Outcomes of patients with solid tumour malignancies treated with first-line immuno-oncology agents who do not meet eligibility criteria for clinical trials
Immuno-oncology (IO)–based therapies have been approved based on randomised clinical trials, yet a significant proportion of real-world patients are not represented in these trials. We sought to compare the outcomes of trial-ineligible vs. -eligible patients with advanced solid tumours treated with...
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| Veröffentlicht in: | European journal of cancer (1990) 2021-07, Vol.151, p.115-125 |
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| creator | Gan, Chun L. Stukalin, Igor Meyers, Daniel E. Dudani, Shaan Grosjean, Heidi A.I. Dolter, Samantha Ewanchuk, Benjamin W. Goutam, Siddhartha Sander, Michael Wells, Connor Pabani, Aliyah Cheng, Tina Monzon, Jose Morris, Don Basappa, Naveen S. Pal, Sumanta K. Wood, Lori A. Donskov, Frede Choueiri, Toni K. Heng, Daniel Y.C. |
| description | Immuno-oncology (IO)–based therapies have been approved based on randomised clinical trials, yet a significant proportion of real-world patients are not represented in these trials. We sought to compare the outcomes of trial-ineligible vs. -eligible patients with advanced solid tumours treated with first-line (1L) IO therapy.
Using the International Metastatic Renal Cell Carcinoma (RCC) Database Consortium and the Alberta Immunotherapy Database, patients with advanced RCC, non–small-cell lung cancer (NSCLC) or melanoma treated with 1L PD-(L)1 inhibition–based therapy were included. Trial eligibility was retrospectively determined as per commonly used exclusion criteria. The outcomes of interest were overall survival (OS), overall response rate (ORR), treatment duration (TD) and time to next treatment (TTNT).
A total of 395 of 1241 (32%) patients were deemed trial-ineligible. The main reasons for ineligibility based on preselected exclusion criteria were Karnofsky performance status 1 (40%, 158 of 395), brain metastases (32%, 126 of 395), haemoglobin |
| doi_str_mv | 10.1016/j.ejca.2021.04.004 |
| format | Article |
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Using the International Metastatic Renal Cell Carcinoma (RCC) Database Consortium and the Alberta Immunotherapy Database, patients with advanced RCC, non–small-cell lung cancer (NSCLC) or melanoma treated with 1L PD-(L)1 inhibition–based therapy were included. Trial eligibility was retrospectively determined as per commonly used exclusion criteria. The outcomes of interest were overall survival (OS), overall response rate (ORR), treatment duration (TD) and time to next treatment (TTNT).
A total of 395 of 1241 (32%) patients were deemed trial-ineligible. The main reasons for ineligibility based on preselected exclusion criteria were Karnofsky performance status <70%/Eastern Cooperative Oncology Group performance status >1 (40%, 158 of 395), brain metastases (32%, 126 of 395), haemoglobin < 9 g/dL (16%, 63 of 395) and estimated glomerular filtration rate <40 mL/min (15%, 61 of 395).
Between the ineligible vs. eligible groups, the median OS, ORR, median TD and median TTNT were 10.2 vs. 39.7 months (p < 0.01), 36% vs. 47% (p < 0.01), 2.7 vs. 6.9 months (p < 0.01) and 6.0 vs. 16.8 months (p < 0.01), respectively. Subgroup analyses showed statistically significant inferior OS, TD and TTNT for trial-ineligible vs. -eligible patients across all tumour types. Adjusted hazard ratios for death in RCC, NSCLC and melanoma were 1.84 (95% confidence interval [CI] 1.22–2.77), 2.21 (95% CI 1.58–3.11) and 1.82 (95% CI 1.21–2.74), respectively..
Thirty-two percent of real-world patients treated with contemporary 1L IO-based therapies were ineligible for clinical trials. Although one-third of the trial-ineligible patients responded to treatment, the overall trial-ineligible population had inferior outcomes than trial-eligible patients. These data may guide patient counselling and temper expectations of benefit.
•The clinical effectiveness of immuno-oncology (IO) agents in the trial-ineligible cancer population is unclear.•A total of 1241 patients with RCC, NSCLC and melanoma treated with first-line IO therapy were studied.•32% of real-world patients were ineligible for IO clinical trials.•These patients had inferior outcomes compared with trial-eligible patients.•These data may temper expectations of treatment benefit in the real-world.]]></description><identifier>ISSN: 0959-8049</identifier><identifier>EISSN: 1879-0852</identifier><identifier>DOI: 10.1016/j.ejca.2021.04.004</identifier><identifier>PMID: 33975059</identifier><language>eng</language><publisher>England: Elsevier Ltd</publisher><subject>Cancer immunotherapy ; Clinical outcomes ; Clinical trial ineligible ; Clinical trials ; Confidence intervals ; Consortia ; Criteria ; Glomerular filtration rate ; Hemoglobin ; IMDC ; Immuno-oncology ; Immunotherapy ; Kidney cancer ; Lung cancer ; Melanoma ; Metastases ; Non-small cell lung carcinoma ; Non–small-cell lung cancer ; Oncology ; Patients ; Real-world patients ; Renal cell carcinoma ; Small cell lung carcinoma ; Solid tumors ; Statistical analysis ; Subgroups ; Trial eligibility ; Tumors</subject><ispartof>European journal of cancer (1990), 2021-07, Vol.151, p.115-125</ispartof><rights>2021 The Authors</rights><rights>Copyright © 2021 The Authors. Published by Elsevier Ltd.. All rights reserved.</rights><rights>Copyright Elsevier Science Ltd. Jul 2021</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c428t-82f7afdc7dd44ad28813e3ed9b416b04321fc9fa6053119b6e1847b345661b9d3</citedby><cites>FETCH-LOGICAL-c428t-82f7afdc7dd44ad28813e3ed9b416b04321fc9fa6053119b6e1847b345661b9d3</cites><orcidid>0000-0002-6250-5368 ; 0000-0003-2576-9310 ; 0000-0002-7619-7327 ; 0000-0001-7655-1405 ; 0000-0002-8449-863X ; 0000-0003-4958-381X ; 0000-0003-2137-9307</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://dx.doi.org/10.1016/j.ejca.2021.04.004$$EHTML$$P50$$Gelsevier$$Hfree_for_read</linktohtml><link.rule.ids>314,780,784,3550,27924,27925,45995</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/33975059$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Gan, Chun L.</creatorcontrib><creatorcontrib>Stukalin, Igor</creatorcontrib><creatorcontrib>Meyers, Daniel E.</creatorcontrib><creatorcontrib>Dudani, Shaan</creatorcontrib><creatorcontrib>Grosjean, Heidi A.I.</creatorcontrib><creatorcontrib>Dolter, Samantha</creatorcontrib><creatorcontrib>Ewanchuk, Benjamin W.</creatorcontrib><creatorcontrib>Goutam, Siddhartha</creatorcontrib><creatorcontrib>Sander, Michael</creatorcontrib><creatorcontrib>Wells, Connor</creatorcontrib><creatorcontrib>Pabani, Aliyah</creatorcontrib><creatorcontrib>Cheng, Tina</creatorcontrib><creatorcontrib>Monzon, Jose</creatorcontrib><creatorcontrib>Morris, Don</creatorcontrib><creatorcontrib>Basappa, Naveen S.</creatorcontrib><creatorcontrib>Pal, Sumanta K.</creatorcontrib><creatorcontrib>Wood, Lori A.</creatorcontrib><creatorcontrib>Donskov, Frede</creatorcontrib><creatorcontrib>Choueiri, Toni K.</creatorcontrib><creatorcontrib>Heng, Daniel Y.C.</creatorcontrib><title>Outcomes of patients with solid tumour malignancies treated with first-line immuno-oncology agents who do not meet eligibility criteria for clinical trials</title><title>European journal of cancer (1990)</title><addtitle>Eur J Cancer</addtitle><description><![CDATA[Immuno-oncology (IO)–based therapies have been approved based on randomised clinical trials, yet a significant proportion of real-world patients are not represented in these trials. We sought to compare the outcomes of trial-ineligible vs. -eligible patients with advanced solid tumours treated with first-line (1L) IO therapy.
Using the International Metastatic Renal Cell Carcinoma (RCC) Database Consortium and the Alberta Immunotherapy Database, patients with advanced RCC, non–small-cell lung cancer (NSCLC) or melanoma treated with 1L PD-(L)1 inhibition–based therapy were included. Trial eligibility was retrospectively determined as per commonly used exclusion criteria. The outcomes of interest were overall survival (OS), overall response rate (ORR), treatment duration (TD) and time to next treatment (TTNT).
A total of 395 of 1241 (32%) patients were deemed trial-ineligible. The main reasons for ineligibility based on preselected exclusion criteria were Karnofsky performance status <70%/Eastern Cooperative Oncology Group performance status >1 (40%, 158 of 395), brain metastases (32%, 126 of 395), haemoglobin < 9 g/dL (16%, 63 of 395) and estimated glomerular filtration rate <40 mL/min (15%, 61 of 395).
Between the ineligible vs. eligible groups, the median OS, ORR, median TD and median TTNT were 10.2 vs. 39.7 months (p < 0.01), 36% vs. 47% (p < 0.01), 2.7 vs. 6.9 months (p < 0.01) and 6.0 vs. 16.8 months (p < 0.01), respectively. Subgroup analyses showed statistically significant inferior OS, TD and TTNT for trial-ineligible vs. -eligible patients across all tumour types. Adjusted hazard ratios for death in RCC, NSCLC and melanoma were 1.84 (95% confidence interval [CI] 1.22–2.77), 2.21 (95% CI 1.58–3.11) and 1.82 (95% CI 1.21–2.74), respectively..
Thirty-two percent of real-world patients treated with contemporary 1L IO-based therapies were ineligible for clinical trials. Although one-third of the trial-ineligible patients responded to treatment, the overall trial-ineligible population had inferior outcomes than trial-eligible patients. These data may guide patient counselling and temper expectations of benefit.
•The clinical effectiveness of immuno-oncology (IO) agents in the trial-ineligible cancer population is unclear.•A total of 1241 patients with RCC, NSCLC and melanoma treated with first-line IO therapy were studied.•32% of real-world patients were ineligible for IO clinical trials.•These patients had inferior outcomes compared with trial-eligible patients.•These data may temper expectations of treatment benefit in the real-world.]]></description><subject>Cancer immunotherapy</subject><subject>Clinical outcomes</subject><subject>Clinical trial ineligible</subject><subject>Clinical trials</subject><subject>Confidence intervals</subject><subject>Consortia</subject><subject>Criteria</subject><subject>Glomerular filtration rate</subject><subject>Hemoglobin</subject><subject>IMDC</subject><subject>Immuno-oncology</subject><subject>Immunotherapy</subject><subject>Kidney cancer</subject><subject>Lung cancer</subject><subject>Melanoma</subject><subject>Metastases</subject><subject>Non-small cell lung carcinoma</subject><subject>Non–small-cell lung cancer</subject><subject>Oncology</subject><subject>Patients</subject><subject>Real-world patients</subject><subject>Renal cell carcinoma</subject><subject>Small cell lung carcinoma</subject><subject>Solid tumors</subject><subject>Statistical analysis</subject><subject>Subgroups</subject><subject>Trial eligibility</subject><subject>Tumors</subject><issn>0959-8049</issn><issn>1879-0852</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2021</creationdate><recordtype>article</recordtype><recordid>eNp9kT2P1DAQQC0E4paFP0CBLNHQZLEd58MSDToBh3TSNVBbjj3ZmyixF9sB7W_hz-JVDgqKq6Z582zNI-Q1ZwfOePt-OsBkzUEwwQ9MHhiTT8iO952qWN-Ip2THVKOqnkl1RV6kNDHGul6y5-SqrlXXsEbtyO-7NduwQKJhpCeTEXxO9Bfme5rCjI7mdQlrpIuZ8eiNt1jQHMFkcBs2Yky5mtEDxWVZfaiCt2EOxzM1x812H6gL1IdMF4BMoahwwBnzmdqIGSIaOoZIbbGgNXN5AM2cXpJnYxnw6mHuyffPn75d31S3d1--Xn-8rawUfa56MXZmdLZzTkrjRN_zGmpwapC8HZisBR-tGk3LmppzNbTAe9kNtWzalg_K1XvybvOeYvixQsp6wWRhno2HsCYtGtFy2dWNKOjb_9CpXMeX3xVKqnLSC7gnYqNsDClFGPUp4mLiWXOmL-n0pC_p9CWdZlKXdGXpzYN6HRZw_1b-tirAhw2AcoufCFGnUsNbcBjBZu0CPub_A7Q7rTM</recordid><startdate>20210701</startdate><enddate>20210701</enddate><creator>Gan, Chun L.</creator><creator>Stukalin, Igor</creator><creator>Meyers, Daniel E.</creator><creator>Dudani, Shaan</creator><creator>Grosjean, Heidi A.I.</creator><creator>Dolter, Samantha</creator><creator>Ewanchuk, Benjamin W.</creator><creator>Goutam, Siddhartha</creator><creator>Sander, Michael</creator><creator>Wells, Connor</creator><creator>Pabani, Aliyah</creator><creator>Cheng, Tina</creator><creator>Monzon, Jose</creator><creator>Morris, Don</creator><creator>Basappa, Naveen S.</creator><creator>Pal, Sumanta K.</creator><creator>Wood, Lori A.</creator><creator>Donskov, Frede</creator><creator>Choueiri, Toni K.</creator><creator>Heng, Daniel Y.C.</creator><general>Elsevier Ltd</general><general>Elsevier Science Ltd</general><scope>6I.</scope><scope>AAFTH</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7TO</scope><scope>7U7</scope><scope>C1K</scope><scope>H94</scope><scope>K9.</scope><scope>NAPCQ</scope><scope>7X8</scope><orcidid>https://orcid.org/0000-0002-6250-5368</orcidid><orcidid>https://orcid.org/0000-0003-2576-9310</orcidid><orcidid>https://orcid.org/0000-0002-7619-7327</orcidid><orcidid>https://orcid.org/0000-0001-7655-1405</orcidid><orcidid>https://orcid.org/0000-0002-8449-863X</orcidid><orcidid>https://orcid.org/0000-0003-4958-381X</orcidid><orcidid>https://orcid.org/0000-0003-2137-9307</orcidid></search><sort><creationdate>20210701</creationdate><title>Outcomes of patients with solid tumour malignancies treated with first-line immuno-oncology agents who do not meet eligibility criteria for clinical trials</title><author>Gan, Chun L. ; Stukalin, Igor ; Meyers, Daniel E. ; Dudani, Shaan ; Grosjean, Heidi A.I. ; Dolter, Samantha ; Ewanchuk, Benjamin W. ; Goutam, Siddhartha ; Sander, Michael ; Wells, Connor ; Pabani, Aliyah ; Cheng, Tina ; Monzon, Jose ; Morris, Don ; Basappa, Naveen S. ; Pal, Sumanta K. ; Wood, Lori A. ; Donskov, Frede ; Choueiri, Toni K. ; Heng, Daniel Y.C.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c428t-82f7afdc7dd44ad28813e3ed9b416b04321fc9fa6053119b6e1847b345661b9d3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2021</creationdate><topic>Cancer immunotherapy</topic><topic>Clinical outcomes</topic><topic>Clinical trial ineligible</topic><topic>Clinical trials</topic><topic>Confidence intervals</topic><topic>Consortia</topic><topic>Criteria</topic><topic>Glomerular filtration rate</topic><topic>Hemoglobin</topic><topic>IMDC</topic><topic>Immuno-oncology</topic><topic>Immunotherapy</topic><topic>Kidney cancer</topic><topic>Lung cancer</topic><topic>Melanoma</topic><topic>Metastases</topic><topic>Non-small cell lung carcinoma</topic><topic>Non–small-cell lung cancer</topic><topic>Oncology</topic><topic>Patients</topic><topic>Real-world patients</topic><topic>Renal cell carcinoma</topic><topic>Small cell lung carcinoma</topic><topic>Solid tumors</topic><topic>Statistical analysis</topic><topic>Subgroups</topic><topic>Trial eligibility</topic><topic>Tumors</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Gan, Chun L.</creatorcontrib><creatorcontrib>Stukalin, Igor</creatorcontrib><creatorcontrib>Meyers, Daniel E.</creatorcontrib><creatorcontrib>Dudani, Shaan</creatorcontrib><creatorcontrib>Grosjean, Heidi A.I.</creatorcontrib><creatorcontrib>Dolter, Samantha</creatorcontrib><creatorcontrib>Ewanchuk, Benjamin W.</creatorcontrib><creatorcontrib>Goutam, Siddhartha</creatorcontrib><creatorcontrib>Sander, Michael</creatorcontrib><creatorcontrib>Wells, Connor</creatorcontrib><creatorcontrib>Pabani, Aliyah</creatorcontrib><creatorcontrib>Cheng, Tina</creatorcontrib><creatorcontrib>Monzon, Jose</creatorcontrib><creatorcontrib>Morris, Don</creatorcontrib><creatorcontrib>Basappa, Naveen S.</creatorcontrib><creatorcontrib>Pal, Sumanta K.</creatorcontrib><creatorcontrib>Wood, Lori A.</creatorcontrib><creatorcontrib>Donskov, Frede</creatorcontrib><creatorcontrib>Choueiri, Toni K.</creatorcontrib><creatorcontrib>Heng, Daniel Y.C.</creatorcontrib><collection>ScienceDirect Open Access Titles</collection><collection>Elsevier:ScienceDirect:Open Access</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Oncogenes and Growth Factors Abstracts</collection><collection>Toxicology Abstracts</collection><collection>Environmental Sciences and Pollution Management</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Nursing & Allied Health Premium</collection><collection>MEDLINE - Academic</collection><jtitle>European journal of cancer (1990)</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Gan, Chun L.</au><au>Stukalin, Igor</au><au>Meyers, Daniel E.</au><au>Dudani, Shaan</au><au>Grosjean, Heidi A.I.</au><au>Dolter, Samantha</au><au>Ewanchuk, Benjamin W.</au><au>Goutam, Siddhartha</au><au>Sander, Michael</au><au>Wells, Connor</au><au>Pabani, Aliyah</au><au>Cheng, Tina</au><au>Monzon, Jose</au><au>Morris, Don</au><au>Basappa, Naveen S.</au><au>Pal, Sumanta K.</au><au>Wood, Lori A.</au><au>Donskov, Frede</au><au>Choueiri, Toni K.</au><au>Heng, Daniel Y.C.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Outcomes of patients with solid tumour malignancies treated with first-line immuno-oncology agents who do not meet eligibility criteria for clinical trials</atitle><jtitle>European journal of cancer (1990)</jtitle><addtitle>Eur J Cancer</addtitle><date>2021-07-01</date><risdate>2021</risdate><volume>151</volume><spage>115</spage><epage>125</epage><pages>115-125</pages><issn>0959-8049</issn><eissn>1879-0852</eissn><abstract><![CDATA[Immuno-oncology (IO)–based therapies have been approved based on randomised clinical trials, yet a significant proportion of real-world patients are not represented in these trials. We sought to compare the outcomes of trial-ineligible vs. -eligible patients with advanced solid tumours treated with first-line (1L) IO therapy.
Using the International Metastatic Renal Cell Carcinoma (RCC) Database Consortium and the Alberta Immunotherapy Database, patients with advanced RCC, non–small-cell lung cancer (NSCLC) or melanoma treated with 1L PD-(L)1 inhibition–based therapy were included. Trial eligibility was retrospectively determined as per commonly used exclusion criteria. The outcomes of interest were overall survival (OS), overall response rate (ORR), treatment duration (TD) and time to next treatment (TTNT).
A total of 395 of 1241 (32%) patients were deemed trial-ineligible. The main reasons for ineligibility based on preselected exclusion criteria were Karnofsky performance status <70%/Eastern Cooperative Oncology Group performance status >1 (40%, 158 of 395), brain metastases (32%, 126 of 395), haemoglobin < 9 g/dL (16%, 63 of 395) and estimated glomerular filtration rate <40 mL/min (15%, 61 of 395).
Between the ineligible vs. eligible groups, the median OS, ORR, median TD and median TTNT were 10.2 vs. 39.7 months (p < 0.01), 36% vs. 47% (p < 0.01), 2.7 vs. 6.9 months (p < 0.01) and 6.0 vs. 16.8 months (p < 0.01), respectively. Subgroup analyses showed statistically significant inferior OS, TD and TTNT for trial-ineligible vs. -eligible patients across all tumour types. Adjusted hazard ratios for death in RCC, NSCLC and melanoma were 1.84 (95% confidence interval [CI] 1.22–2.77), 2.21 (95% CI 1.58–3.11) and 1.82 (95% CI 1.21–2.74), respectively..
Thirty-two percent of real-world patients treated with contemporary 1L IO-based therapies were ineligible for clinical trials. Although one-third of the trial-ineligible patients responded to treatment, the overall trial-ineligible population had inferior outcomes than trial-eligible patients. These data may guide patient counselling and temper expectations of benefit.
•The clinical effectiveness of immuno-oncology (IO) agents in the trial-ineligible cancer population is unclear.•A total of 1241 patients with RCC, NSCLC and melanoma treated with first-line IO therapy were studied.•32% of real-world patients were ineligible for IO clinical trials.•These patients had inferior outcomes compared with trial-eligible patients.•These data may temper expectations of treatment benefit in the real-world.]]></abstract><cop>England</cop><pub>Elsevier Ltd</pub><pmid>33975059</pmid><doi>10.1016/j.ejca.2021.04.004</doi><tpages>11</tpages><orcidid>https://orcid.org/0000-0002-6250-5368</orcidid><orcidid>https://orcid.org/0000-0003-2576-9310</orcidid><orcidid>https://orcid.org/0000-0002-7619-7327</orcidid><orcidid>https://orcid.org/0000-0001-7655-1405</orcidid><orcidid>https://orcid.org/0000-0002-8449-863X</orcidid><orcidid>https://orcid.org/0000-0003-4958-381X</orcidid><orcidid>https://orcid.org/0000-0003-2137-9307</orcidid><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0959-8049 |
| ispartof | European journal of cancer (1990), 2021-07, Vol.151, p.115-125 |
| issn | 0959-8049 1879-0852 |
| language | eng |
| recordid | cdi_proquest_miscellaneous_2526147352 |
| source | Access via ScienceDirect (Elsevier) |
| subjects | Cancer immunotherapy Clinical outcomes Clinical trial ineligible Clinical trials Confidence intervals Consortia Criteria Glomerular filtration rate Hemoglobin IMDC Immuno-oncology Immunotherapy Kidney cancer Lung cancer Melanoma Metastases Non-small cell lung carcinoma Non–small-cell lung cancer Oncology Patients Real-world patients Renal cell carcinoma Small cell lung carcinoma Solid tumors Statistical analysis Subgroups Trial eligibility Tumors |
| title | Outcomes of patients with solid tumour malignancies treated with first-line immuno-oncology agents who do not meet eligibility criteria for clinical trials |
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