Effect of long-term phosphodiesterase-5 inhibitor use on refractory lymphatic malformations in adult and teen patients

Abstract

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Differences in Efficacy and Safety of Sirolimus and Sildenafil in Pediatric Lymphatic Malformations

“…7 The LMs of children treated with sildenafil for pulmonary hypertension improved significantly, and subsequent studies showed the treatment was effective in improving clinical symptoms, softening masses, and reducing the size of the lesions in patients with LMs. [8][9][10][11][12][13] In recent years, basic research has revealed that lymphatic endothelial cell (LEC) somatic gene mutations in LMs lead to persistent activation of the PI3K/AKT/mTOR pathway, which is an essential cause of developing LMs. Sirolimus, an inhibitor of mTOR, has been shown in numerous studies to significantly improve the imaging and clinical symptoms of patients with LMs.…”

“…The drug has been approved for the treatment of pulmonary hypertension in adults, and its application to LMs began in 2012 with a report published in the New England Journal of Medicine 7 . The LMs of children treated with sildenafil for pulmonary hypertension improved significantly, and subsequent studies showed the treatment was effective in improving clinical symptoms, softening masses, and reducing the size of the lesions in patients with LMs 8–13 . In recent years, basic research has revealed that lymphatic endothelial cell (LEC) somatic gene mutations in LMs lead to persistent activation of the PI3K/AKT/mTOR pathway, which is an essential cause of developing LMs.…”