Carfilzomib plus induction chemotherapy for children with relapsed or refractory acute lymphoblastic leukemia.
Franco Locatelli, Musa Karakukcu, Van Huynh et al.
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In 30 seconds
This phase II clinical trial evaluated the efficacy of carfilzomib combined with induction chemotherapy (CFZ-VXLD) in pediatric patients aged 1 month to <21 years with relapsed or refractory acute lymphoblastic leukemia (ALL). The study found postinduction complete remission (CR) rates of 14.8% for CFZ-VXLD versus 7.8% for an external control, but did not meet its primary endpoint of statistical significance.
Key findings
- Postinduction CR rates were 14.8% for CFZ-VXLD vs 7.8% for external control in B-cell ALL.
- In T-cell ALL, CR rates were 13.6% for CFZ-VXLD vs 9.1% for external control.
- Overall response rate (ORR) was 42.6% for CFZ-VXLD vs 26.3% for external control in B-cell ALL.
Why it matters
The study underscores the challenges in treating relapsed or refractory ALL in children, highlighting the need for effective therapies. While CFZ-VXLD showed some promise, the lack of significant improvement in CR rates indicates ongoing unmet medical needs in this population.