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Should young patients with rare sarcoma subtypes be included in adult sarcoma trials?

HemOnc Today |  July 10, 2017 | Ian Judson | See Original Article Here

Click here to read the Cover Story“Deeper understanding of disease biology may expand sarcoma treatment options.”


It is nonsense that clinical trials continue to be performed in diseases like soft tissue sarcoma with a lower age limit that excludes children when many of the individual subtypes — such as synovial sarcoma, alveolar soft part sarcoma and rhabdomyosarcoma — span the child–adult age range. It is true that tolerance of certain drugs is different between children and adults, although for cytotoxic chemotherapy, children usually are more tolerant. There were concerns that some targeted agents, such as angiogenesis inhibitors, might be particularly toxic, and might inhibit growth. However, this has not been our experience to date. Pharmaceutical companies generally have been very slow to conduct trials in children and the lack of safety data is then used as a reason to impose a lower age limit.

There also are clear differences in prognosis across the age range, especially through adolescence. This has often been ascribed to the fact that above a certain age — such as 16 or 18 years — patients are treated by adult physicians. The view of many pediatric oncologists is that adult oncologists do not treat intensively enough. That this is the principal cause of poorer outcomes with increasing age is disputed, because evidence is accruing of important biological differences, such as increasing genetic complexity with age in synovial sarcoma, that is more likely to be responsible.

Teenagers and young adults are poorly represented in clinical trials for a variety of organizational and psychosocial reasons, and due to the lack of available trials. There are a number of initiatives to create clinical trials that will be inclusive, ensure a consistent treatment approach and study the biology across the age spectrum prospectively.

For diseases that occur with reasonable frequency in children, those children should be included in “adult” clinical trials unless there are compelling safety reasons why this cannot be done.

Ian Judson, MA, MD, FRCP, is a visiting researcher at The Institute of Cancer Research. He can be reached at Disclosure: Judson reports research funding from AstraZeneca.