Dataset Information

The experiences of people with haemophilia and their families of gene therapy in a clinical trial setting: regaining control, the Exigency study.

ABSTRACT:

Background

Gene therapy has the potential to change the life experience of people with haemophilia and family members. Few studies have sought to explore the impact of gene therapy on both individuals and families. The aim of this study was to capture real-life experiences of gene therapy in People with haemophilia and their families.

Results

Sixteen participants with severe haemophilia (11 haemophilia A, five haemophilia B), mean age 41.4 years (range 23-75 years), took part in a single qualitative interview; ten were accompanied by a family member. Mean time since transfection was 3.56 years (range 1-10 years). Participants saw their involvement in gene therapy as a positive experience, freeing them from the personal burden of haemophilia and furthering treatment options for the wider haemophilia community. However, participants reported being unprepared for the side effects of immunosuppression. Some also reported feeling unsupported and having little control over what was happening as their factor levels became the focus of the process.

Conclusion

The results suggest that strategies need to be put into place to enable PwH fully to understand the process of gene therapy, and thereby make an informed choice as to whether it is a treatment they might wish for themselves. These include early and ongoing education, increased provision of psychosocial support and ongoing qualitative research.

SUBMITTER: Fletcher S

PROVIDER: S-EPMC8981747 | biostudies-literature | 2022 Apr

REPOSITORIES: biostudies-literature

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Publications

The experiences of people with haemophilia and their families of gene therapy in a clinical trial setting: regaining control, the Exigency study.

Fletcher Simon S Jenner Kathryn K Pembroke Luke L Holland Michael M Khair Kate K

Orphanet journal of rare diseases 20220404 1

<h4>Background</h4>Gene therapy has the potential to change the life experience of people with haemophilia and family members. Few studies have sought to explore the impact of gene therapy on both individuals and families. The aim of this study was to capture real-life experiences of gene therapy in People with haemophilia and their families.<h4>Results</h4>Sixteen participants with severe haemophilia (11 haemophilia A, five haemophilia B), mean age 41.4 years (range 23-75 years), took part in a ...[more]

PMID: 35379267

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Project description:This study explored 12 cancer patients' experiences from participating in an online and on-site Shared Reading group for 16 weeks in Norway. Shared Reading is a practice in which prose and poetry are read aloud in small parts and discussed along the way. The study is a qualitative evaluation study with a particular focus on how the participants experienced the reading group supported their life living with cancer. The study was mainly based on the data collected from focus group discussions with the participants, which was analysed qualitatively through open coding. In total, four themes were identified: (1) open space, (2) disconnecting through connecting, (3) community, and (4) resonances and echoes. The participants expressed that the RG helped them to "balance life and cancer", and "disconnect" from their illness. The cognitive effort needed was beneficial for the participants as a form for "cognitive training." Since many of the participants had, due to their illness, completely stopped reading books, the reading group also brought literature back into the participants' lives. Furthermore, it was essential for the participants to feel they contributed to a community, to feel useful and valuable for others. The texts were also important, as some of them resonated strongly with the participants in the way of activating memories and connecting a text to own experiences. After a session, a text could still have an impact as an echo. The results are synthesised, discussed, and supported through the framework of self-determination theory and, more specifically, the basic psychological need theory. The reading group was experienced as a support for autonomy, competence, and relatedness and promoted a feeling of intrinsic motivation that brought about new dimensions in the participants' lives. The study wishes to increase our knowledge of the benefits of integrating Shared Reading groups as a low-cost, literature-based psychosocial support in cancer organisations.

Project description:BackgroundChronic obstructive pulmonary disease (COPD) presents with physical, emotional and social difficulties that affect quality of life. Multimodal management includes both pharmacological and non-pharmacological strategies, and pulmonary rehabilitation (PR) plays an important role. Recent research has suggested that manual therapies may improve perceptions of dyspnea for people with COPD.MethodsFocus group interviews were conducted as part of a mixed methods study to assess the feasibility of implementing a manual therapy technique-muscle energy technique (MET)-as an adjunct to PR for people with moderate to severe COPD. Focus group interviews were conducted to examine trial participants views of the intervention and the trial design. A thematic analysis was undertaken to explore the data.ResultsTwelve participants with moderate to severe COPD participated in three focus groups. Participants were motivated to participate in the trial to be proactive about their health. They perceived MET to be a gentle, comfortable form of stretching that allowed them to 'breathe easier' and prepared them for PR. A small number of participants reported mild muscular discomfort during MET, but this was short-lasting and was not bothersome. Participants enjoyed the one-on-one contact with researchers and learned more about their breathing while performing spirometric testing. Most participants wanted longer and more frequent MET sessions, and some requested 'homework' stretching exercises.ConclusionsThe findings of this study show that a manual therapy intervention was received well by participants in a clinical trial setting. A small number of participants reported mild musculoskeletal discomfort in relation to the MET treatment. Participant preferences for additional and longer treatment sessions should be carefully considered against available resources in future clinical trials.Trial registrationANZCTR, ACTRN12618000801213. Registered 11 May 2018 - Retrospectively registered. http://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id=374643&isReview=true.

Dataset Information

The experiences of people with haemophilia and their families of gene therapy in a clinical trial setting: regaining control, the Exigency study.

Background

Results

Conclusion

Publications

The experiences of people with haemophilia and their families of gene therapy in a clinical trial setting: regaining control, the Exigency study.

Similar Datasets

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