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Vascular anomalies. A primer for pediatricians
1Harvard Medical School, Boston, Massachusetts, USA. fishman-s@al.tch.harvard.edu
Insights
Advances in vascular anomaly centers and basic science research offer new hope for children. New treatments are emerging for vascular tumors and malformations, driven by genetic insights and antiangiogenic agents.
Area of Science:
- Pediatric vascular medicine
- Vascular biology
- Medical genetics
Background:
- Children with vascular anomalies often face fragmented care across medical specialties.
- Dedicated vascular anomaly centers are improving consultation and management.
- Recent advancements in basic science are illuminating the causes and potential treatments for these conditions.
Purpose of the Study:
- To highlight the progress in managing pediatric vascular anomalies.
- To discuss the impact of basic science research on understanding etiology.
- To outline the development of novel therapeutic strategies.
Main Methods:
- Review of current clinical practices and specialized centers.
- Exploration of emerging insights from molecular genetics and familial studies.
- Discussion of ongoing clinical trials for antiangiogenic agents.
Main Results:
- Improved interdisciplinary care models for vascular anomalies.
- Identification of genetic underpinnings for vascular malformations.
- Development of targeted therapies, including antiangiogenic agents, for vascular tumors.
Conclusions:
- The outlook for children with vascular anomalies is significantly improving.
- Integrated care and ongoing research promise more effective treatments.
- Future research involving animal models will further elucidate pathogenesis and guide novel therapies.
Abstract:
The future is bright for children with vascular anomalies. Children with vascular anomalies no longer have to languish in the interface between various medical specialties. Vascular anomalies centers are available for consultation. Not only are specialists focusing on the conditions, but basic science has brought us to the horizon of new insights into etiology and thus the possibility for new treatment. For children with vascular tumors, newer antiangiogenic agents are already in clinical trials. For children with vascular malformations, research in molecular genetics has begun to uncover the genes responsible for normal fetal vasculogenesis and angiogenesis. Molecular studies of familial vascular malformative syndromes are just beginning to pinpoint causative mutations. Next, animal models of vascular abnormalities will be engineered to elucidate pathogenesis, and these studies, in turn, should lead to development of novel therapies.