Results and outcome of intermittent imatinib (ON/OFF schedule) in children and adolescents with chronic myeloid leukaemia

The treatment of children and adolescents with chronic myeloid leukaemia (CML) is based on that of adult patients, with tyrosine kinase inhibitors (TKIs) being the mainstay and first-line therapies. TKIs have been demonstrated to be highly effective in CML, with life expectancy in patients treated with TKI similar to that of the age-matched healthy population. However, the long-term side effects, the treatment duration in molecular responders, and the poor adherence to therapy remain the main challenges. Delayed growth, changes in bone metabolism, thyroid abnormalities, dysregulation of pubertal development and effects on fertility have been reported in children under chronic exposure to imatinib mesylate (IM), especially in those who started treatment in pre-pubertal age, whatever the administered dose (Mariani et al., 2008; Bansal et al., 2012; Giona et al., 2013; Millot et al., 2014).