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Posted by on Dec 5, 2020 in Leukemia | 0 comments

In a nutshell

This study aimed to investigate the safety and effectiveness of tisagenlecleucel (TL; Kymriah) in a real-world setting as a treatment for acute lymphoblastic leukemia (ALL) and non-Hodgkin lymphoma (NHL).  

This study concluded that TL has similar effectiveness and improved safety in a real-world setting when compared to a trial setting for these patients.  

Some background

TL is a type of immunotherapy called a chimeric antigen receptor T (CAR-T) cell therapy. It involves taking the patient's T cells (a type of immune cell) and modifying them in the laboratory to detect and kill cancer cells. TL has been approved for the treatment of children and young adults with relapsed/refractory (r/r) ALL and for adults with r/r NHL.

This approval was based on results from clinical trials. In clinical trials, most patients selected to participate commonly have no other medical conditions and are in general good health apart from the ALL or NHL. Therefore, the safety and effectiveness of TL in a real-world setting, remain unknown. 

Methods & findings

This study involved 410 patients. 255 patients had r/r ALL and 155 had r/r NHL. All participants received TL therapy. Patients with ALL had an average follow-up of 13.4 months. Patients with NHL had an average follow-up of 11.9 months.  

Of the ALL group, 85.5% of patients had a complete remission (CR – all signs of cancer gone). Of these patients, 99.1% were minimal residual disease (MRD) negative. MRD is the small number of cancer cells left after treatment. MRD negative means there were no cancer cells detected after therapy. After 12 months, 60.9% of patients were still responding to treatment and 77.2% of patients were alive. 

For the NHL group, 61.8% of patients responded to treatment. 39.5% of these had a CR. After 6 months, 55.3% of patients were still responding to treatment and 70.7% were alive.  

55% of the ALL group and 45% of the NHL group experienced cytokine release syndrome (CRS). CRS is a side effect of TL that involves the immune system becoming highly active. Symptoms include fever, low blood pressure, or high heartbeat. Severe CRS was reported in 16.1% of the ALL group and 4.5% of the NHL group. Side effects of the nervous system also occurred. Severe nerve toxicity was reported in 9% of the ALL group and 5.1% of the NHL group.

The bottom line

This study concluded that TL has similar effectiveness and improved safety in a real-world setting when compared to a clinical trial setting for pediatric patients with ALL and adults with NHL.  

The fine print

Some information regarding other therapies were missing. This might have influenced the results.

Published By :

Blood advances

Date :

Nov 10, 2020

Original Title :

Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma.

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