2020
DOI: 10.3390/ph13090258
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Tisagenlecleucel in Children and Young Adults: Reverse Translational Research by Using Real-World Safety Data

Abstract: Tisagenlecleucel has revolutionized the pharmacological approach of relapsed or refractory B-cell acute lymphoblastic leukaemialeukaemia in paediatrics. The safety profile of tisagenlecleucel still needs to be better defined. The aim of this study was a post-marketing evaluation of the safety of tisagenlecleucel through the analysis of the Eudravigilance database with focus on the paediatric population. From 2017 to 2020, one third of Individual Case Safety Reports referring to tisagenlecleucel (117/364) have … Show more

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Cited by 7 publications
(4 citation statements)
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References 32 publications
(45 reference statements)
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“…), suspected medications, codified by ATC classification, indication of use, dosage, and concomitant/interacting medications can be retrieved as a line listing and/or ICSR form. These data are publicly available for transparency through the EMA website ( (accessed on 12 December 2020)) and have been previously described as a valid system for safety surveillance studies [ 39 , 40 , 41 ].…”
Section: Methodsmentioning
confidence: 99%
“…), suspected medications, codified by ATC classification, indication of use, dosage, and concomitant/interacting medications can be retrieved as a line listing and/or ICSR form. These data are publicly available for transparency through the EMA website ( (accessed on 12 December 2020)) and have been previously described as a valid system for safety surveillance studies [ 39 , 40 , 41 ].…”
Section: Methodsmentioning
confidence: 99%
“…Although these strengths exist, several limitations need to be considered when interpreting this type of result. Under-reporting is highly likely in a spontaneous report system and reported adverse events may only be the tip of the iceberg, especially in paediatric patients [ 35 , 36 , 37 ]. Furthermore, ICSRs vary in quality and completeness.…”
Section: Strengths and Limitationsmentioning
confidence: 99%
“…In Europe, an accelerated assessment application through the Priority Medicines (PRIME) scheme for orphan diseases has been granted for CAR-T cells and additional monitoring and post-authorization safety studies have been required by the European Medicines Agency [12] after marketing authorization. Increasing post-marketing data have been recorded for CAR-T-cell safety profile monitoring in a real-world setting, where a significantly larger and heterogeneous population of patients has been treated [13][14][15][16].…”
Section: Introductionmentioning
confidence: 99%