Dr Dan Hart
Consultant haematologist at The Royal London Hospital Haemophilia Centre
New haemophilia treatments could increase protection, cut complication rates, reduce infusions by half – and save the NHS money.
A leap forward in medical engineering could mean a better life for people with haemophilia.
Extended half life (EHL) factor 8 and 9 products could replace the conventional factors used by children and adults to protect themselves from the painful and damaging spontaneous bleeding that characterises haemophilia.
Dr Dan Hart, consultant haematologist at The Royal London Hospital Haemophilia Centre, explains: “Prophylactic treatment currently relies on people injecting synthetic (recombinant) factor 8 or 9 protein every other day or even daily. Children often need injections 180 days a year.
“But in the new EHL products the synthetic proteins have been engineered to last longer, prolonging the protective effect by 1.5 times for factor 8 and three to five times for factor 9.”
A leap forward in medical engineering could mean a better life for people with haemophilia.
This could increase protection, cut complication rates and reduce intravenous infusions by 30 to 50% annually. “That’s 50 to 90 fewer infusions per year,” says Hart. Trials show that EHL products work as well as conventional products to prevent bleeds.
EHL may mean longer, better protection. The need for fewer infusions might increase compliance, reducing complication rates. Community care could become easier both at the start of life and for the increasing number of elderly men who may struggle to self-inject. Hospitalisation for bleeds or surgery could reduce.
“Disability will be reduced over time, improving quality of life, reducing need for joint replacements, minimizing falls risk and cutting chronic care needs,” says Hart.
Much depends on cost. The UK has the cheapest recombinant factor in Europe as a result of a coordinated national tender. “The new products must be priced very close to the existing products to enable uptake,” Hart says. “Some patients using EHL factor 9 have reduced treatment from twice a week to twice a month. Realistically priced EHL products could offer equal or better protection at the same or reduced cost.”
The necessary screening enabling people to switch product already exists, and adverse effects will continue to be monitored.
Other new treatments are also on the horizon. Further results from gene therapy trials are expected at the end of the year, and new subcutaneous delivery systems are on trial that may reduce dosing to once a month.
Hart says: “Treatment options for people with haemophilia will be very different within five to ten years. Potentially exciting new agents still need to prove their efficacy and safety, but extended half life factors already bring the chance of better care and outcomes at no extra cost.”
