Safety and Efficacy of New Hemophilia Treatments Are Most Important, Patients Report


Safety and efficacy were the most important factors for patients with hemophilia learning about gene therapies, although more research is needed to see how collaboration between providers and patients contributes to the best outcomes.

When it comes to hemophilia education for patients, safety and efficacy were discovered to be the most important factors when communicating information about new hemophilia treatments, according to survey findings published in Haemophilia.

The researchers, who leveraged the National Hemophilia Foundation (NHF) Community Voices in Research (CVR) platform, found that efforts need to be made to educate people with hemophilia (PwH) on new therapeutic treatments so that they can make informed and effective decisions with their provider.

Hemophilia treatment centers and patient advocacy groups need to be well-versed in education for PwH regarding components of new therapies that are individually important, particularly safety and efficacy in the short term and long term.

This study was conducted to garner a further understanding of how PwH want to learn and talk about new therapies and how these discussions can be used to inform and educate patients, especially in relation to treatment decisions.

Researchers set out to analyze patient desires and factors contributing to gene therapy and other new hemophilia therapy treatment decisions.

A total of 104 men enrolled in NHF CVR with English proficiency and acute hemophilia A (HA) or hemophilia B (HB) participated in the survey from May to September 2021.

The survey included questions about sociodemographics, clinical characteristics, treatment for hemophilia that was presently being received, how the patient wanted to learn, if they had previous experience or knowledge with new therapies, and how they wanted to make decisions about gene therapy.

Thirty-three percent of the men were Hispanic, 92% of them had not had gene therapy, and 74% were on prophylaxis. Of those who had no prior gene therapy, 73% indicated a high likelihood of considering it, but there were some concerns reported.

When asked what considerations worry them about gene therapy, the PwH mentioned potential safety concerns such as liver impacts or vector shedding, as well as efficacy considerations such as factor level achieved, duration of effect, need for redosing, and impact on bleeding.

Sixty-seven percent of participants said that “It is too early to know all the risks” and 49% said that “I want to wait and see what happens.”

It was found that 50% of patients were uninterested in changing their prophylaxis, which increased to 68% in those currently receiving emicizumab as prophylaxis. About one-third (32%) of participants had no desire to contemplate gene therapy because they were happy with their current treatment.

The survey revealed that PwH of Hispanic ethnicity were more likely to consider a treatment option of gene therapy. Government-issued insurance also increased the likelihood of contemplating gene therapy. A total of 14% of survey participants denoted that cost was a barrier to the consideration of gene therapy.

More research is needed on how patient activation and health literacy contribute to decision- making and how equitable access and valid informed consent for new therapies can be accomplished, according to the study authors.

Learning methods recorded in this survey extended from scientific literature and conferences to groups chatting online and Facebook, so the researchers recommended that providers should use a multifaceted education base for patient reference such as peer-led discussions, printed materials, and e-learning platforms to address diverse learning and language needs.

Twenty-four percent of survey participants indicated anxiety about gene therapy in saying “I don’t know anyone else who’s tried it,” showing the profound impact of connecting with the hemophilia community.

Peer navigation resources would be helpful, as 38% indicated that “friends and family members” and 37% indicated that “support groups” are where PwH are most likely to get updated information about hemophilia and the available treatments.

Some limitations of this study include the fact that participants primarily consisted of PwH in the US Hemophilia Treatment Center network who had high English proficiency and were educated. The authors suggested that future studies include a more diverse patient population with limited English proficiency and lower educational attainment.

“These data identify key elements for patient and provider education and may inform the development of [shared decision-making] tools for patients to help patients have balanced and accurate information about new therapies and to guide informed decisions,” the researchers concluded.


Limjoco J, Calatroni A, Aristizabal P, Thornburg CD. Gene therapy preferences and informed decision-making: results from a National Hemophilia Foundation Community Voices in Research survey. Haemophilia. Published online November 17, 2022. doi:10.1111/hae.14706

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