Reducing infusion time of Vpriv (velaglucerase alfa) enzyme replacement therapy (ERT) in patients with type 1 Gaucher disease from one hour to 10 minutes may be possible using a time-reducing protocol.
Developed by a team led by Ari Zimran, MD, head of the Gaucher Clinic at the Shaare Zedek Medical Centre and professor at the Hadassah-Hebrew University Medical School in Jerusalem, this new protocol allows the administration of Vpriv much faster, either in a clinic or at home, without compromising the safety of the patients or the treatment’s effectiveness.
This may have significant implications not only regarding improvements patients’ quality of life, but also compliance to treatment because of decreased costs.
These findings were reported on a poster presentation titled “Reduction in Intravenous Infusion Duration of Enzyme Replacement Therapy to Ten Minutes with Velaglucerase Alfa in Adult Patients with Type 1 Gaucher Disease” at the 59th ASH Annual Meeting & Exposition held in Atlanta, Georgia in early December.
For many patients with type 1 Gaucher disease ERT is a lifelong commitment that requires weekly or bimonthly visits to infusion centers or clinics. These treatments can be very time-consuming with each appointment taking one hour or more, which represents a major disadvantage in comparison with orally-administered medications.
Previous clinical data have shown that rapid intravenous administration of biological medicines could trigger reactions ranging from mild local irritations to serious life-threatening immunological and inflammatory threats.
The team conducted a “change in practice” study to evaluate the safety and effectiveness of rapid infusions in 15 adult patients with type 1 Gaucher disease who already were receiving Vpriv for at least three months.
All the participants were receiving intravenous ERT (dosing ranging 15 to 60 UI/kg) at home with the standard 60-minute protocol every other week. Next, the team changed the rate of ERT administration in a step-wise manner, starting with one infusion at 30 minutes, followed by one infusion at 20 minutes, and then four infusions over 10 minutes, all in the hospital clinic setting. At the final stage of the study the patients received ERT via 10-minute infusions at home, with clinical follow-up evaluation every three months.
The patients were followed for a mean time of 60 weeks. During this period, no serious adverse events associated with the new treatment were reported, and only one patient experienced mild discomfort in the infused arm.
Despite the changes in infusion rate, all patients maintained their levels of key disease-associated parameters, including organ volume and blood features. Until week 36 no patient had developed antibodies against the treatment.
Overall these results suggest that increasing the administration rate of Vpriv is feasible, since it does not change the treatment effectiveness and it does not represent a risk for patients.
“Willingness of patients to continue with 10 minute infusion confirms the importance of convenience, specifically decreased infusion duration, to patients for whom ERT infusions are currently envisioned to be a life-long commitment,” the researchers stated.