"Clinical trials in HD are creating a sense of optimism and hope within the HD community, with the majority of respondents [to our survey] reporting willingness to participate in trials. As new trials are developed and implemented, it is important to take the opinions and attitudes of the patient population into account," explained Tanya M. Bardakjian, MS, CGC, senior genetic counselor in the department of Neurology at Penn.
Treatments now being tested can be nontraditional, going beyond taking a daily pill. Some require repeated intravenous infusions, lumbar punctures, or brain surgery; some may be administered only at sites far from where the patient lives; and others may result in significant side effects. "In order to optimize feasibility, it is important to first understand how potential study participants feel about these new trial designs, with interventions that might involve a higher potential risk but also higher potential reward," noted Ms. Bardakjian. The authors also wanted to determine whether responses differed if the respondent was a potential disease carrier, an asymptomatic genetic carrier, or showed HD symptoms.
Respondents - Survey - Patients - HD - Mutation
Eighty-seven respondents completed an anonymous survey, including 36 patients diagnosed with HD, 18 pre-manifesting mutation carriers, and 33 asymptomatic participants at risk. The questionnaire included multiple-choice questions concerning hypothetical scenarios. For example, one question asked, "How likely are you to participate in gene therapy trials if treatment was an IV infusion in the arm requiring a clinic...
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