Building a community around vascular anomalies research

May 12, 2013

Cameron Trenor, MD

Research on rare disorders, or in new fields, often follows a particular trajectory. It tends to start out fragmented, carried out by one or two isolated researchers at a few institutions.

But as researchers find each other, identify more patients and start to collaborate systematically, patterns of disease biology emerge, researchers start speaking the same language and new treatments materialize.

The field of complex vascular anomalies—a set of conditions characterized by blood vessels that have not developed normally—is in this kind of early days. In large part this is because they are relatively rare. In addition, few centers worldwide have the multidisciplinary experience to provide comprehensive care to these rare patients.

But a new coalition forming around vascular anomaly research and care could help unravel the biology of vascular anomalies and fashion better treatments for these children by bringing to bear the resources and knowledge of specialists from across the continent.

"There are many similarities between the vascular anomalies field today and solid tumor oncology in the 1980s," says Cameron Trenor III, MD, a hematologist/oncologist with the Vascular Anomalies Center (VAC) at Boston Children's Hospital and Dana-Farber Boston Children's Hospital Cancer and Blood Disorders Center. "There's not much standardization of care. The drugs we use, the processes for delivering care, even the names we use to describe anomalies differ from center to center."

Hopefully not for much longer, though. In a special interest group session held at the 2013 meeting of the American Society of Pediatric Hematology Oncology (ASPHO), Trenor and two of his colleagues—Leonardo Brandáo, MD, MSc, from Toronto's Hospital for Sick Children, and Denise Adams, MD, from Cincinnati Children's Hospital Medical Center—unveiled a new multi-center consortium aimed at bridging gaps in the field: CoSoVA, the Consortium for the Study of Vascular Anomalies.

"We want to build a framework for advancing the care and outcomes of children and young adults with vascular anomalies," Trenor says. "CoSoVA will bring the vascular anomaly research and clinical community together for clinical studies, translational and biomarker research, and to start building standard protocols for vascular anomaly care."

Trenor and his colleagues already have a good head start. Adams, for instance, is leading a clinical trial of sirolimus for complicated vascular anomalies like Kaposiform hemangioendothelioma. Brandáo is investigating the use of anticoagulants in children with vascular anomalies at high-risk of bleeding and thrombotic complications during medical procedures, such as extensive venous malformations, Klippel-Trenaunay syndrome and CLOVES (congenital, lipomatous overgrowth, vascular malformations, epidermal nevi and scoliosis/skeletal/spinal) syndrome. This observational study, which includes patients from Boston and Cincinnati as well, aims to better define bleeding and thrombotic risk and develop standard anticoagulation protocols for vascular anomaly patients during medical procedures.

And Trenor is launching a prospective research registry for collecting and studying clinical and biological data from a broad range of lymphatic anomalies, such as generalized lymphatic anomalies, Kaposiform lymphangiomatosis, Gorham-Stout disease, mixed-type lymphatic malformations and CLOVES.

"The registry should help us develop better clinical and biological descriptions of these conditions, including risk factors, characteristics and natural history," Trenor explains. "We also want to improve diagnosis by creating a staging system, and also collect and analyze treatment response data."

He adds that the Boston Children's VAC has run a retrospective registry for some time. "But like rare pediatric cancers," he says, "collaboration across multiple centers will expedite our learning curve. With more patient data from other centers, we can describe the natural history, responses to therapies, risk factors for poor outcomes and improve care for these rare conditions."

While there are challenges ahead—funding, differences in resources and expertise, variations in patient population and experience—a growing interest in vascular anomalies could give Trenor and his colleagues the push they need.

"We had to cap attendance to our ASPHO special interest group session because we had so much pre-conference interest," he notes. "There is clearly a significant momentum when it comes to these conditions that, if we can tap into it, will be a boon to the patients we see."

If you are a researcher or clinician with interest in vascular anomalies and wish to take part in CoSoVA, contact Cameron Trenor (, Denise Adams ( or Leonardo Brandáo (

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