Treatment choices and outcomes for childhood chronic myeloid leukemia 

This study aimed to investigate treatment outcomes with transplant and tyrosine kinase inhibitors for childhood chronic myeloid leukemia. 

This study concluded that some tyrosine kinase inhibitors provide better outcomes for these patients. 

Chronic myeloid leukemia (CML) is a rare disease in childhood. Hematopoietic stem cell transplant (HSCT) was the treatment of choice for CML prior to 2000. A type of targeted therapy called tyrosine kinase inhibitors (TKIs) is now a frequently used treatment for CML. 

It was unknown if HSCT or TKIs provided better outcomes for children with CML.  

This study involved data from 52 patients who were diagnosed with CML before 18 years of age. These patients received treated between 1985 and 2018. 22 patients were treated before the era of TKIs (before 2002) and mainly received HSCT (82%). The other 30 patients received TKIs such as imatinib (Gleevec; 23 patients) or dasatinib (Sprycel; 4 patients).

The overall survival (OS) rate for patients treated before 2002 was 64% after an average of 6 years. The OS of patients treated in the TKI era (2002 and after) was 90% after an average of 3 years.  

There were three deaths in patients treated in the TKI era and this was due to HSCT complications. The survival of patients who stayed on TKI treatment was 100% compared to 66% for those who underwent HSCT after TKI treatment.  

This study concluded that TKI treatment provides better outcomes for childhood CML when compared to HSCT. It was also concluded that HSCT has increased mortality and that it should only be considered in a small subset of pediatric patients with CML.  

This was a small group of patients treated in pediatric centers. Some adolescent patients may have been treated in adult centers and their information was not included in this analysis.