Molecular, Cytogenic Study Brings Snapshot of Myelodysplastic Neoplasms in Youths

A single-center retrospective analysis was recently conducted to bring insight into myelodysplastic neoplasms among pediatric, adolescent, and young adult patients. Its findings were published in Leukemia Research.

“These findings build on previously reported findings and encourage the use of [stem cell transplant (SCT)] along with molecular and cytogenetic analysis,” wrote lead analyst David McCall, MD, of the University of Texas MD Anderson Cancer Center in Houston.

The analysis examined molecular and cytogenic data from a cohort of 119 pediatric, adolescent, and young adult patients with myelodysplastic neoplasms, excluding individuals with bone marrow failure syndromes.

The 5-year overall survival (OS) rate calculated for the total cohort was 45%, and the analysts found the median OS rate to be shorter in patients younger than 18 years. Acute myeloid leukemia manifested in 31% of the cohort.

The analysis found that, unlike in young adult or older adult patients with myelodysplastic neoplasms, mutations were uncommon among pediatric patients. However, the analysts determined that monosomy 7 or 7q deletion in patients was associated with a significantly shorter median OS rate, while having isolated deletion of 5q or TET2 mutation was associated with a longer median OS.

Therapy-related myelodysplastic neoplasms were identified in 36% of the cohort and were associated with significantly shorter median OS in patients.

Evaluating the effect of SCT, the analysts found that patients who had undergone the procedure at any time had significantly longer median OS. This included patients with de novo myelodysplastic neoplasms, though the analysts also observed in this subgroup that patient OS was not meaningfully affected by whether intensive chemotherapy, hypomethylating agents, or SCT was chosen as the initial treatment.

Dr. McCall wrote that the most favorable OS outcomes in the study cohort were observed in patients who received hematopoietic SCT. He explained, however, that “[m]ore research is needed to determine the optimal timing of hematopoietic SCT, frontline chemotherapy regimens, and the use of maintenance therapy following intensive induction chemotherapy or SCT to induce a deeper remission with improved event-free survival and OS rates.”

Reference

McCall D, Abuasab T, Rodriguez-Sevilla JJ, et al. Characteristics and outcomes of children, adolescent, and young adult patients with myelodysplastic neoplasms: A single-center retrospective analysis. Leuk Res. 2024;144:107563. doi:10.1016/j.leukres.2024.107563