Discontinuation of TKIs

Discontinuation of the TKI is now a standard of care for select adult patients with CML.4 A major advantage of this approach is to limit adverse effects. “If we can stop TKIs for children, that would be very beneficial,” Dr Hijiya said, highlighting that this may be a strategy to help mitigate growth abnormalities or other potential long-term morbidities, as well as help relieve financial burden.

Continue Reading

Dr Hijiya cautioned, however, that the adult standard of care cannot always be applied to children. “Can we apply the knowledge in adult CML to pediatrics? The answer is, we don’t really know because we do not understand the difference of the biology between pediatrics and adults.”

Dr Hijiya highlighted that studies of discontinuing TKIs must be conducted before this approach can be used in children with a deep, sustained molecular remission. She noted that her group is planning to open a study to evaluate this question.

Related Articles


Many unanswered questions about optimal TKI use and long-term outcomes remain in pediatric CML because of its rarity. “We still need more research and we need to increase the awareness of the disease. We need support in many ways,”  Dr Hijiya concluded.


  1. Hijiya N, Schultz KR, Metzler M, et al. Pediatric chronic myeloid leukemia is a unique disease that requires a different approach. Blood. 2016;127:392-399. doi: 10.1182/blood-2015-06-648667
  2. Siegal DA, King J, Tai E, et al. Cancer incidence rates and trends among children and adolescents in the United States, 2001–2009. Pediatrics. 2014;134:e945-e955. doi: 10.1542/peds.2013-3926
  3. Liu H-C, Liang D-C, Yeh T-C, et al. Molecular responses and outcomes of pediatric CML treated with front-line imatinib and a comparison between pediatric and adult cohorts. Blood. 2017;130:2902.
  4. Krumbholz M, Karl M, Tauer JT, et al. Genomic BCR-ABL1 breakpoints in pediatric chronic myeloid leukemia. Genes Chrom Cancer. 2012;51:1045-53. doi: 10.1002/gcc.21989
  5. Radich JP, Deininger M, Abboud CN, et al. Chronic myeloid leukemia, version 4.2018. NCCN Clinical Practice Guidelines in Oncology. January 24, 2018.
  6. US Food and Drug Administration. FDA approves dasatinib for pediatric patients with CML. November 9, 2017. https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm584725.htm. Accessed May 14, 2018.
  7. US Food and Drug Administration. FDA approves nilotinib for pediatric patients with newly diagnosed or resistant/intolerant Ph+ CML in chronic phase. March 22, 2018. https://www.fda.gov/Drugs/InformationOnDrugs/ApprovedDrugs/ucm602264.htm. Accessed May 14, 2018.
  8. Karalexi MA, Baka M, Ryzhov A, et al. Survival trends in childhood chronic myeloid leukaemia in Southern-Eastern Europe and the United States of America. Eur J Cancer. 2016;67:183-190. doi: 10.1016/j.ejca.2016.08.011