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Strategies for New Agent Development and Clinical Trial Considerations

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Childhood Leukemia

Part of the book series: Pediatric Oncology ((PEDIATRICO))

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Abstract

Outcome for children with acute lymphoblastic leukemia (ALL) has improved dramatically, with 5-year survival rates increasing from virtually nil in the early 1960s to approaching 90% by the first decade of the twenty-first century (Horner et al. 2009). While improvements in outcome have not been as impressive for children with acute myeloid leukemia (AML), 5-year survival rates have, nonetheless, increased to approximately 60% (Horner et al. 2009). These improvements are gratifying and represent tens of thousands of children diagnosed with leukemia over the last 20–30 years who have survived to adulthood. Looking forward, there are multiple challenges in study design and conduct in moving toward the goal of curing every child diagnosed with leukemia. These include identifying ways to make sound prioritization decisions about which new treatment approaches should be studied for specific patient populations and identifying ways to develop clinical trial datasets based on limited numbers of patients that allow sufficiently reliable conclusions to be drawn about the clinical benefit that these treatment approaches afford.

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Acknowledgments

This work was supported by NO1CM42216 from the National Cancer Institute (USA) and by Children’s Cancer Institute Australia for Medical Research. Children’s Cancer Institute Australia for Medical Research is affiliated with the University of New South Wales and Sydney Children’s Hospital. It was also supported in part by Children’s Cancer Group grant CA 13539, Pediatric Oncology Group grants CA 29139, 30969, and Children¹s Oncology Group grants CA 98413, 98543.

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Authors and Affiliations

  1. Pediatric Section, CIB, CTEP, National Cancer Institute, 6130 Executive Blvd., Executive Plaza North, Rm 7025, Bethesda, MD, 20892, USA

    Malcolm Smith

  2. Children’s Oncology Group - Data Center, 104 North Main Street, Suite 600, Gainesville, FL, 32601-3330, USA

    Meenakshi Devidas

  3. Cancer Research UK Clinical Trials Unit, School of Cancer Sciences, College of Medical and Dental Sciences, University of Birmingham, Birmingham, B15 2TT, UK

    Keith Wheatley

  4. Leukaemia Biology Program, Children’s Cancer Institute Australia for Medical Research, 81, High Street, Randwick, NSW, 2031, Australia

    Richard B. Lock

  5. Biometrics Research Branch, National Cancer Institute, 6130 Executive Blvd., Executive Plaza North, Rm 8120, Bethesda, MD, 20892, USA

    Sally Hunsberger

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  1. Malcolm Smith
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  3. Keith Wheatley
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  4. Richard B. Lock
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  5. Sally Hunsberger
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Correspondence to Malcolm Smith .

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  1. School of Medicine & Health Sciences, George Washington University, EW. Highway 4600, Bethesda, 20814, Maryland, USA

    Gregory H. Reaman

  2. Cincinnati Children's Hospital, Medical Center (CCHMC), University of Cincinnati, Burnett Avenue 3333, Cincinnati, 45229, Ohio, USA

    Franklin O. Smith

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Smith, M., Devidas, M., Wheatley, K., Lock, R.B., Hunsberger, S. (2011). Strategies for New Agent Development and Clinical Trial Considerations. In: Reaman, G., Smith, F. (eds) Childhood Leukemia. Pediatric Oncology. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-13781-5_8

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