and productivity loss. High costs and HCRU highlight the need for effective management strategies to reduce morbidity, transfusion needs, and economic burden.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE674
Topic
Economic Evaluation, Study Approaches
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Oncology, Rare & Orphan Diseases