Switching to Elocta shown to improve bleed control in real world
Therapy's use by hemophilia A patients also reduced weekly injections
Switching from standard half-life factor replacement therapies to the extended half-life treatment Elocta (efmoroctocog alfa) improves bleed control among people with hemophilia A.
That’s according to an analysis of data from the real-world A-SURE study (NCT02976753), which showed that switching to Elocta, sold as Eloctate in the U.S., also reduced the number of weekly injections among patients, as well as the use of factor replacement products.
“The role of [Elocta] to meet the unmet needs associated with [standard half-life] factor prophylaxis [preventive therapy ] is supported by the real-world evidence presented here,” the researchers wrote.
Indeed, the analysis showed that Elocta more than halved the number of annual bleeding episodes among hemophilia A patients.
The real-world study, “A-SURE: intra-patient comparison of prophylactic effectiveness of a recombinant factor VIII Fc fusion protein versus standard half-life factor VIII in hemophilia A,” was published in the journal Hematology. The work was funded by Sobi, which markets Elocta in the European Union and several countries.
People with hemophilia A lack the blood clotting protein factor VIII, or FVIII, causing excessive, prolonged, or spontaneous bleeding episodes.
Factor replacement therapy is standard treatment in hemophilia A
Factor replacement therapy is the standard treatment for hemophilia A, in which FVIII is routinely infused into the bloodstream to restore normal blood clotting. Recombinant factor replacement therapies are a subset of these treatments that use a lab-made version of FVIII.
There are two types of recombinant factor replacement treatments: standard half-life therapy and extended half-life therapy. As their name suggests, compared with standard half-life products, extended half-life therapies are modified to remain in the bloodstream for longer periods, allowing for less frequent dosing.
Elocta is an extended half-life recombinant factor replacement therapy approved in the European Union to prevent and treat bleeds, including those that occur during surgery, in hemophilia A patients of all ages.
A-SURE was an observational study that directly compared the effectiveness of Elocta with that of standard half-life products in 356 males with hemophilia A treated in real-world clinical practice over the course of two years. All participants had received preventive factor replacement therapy in the year preceding study enrollment.
Data from the study showed that Elocta improved bleeding control, resulting in fewer injections and lower consumption of factor replacement products compared with standard half-life therapies.
Number of bleeding episodes drop after switch to Elocta
Now, a research team analyzed A-SURE data to evaluate the clinical outcomes of 131 participants who switched from standard half-life therapy to Elocta before, at, or after enrollment. These patients, all male, ranged in age from 1 to 76. Most switched for two reasons: better bleed control (47%) or fewer injections (45%). The remaining 8% of patients switched for other reasons.
Before switching, the mean number of bleeding episodes per year was 3.7. After switching, that number dropped to an average of 1.8 — representing a mean of 1.9 fewer bleeds per year across all age groups.
For joint bleeds, switching led to a mean of 1.2 fewer bleeds per year, most prominently in older patients, the data showed.
Further, the researchers noted that the mean number of weekly injections decreased from 3.1 to 2.3 after switching to Elocta. That meant nearly one fewer injection per week across all age groups.
The mean weekly factor consumption was also reduced by a mean of 5.7 international units per kilogram (IU/kg). Importantly, however, among patients younger than 12, weekly factor consumption increased after switching, the data showed.
Switching to [Elocta] from [standard half-life] FVIII prophylaxis reduces [bleeding rates], injection frequency, and factor consumption, thereby meeting important unmet needs associated with [standard half-life] FVIII treatment.
In a subgroup analysis involving 27 participants who switched at enrollment, bleed rates were reduced by a mean of 0.8 per year compared with the year preceding enrollment. Likewise, there were fewer weekly injections, by a mean of 0.8, and less weekly factor consumption, by a mean of 7 IU/kg.
Lastly, the proportion of participants who reported no bleeding episodes was similar, either six months before switching to Elocta or as long as 12 months after switching. These proportions ranged from 55.8% to 58.9%.
Overall, according to the researchers, changing to Elocta showed benefits for the hemophilia patients in the study.
“Switching to [Elocta] from [standard half-life] FVIII prophylaxis reduces [bleeding rates], injection frequency, and factor consumption, thereby meeting important unmet needs associated with [standard half-life] FVIII treatment,” the researchers wrote, adding that “this is consistent with previously reported primary outcomes of the comparative A-SURE study.”
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