Beyond Lesion Control: A Narrative Review of Targeted Therapy and Surgical Decision-Making in Children With Vascular Anomalies Across Function, Growth, and Perioperative Care

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.

Authors

Publication Details

Journal
Cureus
Published
2026-06-09
DOI
https://doi.org/10.7759/cureus.110532
Primary Topic
Vascular Malformations and Hemangiomas
Type
article
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article

Beyond Lesion Control: A Narrative Review of Targeted Therapy and Surgical Decision-Making in Children With Vascular Anomalies Across Function, Growth, and Perioperative Care

Yara Fahad Aljefri, Omer Altamımı, Ibrahim Altamimi, Abdulkreem Al-Juhani et al.
Cureus
Vascular Malformations and Hemangiomas
article

Beyond Lesion Control: A Narrative Review of Targeted Therapy and Surgical Decision-Making in Children With Vascular Anomalies Across Function, Growth, and Perioperative Care

Yara Fahad Aljefri, Omer Altamımı, Ibrahim Altamimi, Abdulkreem Al-Juhani, Eman M Alzahrani, Aseel Ashri, Sultan Khoja, Yara H Essa, Ghaida M Alosaimi, Lamar A Alsaigh, Rahaf Alhowidi
article en

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.

Cureus
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Vascular Malformations and Hemangiomas
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Beyond Lesion Control: A Narrative Review of Targeted Therapy and Surgical Decision-Making in Children With Vascular Anomalies Across Function, Growth, and Perioperative Care — Yara Fahad Aljefri, Omer Altamımı, et al. · Cureus (2026) | TGRS Research Map | TGRS