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The ELIANA trial with tisagenlecleucel, a CD19-specific chimeric antigen receptor T-cell therapy, demonstrated an 81% complete response rate in 75 patients with recurrent pediatric acute lymphoblastic leukemia (ALL), leading to its FDA approval in 2017. now report on "real-world" outcomes in patients with relapsed/refractory pediatric ALL, now almost 5 years out since the initial approval.

In this multicenter retrospective cohort, the authors demonstrate a similar morphologic complete response (CR) rate of 79% compared with the patients on the ELIANA trial (81%); however, this is a bit misleading as almost half the patients in the retrospective cohort had a low burden of disease (< 5% bone marrow lymphoblasts), which was excluded in the ELIANA trial. When comparing the CR of patients with ≥ 5% lymphoblasts, without central nervous system or extramedullary disease, which is most similar to those enrolled on ELIANA, the CR rate fell to 54%, well below the 81% reported on ELIANA.

This is not surprising, given that real-world results are often inferior to trial results, as patients in real-world cohorts often have more comorbidities or higher disease burdens, which may have been disqualifying for trial enrollment.

This retrospective report does highlight the importance of obtaining a low disease burden before heading into CAR T cell therapy, as patients with a high disease burden in this report had worse outcomes and more toxicity.

Michael Kinnaman, MD, is a hematologist/oncologist at Memorial Sloan Kettering Cancer Center in New York City.

Read the study here and an interview about it here.