Imatinib treatment in children with chronic phase chronic myeloid leukemia

This study aimed to investigate the outcomes of imatinib treatment for pediatric patients with chronic-phase chronic myeloid leukemia. This study concluded that imatinib should remain the frontline treatment of choice for these patients.  

Chronic myeloid leukemia (CML) is rare in children and cases that do occur are mainly in adolescence. CML in children is more aggressive than CML seen in adults. Tyrosine kinase inhibitors (TKIs) are a type of targeted treatment that have improved outcomes for CML patients in recent years. Imatinib (Gleevec) is one type of TKI. 

It was unknown if imatinib was the optimal choice for the treatment of pediatric CML.  

This study involved 124 patients aged 18 years or younger with CML. 80% of these patients had chronic phase CML (CP-CML). The response to imatinib was measured. The real-world management of CML in those with a poorer response to imatinib was also investigated. The average follow-up period was 67.4 months. 

At 3 months, 79.7% of patients had a complete hematologic response (CHR). CHR is when all blood cell counts return to normal. At 12 months 54.1% of patients had a complete cytogenetic response (CCyR). CCyR is when there are no Philadelphia chromosomes (genetic abnormality associated with leukemia) present. At 12 months, 50.9% of patients had a major molecular response (MMR). MMR is when there is a low level of BCR-ABL cancer gene.  

The 5-year overall survival (OS) rate was 92%. The 5-year event-free survival (EFS; survival without complication from CML) rate was 64%. Failure to achieve CCyR at 12 months was associated with poor EFS beyond 1 year.  

15 patients did not achieve CHR at 3 months. 87% of these patients were able to obtain CHR after increasing the dose of imatinib. 7 patients had a loss of response to imatinib because of poor adherence to the treatment. 

This study concluded that imatinib should remain the frontline treatment of choice for pediatric CML. The authors suggested that increasing the dose of imatinib is an option for patients with a poor response.  

This study was based on medical records. Some information may have been missing. This may have affected the results.