Outcomes among pediatric patients with cancer who are treated on trial versus off trial : A matched cohort study
© 2020 American Cancer Society..
BACKGROUND: Approximately 50% of children with cancer in the United States who are aged <15 years receive primary treatment on a therapeutic clinical trial. To the authors' knowledge, it remains unknown whether trial enrollment has a clinical benefit compared with the best alternative standard therapy and/or off trial (ie, clinical trial effect). The authors conducted a retrospective matched cohort study to compare the morbidity and mortality of pediatric patients with cancer who are treated on a phase 3 clinical trial compared with those receiving standard therapy and/or off trial.
METHODS: Subjects were aged birth to 19 years; were diagnosed between 2000 and 2010 with acute lymphocytic leukemia (ALL), acute myeloid leukemia (AML), rhabdomyosarcoma, or neuroblastoma; and had received initial treatment at the Children's Hospital of Philadelphia. On-trial and off-trial subjects were matched based on age, race, ethnicity, a diagnosis of Down syndrome (for patients with ALL or AML), prognostic risk level, date of diagnosis, and tumor type.
RESULTS: A total of 428 participants were matched in 214 pairs (152 pairs for ALL, 24 pairs for AML, 32 pairs for rhabdomyosarcoma, and 6 pairs for neuroblastoma). The 5-year survival rate did not differ between those treated on trial versus those treated with standard therapy and/or off trial (86.9% vs 82.2%; P = .093). On-trial patients had a 32% lower odds of having worse (higher) mortality-morbidity composite scores, although this did not reach statistical significance (odds ratio, 0.68; 95% confidence interval, 0.45-1.03 [P = .070]).
CONCLUSIONS: There was no statistically significant difference in outcomes noted between those patients treated on trial and those treated with standard therapy and/or off trial. However, in partial support of the clinical trial effect, the results of the current study indicate a trend toward more favorable outcomes in children treated on trial compared with those treated with standard therapy and/or off trial. These findings can support decision making regarding enrollment in pediatric phase 3 clinical trials.
| Errataetall: |
CommentIn: Cancer. 2021 Feb 15;127(4):649-650. - PMID 33119128 |
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| Medienart: |
E-Artikel |
| Erscheinungsjahr: |
2020 |
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| Erschienen: |
2020 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:126 |
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| Enthalten in: |
Cancer - 126(2020), 15 vom: 01. Aug., Seite 3471-3482 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Schapira, Marilyn M [VerfasserIn] |
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| Links: |
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| Themen: |
Clinical Trial, Phase III |
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| Anmerkungen: |
Date Completed 20.05.2021 Date Revised 07.09.2021 published: Print-Electronic CommentIn: Cancer. 2021 Feb 15;127(4):649-650. - PMID 33119128 Citation Status MEDLINE |
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| doi: |
10.1002/cncr.32947 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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NLM310355052 |
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| 100 | 1 | |a Schapira, Marilyn M |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Outcomes among pediatric patients with cancer who are treated on trial versus off trial |b A matched cohort study |
| 264 | 1 | |c 2020 | |
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| 500 | |a Date Completed 20.05.2021 | ||
| 500 | |a Date Revised 07.09.2021 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a CommentIn: Cancer. 2021 Feb 15;127(4):649-650. - PMID 33119128 | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a © 2020 American Cancer Society. | ||
| 520 | |a BACKGROUND: Approximately 50% of children with cancer in the United States who are aged <15 years receive primary treatment on a therapeutic clinical trial. To the authors' knowledge, it remains unknown whether trial enrollment has a clinical benefit compared with the best alternative standard therapy and/or off trial (ie, clinical trial effect). The authors conducted a retrospective matched cohort study to compare the morbidity and mortality of pediatric patients with cancer who are treated on a phase 3 clinical trial compared with those receiving standard therapy and/or off trial | ||
| 520 | |a METHODS: Subjects were aged birth to 19 years; were diagnosed between 2000 and 2010 with acute lymphocytic leukemia (ALL), acute myeloid leukemia (AML), rhabdomyosarcoma, or neuroblastoma; and had received initial treatment at the Children's Hospital of Philadelphia. On-trial and off-trial subjects were matched based on age, race, ethnicity, a diagnosis of Down syndrome (for patients with ALL or AML), prognostic risk level, date of diagnosis, and tumor type | ||
| 520 | |a RESULTS: A total of 428 participants were matched in 214 pairs (152 pairs for ALL, 24 pairs for AML, 32 pairs for rhabdomyosarcoma, and 6 pairs for neuroblastoma). The 5-year survival rate did not differ between those treated on trial versus those treated with standard therapy and/or off trial (86.9% vs 82.2%; P = .093). On-trial patients had a 32% lower odds of having worse (higher) mortality-morbidity composite scores, although this did not reach statistical significance (odds ratio, 0.68; 95% confidence interval, 0.45-1.03 [P = .070]) | ||
| 520 | |a CONCLUSIONS: There was no statistically significant difference in outcomes noted between those patients treated on trial and those treated with standard therapy and/or off trial. However, in partial support of the clinical trial effect, the results of the current study indicate a trend toward more favorable outcomes in children treated on trial compared with those treated with standard therapy and/or off trial. These findings can support decision making regarding enrollment in pediatric phase 3 clinical trials | ||
| 650 | 4 | |a Clinical Trial, Phase III | |
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Research Support, U.S. Gov't, P.H.S. | |
| 650 | 4 | |a clinical trial | |
| 650 | 4 | |a outcomes assessment | |
| 650 | 4 | |a pediatric oncology | |
| 650 | 4 | |a retrospective studies | |
| 650 | 4 | |a trial effect | |
| 700 | 1 | |a Stevens, Evelyn M |e verfasserin |4 aut | |
| 700 | 1 | |a Sharpe, James E |e verfasserin |4 aut | |
| 700 | 1 | |a Hochman, Lauren |e verfasserin |4 aut | |
| 700 | 1 | |a Reiter, Joseph G |e verfasserin |4 aut | |
| 700 | 1 | |a Calhoun, Shawna R |e verfasserin |4 aut | |
| 700 | 1 | |a Shah, Shivani A |e verfasserin |4 aut | |
| 700 | 1 | |a Bailey, Leonard Charles |e verfasserin |4 aut | |
| 700 | 1 | |a Bagatell, Rochelle |e verfasserin |4 aut | |
| 700 | 1 | |a Silber, Jeffrey H |e verfasserin |4 aut | |
| 700 | 1 | |a Tai, Eric |e verfasserin |4 aut | |
| 700 | 1 | |a Barakat, Lamia P |e verfasserin |4 aut | |
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