SUCCESSFUL TREATMENT OF A CHILD WITH CHRONIC MYELOID LEUKEMIA

A nine year old child is diagnosed in an incidental way with Chronic Myeloid Leukemia in chronic phase. She started treatment with Hydroxyurea, Imatinib and lately Nilotinib and she achived complete molecular remission after 3 months of therapy. Chronic Myeloid Leukemia is a malignant disease which is very rare in pediatric age. This disease is characterised by Philadelphia chromosome , which results by a translocation between chromosome 9 and 22. This translocation results in an abnormal fusion called the oncogene BCR-ABL which codes a chimeric protein called BCR-ABL. This protein is the underlying cause of CML. Nilotinib is a newly licensed drug for CML in adults. Structurally, it is similar to imatinib (the older tyrosine kinase inhibitor), but it is much more potent in inhibiting BCR-ABL due to its much increased affinity for its binding site. Specific guidelines for CML treatment in children have yet to be determined. In our patient, nilotinib was used as an off-label drug because it is not licensed for children. According to the pharmacokinetic response to drugs, children cannot be considered small adults irrespective of their weight. Off-label drug use based on evidence that it is the best treatment available is an important tool in the hands of expert treating physicians.