Unmet needs and quality of life burden in chronic myeloid leukemia: results from a nationwide Italian real-world survey.

BACKGROUND

Optimizing tyrosine kinase inhibitor (TKI) therapy in chronic myeloid leukemia (CML) requires careful consideration of patient quality of life (QoL), which significantly influences treatment adherence and long-term outcomes. Long-term treatment can be associated with persistent symptoms that may negatively impact QoL, adherence, and patient satisfaction. This nationwide Italian survey explored patient experiences, treatment patterns, and factors influencing clinical decision-making in CML, with the aim of better understanding unmet needs and potential barriers to optimal care.

METHODS

We conducted a cross-sectional survey involving 146 adult patients with CML. A structured questionnaire, developed collaboratively by hematologists and patient association representatives, assessed patient-reported outcomes (PROs), treatment-related burden, and care satisfaction. Analyses were descriptive and exploratory.

RESULTS

Despite the availability of effective therapies, a substantial proportion of patients reported impaired QoL due to both disease and treatment-related factors. While most patients reported overall satisfactory QoL, 26% described low or very low QoL. The burden was particularly evident in specific patient subgroups and in later lines of therapy, reflecting a significant physical and psychological impact of CML and its management. Fatigue, weakness, and muscle pain were the most frequently reported symptoms, often persistent and affecting daily functioning. Female patients reported a higher symptom burden and greater psychological distress compared to males. Despite high overall satisfaction with healthcare professionals, relevant unmet needs were identified, particularly regarding information on supportive services, long-term disease management, and fertility-related issues. Treatment-related adverse events were common and frequently chronic, suggesting a substantial cumulative burden over time.

CONCLUSIONS

Despite the effectiveness of TKIs, patients with CML continue to experience a meaningful and multidimensional burden, particularly in later treatment lines and specific subgroups. These findings highlight the importance of greater attention to patient-centered management in CML, with treatment decisions carefully balancing the benefits and limitations of available therapeutic options to optimize both clinical outcomes and quality of life.