Purpose We evaluated the existing risk assessment tools for CML in children. Patients and Methods A total of 55 patients from 1.4 to 18.0 years with newly diagnosed CML between 1996 and 2019 were included. Forty-nine patients presented in the chronic phase, thirty-six of whom were treated with upfront tyrosine kinase inhibitor (CP-TKI group); one presented in the accelerated phase and 4 in the blastic phase. Treatment, survival, responses, and tolerance were evaluated. Results The median follow-up time was 8.7 years (range, 2 months to 24.3 years). All patients in the CP-TKI group received imatinib as their first TKI treatment. Allogenic stem cell transplantation was performed in one patient after complete cytogenetic response was achieved with imatinib and in one patient with imatinib failure. Dasatinib and nilotinib were prescribed as second-line TKI in 5 patients and 4 patients respectively. The 10-year overall survival (OS), progression-free survival (PFS) and event-free survival (EFS) of TKI treated group was 97%, 91.4% and 72.3% respectively. The rates of major molecular response and deep molecular response of TKIs were 81.2% and 67.5% at 60 months. The EUTOS long-term survival (ELTS) risk grouping did not predict OS, PFS or EFS. The IMAFAIL risk groups are correlated with the risk of imatinib failure. Conclusion TKIs resulted in excellent long-term overall and progression-free survival in children and adolescents with newly diagnosed CML in the chronic phase. Further studies are required to modify the existing prognostic scoring system or develop new ones for children.

Background: Pulmonary dysfunction has been reported in patients with β-thalassaemia major but data are conflicting and the association with iron overload remains unclear. Objectives: To determine the pattern of pulmonary dysfunction in patients with β-thalassaemia major and their associations with iron overload. Methods: Subjects with β-thalassaemia major were recruited for lung function assessment. Serum ferritin and magnetic resonance imaging (MRI) measurements of iron status of the myocardium and the liver were used as surrogate indexes of body iron content. A subgroup of this cohort provided data on the longitudinal progress of their lung function. Results: One hundred and one patients were recruited with a mean age of 25.1 years (SD 7.9 years). Thirty-eight (38%) and five (5%) had restrictive and obstructive lung function deficits, respectively. There was a significant correlation between MRI myocardial T2* relaxation time and forced vital capacity (r=0.291, p=0.048). Higher MRI cardiac T2* relaxation time was associated with lower risk of having restrictive lung function deficit (Odds ratio (OR): 0.94; 95% CI: 0.89-0.99; p=0.023) after adjusting for age, gender and BMI. Twenty-three subjects underwent lung function reassessment with a mean follow-up duration of 13 years. Overall, they did not demonstrate significant changes in pulmonary function over time, 3 patients who had normal lung function at baseline developed restrictive abnormality at follow-up. Conclusions: Restrictive lung disease is prevalent in patients with β-thalassaemia major, and the severity correlates with myocardial iron overload. Monitoring of lung function in this group of patients is important, particularly for those with iron overload.