New Medicine Funding Offers Hope For Kiwis Living With Rare Blood Disorder
New Zealanders living with a rare blood disorder which can cause spontaneous, fatal internal bleeding and excruciating arthritis-like symptoms are set to benefit from the funding of a new medicine named Hemlibra® (emicizumab) from December 1, 2020.
Haemophilia A is an inherited blood disorder caused by lack of a clotting protein called factor VIII. It affects eight in every 100,000 Kiwis, with males most likely to be diagnosed with the condition.
According to Consultant Haematologist Associate Professor Paul Ockelford there are around 450 Kiwis living with haemophilia and about 135 have a severe form. The severe form puts them at extreme risk of spontaneous internal bleeding1.
“The bleeding will often pool around the joints which can cause significant levels of pain - in the past this was often treated with morphine which severely impacted the ability to function and led to time off work or school,” he says.
People with haemophilia A are usually treated with factor VIII replacement, which replaces the missing factor VIII and helps control and prevent bleeding. However, the body may develop inhibitors (antibodies) as a reaction to these medicines in around 25-30% of the cases. The inhibitors reduce the medicines’ effect, so bleeding is no longer controlled.
The funding of the new medicine is significant as it is the first treatment for those living with this disease who show inhibitors to factor VIII to be made available in New Zealand in the past 20 years. Hemlibra (emicizumab), works by mimicking the coagulation function of activated factor VIII and can prevent or reduce significantly the frequency of bleeding episodes in adults and children with hemophilia A with factor VIII inhibitors.
The treatment is usually given weekly via a subcutaneous injection, which means it can be injected under the skin without the inconvenience of needing to locate a vein.
Haemophilia Foundation of NZ CEO Sue Ellis says haemophilia can have a significant impact on the quality of life for those living with the condition as well as their families.
“Haemophilia is often picked up at birth, the baby may be born with unusually severe bruising and then tested as a result”
“The day-to-day risk associated with this condition will restrict family life choices including where they live, where they holiday and general family activities,” she says.
Ellis says one of the current treatments for children with the disease may involve regular intravenous injections every couple of days - with infusions taking 20 minutes each time.
Ellis says one of the key goals in treating those living with haemophilia is to prevent damage to joints which is caused by blood pooling in this part of the body.
“Young New Zealanders with haemophilia need to stay on top of the bleeds and work hard to avoid damage to their joints so that when they’re in their early twenties, they do not have to have a joint replacement or a joint fusion”.
“For older people we need to ensure we are supporting them in the right way and enabling them to have the right treatment packages including joint replacements to minimise further bleeding,” says Ellis.
Dr Ockelford says new scientific advancements are helping extend the life expectancy of those living with haemophilia.
“Five decades ago, someone living with severe haemophilia may have lived to be 25, now with new treatments they can have a normal lifespan, which is why we need to continue to support advances in scientific research in this field,” he says.
Roche NZ General Manager Alexander Muelhaupt says the company is proud to have developed Hemlibra and is excited to be able to provide this treatment option to Kiwis who live with haemophilia A with inhibitors.
Muelhaupt says data from one of the largest global studies among people with hemophilia A found an 87 percent reduction in treated bleeds in people who received the new drug as part of their treatment regime.
He says current treatment alternatives in people with haemophilia A who develop inhibitors are time-consuming and often difficult, especially for children and are not always effective. He says there was an unmet medical need for more convenient and effective treatment options.
Muelhaupt says while Hemlibra is now funded to treat those with the condition who have inhibitors, Hemlibra is also now registered for non-inhibitor patients. In the non-inhibitor trial called HAVEN 38, people treated with Hemlibra prophylaxis showed similar significant reductions in bleeding rates as seen in the Haven 1 inhibitor trial9.