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Beyond Lesion Control: A Narrative Review of Targeted Therapy and Surgical Decision-Making in Children With Vascular
Omer Altamimi1, Ibrahim Altamimi2, Rahaf Alhowidi3
1Department of Surgery, Faculty of Medicine, King Abdulaziz University Hospital (KAUH), Jeddah, SAU.
Insights
Targeted therapies like sirolimus are revolutionizing pediatric vascular anomaly treatment. These new drugs can alter surgical timing, extent, and goals, leading to better outcomes for children.
Area of Science:
- Pediatric Surgery
- Vascular Medicine
- Pharmacology
Background:
- Pediatric vascular anomalies present complex challenges, often requiring surgery.
- Surgical decisions in children are complicated by unique factors like growth and recurrence risk.
Purpose of the Study:
- To review how targeted therapies influence surgical decision-making for pediatric vascular anomalies.
- To propose a child-centered model for integrating targeted therapy and surgery.
- To introduce the PERFORM framework for multidisciplinary assessment.
Main Methods:
- Narrative review of current literature on targeted therapies and surgical management.
- Analysis of how sirolimus and alpelisib impact treatment paradigms.
- Development of a decision-making framework (PERFORM).
Main Results:
- Targeted therapies are shifting the management paradigm for pediatric vascular anomalies.
- These therapies can be used to facilitate, delay, avoid, or modify surgical interventions.
- The PERFORM framework offers guidance for multidisciplinary patient assessment.
Conclusions:
- Targeted therapies offer new possibilities beyond surgical alternatives for pediatric vascular anomalies.
- A child-centered approach integrating medical and surgical strategies is crucial.
- Future research should focus on functional outcomes, quality of life, and family burden.
Abstract:
Pediatric vascular anomalies are heterogeneous disorders that may cause pain, bleeding, infection, deformity, airway compromise, impaired mobility, psychosocial distress, and long-term functional morbidity. Surgery has traditionally played a central role in selected lesions through excision, debulking, staged reconstruction, and functional restoration. However, in children, operative decision-making is complicated by infiltrative anatomy, bleeding risk, lymphatic leakage, recurrence, scarring, growth potential, and the possibility of repeated procedures across childhood. The emergence of targeted therapies, particularly sirolimus for complex slow-flow malformations and alpelisib for PIK3CA-related overgrowth spectrum and selected vascular malformations, has shifted the management paradigm. These agents should not be viewed only as medical alternatives to surgery; rather, they may reshape surgical timing, extent, risk, and goals. This narrative review examines how targeted therapy may influence reconstructive surgical decision-making in children with vascular anomalies. We propose a child-centered model in which surgery may be performed, facilitated, delayed, avoided, or modified after targeted therapy. We also introduce the PERFORM framework to guide multidisciplinary assessment of patient age, lesion extent, treatment response, functional threat, operative risk, reconstruction required, and long-term follow-up. Future studies should move beyond radiologic response and report pediatric outcomes that matter: function, growth, quality of life, recurrence, revision surgery, and family burden.
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