Acute lymphoblastic leukemia is one of the most frequent malignancies in pediatrics.
Acute lymphoblastic leukemia (ALL) is one of the most frequent oncological diseases in pediatrics and mutations in the KMT2A gene have been detected in 70 percent of patients under the age of 1 year. Chemotherapy has so far been the go-to against the disease, and research yields a new treatment approach for a specific T-cell molecule targeting CD19 that allows survival after a two-year follow-up.
The study, published in The New England Journal of Medicine (NEJM), noted that although event-free survival among older children has improved over time and is now more than 85 percent, all diagnosed in the first year of life Babies who do are still alive. Bad forecast.
In childhood leukemic cells leukemia express CD19, and through a bispecific molecule, blinatumomab, a response is elicited against these malignant cells. Studies have shown that blinatumomab is a safe and effective treatment for older children and adults with relapsed or refractory B-lineage after intensive chemotherapy, so a prospective study was conducted in patients with newly reconstituted KMT2A ALL. Diagnosed in the first year of life.
The Pediatric Acute Lymphoblastic Leukemia Investigation combined the molecule with chemotherapy in 30 patients younger than 1 year old with ALL and, after a median follow-up of 26.3 months, had a 2-year disease-free survival of 81.6 percent and a two-year overall survival. 93.3 percent. These results demonstrate, as the research says, a very promising outcome, given the poor survival and lack of improvement in outcomes among infants with KMT2A-rearranged ALL in recent decades.
One of the main findings of the study is that blinatumomab has fewer acute adverse effects than chemotherapy. The toxic effect profile in the infant group was consistent with previous studies of blinatumomab in older children and adults. Due to the low tumor burden, severe cytokine release syndrome did not occur and neurological events were not reported, although underreporting of mild neurological symptoms that could not be identified in infants cannot be ruled out.
Following these results, the investigators believe that longer follow-up should be performed, but the lofollow-upsce of relapse after blinatumomab treatment is noteworthy, given that relapses occur frequently and early during treatment in historical controls.
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