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Medical Therapies for Pediatric Lymphatic Malformations: A Systematic Review
Dylan Hollman1, Harleen Cheema2, Adam C Yu3
1Division of Dermatology, Department of Medicine, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada.
Journal of Cutaneous Medicine and Surgery
|October 31, 2025
Summary
New drug therapies show promise for treating pediatric lymphatic malformations (LM). Sirolimus and alpelisib demonstrated significant reductions in LM size, though more research is needed for long-term efficacy and safety.
Area of Science:
- Vascular Anomalies
- Pediatric Medicine
- Pharmacology
Background:
- Lymphatic malformations (LM) are complex vascular anomalies in children.
- Management of pediatric soft tissue LMs presents significant challenges.
- Emerging medical therapies offer new treatment avenues.
Purpose of the Study:
- To systematically review current medical therapies for pediatric soft tissue lymphatic malformations.
- To evaluate the efficacy of various drug treatments for LM.
- To identify promising therapeutic agents for LM management.
Main Methods:
- Systematic literature search of MEDLINE, Embase, Cochrane Library, and SCOPUS.
- Keywords included "lymphatic malformation," "drug therapy," and "pediatric."
- Treatment success defined as >10% lesion size reduction; complete response (CR) as >90% reduction.
Main Results:
- 77 studies were included from 4937 initial titles/abstracts.
- Sirolimus (oral and topical) and alpelisib (oral) showed high success rates.
- Other agents like acetylsalicylic acid, propranolol, and sildenafil also demonstrated efficacy; CR noted for isotretinoin, cyclophosphamide, and others.
Conclusions:
- Alpelisib and sirolimus show significant promise in reducing pediatric LM size.
- Further long-term studies are essential to confirm efficacy and safety profiles.
- Study limitations include data heterogeneity and potential bias.
