title: Patient and physician attitudes regarding clinical trials in neurofibromatosis 1.
abstract: Neurofibromatosis 1 (NF1) is a multisystem genetic disorder that primarily affects the skin (freckling and café-au-lait macules), nervous system (neurofibromas, optic gliomas, and learning disabilities), and skeletal system (pseudoarthroses). The interest in pharmacological intervention for patients with NF1 has grown in recent years. However, little is known about the attitudes and priorities of patients, families, and physicians regarding participation in clinical trials. We surveyed 74 adult patients or parents of patients with NF1 and 69 care providers participating in a neurofibromatosis clinic to assess their willingness to participate in clinical trials and their opinions about which conditions they thought were most important to treat. Both patients and care providers are willing to participate in clinical trials for NF1 and both groups rate malignant peripheral nerve sheath tumors as the most urgent for new treatments. There are concordant views among patients and physicians concerning clinical trials for NF1, and patients do not dismiss participation in placebo-controlled trials. Neuroscience nurses are poised to facilitate the research process from conception through implementation as they take the viewpoints of our study populations into consideration.
authors.affiliation: Wardenburg Health Center at University of Colorado at Boulder, USA.
authors.forename: Mary
authors.initials: M
authors.lastname: McQueen
authors.forename: Mia
authors.initials: M
authors.lastname: MacCollin
authors.forename: James
authors.initials: J
authors.lastname: Gusella
authors.forename: Scott R
authors.initials: SR
authors.lastname: Plotkin
mesh_terms: D000328:Adult; D001291:Attitude of Health Personnel; D001294:Attitude to Health; D001900:Boston; D002986:Clinical Trials as Topic / Q000451:nursing / Q000523:psychology*; D005260:Female; D005826:Genetics, Medical; D007722:Health Knowledge, Attitudes, Practice; D006301:Health Services Needs and Demand; D006801:Humans; D016015:Logistic Models; D008297:Male; D009042:Motivation; D009456:Neurofibromatosis 1 / Q000235:genetics / Q000523:psychology* / Q000628:therapy; D009462:Neurology; D024802:Nurse's Role; D015404:Nursing Methodology Research; D018579:Patient Selection; D010372:Pediatrics; D010820:Physicians / Q000523:psychology*; D012720:Severity of Illness Index; D011795:Surveys and Questionnaires