Molecular diagnosis and risk-adjusted therapy in pediatric hematologic malignancies: a primer for pediatricians

Abstract

Progress in the care of the hematologic malignancies of childhoond has been one of the proudest success stories in modern pediatrics. The cure rates of these diseases have improved from essentially zero in the 1950's and early 1960's to cure rates that range from 65%-90% in modern centers. While the largest improvements have been made in the most common (and the lower risk subtypes) of Acute Lymphoblastic Leukemia (ALL), there has also been significant progress in both the higher risk forms of ALL (i.e. Philadelphia chromosome positive, Ph+ ALL) and in Acute Myeloid Leukemia (AML). This progress has been achieved by the careful and stepwise identification of clinical, cytogenetic, molecular, and most recently response-based prognostic criteria, that now allow oncologists to focus the intensity of the therapy more closely to what is required to cure individual subgroups of patients.

Conclusion: Pediatricians need to be familiar with the changes in diagnostic and therapeutic approaches, because these changes have impact on: the laboratory tests that should be ordered at the time of specialist referral; counseling of patients and their families; and with the advent of "shared care models" pediatricians will need to be more involved in the general, supportive, and long-term care of these patients.