Clinical trial design in neurofibromatosis type 1 as a model for other tumor predisposition syndromes.

ABSTRACT: Up to 10% of all pediatric cancer patients may have an underlying germline mutation which predisposed them to develop a malignancy. With more patients being tested for and diagnosed with genetic tumor predisposition syndromes, there has been improved characterization of their many nonmalignant manifestations. However, designing and implementing clinical trials to treat the nonmalignant tumor and non-tumor manifestations of these syndromes poses many unique challenges. Unlike trials for malignancies where tumor response and survival can be used as straightforward trial endpoints, the nonmalignant manifestations are often chronic, evolve more slowly over time, and may not be immediately life-threatening. Therefore, they will likely require a different approach to both testing and treatment w

SUBMITTER: Gross AM 

PROVIDER: S-EPMC7317058 | biostudies-literature | 2020 Jul

REPOSITORIES: biostudies-literature