title: An open-label study to evaluate sildenafil for the treatment of lymphatic malformations.
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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Department of Dermatology, Lucile Packard Children's Hospital at the Stanford University School of Medicine, Palo Alto, California.
authors.forename: Christina
authors.initials: C
authors.lastname: Danial
authors.affiliation: Department of Dermatology, Lucile Packard Children's Hospital at the Stanford University School of Medicine, Palo Alto, California.
authors.forename: Andrea L
authors.initials: AL
authors.lastname: Tichy
authors.affiliation: Department of Radiology, Stanford University School of Medicine, Stanford, California.
authors.forename: Umar
authors.initials: U
authors.lastname: Tariq
authors.affiliation: Department of Dermatology, Lucile Packard Children's Hospital at the Stanford University School of Medicine, Palo Alto, California.
authors.forename: Glenda L
authors.initials: GL
authors.lastname: Swetman
authors.affiliation: Department of Dermatology, Lucile Packard Children's Hospital at the Stanford University School of Medicine, Palo Alto, California.
authors.forename: Phuong
authors.initials: P
authors.lastname: Khuu
authors.affiliation: Department of Dermatology, Lucile Packard Children's Hospital at the Stanford University School of Medicine, Palo Alto, California.
authors.forename: Thomas H
authors.initials: TH
authors.lastname: Leung
authors.affiliation: Department of Dermatology, Lucile Packard Children's Hospital at the Stanford University School of Medicine, Palo Alto, California.
authors.forename: Latanya
authors.initials: L
authors.lastname: Benjamin
authors.affiliation: Department of Dermatology, Lucile Packard Children's Hospital at the Stanford University School of Medicine, Palo Alto, California.
authors.forename: Joyce
authors.initials: J
authors.lastname: Teng
authors.affiliation: Department of Radiology, Stanford University School of Medicine, Stanford, California.
authors.forename: Shreyas S
authors.initials: SS
authors.lastname: Vasanawala
authors.affiliation: Department of Dermatology, Lucile Packard Children's Hospital at the Stanford University School of Medicine, Palo Alto, California. Electronic address: alfred.lane@stanford.edu.
authors.forename: Alfred T
authors.initials: AT
authors.lastname: Lane
mesh_terms: D000284:Administration, Oral; D002648:Child; D002675:Child, Preschool; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D044148:Lymphatic Abnormalities / Q000175:diagnosis* / Q000188:drug therapy*; D008279:Magnetic Resonance Imaging / Q000379:methods; D008297:Male; D010879:Piperazines / Q000627:therapeutic use*; D011446:Prospective Studies; D011687:Purines / Q000627:therapeutic use; D012720:Severity of Illness Index; D000068677:Sildenafil Citrate; D013450:Sulfones / Q000627:therapeutic use*; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D010879:Piperazines; D011687:Purines; D013450:Sulfones; D000068677:Sildenafil Citrate
keywords: congenital; lymphatic malformation; macrocystic; magnetic resonance imaging; microcystic; phosphodiesterase-5 inhibitors; sclerotherapy