Frontiers | Precision medicine in myelofibrosis: from molecular profiling to personalized therapy

Cancer research · Myelofibrosis

Ruxolitinib remains the anchor first‑line option for symptomatic proliferative disease.

Precision medicine in myelofibrosis (MF) has moved beyond confirmation of a driver mutation toward an integrated interpretation of marrow morphology, molecular profile, cytogenetics, clinical phenotype, symptom burden, and transplant fitness. MF is biologically heterogeneous across both primary MF and secondary MF arising after polycythemia vera or essential thrombocythemia, with clinically relevant differences in disease origin, driver distribution, prognostic model validation, phenotype, and outcome. Current precision care is actionable now in diagnosis, integrated risk assessment, phenotype‑adapted JAK inhibitor selection, structured symptom assessment, and transplant timing. Ruxolitinib remains the anchor first‑line option for symptomatic proliferative disease; fedratinib provides an alternative or post‑ruxolitinib option in selected patients with preserved platelet reserve; pacritinib is particularly relevant in severe thrombocytopenia; and momelotinib has a differentiated role in anemia‑dominant MF. Allogeneic hematopoietic cell transplantation remains the only curative strategy and should be discussed early for biologically or clinically high‑risk disease.

This sentence highlights the current first‑line standard of care for symptomatic proliferative myelofibrosis, a key decision point in precision‑medicine management.

Evidence level: Klinikai vizsgálat. Formális klinikai vizsgálati eredmény.

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