PERSPECTIVES: Stroke survivors' views on the design of an early‐phase cell therapy trial for patients with chronic ischaemic stroke

Abstract

BackgroundStem cell research holds the potential for a paradigm shift in the management of diseases such as stroke. Patient and public involvement in research (PPIR) can bring a focus to issues of clinical relevance and accelerate translation to real‐world clinical practice.ObjectiveA qualitative thematic analysis of the perspectives of stroke survivors regarding the conduct and design aspects of a proposed phase I clinical cell therapy study in stroke.DesignTwelve stroke survivors were purposively recruited in July 2016–August 2017 and participated in semi‐structured, face‐to‐face interviews for input into the design of a proposed phase I clinical study of autologous dental pulp stem cells. Concurrent thematic analysis was conducted until data saturation was achieved.Discussion and conclusionsParticipants conveyed that the most relevant outcomes to them were regaining participation, decreased dependence on caregivers and improvement in cognition, memory, mood, pain and fatigue. The perception of risk vs. benefit was likely influenced by the time elapsed since stroke, with participants being more willing to accept a higher level of risk early in the post‐stroke disease course. They believed that all stroke survivors should be given an opportunity to participate in research, irrespective of their cognitive capacity. A relatively small sample population of 12 stroke survivors was studied as thematic saturation was achieved. PERSPECTIVES study applied principles of PPIR to early‐phase cell research. Incorporation of outcomes relevant to patients' need within the study design is critical to generate data that will enable personalized application of regenerative medicine in stroke.

Clinical Translation of Cell Therapies in Stroke (CT2S) Checklist—a pragmatic tool to accelerate development of cell therapy products

“…Studies have reported a general positive attitude towards participation in cell therapy research, although with a wide divergence in efficacy expectations between study participants and research teams [ 39 ]. Our study revealed that patients had a pragmatic realistic approach to anticipated benefits [ 18 ]. It was interesting to note that patients were more worried about a loss of function as a possible risk as compared to more widely discussed risks such as tumorigenicity and death [ 18 ].…”

“…Our study revealed that patients had a pragmatic realistic approach to anticipated benefits [ 18 ]. It was interesting to note that patients were more worried about a loss of function as a possible risk as compared to more widely discussed risks such as tumorigenicity and death [ 18 ]. The importance of measuring changes in often neglected outcomes such as cognition, mood, and overall ability to restore “normal” pre-stroke participation levels is a key insight that should inform future trial designs [ 18 ].…”

“…A qualitative interview-based study was undertaken [ 18 ] with stroke survivors to collect insights and understand ethical perspectives on the research design of our proposed early phase cell therapy study in chronic ischemic stroke [ 19 ]. This was conducted via semi-structured, face-to-face interviews on specific aspects of the design of the study while a concurrent thematic analysis was conducted to identify key perspectives until data saturation was achieved.…”

“…Dr. Stuart Howell, University of Adelaide, Adelaide, South Australia [11,16] and Dr. Susan W Kim, South Australian Health and Medical Research Institute, Adelaide, South Australia [18,19] for statistical analysis.…”

Muscle strengthening intervention for boys with haemophilia: Developing and evaluating a best‐practice exercise programme with boys, families and health‐care professionals

“…Due to the small‐sized patient population and limited validated outcome measures, large randomized controlled trials (RCT) to establish whether an intervention is effective in rare diseases like haemophilia are often difficult to conduct without establishing whether a study is feasible 5,6 . In addition, resource use may not be optimally rationalized when a treatment or intervention is found to be ineffective or unsafe, or conversely, a treatment or intervention is not provided if it turns out to be effective 57 …”