Chronic Myeloid Leukemia in Children: Challenges and Opportunities : Based on 7th Dr. I. C. Verma Excellence Award for Young Pediatricians Delivered as Oration on 29th Sept. 2019.

Abstract

Treatment with tyrosine kinase inhibitors (TKI) is an effective therapy for children and adolescents with chronic phase of chronic myeloid leukemia (CML). For the majority of patients with CML in low- and middle-income countries (LMIC), imatinib is the TKI of choice for reasons of cost, availability, and experience. Children are exposed to therapy for a greater proportion of their lifetime as compared to adults. The adverse effects of prolonged administration of TKI is a subject of ongoing research, as more experience is collected. Therapy with TKI is currently considered to be life-long. Trials on stopping treatment are ongoing to explore if it may be feasible in selected patients, as reported in adults. Growth-failure is a concerning adverse effect. Currently, it seems unclear if the final height attained is within the expected range of the mid-parenteral height and growth standards. Whether the children will achieve a normal height at the end of their growth potential or remain below the predicted range is critical to decide if therapeutic interventions (E.g., growth hormone therapy, or interruption in TKI) should be considered during the period of growth potential. Research on CML in children is at a relatively slow pace, largely due to the rarity of the disease. This provides a unique opportunity for research in population-dense LMICs, as several tertiary centers tend to have a sizable cohort of children and adolescents with CML on follow-up. This narrative summarises the challenges and opportunities in dealing with CML in children, particularly in reference to a center in LMIC.