CAR-T treatment is safe and effective in adult patients with Down syndrome and B-cell acute lymphoblastic leukemia (B-ALL)

We reviewed data from PubMed including both randomized and real-life studies filtering for “CAR-T” and “acute lymphoblastic leukemia” between January 2018 and May 2024. We reviewed 90 papers, among which only 4 reported data on 18 DS patients (age 3-21 years) with B-ALL treated with CAR-T (tisa-cel in 17 patients, a CD19.28zeta-CAR T in 1 patient) [1, 14, 15]. In all studies, the authors observed no differences in overall response rate and early and late CAR-T toxicities between patients with or without DS. Adult patients with DS were admitted in the ZUMA-3 trial with KTE-X19, but specific data on those patients are not disclaimed by the authors. Here, we present two cases of adult patients with DS (summarized in Table 1) affected by B-ALL safely treated with brexucabtagene autoleucel, based on data reported in the ZUMA-3 trial.