title: A review of contemporary options for medical management of hemangiomas, other vascular tumors, and vascular malformations.
abstract: Vascular anomalies include vascular tumors and vascular malformations. With growing pharmacologic options and parallels to cancer treatment and biology, the hematologist-oncologist has assumed a more prominent role in clinical care and research relating to these diagnoses. This also is a growing area for targeted therapies and drug repositioning. We performed a review of contemporary options for medical management of these lesions. PubMed was searched for "vascular anomaly", "hemangioma", "vascular malformation", "arteriovenous malformation", "capillary malformation", "cerebral cavernous malformation", "lymphatic malformation", and "venous malformation", each with "drug treatment" as a modifier. Manuscripts were reviewed to verify diagnoses, indications for treatment, dose-schedules, evidence of effectiveness, toxicities, and mechanisms of action. ClinicalTrials.gov also was reviewed for relevant trials. More than 20 agents were identified which have been used to treat vascular anomalies. Rigorous studies are lacking for many of these. The rarity of these tumors has limited development of medical approaches to treatment. Cooperative group trials will be needed to prove the effectiveness of drugs which have shown promise in cases and small series. The observant clinician remains a powerful tool for identifying potential new treatments for vascular tumors and malformations.
authors.affiliation: Division of Pediatric Hematology Oncology, the University of North Carolina, Chapel Hill, NC, USA. jblat@med.unc.edu
authors.forename: Julie
authors.initials: J
authors.lastname: Blatt
authors.forename: Thomas W
authors.initials: TW
authors.lastname: McLean
authors.forename: Sharon M
authors.initials: SM
authors.lastname: Castellino
authors.forename: Craig N
authors.initials: CN
authors.lastname: Burkhart
mesh_terms: D002986:Clinical Trials as Topic; D015195:Drug Design; D006391:Hemangioma / Q000188:drug therapy* / Q000473:pathology; D006801:Humans; D058990:Molecular Targeted Therapy; D054079:Vascular Malformations / Q000188:drug therapy* / Q000473:pathology; D019043:Vascular Neoplasms / Q000188:drug therapy* / Q000473:pathology
keywords: <5 cases demonstrating efficacy; AVM; C; CCM; CM; Drug treatment; FGF; HHT; Hemangioma; IH; KMS; Kasabach–Merritt syndrome; LM; MMP; R; S; VEGF; VM; Vascular anomaly; Vascular malformation; arteriovenous malformation; capillary malformation; cerebral cavernous malformation; fibroblast growth factor; hemangioma of infancy; hereditary hemorrhagic telangiectasia; lymphatic malformation; mTOR; mammalian target of rapamycin; matrix metalloproteinase; randomized clinical trial; retrospective series≥5 cases demonstrating efficacy; rs; single arm clinical trial; vascular endothelial growth factor; venous malformation