In this installment of the “How I treat ...” series in Blood, the authors outline the somatic or germline mutations that are the underlying cause of juvenile myelomonocytic leukemia (JMML) and describe their treatment algorithm for this aggressive hematopoietic disorder of infancy/early childhood. They note that allogeneic HCT “remains the therapy of choice for most patients with JMML, curing more than 50% of affected children” and they recommend that this option be promptly offered to any child with PTPN-11-, K-RAS-, or NF1-mutated JMML and to the majority of those with N-RAS mutations. They also discuss the recent use of the hypomethylating agent azacytidine in treating JMML, and its ability to induce hematologic/molecular remissions in some children with the disorder and possible use in reducing leukemia burden prior to HCT.
Review: How I Treat JMML
Feb 2015