Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma Journal Article
| Authors: | Pasquini, M. C.; Hu, Z. H.; Curran, K.; Laetsch, T.; Locke, F.; Rouce, R.; Pulsipher, M. A.; Phillips, C. L.; Keating, A.; Frigault, M. J.; Salzberg, D.; Jaglowski, S.; Sasine, J. P.; Rosenthal, J.; Ghosh, M.; Landsburg, D.; Margossian, S.; Martin, P. L.; Kamdar, M. K.; Hematti, P.; Nikiforow, S.; Turtle, C.; Perales, M. A.; Steinert, P.; Horowitz, M. M.; Moskop, A.; Pacaud, L.; Yi, L.; Chawla, R.; Bleickardt, E.; Grupp, S. |
| Article Title: | Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma |
| Abstract: | Tisagenlecleucel is a CD19 chimeric antigen receptor (CAR) T-cell therapy approved for treatment of pediatric and young adult patients with relapsed/refractory acute lymphoblastic leukemia (ALL) and adults with non-Hodgkin lymphoma (NHL). The initial experience with tisagenlecleucel in a real-world setting from a cellular therapy registry is presented here. As of January 2020, 511 patients were enrolled from 73 centers, and 410 patients had follow-up data reported (ALL, n 5 255; NHL, n 5 155), with a median follow-up of 13.4 and 11.9 months for ALL and NHL, respectively. Among patients with ALL, the initial complete remission (CR) rate was 85.5%. Twelve-month duration of response (DOR), event-free survival, and overall survival (OS) rates were 60.9%, 52.4%, and 77.2%, respectively. Among adults with NHL, the best overall response rate was 61.8%, including an initial CR rate of 39.5%. Six-month DOR, progression-free survival, and OS rates were 55.3%, 38.7%, and 70.7%, respectively. Grade $3 cytokine release syndrome and neurotoxicity were reported in 11.6% and 7.5% of all patients, respectively. Similar outcomes were observed in patients with in-specification and out-of-specification products as a result of viability,80% (range, 61% to 79%). This first report of tisagenlecleucel in the real-world setting demonstrates outcomes with similar efficacy and improved safety compared with those seen in the pivotal trials. © 2020 by The American Society of Hematology |
| Keywords: | adolescent; cancer survival; event free survival; treatment outcome; human cell; major clinical study; overall survival; fludarabine; neutropenia; cancer recurrence; drug efficacy; neurotoxicity; outcome assessment; t lymphocyte; cell viability; progression free survival; basal cell carcinoma; thrombocytopenia; oxygen; oxygen therapy; cytogenetics; cyclophosphamide; acute lymphoblastic leukemia; nonhodgkin lymphoma; drug response; cell therapy; cell count; corticosteroid; chromosome 21; philadelphia 1 chromosome; cytokine release syndrome; tocilizumab; blinatumomab; human; male; female; priority journal; article; positive pressure ventilation; tisagenlecleucel t |
| Journal Title: | Blood Advances |
| Volume: | 4 |
| Issue: | 21 |
| ISSN: | 2473-9529 |
| Publisher: | American Society of Hematology |
| Date Published: | 2020-11-10 |
| Start Page: | 5414 |
| End Page: | 5424 |
| Language: | English |
| DOI: | 10.1182/bloodadvances.2020003092 |
| PUBMED: | 33147337 |
| PROVIDER: | scopus |
| PMCID: | PMC7656920 |
| DOI/URL: | |
| Notes: | Article -- Export Date: 4 January 2021 -- Source: Scopus |
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