Dr Cuglievan on Results for Revumenib in Pediatric R/R KMT2A+ Acute Leukemia

“To see some of these [pediatric patients with R/R KMT2AR-acute leukemias] alive now is amazing. The key now is to move [revumenib] to the frontline.”

Branko Cuglievan, MD, an associate professor in the Department of Leukemia, associate professor in the Department of Pediatrics, and section chief of Department of Pediatrics Patient Care, all of which are at The University of Texas MD Anderson Cancer Center, discussed long-term follow up data from the phase 1/2 AUGMENT-101 trial (NCT04065399) for the oral menin inhibitor, revumenib [Revuforj] in pediatric patients with relapsed or refractory acute leukemias with KMT2A-rearrangements which were presented at the 2026 EHA Congress. In addition to outlining the data from the trial, Cuglievan also dove into how these data will impact the pediatric relapsed or refractory KMT2A-rearranged treatment paradigm.

Among patients in the pediatric and young adult subgroup in the trial who received revumenib (n = 36), the complete response (CR)/CR with partial hematologic recovery (CRh) rate was 27.8% (95% CI, 14.2%-45.2%), with a median duration of 32.3 months (95% CI, 1.8-not reached [NR]). Additionally patient achieved amedian time to CR/CRh of 1.9 months (95% CI, 1.0-5.6) with the composite CR (CRc) rate being 30.6% (95% CI, 16.3%-48.1%). Additionally, the overall response rate (ORR) for these patients was 55.6% (95% CI, 38.1%-72.1%), the median event-free survival and median overall survival being 3.6 months (95% CI, 1.9-6.6) and 6.4 months (95% CI, 2.9-34.2), respectively.

Cuglievan noted that most patients in the subgroup had acute myeloid leukemia (AML) while less had acute lymphoblastic leukemia (ALL), pointing out that patients with AML had slightly better CR/CRh rates than those with ALL. Although, patients with mixed-phenotype acute leukemia also demonstrated strong CR/CRh rates, he added. Cuglievan then underscored how many patients in this subgroup were able to receive transplants and of those who did receive transplants, many were able to continue revumenib treatments.

Ultimately, Cuglievan concluded by emphasizing the fact that most of the patients in the pediatric and young adult subgroup did not experience death and that the next step for revumenib is in the frontline for these patients.