Gaucher Outcome Survey Helps Track Global Treatment Strategies

Gaucher Outcome Survey Helps Track Global Treatment Strategies

Medical professionals in several countries use a range of approaches to treat Gaucher disease (GD), and data from these strategies can help determine the impact of different treatment patterns on real-world outcomes, a recent study said.

The findings were reported at WORLDSymposium 2018 in San Diego, California, in a poster titled “Treatment patterns from 647 patients with Gaucher disease: An analysis from the Gaucher Outcome Survey.”

To date, six therapies have been approved for the treatment of GD, including four enzyme replacement therapies (ERTs) and two substrate reduction therapies (SRTs). ERTs tend to be the standard of care for treating the manifestations of symptomatic type 1 GD.

Analysis of treatment patterns from around the world can help to provide a picture of how GD management varies among countries. It also can serve as a way to assess data from clinical outcomes regarding the effectiveness of different therapeutic approaches.

The Gaucher Outcome Survey (GOS) is an international GD-specific registry that collects real-world data from patients, including information on disease manifestations and treatment history. The registry includes data from 34 treatment centers in 10 countries involving 1,003 patients.

At the time of the analysis, results showed that VPRIV (velaglucerase alfa) and Cerezyme (imiglucerase) were most widely used. Among patients on treatment, more than 95 percent were receiving an ERT.

Analysis also showed that 68.9 percent of patients had been treated longer than five years, and 56.9 percent had received only one GD-specific therapy.

However, while most patients received treatment with a single therapy, changes from one ERT to another accounted for the majority of treatment changes.

A total of 377 patients had received at least one dose of VPRIV at any given time. The VPRIV dose most commonly used was 60 U/kg every other week.

However, differences were noted in dosing between the three highest-enrolling countries: Israel, the United Kingdom, and the United States. Most patients in Israel received less than 20 U/kg, while most patients in the U.K. received between 20 and 40 U/kg. Most patients in the U.S. received 60 U/kg.

“Overall, our data indicate a range of approaches toward GD management globally, which can be influenced by a number of interacting factors including drug availability, treatment cost, local prescribing practices, and disease severity,” the investigators concluded. “Further analyses from GOS can help examine the impact of these treatment patterns on real-world outcomes.”

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