CHARACTERIZING THE CLINICAL AND HUMANISTIC BURDEN OF MYELOFIBROSIS: EVIDENCE FROM A SYSTEMATIC LITERATURE REVIEW
Author(s)
Natalia Curto-Garcia, MD1, Claire Harrison, MD,PhD1, Andrii Danyliv, PhD2, Kavita Rodha, M.Pharm3, Claudia Gorcea-Carson, MD4, Aditi Kataria, M.Pharm5, Rahul Kumar, MS5, Elisabetta Abruzzese, PhD6.
1Guys and St Thomas' NHS Foundation Trust, London, United Kingdom, 2Novartis, Basel, Switzerland, 3Novartis, Dublin, Ireland, 4Novartis Pharmaceuticals UK Ltd, London, United Kingdom, 5Novartis Healthcare Pvt. Ltd., Hyderabad, India, 6S Eugenio Hospital, Tor Vergata University, Rome, Italy.
1Guys and St Thomas' NHS Foundation Trust, London, United Kingdom, 2Novartis, Basel, Switzerland, 3Novartis, Dublin, Ireland, 4Novartis Pharmaceuticals UK Ltd, London, United Kingdom, 5Novartis Healthcare Pvt. Ltd., Hyderabad, India, 6S Eugenio Hospital, Tor Vergata University, Rome, Italy.
OBJECTIVES: Myelofibrosis (MF) is a rare myeloproliferative neoplasm marked by bone marrow fibrosis, cytopenias, and substantial symptom burden. This systematic literature review assessed the clinical and humanistic burden of MF.
METHODS: Embase® and MEDLINE® were searched (inception to February 2026). Eligible studies from Europe, China, Canada, Japan, Brazil, and Mexico reporting overall survival (OS), disease progression, transfusion dependence (TD), splenomegaly, symptoms, and patient-reported outcomes (PROs) were included.
RESULTS: A total of 107 studies (99 clinical; 2 humanistic; 6 both) were included, predominantly from Italy (n=29) and China (n=13). Median OS ranged from 20.6 months (Italy; severe anemia) to 256.8 months (Canada; small MF cohort; n=24) and varied by anemia, TD, and mutation/risk. Survival was shorter in higher-risk, cytopenic, or TD populations (21-58.9 months) and longer in lower-risk or early-stage disease (8.6-28 years). In Chinese cohorts, median OS was 110/37/21 months in intermediate-1/intermediate-2/high-risk groups. Rates of leukemic transformation ranged from 1.9% (early-stage, favourable mutation) to 72.4% (high-risk smaller cohort) and blastic transformation from 14.1% (early-stage) to 62.5% (pre-transplant), reaching 100% (advanced disease). TD ranged from 12% (Italy; large cohort) to 100% (Spain; pre-transplant smaller cohort). It increased from 40% (at diagnosis) to 60% (19-month follow-up) in Greece. Symptom burden remained high. Splenomegaly ranged from 7.1% (Italy) to 95% (Japan), reaching 100% in pre-transplant patients, driven by progression and advanced MF. Fatigue was reported in up to 81% of patients. Quality of life was substantially impaired, especially in overt and secondary MF. Common PRO instruments included MPN-SAF TSS, EORTC QLQ-C30, and MPN-10 TSS.
CONCLUSIONS: MF imposes substantial, multi-dimensional clinical and humanistic burden, across the disease spectrum and geographies, with poor survival, high progression rates and TD, persisting despite available therapies. Limited humanistic evidence highlights a key gap, supporting earlier intervention, standardized PRO assessment, alongside evaluation of caregiver and societal burden.
METHODS: Embase® and MEDLINE® were searched (inception to February 2026). Eligible studies from Europe, China, Canada, Japan, Brazil, and Mexico reporting overall survival (OS), disease progression, transfusion dependence (TD), splenomegaly, symptoms, and patient-reported outcomes (PROs) were included.
RESULTS: A total of 107 studies (99 clinical; 2 humanistic; 6 both) were included, predominantly from Italy (n=29) and China (n=13). Median OS ranged from 20.6 months (Italy; severe anemia) to 256.8 months (Canada; small MF cohort; n=24) and varied by anemia, TD, and mutation/risk. Survival was shorter in higher-risk, cytopenic, or TD populations (21-58.9 months) and longer in lower-risk or early-stage disease (8.6-28 years). In Chinese cohorts, median OS was 110/37/21 months in intermediate-1/intermediate-2/high-risk groups. Rates of leukemic transformation ranged from 1.9% (early-stage, favourable mutation) to 72.4% (high-risk smaller cohort) and blastic transformation from 14.1% (early-stage) to 62.5% (pre-transplant), reaching 100% (advanced disease). TD ranged from 12% (Italy; large cohort) to 100% (Spain; pre-transplant smaller cohort). It increased from 40% (at diagnosis) to 60% (19-month follow-up) in Greece. Symptom burden remained high. Splenomegaly ranged from 7.1% (Italy) to 95% (Japan), reaching 100% in pre-transplant patients, driven by progression and advanced MF. Fatigue was reported in up to 81% of patients. Quality of life was substantially impaired, especially in overt and secondary MF. Common PRO instruments included MPN-SAF TSS, EORTC QLQ-C30, and MPN-10 TSS.
CONCLUSIONS: MF imposes substantial, multi-dimensional clinical and humanistic burden, across the disease spectrum and geographies, with poor survival, high progression rates and TD, persisting despite available therapies. Limited humanistic evidence highlights a key gap, supporting earlier intervention, standardized PRO assessment, alongside evaluation of caregiver and societal burden.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
CO200
Topic
Clinical Outcomes, Patient-Centered Research, Study Approaches
Topic Subcategory
Clinical Outcomes Assessment
Disease
Oncology, Rare & Orphan Diseases