Pharmakon: Patient Journey e Real World Effectiveness nel paziente con patologia rara: il caso della Leucemia Mieloide Cronica

Abstract

In the current context characterised by the growing, and often hypertrophic, availability of healthcare data, the mere accumulation of information does not necessarily translate into evidence that is useful for decision-making processes. This issue is particularly relevant in the field of rare diseases, where the limited number of cases and the need for highly specialised care pathways make it essential to adopt a methodological approach capable of integrating heterogeneous sources of information. In this scenario, chronic myeloid leukaemia (CML) assumes particular significance. This rare haematological malignancy has undergone a profound transformation in its clinical course following the introduction of tyrosine kinase inhibitors, a therapeutic breakthrough that has rendered systematic monitoring of treatment effectiveness indispensable in real-world clinical practice.