Clinically important improvements in 6-minute walk distance and forced vital capacity in adults with late-onset Pompe disease switching from alglucosidase alfa to cipaglucosidase alfa plus miglustat in the PROPEL study.

What was studied

This was a post hoc analysis of the PROPEL trial. It involved adults with late-onset Pompe disease. It included 95 patients. They either switched to cipaglucosidase alfa plus miglustat or stayed on alglucosidase alfa plus placebo. The team counted who had clinically important changes. They looked at 6-minute walk distance and forced vital capacity (FVC). They used published cut-offs for what counts as important.

What they found

More patients improved in walking distance after the switch. Fewer patients got worse. For FVC, some patients improved with the switch. None improved on the old drug plus placebo. More got worse on the old drug plus placebo. For both measures together, more patients improved with the switch. The odds of a better response category were 4.05 times higher with the switch.

What it means for patients

The authors conclude that adults who switch have greater chances of clinically relevant gains. These are gains in movement and lung function. They compare them with those who stay on the old drug.

Why it is not certain yet

This was a post hoc analysis, done after the trial. It included 95 patients. The cut-offs came from published work, not from this trial. The abstract does not say how long patients were followed.

Source

Clinically important improvements in 6-minute walk distance and forced vital capacity in adults with late-onset Pompe disease switching from alglucosidase alfa to cipaglucosidase alfa plus miglustat in the PROPEL study.

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