Elocta found superior to standard half-life products: Real-world study
Hemophilia A patients had better bleed control, fewer injections over 2 years
Real-world use of the extended half-life factor replacement therapy Elocta (efmoroctocog alfa) was associated with better bleed control than standard half-life products among people with hemophilia A, according to findings from an observational study in Europe.
Elocta, sold as Eloctate in the U.S., was also associated with fewer injections and lower consumption of factor replacement products over nearly two years of follow-up.
“These real-world results support the contribution of [Elocta] to address the unmet needs in patients” on standard half-life factor prophylaxis, or preventive treatment, researchers wrote.
The study, “Superior Prophylactic Effectiveness of a Recombinant FVIIIFc Over Standard Half-Life FVIII in Hemophilia A: A-SURE Study,” was published in the European Journal of Haematology. It was funded by Sobi, which markets Elocta in Europe and several other regions.
Hemophilia A patients lack enough of an important blood clotting protein called factor VIII (FVIII), leading to excessive, prolonged, or spontaneous bleeding episodes.
Factor replacement therapy is standard for hemophilia
Standard-of-care treatment involves factor replacement therapy, where a version of the missing FVIII protein is routinely infused into the bloodstream as a way of restoring normal blood clotting.
These treatments come in two forms: standard half-life (SHL) and extended half-life (EHL). Compared with SHL therapies, EHL products are modified in ways that help them last longer in the bloodstream, enabling less frequent dosing.
Elocta is an EHL therapy that’s been approved in the European Union since 2015 for preventing and treating bleeds, including those happening during surgery, in hemophilia A patients of all ages. It’s been similarly approved in the U.S. since 2014.
The A-SURE observational study (NCT02976753) was designed to compare the clinical outcomes of male hemophilia A patients treated with Elocta (184 people) or standard half-life products (170 people) in real-world clinical practice at European centers over a two-year period.
All participants had received preventive factor replacement therapy in the year before enrolling in the study and had at least a year’s worth of clinical data available leading up to the study. Participants on Elocta were matched to those on standard products in terms of clinical factors such as age and previous replacement therapy use.
The study had three main goals: to evaluate bleed rates, injection frequency, and overall factor consumption. Patients in either group were followed for a mean of a little less than two years.
Patients on Elocta experienced significantly lower annualized bleed rates
Results showed patients on Elocta experienced significantly lower annualized bleed rates than those on conventional products over that period. Specifically, the mean number of bleeds per year in the Elocta group was 1.5, compared with 2.3 in the standard half-life group, meaning that those on Elocta experienced, on average, nearly one less bleeding event per year than those on other products.
Moreover, the annualized number of required injections per year was significantly lower with Elocta — a mean of 114.4 yearly injections compared to 169.2 with conventional therapies.
This was accompanied by a lower overall consumption of factor products, with those on Elocta using a mean of 243,024 international units (IUs) annually and those on conventional products using a mean of 288,718 IUs.
Secondary outcome measures also favored Elocta over conventional products, with patients on the EHL product having significantly lower annualized rates of joint bleeds.
Among the 27 people who started on Elocta for the first time at the start of the two-year study, bleed rates were reduced by a mean of 0.8 per year compared to the year before enrollment. Overall factor consumption and injection frequency were also reduced after the switch.
Other patient-reported outcome measures were collected, but results were inconclusive due to a limited number of respondents, according to the team. No significant differences were found between groups in terms of hospitalizations or outpatient clinical visits.
Safety data were consistent with the known profiles of both types of replacement therapies. No serious adverse events related to Elocta were reported.
“Overall, our findings show clear benefits of [Elocta] …, with improved protection against bleeds, fewer injections, and lower factor product consumption,” the researchers wrote. “Further real-world studies comparing products directly are warranted and would be important to help inform physicians and guide decision-making.”
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