title: Outcomes Following Bone Marrow Transplantation in Children With Accelerated Phase or Blast Crisis Chronic Myelogenous Leukemia in the Era of Tyrosine Kinase Inhibitors.
abstract: 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.
authors.affiliation: *Boston Children's Hospital †Pediatric Stem Cell Transplantation Center, Dana-Farber Cancer Institute/Boston Children's Hospital, Boston, MA.
authors.forename: David S
authors.initials: DS
authors.lastname: Shulman
authors.forename: Michelle A
authors.initials: MA
authors.lastname: Lee
authors.forename: Leslie E
authors.initials: LE
authors.lastname: Lehmann
authors.forename: Steven P
authors.initials: SP
authors.lastname: Margossian
mesh_terms: D000293:Adolescent; D001752:Blast Crisis / Q000628:therapy; D016026:Bone Marrow Transplantation / Q000379:methods; D002648:Child; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods; D006801:Humans; D015464:Leukemia, Myelogenous, Chronic, BCR-ABL Positive / Q000473:pathology / Q000628:therapy*; D015465:Leukemia, Myeloid, Accelerated Phase / Q000628:therapy; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use*; D012074:Remission Induction; D012189:Retrospective Studies; D016896:Treatment Outcome
chemical_list: D047428:Protein Kinase Inhibitors