Enzyme replacement therapy compared with best supportive care for the treatment of Pompe Disease: a systematic review and network meta-analysis.

What was studied

This review compared enzyme replacement therapy with supportive care in late-onset Pompe disease. It included 60 studies. Searches ran up to May 2024. It used a network analysis to compare drugs that were not tested head to head. It looked at the 6-minute walk test and a lung measure called forced vital capacity.

What they found

After about 1 year, people new to the therapy walked farther than with placebo. Gains were about 25 m with alglucosidase alfa. They were about 54 m with avalglucosidase alfa. No significant gaps were found for the lung measure. In single-group studies, early gains held for 1-3 years. Then walking and lung measures fell slowly over 10 to 15 years.

What it means for patients

The authors say the therapy modestly improves the walk test after 1 year versus placebo. They say evidence for real gaps between the drugs is limited. They say long-term gains are uncertain.

Why it is not certain yet

Small samples and missing data add doubt. Some trials had a high risk of bias. Long-term comparison with supportive care was not possible. Few people new to cipaglucosidase alfa were in the analyses. The long-term effect on disease progression is still uncertain.

Source

Enzyme replacement therapy compared with best supportive care for the treatment of Pompe Disease: a systematic review and network meta-analysis.

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