Gene therapy for cystic fibrosis: a psychosocial study of trial participants

Blair, Caroline; Kacser, E.; Porteous, David J.

doi:10.1038/sj.gt.3300587

Cited by 12 publications

(35 citation statements)

References 11 publications

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“…We should begin with more studies on the psychosocial aspects of gene therapy with trial participants and their families [21]. We also need to examine whether attitudes differ between different sub-groups in the population, and to explore further the extent to which opinions are contingent upon such things as health status and exposure to genetic conditions.…”

Section: Discussionmentioning

confidence: 99%

Public Attitudes to Human Gene Therapy: A Pilot Study in Wales

Iredale

Dolan²,

McDonald³

et al. 2003

Public Health Genomics

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Objective: This study aimed to explore some factors influencing perceptions of human gene therapy. Method: A small qualitative study using two semi-structured interviews per participant (n = 22). The groups comprised (1) people with cystic fibrosis and members of their family (n = 9), and (2) students from a science evening class as well as lay members of the public selected from the practice list of a local general practitioner (n = 13). Results: This pilot study demonstrates support for somatic gene therapy and ambivalence about germline gene therapy. A clear distinction is drawn between therapy and enhancement, with the majority opposing gene enhancement. Conclusions: Attitudes towards the acceptability of gene therapy are not necessarily determined by experience of, or exposure to, a genetic condition. More research is needed with the general public to determine what is perceived to be acceptable public policy in this field.

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Section: Discussionmentioning

confidence: 99%

Public Attitudes to Human Gene Therapy: A Pilot Study in Wales

Iredale

Dolan²,

McDonald³

et al. 2003

Public Health Genomics

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“…However, we feel this is a neglected area and could provide valuable information about the progress of blinded, placebo-controlled, phase 2/3 gene therapy trials. We believe this aspect could best be approached through the use of a validated quality of life questionnaire building on previously published experience [85].…”

Section: Non-invasive Secondary Endpointsmentioning

confidence: 99%

Pre-clinical and clinical endpoint assays for cystic fibrosis gene therapy

Griesenbach

Boyd

2005

Journal of Cystic Fibrosis

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The credibility and hence value of pre-clinical and clinical cystic fibrosis gene therapy studies depend on the assays used to evaluate gene transfer. Awareness of assay suitability, sensitivity and variability is therefore crucial to the design of experimental programmes. Here, we review the assays that are in use to assess the efficacy of gene transfer in pre-clinical and clinical CF gene therapy research, highlight their weaknesses and suggest possible new strategies that may help to overcome current limitations.

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“…Those patients had concerns about the safety, efficacy and unpleasant invasiveness of gene therapy. Such information can be useful for professionals as they can give more attention to patient's emotional needs [21]. In the past, several studies detected higher rates of depression in visually disabled patients [23,24].…”

Section: Introductionmentioning

confidence: 99%

“…A British study assessed the psychological functioning of 16 patients with cystic fibrosis, who were included in a phase I safety trial of gene therapy [21]. Results showed that most patients were in good psychological health.…”

Section: Introductionmentioning

confidence: 99%

“…During the last years, several studies investigated the patient-physician communication and decision-making. The so-called shared decision-making model ensures that health care professionals do not make decisions solely on the basis of knowledge, experience and the latest scientific evidence, but that they also inform patients broadly and let them take part in all important aspects of the medical decision [21]. Regarding shared decision-making, patients' preferences for participation and their information needs are important, as there are patients who prefer an active role in their care and patients who desire a more passive role [22].…”

Section: Introductionmentioning

confidence: 99%

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Shared Decision-Making, Control Preferences and Psychological Well-Being in Patients with RPE65 Deficiency Awaiting Experimental Gene Therapy

et al. 2015

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Purpose: Retinal gene therapy trials are currently ongoing in a small number of inherited retinal disorders and this number is expected to rise significantly. The aim of this study was to analyze the psychological aspects of patients with RPE65 deficiency awaiting potential enrollment in gene therapy trials. Methods: Five patients with genetically proven RPE65 deficiency took part in this study. They were asked to complete the German versions of (i) the Patient Health Questionnaire (PHQ-D), (ii) the National Eye Institute Visual Function Questionnaire (NEI-VFQ), (iii) the Shared Decision Making Questionnaire (PEF-FB-9), and (iv) the Autonomy Preference Index (API-Dm), and in addition they took part in qualitative interviews. Results: The evaluations of the questionnaires and the interviews showed that the patients have quite high information needs and wish to take part in medical decisions. The perspective to participate in gene therapy trials does not seem to cause pronounced worries. Only the insecurity about if and when enrollment in a trial takes place may be burdensome. Discussion: This study generated important data about the psychological situation of patients awaiting potential enrollment in clinical trials, which can be used to improve patient care in the increasing number of future gene therapy trials around the world.

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Gene therapy for cystic fibrosis: a psychosocial study of trial participants

Cited by 12 publications

References 11 publications

Public Attitudes to Human Gene Therapy: A Pilot Study in Wales

Public Attitudes to Human Gene Therapy: A Pilot Study in Wales

Pre-clinical and clinical endpoint assays for cystic fibrosis gene therapy

Shared Decision-Making, Control Preferences and Psychological Well-Being in Patients with RPE65 Deficiency Awaiting Experimental Gene Therapy

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