Alglucosidase Alfa Benefits Pompe's Disease Patients

Treatment group showed improved walking distance, stabilization of pulmonary function

WEDNESDAY, April 14 (HealthDay News) -- The use of alglucosidase alfa is associated with longer walking distance and stabilization of pulmonary function in patients with late-onset Pompe's disease, according to research published in the April 15 issue of the New England Journal of Medicine.

Ans T. van der Ploeg, M.D., of the Erasmus MC University Medical Center in Rotterdam, Netherlands, and colleagues analyzed data from 90 patients, aged 8 years and older, with late-onset Pompe's disease, which is caused by deficiency of acid α-glucosidase. Subjects were ambulatory and free of invasive ventilation and were randomized to receive placebo or biweekly intravenous alglucosidase alfa for 78 weeks.

The researchers found that those in the treatment group had increased walking distance on a six-minute walk test (increase of 28.1 m), and an absolute increase of 3.4 percentage points in forced vital capacity. Adverse events that occurred only in the treatment group included anaphylactic reactions and infusion-associated urticaria, chest discomfort, vomiting, flushing, hyperhidrosis and increased blood pressure, though similar proportions of patients in each group had adverse events, serious adverse events, and infusion-associated reactions.

"In summary, our data indicate that alglucosidase alfa treatment, as compared with placebo, has a positive, if modest, effect on walking distance and pulmonary function in patients with late-onset Pompe's disease and may stabilize proximal limb and respiratory muscle strength," the authors conclude.

The study was funded by the Genzyme Corporation, which employs several co-authors.

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