This rapid response report from the Canadian Agency for Drugs and Technologies in Health reviews the clinical effectiveness and safety of enzyme replacement therapy (ERT: imiglucerase and velaglucerase alfa) and substrate reduction therapy (SRT: miglustat and investigational agent eliglustat tartrate) as monotherapy or in combination therapy for patients with Gaucher disease.

It found that “limited evidence supports the use of ERT for type 1 Gaucher disease and shows no additional benefit of combination with SRT The evidence is insufficient to draw conclusions regarding the safety and efficacy of substrate reduction treatment for Type 1 Gaucher disease.”

The Rapid Response Service is an information service for those involved in planning and providing health care in Canada. Rapid responses are based on a limited literature search and are not comprehensive, systematic reviews. The intent is to provide a list of sources and a summary of the best evidence on the topic that CADTH could identify using all reasonable efforts within the time allowed. Rapid responses should be considered along with other types of information and health care considerations