Malignant peripheral nerve sheath tumors in neurofibromatosis type 1: a multicenter project with 3 clinical trials

A major goal of this CTDA proposal is to optimize subject recruitment in each of the clinical trials. Using the prevalence estimate for NF1 as 1 in 3,500 individuals in the population at large coupled with the cross-sectional estimate of 5% affected by MPNST, we acknowledge that few centers will have more than 2 patients with MPNST and NF1 in any given year. We anticipate 1 in 1,750,000 people will develop MPNST and NF1 on an annual basis, thus the populations of the US, Canada, and Europe will provide a maximum of 50 cases per year. Our goal is to recruit at least 2/3 of this cohort for enrollment in at least 1 of the 3 clinical trials. By developing a well-publicized network of NF1 Clinic Centers and Sarcoma Centers, we plan to offer enrollment to every individual in North America and Europe who has MPNST and NF1 into the case-control trial to identify risk factors for MPNST (clinical trial project 1). Based on inclusion and exclusion criteria, some individuals will be eligible for the clinical trial of neoadjuvant chemotherapy, and this will be offered to them as a treatment option (clinical trial project 3).