Medical Therapies for Pediatric Lymphatic Malformations: A Systematic Review
Abstract
Introduction: Lymphatic malformations (LM) are vascular anomalies that can be challenging to manage with new medical therapies emerging. This systematic review examines current medical therapies for pediatric patients with LMs that involve the soft tissues.
Materials and methods: MEDLINE, Embase, Cochrane Library, and SCOPUS were searched on April 12, 2024, using variations of the keywords "lymphatic malformation" AND "drug therapy" AND "pediatric." Language was limited to English, and no date restriction was applied. Treatment success was defined as a reduction in lesion size of over 10%, with complete response (CR) defined as a reduction in size of over 90%.
Results: Our review encompassed 4937 title/abstracts, 436 full-texts and ultimately included 77 studies. Reported success rates were variable, with notable results for alpelisib (oral) (n = 9/9), sirolimus (oral) (n = 257/287), sirolimus (topical) (n = 13/15), acetylsalicylic acid (oral) (n = 18/23), propranolol (oral) (n = 19/29), and sildenafil (oral) (n = 33/71). CR was reported with isotretinoin (n/a) (n = 1/1), cyclophosphamide (iv) (n = 1/2), acetylsalicylic acid (oral) (n = 4/23), sirolimus (topical) (n = 2/15), and sirolimus (oral) (n = 17/287).
Conclusion: Overall, therapies such as alpelisib and sirolimus showed promising results in the reduction of pediatric LM size; however, additional long-term data are needed to validate their efficacy and safety profile. Limitations of our study include heterogeneity and…
How strong is the evidence?
Evidence Hub reads this as a systematic review, done in people. Its evidence level is 1 of 5 (Highest · pooled evidence). Pooled evidence from several studies sits at the top of the evidence pyramid. Reported size: 257 participants or records.
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