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Review
. 2020 Feb;32(1):7-12.
doi: 10.1097/MOP.0000000000000857.

Challenges and solutions to the study of rare childhood tumors

Douglas Fair  1 , Samara L Potter  2 , Rajkumar Venkatramani  2
Affiliations
Review

Challenges and solutions to the study of rare childhood tumors

Douglas Fair et al. Curr Opin Pediatr. 2020 Feb.

Abstract

Purpose of review: The majority of progress made in pediatric oncology over the past 50 years has been achieved in the most common cancers. Rare pediatric cancers, which collectively comprise more than 10% of all pediatric cancers, pose multiple challenges to researchers and clinicians, all which stem from the infrequency of these cancers. There has been a tremendous increase in focus on rare pediatric cancers by international consortia and registries, disease-specific clinics, and divisions of academic children's hospitals in the last 10 years. This focus, along with the progress made in cancer genomics, has changed the landscape for the study and treatment of rare pediatric cancers. This review focuses on the past, present, and future of the study and treatment of rare pediatric cancers.

Recent findings: Cancer genomics is changing the way some cancers are being diagnosed, categorized, and treated. Rare pediatric cancers potentially stand to greatly benefit from advances in precision diagnosis and treatment.

Summary: The challenges of studying rare pediatric cancers are well known. By utilizing similar techniques that allowed for progress in the common pediatric malignancies, namely collaboration, increased focus, greater funding, and utilization of cancer genomics, progress in the study and treatment of rare pediatric cancers is promising.

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References

    1. Gadalla SM, Widemann BC. Editorial: US cancer statistics of survival: achievements, challenges, and future directions. J Natl Cancer Inst 2017; 109: doi:10.1093/jnci/djx070. - DOI
    1. Jemal A, Ward EM, Johnson CJ, et al. Annual report to the nation on the status of cancer, 1975–2014, featuring survival. J Natl Cancer Inst 2017; 109: doi:10.1093/jnci/djx030. - DOI
    1. Shimkus J. H.R.4013 - 107th Congress (2001-2002): Rare Diseases Act of 2002. https://www.congress.gov/bill/107th-congress/house-bill/4013. Published November 6, 2002. Accessed November 14, 2019.
    1. Genetic and Rare Diseases Information Center (GARD) – an NCATS Program | Providing information about rare or genetic diseases. https://rarediseases.info.nih.gov/. Accessed April 5, 2019.
    1. Moliner AM, Waligora J. The European Union Policy in the Field of Rare Diseases. Adv Exp Med Biol 2017; 1031:561–87.