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Outcomes Following Bone Marrow Transplantation in Children With Accelerated Phase or Blast Crisis Chronic Myelogenous Leukemia in the Era of Tyrosine Kinase Inhibitors

Shulman, David S. MD; Lee, Michelle A. MD, PhD; Lehmann, Leslie E. MD; Margossian, Steven P. MD, PhD

Journal of Pediatric Hematology/Oncology: November 2016 - Volume 38 - Issue 8 - p 610–614
doi: 10.1097/MPH.0000000000000636
Original Articles

The management of chronic myelogenous leukemia (CML) in children changed dramatically with the introduction of tyrosine kinase inhibitors (TKIs). Unfortunately, outcomes for patients presenting in an advanced stage—accelerated phase or blast crisis CML—continues to be poor, requiring chemotherapy and allogeneic hematopoietic stem cell transplant (HSCT) to attempt cure. Integration of TKIs in the therapy of advanced CML is still an area of active investigation. There are little published data on TKI use in children with advanced stage CML. We performed a retrospective review of all children treated at our institution between January 1, 2010 and June 30, 2013, and identified 5 children, age 12 to 18 years, with advanced stage CML. All patients were treated with a TKI before HSCT and TKIs were restarted post-HSCT in 4/5 with a goal of continuing until 2 years posttransplant. At time of HSCT all were in a morphologic and cytogenetic remission; 1 patient had also achieved molecular remission. All patients are alive and in molecular remission at an average of 38 months (range, 14 to 51 mo) following transplant. Our experience indicates that TKIs are safe and well tolerated in children both pretransplant and posttransplant and may improve outcomes in this aggressive disease.

*Boston Children’s Hospital

Pediatric Stem Cell Transplantation Center, Dana-Farber Cancer Institute/Boston Children’s Hospital, Boston, MA

The authors declare no conflict of interest.

Reprints: David S. Shulman, MD, Boston Children’s Hospital, 300 Longwood Ave, Boston, MA 02115 (e-mail:

Received February 10, 2016

Accepted June 7, 2016

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