James Slade is the Advanced Practice Nurse, Haemophilia & Bleeding Disorders at The Canberra Hospital. He works as part of the Haemophilia Treatment Centre team caring for both paediatric and adult patients diagnosed with a bleeding disorder
The WFH World Congress is an opportunity for clinicians from all fields of care, patients and their carers along with industry to share their research, innovation and ideas on a global stage. The Congress provides an incredible learning opportunity that for me resonates long after its conclusion.
Sunday was set aside as an opportunity for professional development, with a nursing workshop set around key areas of treatment. This year there was also an interesting opportunity to discuss with the editors of the journal Haemophilia who provided tips on getting your research published. For the first time this year the WFH nurses working group were able to incorporate nursing sessions daily, as well as a full day’s workshop on Sunday, providing us with an even greater platform for presenting our work. This is a trend I hope is continued in Congresses to come.
A couple other sessions that were further highlights for me were surrounding new and innovative products currently under clinical trial: one for haemophilia B and the other for haemophilia A.
Once-weekly dosing with a long-acting glycoPEGylated factor IX, nonacog beta pegol (N9-GP), maintains time with high mean trough levels in previously treated adult, adolescent, and pediatric patients with severe/moderate hemophilia B – Results from two phase 3 clinical trials
– Guy Young, USA
Dr Guy Young on behalf of his group presented updated data on two phase 3 clinical trials for a long-acting factor IX product. The focus of this presentation was on going beyond the current common idea that prophylaxis with standard factor IX should be aiming at trough levels above 1%. Dr Young asked are we setting targets too low and with the use of longer acting products could we as a treating team aim for trough levels greater than 15% and what would this mean for our patients. The results of this trial are encouraging: in two of the phase 3 trials the N9-GP factor has been providing effective once weekly prophylaxis in both adults and adolescents as well as in children. The thoughts behind these trials were aiming to increase trough levels while keeping to a minimal infusion schedule, but at the same time maintaining or exceeding current expectations of treatment. So far results are very exciting and encouraging with the once weekly regime potentially shifting patients from a severe/moderate haemophilia B range into a non-haemophilic range for a substantial time period throughout the week.
Updated results of an ongoing long-term phase 1/2 study of emicizumab (ACE910) in hemophilia A patients with or without inhibitors
– Keiji Nogami, Japan
Another exciting product in development is the ACE910 monoclonal antibody that was created as a humanised bispecific agent (binding to two different agents in humans) against factor IXa and factor X. Its main purpose is to mimic activated factor VIIIa – to carry out a key role in the clotting cascade that is normally carried out by factor VIII, which is deficient in haemophilia A. Factor VIIIa is the activated FVIII molecule which then continues the activation pathway in the coagulation cascade. The principle of this bispecific antibody is based on the hypothesis that, in the chain of steps in the clotting cascade where factor VIII is a co-factor for factor IXa (the activated form of factor IX), the next steps in the clotting cascade are enhanced by interactions between factor IXa and factor X. ACE910 is therefore designed to promote blood clotting in people with haemophilia A, and – because it is different in structure to factor VIII, it is anticipated that it will not create inhibitors to factor VIII.
Pharmacokinetic parameters indicated that the half-life of AC910 was approximately 3 weeks for both single intravenous and subcutaneous administrations which in the current and future potential of haemophilia A treatment is very exciting. It was also presented that effective haemostatic levels might be maintained by once-weekly subcutaneous (injection just under the skin) administration of ACE910. Being able to inject subcutaneously is an important option for people with haemophilia A who are struggling with vein care. ACE910 can be used to treat both people with or without inhibitors effectively.
This is the second time I have had the opportunity to attend a WFH World Congress; the first was Melbourne in 2014 when my involvement in bleeding disorders had only just begun. WFH Congress in Orlando this time around was truly inspiring, providing on more than one occasion information overload, and further highlighted that there are nowhere near enough hours in the day to do it all. Yet it is comforting to know that there are so many of us that work so hard and are just as passionate about bleeding disorders to continue to produce such high quality education as demonstrated here. Bring on WFH Congress 2018 in Glasgow Scotland.
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