International patterns of childhood chronic myeloid leukemia: Comparisons between the United States and resource-restricted nations

Abstract

Background: Chronic myeloid leukemia (CML) is a rare disease in children and represents approximately 2% of all childhood leukemia. This results in difficulty creating large cohorts of patients for pediatric CML research. The Glivec International Patient Assistance Program (GIPAP) is a patient-access program sponsored by Novartis Oncology and administered by The Max Foundation (MAX) that provides imatinib free of charge to patients in resource-restricted countries who are not able to afford this treatment. Procedures: GIPAP highlights a cohort of children (n=3,188) with CML that provides novel insight into international trends in diagnosis, treatment, and survival. These trends can be compared to outcomes in developed nations to crudely assess the impact of an extended access program for CML treatment such as GIPAP. Results: Overall survival values for children treated for CML within the GIPAP (89%) suggest that imatinib is very effective in middle and low-income countries. Conclusions: This may allow for increased international awareness within the scientific community to consider possible reasons for the differences in overall survival in pediatric CML within the United States versus other nations with fewer resources. © 2014 Wiley Periodicals, Inc.