Parents, Lacking Information, Wary of Gene Therapies, UK Survey Finds 

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Parents of children with hemophilia in the U.K. are generally aware of gene therapy, but many lack both the knowledge necessary for an informed decision and confidence regarding this form of treatment, according to a questionnaire and interview-based study.

Most of those interviewed were unaware that gene therapies are not currently being tested in children with hemophilia.

The study, “Parental perspectives on gene therapy for children with haemophilia: The Exigency study” was published in the journal Haemophilia.

Gene therapies for hemophilia are intended to introduce a working copy of the gene that encodes the missing clotting protein. Although not available for children, results from clinical trials in adults with severe disease suggest that gene therapy can have a long-lasting effect and significantly increase the activity of the missing clotting factor.

Lack of information on the therapy’s long-term safety and durability is currently an obstacle to its testing in children with hemophilia. However, this type of treatment should be considered in the future, especially for those with limited access to standard treatment, the researchers said.

But “there is little information about parental understanding of gene therapy in haemophilia, or about their attitudes to its potential as a treatment for their children,” they wrote.

Researchers at Haemnet, which specializes in work with bleeding disorders, led a study to assess these adults attitudes and opinions concerning gene therapy as a potential future treatment. They evaluated parents’ levels of therapy awareness, as well as their questions and concerns.

The study, sent to 158 parents of a child with hemophilia, included an online questionnaire and interviews. Sixty-three parents (39%) completed the survey, taking about five minutes on average. The high non-completion rate was due largely to respondents not residing in the U.K., having no knowledge of gene therapy, or not having children with hemophilia.

Sixty participants were mothers, including 31 with a family history of the disease.

Nearly all who completed the survey (60 of the 63) had heard of gene therapy for hemophilia. A majority, 76.6% or 46 respondents, said their information came from social media focused on hemophilia. Seventeen (28.3%) felt they had a good understanding, and a favorable attitude toward gene therapies.

Fifty-three respondents (88.3%) were open to considering a gene therapy for their child, but a majority — 63.3% or 38 people — did not know that gene therapy is currently unavailable for children with hemophilia.

Thirty-eight parents (63.3%) also said their discussions about gene therapy took place within the family, while 25 others (41.6%) had talked about this treatment with healthcare professionals. Thirteen study participants, 26.6%, said their children had asked for gene therapy.

“All participants felt their attitude towards gene therapy was conflicted,” the researchers wrote. “Individual participants felt full of hope and excitement but also worried about loss of control: gene therapy was life-changing but also scary.”
Ten mothers, with a total of 12 children with severe hemophilia, took part in the study’s online video interview focus group. They joined in two video interviews of 90 minutes, during which they were shown visual prompts of a gene therapy procedure and given a short explanation.

The women were equally divided in two groups. All their children were on prophylaxis treatment, and three were being treated with Hemlibra (emicizumab), an approved treatment for type A patients marketed by Roche.

Focus group discussion showed a high awareness but “lack of consistency in how families heard and learned about gene therapy,” the investigators wrote.

Mothers of older children (ages 9 and older) reported having long been aware of gene therapy, while those with younger children had heard about the treatment in the last two years. Most felt they were not well informed.

Knowledge was obtained mostly through the participants’ own research or randomly, rather from formal medical sources. Caregivers who used online sources reported the information they found was scientific and hard to understand.

Nurses with a hemophilia specialty were seen as the “most trusted” sources of “tailored and age-appropriate” advice about these therapies, followed by patient organizations.

After watching the video of the gene therapy procedure, mothers said their understanding had improved. “It looks a lot more simple. I know it’s meant to in the picture. But it’s definitely not what I thought it was,” one said.

Lack of information on long-term effects of gene therapy was the main cause of fear and concern among parents. Ethical considerations due to a treatment still in testing phases were also mentioned, both regarding the potential for a poor outcome and about a child going through a trial’s eligibility check only to be denied the therapy.

Parents not considering gene therapy at this point, however, also thought it important to learn more about this treatment.

In general, the researchers reported a lack of reliable and comprehensive sources of information about gene therapy, including updated reports on clinical trials and their outcomes.

Parents saw being better informed a way to not only stay hopeful, but also to better prepare for when gene therapy is available for children.

“The more exposure they have and we have every time we go to the centre, and go over it and learn a bit more, it gets time for us to take in that information bit by bit over the years, rather than all in one go,” one parent said.

Overall, “using a combination of information from relevant, trusted sources may be the best way to ensure that people with haemophilia and their families are able to obtain the knowledge needed to support informed decision-making about gene therapy now and in the future,” the researchers wrote.