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The trial effect in patients with glioblastoma: effect of clinical trial enrollment on overall survival

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Abstract

Purpose

To determine whether participation in a clinical trial was associated with improved survival in patients with glioblastoma (GBM).

Methods

Following IRB approval, patients were identified using CPT and ICD codes. Data was collected using retrospective review of electronic medical records. When necessary, death data was obtained from online obituaries. Inverse propensity score matching was utilized to transform the two cohorts to comparable sets. Survival was compared using Kaplan-Meyer curves and Wilcoxon Rank Sum Test.

Results

In this cohort of 365 patients, 89 were enrolled in a clinical trial and 276 were not. Patients enrolled in clinical trials had a significantly higher mean baseline KPS score, higher proportion of surgical resections, and were more likely to receive temozolomide treatment than patients not enrolled in a clinical trial. After inverse propensity score matching, patients enrolled in a clinical trial lived significantly longer than those not enrolled (28.8 vs 22.2 months, p = 0.005). A potential confounder of this study is that patients not in a clinical trial had significantly fewer visits with neuro-oncologists than patients enrolled in a clinical trial (7 ± 8 vs 12 ± 9, p < 0. 0001).

Conclusions

Clinical trials enroll patients with the most favorable prognostic features. Even when correcting for this bias, clinical trial enrollment is an independent predictor of increased survival regardless of treatment arm.

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Acknowledgements

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Funding

The authors have not disclosed any funding.

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Authors and Affiliations

  1. Department of Neurological Surgery, University of Florida, Gainesville, FL, USA

    Kaitlyn F. Melnick, Patricia Miller, Ethan Carmichael, Ashley Ghiaseddin, David D. Tran & Maryam Rahman

  2. University of Florida College of Medicine, Gainesville, FL, USA

    Kyle McGrath

Authors
  1. Kaitlyn F. Melnick
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  2. Patricia Miller
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  3. Ethan Carmichael
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  4. Kyle McGrath
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  5. Ashley Ghiaseddin
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  7. Maryam Rahman
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Contributions

KM wrote the manuscript text and oversaw data collection PM assisted in data collection EC assisted in data collection KM revised the manuscript AG reviewed the manuscript DT reviewed the manuscript MR conceptualized the projected, reviewed the manuscript, prepared figures and supervised all aspects of the project

Corresponding author

Correspondence to Kaitlyn F. Melnick.

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Conflicts of interest

DDT has received research funding from Novocure, Merck, Sarepta, and Lacerta. AG is on the Advisory Board for Novocure and Neosoma.

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Melnick, K.F., Miller, P., Carmichael, E. et al. The trial effect in patients with glioblastoma: effect of clinical trial enrollment on overall survival. J Neurooncol 159, 479–484 (2022). https://doi.org/10.1007/s11060-022-04083-8

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  • DOI: https://doi.org/10.1007/s11060-022-04083-8

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