Patient-Reported Functional Outcomes in Patients With Chronic Myeloid Leukemia After Stopping Tyrosine Kinase...

Area of Science:

• Hematology
• Oncology
• Patient-Reported Outcomes

Background:

• Treatment-free remission (TFR) is a key goal for patients with chronic myeloid leukemia (CML).
• Functional outcomes following tyrosine kinase inhibitor (TKI) discontinuation in CML are not well-documented.
• Understanding patient-reported outcomes is crucial for assessing quality of life after CML treatment cessation.

Purpose of the Study:

• To evaluate functional outcomes in adult patients with chronic phase CML during treatment-free remission (TFR).
• To assess changes in social, physical, cognitive, and sexual function after discontinuing TKI therapy.
• To compare functional trajectories between patients in TFR and those who restart TKI treatment.

Main Methods:

• Prospective assessment of 172 adult patients with chronic phase CML across 14 sites.
• Utilized Patient-Reported Outcomes Measurement Information System (PROMIS) measures for social, physical, cognitive, and sexual function.
• Employed linear mixed-effects models to analyze functional trajectories over 36 months, comparing on-TKI, TFR, and restart-TKI groups.

Main Results:

• Of 112 patients in TFR at 12 months, 92% showed improved social function and 71.4% reduced social isolation.
• Minor improvements were observed in satisfaction with sex life (9.8%) and physical function (3.6%).
• No significant improvements were noted in cognitive function or sexual interest; scores declined upon restarting TKI therapy.

Conclusions:

• Treatment-free remission in CML is associated with substantial improvements in social well-being.
• Functional gains in physical and sexual domains after TKI discontinuation are limited.
• These findings provide valuable insights for shared decision-making regarding TKI cessation in CML management.