Clinical and Humanistic Burden in Patients With Chronic Myeloid Leukemia in Early Lines of Treatment: A Literature Review

OBJECTIVES: Patients with chronic phase-chronic myeloid leukemia (CP-CML) receive tyrosine kinase inhibitors as initial treatment. However, the disease burden is increased by the treatment resistance, intolerability, and side-effects. We conducted a literature review examining this burden in CP-CML patients receiving early line treatment.

METHODS: Embase^® and MEDLINE^® were searched for English language publications from database inception until June 16, 2023. Complementary searches involved hand-searching relevant conferences and bibliographical reviews. Publications meeting the pre-defined inclusion criteria, reporting clinical and humanistic burden in CP-CML patients were included.

RESULTS: Ninety-one studies reported clinical burden, while 39 studies reported humanistic burden. Majority of studies (74%) reported all-cause mortality in less than 50% of patients receiving early line treatment. Proportion of patients progressing from CP to later disease stages was variable (2%-77%). Majority of studies reported over 50% of patients experiencing any grade adverse events (AEs). Commonly reported AEs included nausea/vomiting, myalgia, and skin-rash. AEs, intolerance, and treatment resistance led to treatment discontinuations and switching in up to 83% and 100% of patients, respectively. SF-36 and EORTC QLQ-C30 were commonly used instruments assessing Quality of life (QoL) showing negative impact on role, social, and emotional functioning in CP-CML patients compared to general population. Fatigue, anxiety, depression, and gastrointestinal symptoms contributed to high burden. Poor QoL correlated with intolerance due to AEs and lower treatment adherence.

CONCLUSIONS: As a chronic disease, CML requires long-term therapy, underscoring need for effective and tolerable treatments. However, despite the availability of multiple treatment options including tyrosine kinase inhibitors, CP-CML carries, significant clinical burden and reduces QoL due to escalating symptoms, treatment intolerance, and resistance. Effective and safe treatments are required to address disease burden at early treatment stages of CP-CML to limit treatment switching, development of treatment-resistant mutation and to increase the number of patients eligible for treatment free remission.