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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. 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.

SUBMITTER: Danial C

PROVIDER: S-EPMC4024322 | biostudies-literature | 2014 Jun

REPOSITORIES: biostudies-literature

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An open-label study to evaluate sildenafil for the treatment of lymphatic malformations.

Danial Christina C Tichy Andrea L AL Tariq Umar U Swetman Glenda L GL Khuu Phuong P Leung Thomas H TH Benjamin Latanya L Teng Joyce J Vasanawala Shreyas S SS Lane Alfred T AT

Journal of the American Academy of Dermatology 20140320 6

<h4>Background</h4>Lymphatic malformations can be challenging to treat. Mainstay interventions including surgery and sclerotherapy are invasive and can result in local recurrence and complications.<h4>Objective</h4>We sought to assess the effect of 20 weeks of oral sildenafil on reducing lymphatic malformation volume and symptoms in children.<h4>Methods</h4>Seven children (4 boys, 3 girls; ages 13-85 months) with lymphatic malformations were given oral sildenafil for 20 weeks in this open-label ...[more]

PMID: 24656411

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Project description:IntroductionIn the pivotal RE-LY trial, dabigatran etexilate (DE) at the dose of 150-mg twice daily (BID), significantly reduced total stroke and ischemic stroke compared with warfarin in patients with non-valvular atrial fibrillation (NVAF), while the 110-mg BID dose had efficacy equivalent to warfarin, and major bleeds were significantly reduced. Both DE regimens were generally well tolerated; however, approximately 4% of the patients discontinued treatment with DE due to gastrointestinal (GI) discomfort.MethodsClinical trial NCT01493557 was a multicenter, randomized, active control, open-label study to assess the efficacy of two simple GI symptom (GIS) management strategies in DE-treated patients who developed GIS: (1) concurrent treatment with the proton pump inhibitor pantoprazole (DE-P), or (2) ingestion of DE after a meal (DE-M). Patients were initially randomized to either GIS management strategy. If the first did not resolve their GIS, patients had the option to "add on" the alternative strategy.ResultsA total of 1067 patients with NVAF received DE therapy BID for 3 months (United States, 150-mg or 75-mg; Canada, 150-mg or 110-mg). Of these, 117 (11%) patients reported GIS and were randomized to one of two GIS management strategies. At 4 weeks, a significantly higher rate of complete or partial effectiveness was observed in patients on DE-P than in those receiving DE-M, [50/58 (86.2%) versus 40/59 (67.8%), respectively; p = 0.0273]. Patients with ongoing GIS were asked to "add on" the alternate strategy for an additional 4 weeks. Overall, 92/117 (78.6%) of randomized patients experienced complete or partial effectiveness using either the initial strategy or a combination of the two strategies: DE-P, 47 (81.0%); and DE-M, 45 (76.3%, no significant difference) (by initial strategy).ConclusionThe majority of patients enrolled either did not experience GIS at all, or their GIS resolved using either one individually, or a combination of the two strategies described.Trial registrationhttp://www.ClinicalTrials.gov identifier: NCT01493557.

Project description:BackgroundPrevious studies with extended-release (ER) paliperidone have reported an effective outcome in terms of personal and social functioning improvement and also reported schizophrenia symptom improvement. The main objectives of this study were to further investigate improvements in symptom control and social functioning of paliperidone ER and acknowledge the safety profile of paliperidone ER in Thai patients with schizophrenia.Patients and methodsPatients with schizophrenia according to Diagnostic and Statistical Manual of Mental Disorders criteria were allowed flexible 3-12 mg/day dosing during the 10-week study duration. Patients were interviewed and assessed in social functioning using the Personal and Social Performance (PSP) scale. Patients were also rated on overall severity of illness using the Clinical and Global Impressions - Severity (CGI-S) scale.ResultsIn total, 40 patients were enrolled, 80% of enrolled patients (n = 32) completed the 10-week study period. Thirty-eight eligible patients were included in the intention-to-treat analysis set (male 39.5%, female 60.5%). One patient was lost to follow-up without postbaseline-efficacy measurements. Another patient was terminated early due to a change in diagnosis during the trial. Statistically significant improvements from baseline in PSP total score were observed at all time points. Clinically relevant improvement in PSP (increase of at least one 10-point category) was observed in 47.40% of patients at end point. Improvement in CGI-S was observed at end point (P < 0.001). The mean reduction ± standard deviation at end point in CGI-S was 0.8 ± 1.04 (95% confidence interval 0.48-1.16). The most commonly reported adverse events (≥5% of patients) were daytime drowsiness (15%) and headache (15%). Three subjects (7.5%) discontinued due to adverse events.ConclusionThis study suggests that paliperidone ER is well tolerated in Thai patients with schizophrenia. Paliperidone ER showed improvement in schizophrenic symptom control and social functioning.

Dataset Information

An open-label study to evaluate sildenafil for the treatment of lymphatic malformations.

Publications

An open-label study to evaluate sildenafil for the treatment of lymphatic malformations.

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