Use of External Control Cohorts in Pediatric Brain Tumor Clinical Trials

Margol, Ashley S.; Molinaro, Annette M.; Onar-Thomas, Arzu; Resnick, Adam; Hanson, Derek; Kieran, Mark; Mishra-Kalyani, Pallavi; Rivera, Donna; Barone, Amy; Arons, David; Meehan, Clair; Prados, Michael

doi:10.1200/jco.23.01084

JCO

2024

DOI: 10.1200/jco.23.01084

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Use of External Control Cohorts in Pediatric Brain Tumor Clinical Trials

Ashley S. Margol,

Annette M. Molinaro,

Arzu Onar-Thomas

et al.

Abstract: Why, when, and how to consider external control cohorts in pediatric brain tumor clinical trials.

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Treatment-related survival patterns in diffuse intrinsic pontine glioma using a historical cohort: A report from the European Society for Pediatric Oncology DIPG/DMG Registry

Baugh,

Veldhuijzen van Zanten,

Fiocco

et al. 2024

Neuro-Oncology Advances

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Background Our aim is to investigate the association of treatment with survival in patients with diffuse intrinsic pontine glioma (DIPG) by examining 6 historical treatment paths. Methods We retrospectively analyzed data from 409 patients with radiologically centrally reviewed DIPG, sourced from the German Society of Pediatric Oncology and Hematology HIT-HGG trial database and the SIOPE-DIPG/DMG Registry. Survival outcomes were estimated using the Kaplan–Meier method, and univariable and multivariable Cox proportional hazard models were estimated to study treatment effects. Results The median overall survival (OS) from diagnosis was 11.2 months (95% confidence interval [CI], 10.5–11.9). Patients who by choice received no frontline treatment had an OS of 3.0 months (95% CI, 2.0–4.0), while those treated with radiation therapy (RT) alone had a median OS of 10.4 months (95% CI, 9.1–11.8). Those receiving RT combined with chemotherapy had the longest median OS of 11.7 months (95% CI, 10.8–12.6). The median post-progression survival (PPS) was 4.1 months (95% CI, 3.5–4.7). Patients who relapsed and did not receive treatment had a PPS of 2.2 months (95% CI, 1.8–2.6), while those treated with chemotherapy alone had a PPS of 4.4 months (95% CI, 3.7–5.0), and those who underwent reirradiation, with or without chemotherapy, had the longest survival after relapse of 6.6 months (95% CI, 5.3–8.0). Treatment differences remained significant in multivariable analysis adjusted for age and symptom duration in both diagnosis and relapse setting. Conclusions This study shows increased survival outcomes associated with radiation and chemotherapy treatment or a combination thereof, at diagnosis and relapse, in a historical DIPG cohort.

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Treatment-related survival patterns in diffuse intrinsic pontine glioma using a historical cohort: A report from the European Society for Pediatric Oncology DIPG/DMG Registry

Baugh,

Veldhuijzen van Zanten,

Fiocco

et al. 2024

Neuro-Oncology Advances

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Use of External Control Cohorts in Pediatric Brain Tumor Clinical Trials

Abstract: Why, when, and how to consider external control cohorts in pediatric brain tumor clinical trials.

Cited by 1 publication

References 22 publications

Treatment-related survival patterns in diffuse intrinsic pontine glioma using a historical cohort: A report from the European Society for Pediatric Oncology DIPG/DMG Registry

Treatment-related survival patterns in diffuse intrinsic pontine glioma using a historical cohort: A report from the European Society for Pediatric Oncology DIPG/DMG Registry

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