Abstract 79P
Background
Chronic myeloid leukaemia (CML) is a rare form of childhood cancer, accounting for just 2-3% of paediatric leukaemia’s. While the introduction of tyrosine kinase inhibitors (TKIs) has significantly improved treatment outcomes, their long-term impact on endocrine functions and quality of life (QoL) in children is still not fully understood. This study investigates the off-target effects of TKIs on growth, bone health, metabolism, and QoL in paediatric CML patients.
Methods
This prospective observational study includes 82 paediatric and young adult CML patients who began TKI therapy before 18 years of age between 2004-2024 at AIIMS and NCI, India. Of these, 71 were on TKI therapy for over 2 years, and 11 were newly initiated during the study. Excluding those treated with chemotherapy or undergoing bone marrow transplant, the study assesses endocrine functions (BMD, thyroid, growth, pubertal development, gonadal, adrenal function, glucose/lipid metabolism) and QoL (EORTC QLQ-C30 for adults, PedsQL for children).
Results
A majority of patients were male (80.5%), and the median age at diagnosis was 12 years, with 66% presenting in chronic phase. The median age of the patients during study was 20 years, and the median duration of TKI exposure was 7.5 years. Short stature occurred in 15% of patients, mostly prepubertal at TKI initiation, linked to low IGF-1 levels but not growth hormone deficiency. Low BMD was noted in 35.2%, and 93% had vitamin D deficiency. Subclinical hypothyroidism was observed in 10% of patients, while dysglycemia and dyslipidaemia affected 11% and 8%, respectively. Infertility was noted in only one patient. QoL assessment revealed good physical and emotional functioning in younger patients, but poorer social functioning and older patients reported financial concerns and fatigue.
Conclusions
Prolonged TKI use in paediatric CML patients causes significant endocrine and metabolic complications, especially affecting growth and bone health. These effects are more severe in prepubertal patients. Regular monitoring of growth, bone density and thyroid function is vital and evaluation of all other endocrine functions can be done as clinically indicated.
Clinical trial identification
Editorial acknowledgement
Legal entity responsible for the study
Rajivmohan Nannapaneni.
Funding
Has not received any funding.
Disclosure
All authors have declared no conflicts of interest.