Blinatumomab promising for treatment of aggressive infant leukemia with 81.6% disease-free survival rate: NEJM

Netherlands: In a study conducted by researchers, Blinatumomab, a bispecific T-cell engager molecule, to be safe and efficacious for infants with newly diagnosed KMT2A- rearranged acute lymphoblastic leukemia (ALL). The study was published in NEJM Medicine.

KMT2A-rearranged acute lymphoblastic leukemia (ALL) in neonates is a deadly disease with a three-year event-free survival rate of less than 40%. Relapses occur in two-thirds of patients within a year and 90% within 2 years of diagnosis. The study aimed to study the safety and efficacy of the Blinatumomab molecule targeting CD19 in infants with KMT2A-rearranged ALL.

The study administered one postinduction course of blinatumomab to 30 infants with KMT2A-rearranged ALL, alongside Interfant-06 chemotherapy. The primary endpoint was safety, and minimal residual disease was measured by polymerase chain reaction. Data on adverse events were collected and compared with historical control data from the Interfant-06 trial.

The following clinical findings were revealed in the study:

1. No toxic effects met the primary endpoint.

2. Ten serious adverse events were reported, consistent with those reported in older patients.

3. 93% of patients were MRD-negative or had low levels of MRD after blinatumomab infusion.

4. All patients who continued chemotherapy became MRD-negative during further treatment.

5. Two-year disease-free survival was 81.6%, compared to 49.4% in the Interfant-06 trial.

6. Corresponding values for overall survival were 93.3% compared to 65.8% in the Interfant-06 trial.

Blinatumomab, which targets CD19, has shown promising results in treating KMT2A-rearranged ALL in adults, and this study shows that it may be a viable treatment option for infants as well.

“The study's results are compared with historical control data from the Interfant-06 trial, and blinatumomab showed a significant improvement in disease-free survival and overall survival rates. The findings suggest that blinatumomab may be an effective treatment option for infants with KMT2A-rearranged ALL, and further studies may be conducted to validate the results.” said the researchers of the study.

Reference:

Van der Sluis I., de Lorenzo P., Kotecha R., Attarbaschi A., et al.; Blinatumomab Added to Chemotherapy in Infant Lymphoblastic Leukemia; N Engl J Med 2023; 388:1572-1581; doi: 10.1056/NEJMoa2214171