WEDNESDAY, March 16 (HealthDay News) -- Sirolimus, a mammalian target of rapamycin inhibitor, appears to be an effective therapy for stabilizing lung function and improving symptoms and quality of life in patients with lymphangioleiomyomatosis (LAM), according to research published online March 16 in the New England Journal of Medicine.
Francis X. McCormack, M.D., of the University of Cincinnati, and colleagues randomized 89 patients with LAM and moderate lung impairment to 12 months of sirolimus or placebo followed by a 12-month observation period.
During treatment, the sirolimus group experienced a change in forced expiratory volume in one second of +1 ml per month, compared with −12 ml per month in the placebo group. The treatment group also experienced improved measurements of forced vital capacity, serum vascular endothelial growth factor D (VEGF-D), functional residual capacity, and quality of life and functioning. Adverse events were more common in the treatment group, but the frequency of serious adverse events did not differ significantly between the two groups.
"In patients with LAM, sirolimus stabilized lung function, reduced serum VEGF-D levels, and was associated with a reduction in symptoms and improvement in quality of life. Therapy with sirolimus may be useful in selected patients with LAM," the authors write.
The study was supported in part by Pfizer; several authors disclosed financial relationships with pharmaceutical companies, including Pfizer. One author disclosed involvement with several patents related to the study.
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