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Clinical research tools in pediatric oncology: challenges and opportunities

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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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Authors and Affiliations

  1. Medical Department, EORTC Headquarters, Av. E. Mounier 83/11, 1200, Brussels, Belgium

    Teresa de Rojas

  2. Statistics Department, EORTC Headquarters, Av. E. Mounier 83/11, 1200, Brussels, Belgium

    Anouk Neven

  3. Department of Radiology, Cincinnati Children’s Hospital, 3333 Burnet Ave, Cincinnati, OH, 45229, USA

    Alexander J. Towbin

  4. Department of Radiology, University of Cincinnati College of Medicine, 230 Albert Sabin Way, Cincinnati, OH, 45229, USA

    Alexander J. Towbin

  5. Children & Young People’s Unit, The Royal Marsden NHS Foundation Trust, Downs Rd, Sutton, London, SM2 5PT, UK

    Fernando Carceller

  6. Division of Clinical Studies, The Institute of Cancer Research, 15 Cotswold Road, Sutton, London, SM2 5NG, UK

    Fernando Carceller

  7. Pediatric Oncology, Haematology and Stem Cell Transplantation Department, Hospital Niño Jesús, Av. Menéndez Pelayo 65, 28009, Madrid, Spain

    Francisco Bautista

  8. University Hospital for Medical Psychology, Medical University of Innsbruck, Christoph-Probst-Platz 1, Innrain 52, 6020, Innsbruck, Austria

    David Riedl

  9. School of Health Sciences, University of Southampton, Southampton, SO17 1BJ, UK

    Samantha Sodergren & Anne-Sophie Darlington

  10. Pediatric Onco-Hematology Unit, Hospital Universitario Virgen Macarena, Av. Dr. Fedriani s/n, 41009, Sevilla, Spain

    Ana Fernandez-Teijeiro

  11. Paediatric Oncology & Haematology Department, Vall d’Hebron University Hospital, Passeig de la Vall d’Hebron 119, 129, 08035, Barcelona, Spain

    Lucas Moreno

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  1. Teresa de Rojas
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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 39, 149–160 (2020). https://doi.org/10.1007/s10555-020-09856-z

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