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Clinical research tools in pediatric oncology: challenges and opportunities

Abstract

Survival for childhood cancers has improved significantly over the last decades. However, patient outcomes have plateaued over the last decade for difficult-to-treat diseases. With high cure rates, decreasing long-term toxicities and sequelae remains crucial. Since many advances in childhood cancer research come from the adult oncology world, one of the key areas is improving the adaptation of tools that are essential for clinical trial conduct that were developed for adults into pediatrics. These include tools to evaluate toxicity, quality of life, radiological response, statistical methodology, or indicators of cancer care quality. In this review, we present ongoing international efforts to validate and adapt these tools for children and adolescents and discuss remaining challenges. These efforts will hopefully accelerate and improve the quality of pediatric oncology research in the upcoming years.

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Acknowledgments

Teresa de Rojas’ work as Fellow at EORTC Headquarters was supported by a grant from EORTC Cancer Research Fund (ECRF) from Belgium. Fernando Carceller is supported by George and the Giant Pledge via the Royal Marsden Cancer Charity.

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Correspondence to Teresa de Rojas.

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Conflict of interest

Francisco Bautista had a consultant or advisory role for Bayer, Amgen, and EusaPharma, received honoraria for speaking at symposia from Amgen and Jazz Pharmaceuticals and support for attending symposia from Takeda, EusaPharma, Shire, and Jazz Pharmaceuticals. Ana Fernández-Teijeiro has had a consulting or advisory role for Amgen, Novartis, Takeda, SOBI and Bayer. She received honoraria from Takeda and Amgen for educational events and travel expenses from Servier, Shire, and Gilead. Alexander Towbin has received grants from Guerbet and the Cystic Fibrosis Foundation. He receives royalties from Elsevier. He has served as consultant for Applied Radiology, IBM Watson Health Imaging, and KLAS. Lucas Moreno has served in a consulting or advisory role for Novartis, AstraZeneca, Roche Genentech, Bayer, Amgen, and MundiPharma; has received honoraria for educational events from Celgene and Novartis; and has received travel expenses from MundiPharma, Celgene, and Amgen. The rest of the authors declare that they have no conflict of interest.

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de Rojas, T., Neven, A., Towbin, A.J. et al. Clinical research tools in pediatric oncology: challenges and opportunities. Cancer Metastasis Rev (2020). https://doi.org/10.1007/s10555-020-09856-z

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Keywords

  • RECIST
  • CTCAE
  • Quality of life
  • Quality assurance
  • Childhood cancer
  • AYA oncology