Dr. Pasquini on Tisagenlecleucel in Real-World and Clinical Trial Settings in ALL and DLBCL

Marcelo Pasquini, MD, associate professor of medicine, Medical College of Wisconsin, Center for International Blood and Marrow Transplant Research, discusses the similarities between real-world experience with tisagenlecleucel (Kymriah) and in the ELIANA and JULIET trials.

The real-world study was a registry-based analysis; results were found to be similar when compared with the pivotal ELIANA and JULIET trials on pediatric and young adult patients with acute lymphoblastic leukemia (ALL) and adults with relapsed/refractory diffuse large B-cell lymphoma. Researchers noticed similar patient populations in the studies. For example, in the ELIANA trial, patients were ≤21 years old; in the real-world study, patients were ≤26 years old.

The JULIET trial enrolled patients ≥18 years old and there was a higher proportion of patients with high-risk B-cell malignancies than in the real-world tisagenlecleucel trial. This has to be taken into account when assessing the effect of tisagenlecleucel because now researchers have a population that is much more aggressive in disease, says Pasquini.

In terms of outcomes, the safety captured in the real-world tisagenlecleucel trial had very similar rates compared with that of the pivotal trials. Although, one of the interesting aspects with the current analysis is that there were not as many cases with severe cytokine release syndrome and neurotoxicity as the JULIET and ELIANA trials, concludes Pasquini.