An open-label study to evaluate sildenafil for the treatment of lymphatic malformations - 14/05/14
Abstract |
Background |
Lymphatic malformations can be challenging to treat. Mainstay interventions including surgery and sclerotherapy are invasive and can result in local recurrence and complications.
Objective |
We sought to assess the effect of 20 weeks of oral sildenafil on reducing lymphatic malformation volume and symptoms in children.
Methods |
Seven children (4 boys, 3 girls; ages 13-85 months) with lymphatic malformations were given oral sildenafil for 20 weeks in this open-label study. The volume of the lymphatic malformation was calculated blindly using magnetic resonance imaging performed before and after 20 weeks of sildenafil. Lymphatic malformations were assessed clinically on weeks 4, 12, 20, and 32. Both the physician and parents evaluated the lymphatic malformation in comparison with baseline.
Results |
Four subjects had a lymphatic malformation volume decrease (1.0%-31.7%). In 2 subjects, despite a lymphatic malformation volume increase (1.1%-3.7%), clinical improvement was noted while on sildenafil. One subject had a 29.6% increase in lymphatic malformation volume and no therapeutic response. Lymphatic malformations of all 6 subjects who experienced a therapeutic response on sildenafil softened and became easily compressible. Adverse events were minimal.
Limitations |
A randomized controlled trial will be necessary to verify the effects of sildenafil on lymphatic malformations.
Conclusions |
Sildenafil can reduce lymphatic malformation volume and symptoms in some children.
Il testo completo di questo articolo è disponibile in PDF.Key words : congenital, lymphatic malformation, macrocystic, magnetic resonance imaging, microcystic, phosphodiesterase-5 inhibitors, sclerotherapy
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| Funding sources: The project described was supported by the National Center for Research Resources (NCRR) and the National Center for Advancing Translational Sciences, National Institutes of Health (NIH), through UL1 TR000093 (formerly UL1 RR025744). The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH. All phases of this study were supported by the following: SPARK, Spectrum–the Stanford Center for Clinical and Translational Research and Education; Lucile Packard Foundation for Children's Health; and the Stanford NIH/NCRR Clinical Translational Science Award grant number TLI RR025742. Ms Danial was supported by the Stanford University Medical Scholars Research Program. We thank Pfizer for its support in providing Revatio. |
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| Reprints not available from the authors. |
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| Conflicts of interest: None declared. |
Vol 70 - N° 6
P. 1050-1057 - giugno 2014 Ritorno al numero
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