Development of a haemophilia A gene therapy shared decision‐making tool for clinicians

Limjoco, Jacqueline; Thornburg, Courtney D.

doi:10.1111/hae.14822

Cited by 10 publications

(4 citation statements)

References 30 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Education and support of PwH during the SDM process using currently available and developing SDM tools [27,30,31] are paramount to ensure fully informed individual decision-making and consent. The development of tools to ensure equitable access to education and information about haemophilia gene therapy and the patient journey are now starting to be developed.…”

Section: Discussionmentioning

confidence: 99%

“…Having a point of reference of this kind can help to ensure that all elements that need to be discussed in clinic are discussed [18] . In the US, Limjoco and Thornburg have consulted people with haemophilia A and HCPs on the development of an SDM tool or tools for gene therapy, incorporating a checklist and taking both perspectives into account [29,30] . PwH felt that having access to an SDM tool laying out the pros and cons of gene therapy would support their decision-making outside of clinic through enabling further review and discussion with their families.…”

Section: How Do We Ensure Shared Decision-making Is a Reality?mentioning

confidence: 99%

“…The proposed checklist included education about gene therapy, risks, comparison with other treatments, follow up, impact on mental health and quality of life [29] . A tool aimed at facilitating HCP discussion has been developed, which can be used during patient discussions on gene therapy in combination with the SDM tool developed by the World Federation of Hemophilia (WFH) [27,30,31] . The WFH SDM tool is designed to assist with treatment selection and includes a significant number of downloadable patient education materials comparing currently available treatments alongside gene therapy [31] .…”

Section: How Do We Ensure Shared Decision-making Is a Reality?mentioning

confidence: 99%

See 2 more Smart Citations

Shared decision-making for gene therapy in haemophilia care

Fletcher,

Jenner,

Khair

2023

The Journal of Haemophilia Practice

View full text Add to dashboard Cite

Shared decision-making (SDM) is an important part of patient-centred care in which healthcare professionals (HCPs) and patients/caregivers jointly reach care decisions through a two-way exchange and synthesis of information based on clinical evidence and patient preference. SDM was described in haemophilia care in 2014 as two-sided intervention to aid patient decision-making. However, as the range of haemophilia treatments has expanded, identifying the optimal haemophilia treatment for an individual has become more complex. This is particularly so in the case of gene therapy, a onetime-only, irreversible treatment. In this context, it is vital that people with haemophilia (PwH) and their families continue to be involved in care decisions in an informed and interactive way. For gene therapy, this must include being well informed about the gene therapy process, enabling the patient to engage in fully informed SDM and consent, and ensuring that issues around long-term durability, potential side effects, the need for long-term follow-up are understood with a recognition that the ‘unknown unknowns’ are also unknown to HCPs. Both HCPs and patient organisations have a key role to play in providing PwH with access to relevant information and education, tailored to individual needs and free of jargon. Considerable education and support are required before PwH can make a truly informed decision about having gene therapy. Use of structured SDM tools such as the SHARE approach can help to support this. There is a need for SDM educational tools that include written/visual information and the use of standardised checklists may be helpful for both PwH and HCPs. The most important part of this process is that PwH want to undergo gene therapy – and this is only an option if they are fully educated and informed by fully educated and informed healthcare teams.

show abstract

Section: Discussionmentioning

confidence: 99%

Section: How Do We Ensure Shared Decision-making Is a Reality?mentioning

confidence: 99%

Section: How Do We Ensure Shared Decision-making Is a Reality?mentioning

confidence: 99%

See 1 more Smart Citation

Shared decision-making for gene therapy in haemophilia care

Fletcher,

Jenner,

Khair

2023

The Journal of Haemophilia Practice

View full text Add to dashboard Cite

show abstract

“…The multidisciplinary teams of haemophilia care centres must provide objective information for these irreversible treatments that cannot be stopped once administered. Patient education and the development of shared decision‐making tools will be mandatory to allow patients to reach a decision liable to ‘change their life’ after being well aware of transparent information about efficacy, safety, uncertainties and unknowns of HGT 11–14 . The World Federation of Haemophilia has developed a shared decision‐making tool to guide patients and caregivers in discussing treatment options (https://sdm.wfh.org/).…”

Section: Discussionmentioning

confidence: 99%

Perspectives and perception of haemophilia gene therapy by French patients

Pietu,

Giraud,

Chamouard

et al. 2023

Haemophilia

View full text Add to dashboard Cite

Introduction and aimA national survey was initiated by representatives of French patients with haemophilia (AFH) and the French reference centre for haemophilia, in order to appreciate the awareness and knowledge of these patients regarding haemophilia gene therapy (HGT) and understand better their position about this innovative treatment that will soon become available.ResultsOf 143 answers received, 137 could be analysed, representing about 3.5% of patients with severe or moderate haemophilia over 16year‐old. They were 80.3% with haemophilia A and 19.7 % with haemophilia B, with a severe form of the disease for 80.3 % of them. Curiosity for HGT was formulated by 64.2% of the participants, 33.6 % being interested by this approach as soon as it will be available and 38.7 % preferring to wait until more patients have been treated. Only 3.6 % of the participants would never consider receiving HGT. The level of awareness and knowledge was estimated to be limited by 39.5 % of the patients. More than 60 % of them declared having never or almost never discussed HGT with the team of their haemophilia centre. Before deciding to get HGT, 54.4 % of the participants considered that it will be very important to compare it with their current treatment and 53.7 % would like to be better informed by their care providers.ConclusionsThese results highlight the need for training and education for patients, but also for professionals at haemophilia centres, about HGT and the shared decision‐making process. Objective, unbiased and transparent information must be available for patients about this very promising therapy which nonetheless carries more uncertainty and unknowns compared to other haemophilia treatments.

show abstract