How I treat childhood CML

Abstract

Chronic myeloid leukemia (CML) is composed of 3% of pediatric leukemias, making evidence-based recommendations difficult. Imatinib has revolutionized the treatment for adult CML by eliminating allogeneic stem cell transplantation for almost all patients in chronic phase. Shown effective in pediatric CML, imatinib and successive tyrosine kinase inhibitors (TKI) have provided more therapeutic options. Because stem cell transplantation has been better tolerated in children and adolescents, the decision to treat by either TKI or transplantation is controversial. We present a recent case of a 12-month-old boy diagnosed with BCR-ABL ؉ CML to highlight the controversies in treatment recommendations. We review the pediatric stem cell transplantation outcomes as well as the pediatric experience with imatinib and other TKIs. Finally, we compare the side effects as well as costs associated with allogeneic stem cell transplantation versus TKI therapy. We recommend that frontline therapy for pediatric CML in chronic phase is TKI therapy without transplantation. Patients in accelerated or blast crisis or who fail to reach landmarks on TKIs either because of intolerance or resistance should pursue stem cell transplantation. Although we recommend adopting adult clinical experience to guide therapeutic decision making, the issues of infant CML, drug formulation, pharmacokinetics, and adolescent compliance merit clinical investigation.

Single-centre experience of allogeneic haemopoietic stem cell transplant in paediatric patients in Cape Town, South Africa

“…[1] There is still a role for sibling matched Allo-HSCT in chronic myeloid leukaemia (CML), despite the good long-term survival demonstrated with the use of tyrosine kinase inhibitors. [2] Allo-HSCT is also used as salvage therapy in the treatment of paediatric lymphoma. [3] In 2007, ~150 new cases of leukaemia per annum in children aged <14 years were being captured on the South African Children's Cancer Group registry.…”

Health-related quality of life of patients with newly diagnosed chronic myeloid leukemia treated with allogeneic hematopoietic SCT versus imatinib

“…Therefore, the use of imatinib therapy in young patients remains controversial. 31,32 In the present study, we demonstrated that patients treated with ISD HSCT have HRQOL comparable to that of patients treated with imatinib. HSCT remains the only proven cure for CML, and the outcome of HSCT for CML has improved significantly.…”

BCR-ABL Positive Childhood Chronic Myeloid Leukemia

“…This represents about 3-5% of all cases of newly diagnosed childhood leukemia [4]. The clinical course and prognosis are similar to that of adult CML.…”

“…The clinical course and prognosis are similar to that of adult CML. However, unlike the adult CML, ionizing radiation and other such environmental predisposing factors have not been shown to have a causal effect in its occurrence [4].…”

“…It constitute about 3-5% of all childhood leukemia with annual incidence of about 1 per million children [4]. However, the molecular biology of the disease in childhood is the same as adults; i.e.…”