Abstract
There has been great progress in Huntington’s disease (HD) research. Yet, effective treatments to halt disease before the onset of disabling symptoms are still unavailable. Scientific breakthroughs require an active and lasting commitment from families. However, they are traditionally less involved and heard in studies. Accordingly, the European Huntington Association (EHA) surveyed individuals at risk (HDRisk) and with premanifest HD (PreHD) to determine which factors affect their willingness to participate in research. Questions assessed research experience and knowledge, information sources, reasons for involvement and noninvolvement, and factors preventing and facilitating participation. The survey included 525 individuals, of which 68.8% never participated in studies and 38.6% reported limited research knowledge. Furthermore, 52% trusted patient organizations to get research information. Reasons for involvement were altruistic and more important than reasons for noninvolvement, which were related to negative emotions. Obstacles included time/financial constraints and invasive procedures, while professional support was seen as a facilitator. PreHD individuals reported less obstacles to research participation than HDRisk individuals. Overall, a high motivation to participate in research was noted, despite limited experience and literacy. This motivation is influenced by subjective and objective factors and, importantly, by HD status. Patient organizations have a key role in fostering motivation through education and support.
Highlights
Over the last decade, there have been significant advances in Huntington’s disease (HD) research, which have culminated in several new therapeutic compounds either in clinical evaluation or on the verge of being so [1,2]
We found that the HDRisk group relied significantly more on family members to get information about HD research than the premanifest HD stage (PreHD) group (χ2 (1) = 8.321, p = 0.004). These results reveal that the PreHD group reported an increased knowledge about HD research compared to the HDRisk group, which depended more on family members to get information about HD studies and trials
A higher number of individuals with HDRisk than individuals with PreHD signaled the following facilitating factors: “I would have psychological and social care available” (χ2 (1) = 4.953, p = 0.026), and “I would not have to go through invasive procedures” (χ2 (1) = 6.994, p = 0.008). These results suggest that respondents with PreHD found fewer obstacles to research participation than respondents with HDRisk, who seemed to need more support to get engaged in studies
Summary
There have been significant advances in Huntington’s disease (HD) research, which have culminated in several new therapeutic compounds either in clinical evaluation or on the verge of being so [1,2]. One of the key messages has been that successful treatments for HD cannot be developed without an active and long-lasting commitment from patients and families In this context, the EHA has been involved in the design and implementation of several initiatives aimed at broadening and improving the clinical research knowledge of the European HD community. While being a rare disease, HD may help to develop therapeutic compounds or strategies applicable in time to more prevalent diseases. It has underpinned the rapid pace and dissemination of encouraging preliminary findings of some studies, such as Generation HD1 [10,11]. Many of these have had input from representatives of the EHA member associations from their very beginning (e.g., [12,13,14,15])
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