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