WFH Summit: new therapies and approaches

LIANE KHOO

Dr Liane Khoo is a haematologist and Haemophilia Treatment Centre Director at the Royal Prince Alfred Hospital, Sydney.

Health literacy, new VWD therapies, women

The World Federation of Haemophilia (WFH) 2025 Comprehensive Care Summit took place in Dubai, UAE from 23-25 April 2025. It brought together over 800 attendees from 93 countries; and I was fortunate enough to be able to attend it.

The Summit covered a wide range of topics, and I was particularly interested in the sessions on:

(i) Improving health literacy to improve shared decision making
(ii) Raising awareness and new therapies for von Willebrand disease (VWD)
(iii) Women and girls with bleeding disorders, especially with the newly formed HFA Women and girls Advisory Group.

HEALTH LITERACY AND SHARED DECISION-MAKING

New paradigm in patient education with new treatments ~ Kate Khair, UK

Dr Kate Khair, Director of Research, Haemnet, discussed a new paradigm that has developed in patient education with the new therapies.

She highlighted that in the last twenty years treatments have evolved from plasma and plasma derived products through recombinant factors (standard, extended, ultra-extended replacement therapy), monoclonal antibody therapy and gene therapy; with many other innovative therapies currently in clinical trials.

For those with haemophilia and their caregivers this raises issues of therapeutic choice.

Patient education is now more imperative than ever before, to enable informed shared decision-making about current and potential future treatments. Health literacy (a person’s ability to understand and use information to make decisions about their health) is necessary to underpin these decisions and we as a community need to be part of this journey. It is exciting that in Australia HFA is being proactive in this space with the ongoing development of the GETS (Gene and Emerging Therapies) Hub website.

VWD NEW THERAPIES

Novel VWD treatment ~ Caterina Casari, France
VWD & rare bleeding disorders – new technologies ~ Paula James, Canada

Continuing the theme on new innovations, there was a focus on new therapies for von Willebrand disease (VWD), presented by Dr Caterina Casari and Dr Paula James.

These new therapies include:

1. Increasing endogenous VWF
   Molecules: HMB-002, BT200, KB-V13A12
2. Increasing procoagulant potential
   FVIII (8) mimetics: emicizumab, Mim8, BIV001, NXT007
   General haemostatic agents: platelet-inspired nanoparticles, fitusiran, VGA039, marstacimab/concizumab
3. Correcting genetic alterations
   siRNA, gene therapy, CRISPR-Cas9-based editing
4. Preventing VWF degradation
   Anti-VWF Ab50 8, Anti-ATS13 Ab17C7

This is an exciting time for people with VWD as there are a lot more therapies that are becoming available in clinical trials.

We are fortunate in Australia to have several of these new treatments available as part of a clinical trial.

If you are interested in new VWD therapies, speak to your local Haemophilia Treatment Centre or haematologist about what clinical trials are available.

WOMEN AND GIRLS

There were also great sessions focusing on Women and Girls with Bleeding disorders, including sessions on heavy menstrual bleeding. Talks acknowledged the current challenges, barriers, underdiagnosis, taboos, medical sexism etc..; with an important call to action in this area. There was a good reference on strategies on how we can identify patients in clinic (see figure 1).

There were lots of other areas covered at the Summit, including health equity, machine learning, ageing, pain… and many more interesting topics.

I look forward to seeing a good Australian contingent at the next WFH Congress in Kuala Lumpur, Malaysia – 19-22 April 2026!

REFERENCES

1. Hermans C. A systematic clinic approach to the identification of women and girls with bleeding disorders. Haemophilia 2025; 31:3–20. https://doi.org/10.1111/hae.70031