Medical Therapies for Pediatric Lymphatic Malformations: A Systematic Review
Bibliographic record
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 a potential risk of bias.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.004 | 0.017 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.006 | 0.006 |
| Bibliometrics | 0.008 | 0.011 |
| Science and technology studies | 0.001 | 0.001 |
| Scholarly communication | 0.002 | 0.002 |
| Open science | 0.002 | 0.001 |
| Research integrity | 0.002 | 0.001 |
| Insufficient payload (model declined to judge) | 0.006 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".