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Long-Term Follow-Up of Lymphatic Malformations in Children Treated with Sildenafil
Joanna H Tu1,2, Elidia Tafoya2, Michael Jeng3
1College of Physicians and Surgeons, Columbia University, New York, New York.
Insights
Sildenafil shows long-term benefits for pediatric lymphatic malformations (LMs), improving size and compressibility. Most patients experienced positive outcomes with minimal side effects, suggesting its value in LM symptom management.
Area of Science:
- Vascular Anomalies
- Pediatric Surgery
- Pharmacology
Background:
- Lymphatic malformations (LMs) present significant treatment challenges.
- Previous reports on sildenafil for LM management have yielded mixed results.
- Long-term clinical outcomes of pediatric LM patients treated with sildenafil require further evaluation.
Purpose of the Study:
- To evaluate the long-term clinical outcomes of pediatric patients with lymphatic malformations (LMs) following sildenafil treatment.
- To assess the efficacy and safety of sildenafil in managing symptomatic LMs in children.
Main Methods:
- Retrospective chart review of pediatric LM patients treated with sildenafil over the past five years.
- Follow-up surveys conducted with patients to gather comprehensive data on LM status and subsequent interventions post-sildenafil.
Main Results:
- Ten out of twelve (83.3%) surveyed patients reported a positive therapeutic response to sildenafil.
- Improvements in LM size and compressibility were observed during post-treatment clinical visits.
- Most patients experienced only minor, transient side effects during sildenafil treatment, with no adverse effects reported at long-term follow-up.
Conclusions:
- This study provides the first long-term follow-up data on pediatric LM patients treated with sildenafil.
- Findings suggest sildenafil is beneficial for the symptomatic treatment of lymphatic malformations.
- Further analysis is needed to determine sildenafil's role as adjuvant therapy for complex LMs.
Background/Objectives:
Lymphatic malformations (LMs) are challenging to treat. Reports on the benefits of sildenafil for LM management have been mixed. This study evaluated long-term clinical outcomes of pediatric LM patients after sildenafil treatment.
Methods:
A retrospective chart review was performed on pediatric LM patients treated with sildenafil in the past 5 years. Patients were also contacted to complete a survey of comprehensive questions about their LM after sildenafil and subsequent interventions.
Results:
Of 12 patients identified, 10 (83.3%) participated in the follow-up survey. The average age was 8 years (range 4-16 yrs), median treatment duration was 9 months, and the average time of follow-up after sildenafil was 4 years; one patient is still taking sildenafil. Ten patients surveyed (83.3%) reported positive therapeutic response, with improvement in the size and compressibility of their LM during posttreatment clinical visits. Six received additional interventions (four sirolimus, one sclerotherapy, one surgery) after sildenafil was discontinued, with all but one reporting a positive response to subsequent interventions. Minor side effects at the time of sildenafil treatment included mild flushing, dizziness, and transient nausea, but no adverse effects were reported at the long-term follow-up.
Conclusion:
This is the first report of long-term follow-up of pediatric LM patients treated with sildenafil. Our findings suggest that sildenafil is beneficial for the symptomatic treatment of LMs. Additional analysis on the role of sildenafil as adjuvant therapy is necessary to optimize the use of this medication in the management of complex LMs.
