Abstract
Targeted therapy has revolutionized the treatment of poor-prognosis pediatric acute lymphoblastic leukemia (ALL) with specific genetic abnormalities. It is still being described as a new landmark therapeutic approach. The main purpose of the use of molecularly targeted drugs and immunotherapy in the treatment of ALL is to improve the treatment outcomes and reduce the doses of conventional chemotherapy, while maintaining the effectiveness of the therapy. Despite promising treatment results, there is limited clinical research on the effect of target cell therapy on the potential toxic events in children and adolescents. The recent development of highly specific molecular methods has led to an improvement in the identification of numerous unique expression profiles of acute lymphoblastic leukemia. The detection of specific genetic mutations determines patients’ risk groups, which allows for patient stratification and for an adjustment of the directed and personalized target therapies that are focused on particular molecular alteration. This review summarizes the knowledge concerning the toxicity of molecular-targeted drugs and immunotherapies applied in childhood ALL.
Highlights
Acute lymphoblastic leukemia (ALL) is the most commonly diagnosed pediatric neoplasm that belongs to the family of hematologic malignancies
The minimal residual disease (MRD) status is evaluated with the use of sensitive techniques, such as multiparameter flow cytometry (MFC) and polymerase chain reaction (PCR), which enable quantification in more than 90% of ALL pediatric patients [9]
In comparison with the chemotherapy-regimen group, the bispecific antibody presented a significantly improved SR [60]. Another crucial study resulted in an overall survival of 71.3% for the blinatumomab group vs. 58.4% for the chemotherapy group
Summary
Acute lymphoblastic leukemia (ALL) is the most commonly diagnosed pediatric neoplasm that belongs to the family of hematologic malignancies. It arises from the uncontrolled proliferation of immature precursor lymphocytic cells originating in the bone marrow. A share of 60.3% of all newly diagnosed ALL cases were found in the pediatric population aged 0–19 years, which suggests a strong correlation with patient age. The highest incidence rate per 100,000 people was found among patients aged 0–4, of approximately 5.5 in females and 6.5 in males. Among people aged 15–19, the incidence rate per 100,000 people decreases to 1.0 and 2.1 cases in females and males, respectively.
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