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ASH 2020 | Out of specification tisagenlecleucel in real-world treatment of pediatric B-ALL

Jenna Rossoff, MD, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, shares the findings of a retrospective study of outcomes in pediatric and young adult B-cell acute lymphoblastic leukemia patients (B-ALL) who received out of specification (OOS) tisagenlecleucel. OOS tisangenlecleucel – that is tisangenlecleucel not meeting commercial release specifications- can be provided through the Managed Access Program (MAP) or a single-patient Investigational New Drug (IND) application. 185 relapsed/refractory B-ALL patients treated outside of clinical trials were identified for inclusion from the Pediatric Real-World CAR Consortium database, of whom 13% were categorized as MAP/IND. The results showed that 12-month overall survival was 83% in the MAP/IND cohort compared to 70% in those receiving products that met all criteria. Event-free survival and probability of continued remission results were closely comparable between both arms, as were toxicity results. The study suggests the use of OSS products does not compromise the efficacy or safety of tisagenlecleucel. This interview took place during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, 2020.

Transcript (edited for clarity)

I am presenting, like you mentioned, a sub-analysis of our greater pediatric real-world CAR consortium effort that involves 15 U.S. institutions and 185 pediatric and young adult patients who were infused commercially with tisagenlecleucel, obviously outside of a clinical trial to evaluate the outcomes and compare them to those seen in trials, such as the ELIANA trial.

My sub-analysis looks at the 24 patients, which is about 13% of our cohort that received their product either through the manage access program or their physician applied for a single patient IND because their product didn’t meet a variety of commercial specifications, the most common one being cell viability less than 80%, which is one of the FDA requirements for commercial release of tisagenlecleucel...

I am presenting, like you mentioned, a sub-analysis of our greater pediatric real-world CAR consortium effort that involves 15 U.S. institutions and 185 pediatric and young adult patients who were infused commercially with tisagenlecleucel, obviously outside of a clinical trial to evaluate the outcomes and compare them to those seen in trials, such as the ELIANA trial.

My sub-analysis looks at the 24 patients, which is about 13% of our cohort that received their product either through the manage access program or their physician applied for a single patient IND because their product didn’t meet a variety of commercial specifications, the most common one being cell viability less than 80%, which is one of the FDA requirements for commercial release of tisagenlecleucel.

And so what this analysis looked at is comparing outcomes of patients who received standard of care tisagenlecleucel, meaning they met all commercial release specifications to the 24 patients who received it through the manage access program or a single patient IND, as this is a very common real-world problem where say cell viability is just under the threshold of 80% to see are these products still safe and efficacious.

And overall, we found that they were. Both overall survival and event-free survival at 6 and 12 months were not different between the two groups. For those patients who achieved a CR at the 20 day evaluation, there was not a difference in the probability of continued remission at 12 months for these patients. So overall looks at efficacy. They are fairly similar between these two groups.

And then in terms of toxicities, there were also none, no differences there, no difference in CRS of any grade, severe CRS. So just looking at grade three or greater, no difference in I-CANS, and no difference in infectious complications.

So overall we concluded that the use of tisagenlecleucel products that are slightly outside of the specifications needed for commercial release is still safe and efficacious. The disclaimer to that is there are further studies that should be done to look at what cutoffs are actually needed to preserve this safety and efficacy.

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