Investigating the therapeutic profile of velaglucerase alfa in paediatric patients with Gaucher disease: a systematic review across all paediatric age groups.

What was studied

This review looked at velaglucerase alfa in children with Gaucher disease. It covered type 1 and type 3. It searched medical databases and conference reports. In all, 23 studies with 159 children were included. It looked at safety and at how well the drug worked.

What they found

In type 1, the drug was described as well tolerated. Blood, organ, bone and marker results improved in children new to treatment. So did quality of life. Children who switched from imiglucerase stayed stable. Home therapy was reported to improve quality of life for children and caregivers. In type 3, safety looked good in 7 papers. Non-nerve symptoms improved.

What it means for patients

The authors say the drug may help in type 1 across all child ages. They say it might be an option for non-nerve symptoms of type 3.

Why it is not certain yet

The authors call the evidence limited. For type 3, it is scarce and exploratory. Effects in type 3 were reported for only 16 patients. They call for more research in those patients.

Source

Investigating the therapeutic profile of velaglucerase alfa in paediatric patients with Gaucher disease: a systematic review across all paediatric age groups.

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