Lay Stakeholders in Science and Research Initiative: A Science Advisory From the American Heart Association.

The American Heart Association (AHA) is the largest not-for-profit funder of cardiovascular and cerebrovascular disease research in the United States. It has supported research of independent scientists for 7 decades with the goal of finding novel discoveries that will reduce death and disability from these diseases and ultimately improve overall health. In 2014, the AHA approved a pilot initiative to include lay stakeholders (patients, caregivers, and passionate advocates) in its research and science operations. The initiative was based on the premise that lay stakeholders would add a unique and necessary perspective that would improve decisions concerning research funding, research direction, and scientific guidelines. The AHA developed a framework for the initiative that defined lay stakeholder, created a volunteer recruitment and training program, established policies for incorporating lay stakeholders into science operations, and set metrics for evaluating the initiative over time. It has instituted creative ways to engage lay volunteers and to foster lay and scientist cooperation. Program assessments have been consistently positive and have identified needed future improvements. The benefits of lay/scientist collaboration have far exceeded the AHA's expectations. The AHA will continue to strengthen lay volunteer engagement throughout its science and research operations; to focus on developing a larger, diverse group of qualified lay stakeholders; to educate scientists on how to communicate research effectively to the public and donors; and to retain the respect of donors for the rigors of its research funding, scientific statements, and clinical guidelines.

Toxicity and efficacy of the acetylcholinesterase (AChe) inhibitor donepezil in childhood brain tumor survivors: a pilot study.

BACKGROUND: Neurocognitive deficits are a recognized late effect of curative brain tumor therapy. We evaluated the feasibility, tolerance, and impact of a pilot pharmacologic intervention with the acetylcholinesterase (AChe) inhibitor, donepezil, in pediatric brain tumor (BT) survivors at risk for neurocognitive dysfunction. PROCEDURE: A single institution open-label pilot study was conducted in childhood BT survivors: ≥1 year from cancer treatment; and who received >23.5 Gy cranial radiation therapy (RT). Toxicity, adherence and neurocognitive outcomes were evaluated at baseline and serially during 24 weeks of donepezil, and following a 12-week washout period off drug. RESULTS: From a pool of subjects, 13 were successfully contacted and screened, and 11 met all eligibility criteria to initiate donepezil at a median of 4.7 (1.9-11.9) years from RT. Seventy-two percent of patients completed the 24-week drug study visit. Despite transient gastrointestinal toxicity (vomiting and diarrhea) in 30% of patients there was no weight loss on donepezil. Significant improvement in performance was noted at 24 weeks on the Dellis-Kaplan Executive Function (D-KEF) Tower test (P < 0.001), the Wide Range Assessment of Memory and Learning, 2nd Edition (WRAML-2) Visual memory (P = 0.007), and the Number/Letter task (P = 0.018). CONCLUSIONS: Donepezil was well tolerated among childhood BT survivors who had received substantial prior therapy. Based on improved executive function and memory performance in this pilot trial, a randomized placebo controlled trial of this pharmacologic agent is warranted to fully evaluate its efficacy in remediating neurocognitive dysfunction.