Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma
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...
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| Veröffentlicht in: | Blood advances 2020-11, Vol.4 (21), p.5414-5424 |
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| creator | Pasquini, Marcelo C. Hu, Zhen-Huan Curran, Kevin Laetsch, Theodore Locke, Frederick Rouce, Rayne Pulsipher, Michael A. Phillips, Christine L. Keating, Amy Frigault, Matthew J. Salzberg, Dana Jaglowski, Samantha Sasine, Joshua P. Rosenthal, Joseph Ghosh, Monalisa Landsburg, Daniel Margossian, Steven Martin, Paul L. Kamdar, Manali K. Hematti, Peiman Nikiforow, Sarah Turtle, Cameron Perales, Miguel-Angel Steinert, Patricia Horowitz, Mary M. Moskop, Amy Pacaud, Lida Yi, Lan Chawla, Raghav Bleickardt, Eric Grupp, Stephan |
| description | 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 = 255; NHL, n = 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 |
| doi_str_mv | 10.1182/bloodadvances.2020003092 |
| format | Article |
| fullrecord | <record><control><sourceid>elsevier_pubme</sourceid><recordid>TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_7656920</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><els_id>S2473952920319364</els_id><sourcerecordid>S2473952920319364</sourcerecordid><originalsourceid>FETCH-LOGICAL-c600t-9189bede982b15f25fad581b58f5a22dd4b3ffaeb570b84b5a9e2e6d74971ea43</originalsourceid><addsrcrecordid>eNqFkV9rFDEUxYMottR-BckXmJo_k5nJi6BFrVAQRJ_DTXKzjZuZLMnsSr99U7au9smnXHJ_51w4hxDK2RXnk3hnU84e_AEWh_VKMMEYk0yLF-Rc9KPstJLjy9Ms9Bm5rPVXg_g4SKXFa3ImJW9bOZ6T7XeE1P3OJXmKh-ixmdIc6BorbHBJ6BLuHSYacqE79BHWEh0Ft1-Rpvt5d5dtgrq2vwZucY5AYfF0yUt3k_1mG5cnbIY35FWAVPHy6b0gPz9_-nF9091--_L1-sNt5wbG1k7zSVv0qCdhuQpCBfBq4lZNQYEQ3vdWhgBo1cjs1FsFGgUOfuz1yBF6eUHeH313ezujd7isBZLZlThDuTcZonm-WeKd2eSDGQc1aMGawXQ0cCXXWjCctJyZxw7Msw7M3w6a9O2_t0_CP4k34OMRwJbAIWIx1cXH1H0s6Fbjc_z_lQeBn6Ie</addsrcrecordid><sourcetype>Open Access Repository</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype></control><display><type>article</type><title>Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma</title><source>MEDLINE</source><source>DOAJ Directory of Open Access Journals</source><source>Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals</source><source>PubMed Central</source><source>Alma/SFX Local Collection</source><creator>Pasquini, Marcelo C. ; Hu, Zhen-Huan ; Curran, Kevin ; Laetsch, Theodore ; Locke, Frederick ; Rouce, Rayne ; Pulsipher, Michael A. ; Phillips, Christine L. ; Keating, Amy ; Frigault, Matthew J. ; Salzberg, Dana ; Jaglowski, Samantha ; Sasine, Joshua P. ; Rosenthal, Joseph ; Ghosh, Monalisa ; Landsburg, Daniel ; Margossian, Steven ; Martin, Paul L. ; Kamdar, Manali K. ; Hematti, Peiman ; Nikiforow, Sarah ; Turtle, Cameron ; Perales, Miguel-Angel ; Steinert, Patricia ; Horowitz, Mary M. ; Moskop, Amy ; Pacaud, Lida ; Yi, Lan ; Chawla, Raghav ; Bleickardt, Eric ; Grupp, Stephan</creator><creatorcontrib>Pasquini, Marcelo C. ; Hu, Zhen-Huan ; Curran, Kevin ; Laetsch, Theodore ; Locke, Frederick ; Rouce, Rayne ; Pulsipher, Michael A. ; Phillips, Christine L. ; Keating, Amy ; Frigault, Matthew J. ; Salzberg, Dana ; Jaglowski, Samantha ; Sasine, Joshua P. ; Rosenthal, Joseph ; Ghosh, Monalisa ; Landsburg, Daniel ; Margossian, Steven ; Martin, Paul L. ; Kamdar, Manali K. ; Hematti, Peiman ; Nikiforow, Sarah ; Turtle, Cameron ; Perales, Miguel-Angel ; Steinert, Patricia ; Horowitz, Mary M. ; Moskop, Amy ; Pacaud, Lida ; Yi, Lan ; Chawla, Raghav ; Bleickardt, Eric ; Grupp, Stephan</creatorcontrib><description>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 = 255; NHL, n = 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.
•Represents the largest set of safety and efficacy data for tisagenlecleucel.•Outcomes in the real-world setting are similar to results in the pivotal trials.
[Display omitted]</description><identifier>ISSN: 2473-9529</identifier><identifier>EISSN: 2473-9537</identifier><identifier>DOI: 10.1182/bloodadvances.2020003092</identifier><identifier>PMID: 33147337</identifier><language>eng</language><publisher>United States: Elsevier Inc</publisher><subject>Adult ; Antigens, CD19 ; Humans ; Immunobiology and Immunotherapy ; Immunotherapy, Adoptive ; Lymphoma, Non-Hodgkin - therapy ; Precursor Cell Lymphoblastic Leukemia-Lymphoma - therapy ; Receptors, Antigen, T-Cell - therapeutic use</subject><ispartof>Blood advances, 2020-11, Vol.4 (21), p.5414-5424</ispartof><rights>2020 American Society of Hematology</rights><rights>2020 by The American Society of Hematology.</rights><rights>2020 by The American Society of Hematology 2020</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c600t-9189bede982b15f25fad581b58f5a22dd4b3ffaeb570b84b5a9e2e6d74971ea43</citedby><cites>FETCH-LOGICAL-c600t-9189bede982b15f25fad581b58f5a22dd4b3ffaeb570b84b5a9e2e6d74971ea43</cites><orcidid>0000-0002-0863-5655 ; 0000-0002-8497-3138 ; 0000-0003-3030-8420 ; 0000-0001-9866-462X ; 0000-0002-4335-2554 ; 0000-0002-7803-2366 ; 0000-0003-1579-2293</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC7656920/pdf/$$EPDF$$P50$$Gpubmedcentral$$H</linktopdf><linktohtml>$$Uhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC7656920/$$EHTML$$P50$$Gpubmedcentral$$H</linktohtml><link.rule.ids>230,314,724,777,781,861,882,27905,27906,53772,53774</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/33147337$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Pasquini, Marcelo C.</creatorcontrib><creatorcontrib>Hu, Zhen-Huan</creatorcontrib><creatorcontrib>Curran, Kevin</creatorcontrib><creatorcontrib>Laetsch, Theodore</creatorcontrib><creatorcontrib>Locke, Frederick</creatorcontrib><creatorcontrib>Rouce, Rayne</creatorcontrib><creatorcontrib>Pulsipher, Michael A.</creatorcontrib><creatorcontrib>Phillips, Christine L.</creatorcontrib><creatorcontrib>Keating, Amy</creatorcontrib><creatorcontrib>Frigault, Matthew J.</creatorcontrib><creatorcontrib>Salzberg, Dana</creatorcontrib><creatorcontrib>Jaglowski, Samantha</creatorcontrib><creatorcontrib>Sasine, Joshua P.</creatorcontrib><creatorcontrib>Rosenthal, Joseph</creatorcontrib><creatorcontrib>Ghosh, Monalisa</creatorcontrib><creatorcontrib>Landsburg, Daniel</creatorcontrib><creatorcontrib>Margossian, Steven</creatorcontrib><creatorcontrib>Martin, Paul L.</creatorcontrib><creatorcontrib>Kamdar, Manali K.</creatorcontrib><creatorcontrib>Hematti, Peiman</creatorcontrib><creatorcontrib>Nikiforow, Sarah</creatorcontrib><creatorcontrib>Turtle, Cameron</creatorcontrib><creatorcontrib>Perales, Miguel-Angel</creatorcontrib><creatorcontrib>Steinert, Patricia</creatorcontrib><creatorcontrib>Horowitz, Mary M.</creatorcontrib><creatorcontrib>Moskop, Amy</creatorcontrib><creatorcontrib>Pacaud, Lida</creatorcontrib><creatorcontrib>Yi, Lan</creatorcontrib><creatorcontrib>Chawla, Raghav</creatorcontrib><creatorcontrib>Bleickardt, Eric</creatorcontrib><creatorcontrib>Grupp, Stephan</creatorcontrib><title>Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma</title><title>Blood advances</title><addtitle>Blood Adv</addtitle><description>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 = 255; NHL, n = 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.
•Represents the largest set of safety and efficacy data for tisagenlecleucel.•Outcomes in the real-world setting are similar to results in the pivotal trials.
[Display omitted]</description><subject>Adult</subject><subject>Antigens, CD19</subject><subject>Humans</subject><subject>Immunobiology and Immunotherapy</subject><subject>Immunotherapy, Adoptive</subject><subject>Lymphoma, Non-Hodgkin - therapy</subject><subject>Precursor Cell Lymphoblastic Leukemia-Lymphoma - therapy</subject><subject>Receptors, Antigen, T-Cell - therapeutic use</subject><issn>2473-9529</issn><issn>2473-9537</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2020</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNqFkV9rFDEUxYMottR-BckXmJo_k5nJi6BFrVAQRJ_DTXKzjZuZLMnsSr99U7au9smnXHJ_51w4hxDK2RXnk3hnU84e_AEWh_VKMMEYk0yLF-Rc9KPstJLjy9Ms9Bm5rPVXg_g4SKXFa3ImJW9bOZ6T7XeE1P3OJXmKh-ixmdIc6BorbHBJ6BLuHSYacqE79BHWEh0Ft1-Rpvt5d5dtgrq2vwZucY5AYfF0yUt3k_1mG5cnbIY35FWAVPHy6b0gPz9_-nF9091--_L1-sNt5wbG1k7zSVv0qCdhuQpCBfBq4lZNQYEQ3vdWhgBo1cjs1FsFGgUOfuz1yBF6eUHeH313ezujd7isBZLZlThDuTcZonm-WeKd2eSDGQc1aMGawXQ0cCXXWjCctJyZxw7Msw7M3w6a9O2_t0_CP4k34OMRwJbAIWIx1cXH1H0s6Fbjc_z_lQeBn6Ie</recordid><startdate>20201110</startdate><enddate>20201110</enddate><creator>Pasquini, Marcelo C.</creator><creator>Hu, Zhen-Huan</creator><creator>Curran, Kevin</creator><creator>Laetsch, Theodore</creator><creator>Locke, Frederick</creator><creator>Rouce, Rayne</creator><creator>Pulsipher, Michael A.</creator><creator>Phillips, Christine L.</creator><creator>Keating, Amy</creator><creator>Frigault, Matthew J.</creator><creator>Salzberg, Dana</creator><creator>Jaglowski, Samantha</creator><creator>Sasine, Joshua P.</creator><creator>Rosenthal, Joseph</creator><creator>Ghosh, Monalisa</creator><creator>Landsburg, Daniel</creator><creator>Margossian, Steven</creator><creator>Martin, Paul L.</creator><creator>Kamdar, Manali K.</creator><creator>Hematti, Peiman</creator><creator>Nikiforow, Sarah</creator><creator>Turtle, Cameron</creator><creator>Perales, Miguel-Angel</creator><creator>Steinert, Patricia</creator><creator>Horowitz, Mary M.</creator><creator>Moskop, Amy</creator><creator>Pacaud, Lida</creator><creator>Yi, Lan</creator><creator>Chawla, Raghav</creator><creator>Bleickardt, Eric</creator><creator>Grupp, Stephan</creator><general>Elsevier Inc</general><general>American Society of Hematology</general><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>5PM</scope><orcidid>https://orcid.org/0000-0002-0863-5655</orcidid><orcidid>https://orcid.org/0000-0002-8497-3138</orcidid><orcidid>https://orcid.org/0000-0003-3030-8420</orcidid><orcidid>https://orcid.org/0000-0001-9866-462X</orcidid><orcidid>https://orcid.org/0000-0002-4335-2554</orcidid><orcidid>https://orcid.org/0000-0002-7803-2366</orcidid><orcidid>https://orcid.org/0000-0003-1579-2293</orcidid></search><sort><creationdate>20201110</creationdate><title>Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma</title><author>Pasquini, Marcelo C. ; Hu, Zhen-Huan ; Curran, Kevin ; Laetsch, Theodore ; Locke, Frederick ; Rouce, Rayne ; Pulsipher, Michael A. ; Phillips, Christine L. ; Keating, Amy ; Frigault, Matthew J. ; Salzberg, Dana ; Jaglowski, Samantha ; Sasine, Joshua P. ; Rosenthal, Joseph ; Ghosh, Monalisa ; Landsburg, Daniel ; Margossian, Steven ; Martin, Paul L. ; Kamdar, Manali K. ; Hematti, Peiman ; Nikiforow, Sarah ; Turtle, Cameron ; Perales, Miguel-Angel ; Steinert, Patricia ; Horowitz, Mary M. ; Moskop, Amy ; Pacaud, Lida ; Yi, Lan ; Chawla, Raghav ; Bleickardt, Eric ; Grupp, Stephan</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c600t-9189bede982b15f25fad581b58f5a22dd4b3ffaeb570b84b5a9e2e6d74971ea43</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2020</creationdate><topic>Adult</topic><topic>Antigens, CD19</topic><topic>Humans</topic><topic>Immunobiology and Immunotherapy</topic><topic>Immunotherapy, Adoptive</topic><topic>Lymphoma, Non-Hodgkin - therapy</topic><topic>Precursor Cell Lymphoblastic Leukemia-Lymphoma - therapy</topic><topic>Receptors, Antigen, T-Cell - therapeutic use</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Pasquini, Marcelo C.</creatorcontrib><creatorcontrib>Hu, Zhen-Huan</creatorcontrib><creatorcontrib>Curran, Kevin</creatorcontrib><creatorcontrib>Laetsch, Theodore</creatorcontrib><creatorcontrib>Locke, Frederick</creatorcontrib><creatorcontrib>Rouce, Rayne</creatorcontrib><creatorcontrib>Pulsipher, Michael A.</creatorcontrib><creatorcontrib>Phillips, Christine L.</creatorcontrib><creatorcontrib>Keating, Amy</creatorcontrib><creatorcontrib>Frigault, Matthew J.</creatorcontrib><creatorcontrib>Salzberg, Dana</creatorcontrib><creatorcontrib>Jaglowski, Samantha</creatorcontrib><creatorcontrib>Sasine, Joshua P.</creatorcontrib><creatorcontrib>Rosenthal, Joseph</creatorcontrib><creatorcontrib>Ghosh, Monalisa</creatorcontrib><creatorcontrib>Landsburg, Daniel</creatorcontrib><creatorcontrib>Margossian, Steven</creatorcontrib><creatorcontrib>Martin, Paul L.</creatorcontrib><creatorcontrib>Kamdar, Manali K.</creatorcontrib><creatorcontrib>Hematti, Peiman</creatorcontrib><creatorcontrib>Nikiforow, Sarah</creatorcontrib><creatorcontrib>Turtle, Cameron</creatorcontrib><creatorcontrib>Perales, Miguel-Angel</creatorcontrib><creatorcontrib>Steinert, Patricia</creatorcontrib><creatorcontrib>Horowitz, Mary M.</creatorcontrib><creatorcontrib>Moskop, Amy</creatorcontrib><creatorcontrib>Pacaud, Lida</creatorcontrib><creatorcontrib>Yi, Lan</creatorcontrib><creatorcontrib>Chawla, Raghav</creatorcontrib><creatorcontrib>Bleickardt, Eric</creatorcontrib><creatorcontrib>Grupp, Stephan</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Blood advances</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Pasquini, Marcelo C.</au><au>Hu, Zhen-Huan</au><au>Curran, Kevin</au><au>Laetsch, Theodore</au><au>Locke, Frederick</au><au>Rouce, Rayne</au><au>Pulsipher, Michael A.</au><au>Phillips, Christine L.</au><au>Keating, Amy</au><au>Frigault, Matthew J.</au><au>Salzberg, Dana</au><au>Jaglowski, Samantha</au><au>Sasine, Joshua P.</au><au>Rosenthal, Joseph</au><au>Ghosh, Monalisa</au><au>Landsburg, Daniel</au><au>Margossian, Steven</au><au>Martin, Paul L.</au><au>Kamdar, Manali K.</au><au>Hematti, Peiman</au><au>Nikiforow, Sarah</au><au>Turtle, Cameron</au><au>Perales, Miguel-Angel</au><au>Steinert, Patricia</au><au>Horowitz, Mary M.</au><au>Moskop, Amy</au><au>Pacaud, Lida</au><au>Yi, Lan</au><au>Chawla, Raghav</au><au>Bleickardt, Eric</au><au>Grupp, Stephan</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma</atitle><jtitle>Blood advances</jtitle><addtitle>Blood Adv</addtitle><date>2020-11-10</date><risdate>2020</risdate><volume>4</volume><issue>21</issue><spage>5414</spage><epage>5424</epage><pages>5414-5424</pages><issn>2473-9529</issn><eissn>2473-9537</eissn><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 = 255; NHL, n = 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.
•Represents the largest set of safety and efficacy data for tisagenlecleucel.•Outcomes in the real-world setting are similar to results in the pivotal trials.
[Display omitted]</abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>33147337</pmid><doi>10.1182/bloodadvances.2020003092</doi><tpages>11</tpages><orcidid>https://orcid.org/0000-0002-0863-5655</orcidid><orcidid>https://orcid.org/0000-0002-8497-3138</orcidid><orcidid>https://orcid.org/0000-0003-3030-8420</orcidid><orcidid>https://orcid.org/0000-0001-9866-462X</orcidid><orcidid>https://orcid.org/0000-0002-4335-2554</orcidid><orcidid>https://orcid.org/0000-0002-7803-2366</orcidid><orcidid>https://orcid.org/0000-0003-1579-2293</orcidid><oa>free_for_read</oa></addata></record> |
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| source | MEDLINE; DOAJ Directory of Open Access Journals; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals; PubMed Central; Alma/SFX Local Collection |
| subjects | Adult Antigens, CD19 Humans Immunobiology and Immunotherapy Immunotherapy, Adoptive Lymphoma, Non-Hodgkin - therapy Precursor Cell Lymphoblastic Leukemia-Lymphoma - therapy Receptors, Antigen, T-Cell - therapeutic use |
| title | Real-world evidence of tisagenlecleucel for pediatric acute lymphoblastic leukemia and non-Hodgkin lymphoma |
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