Beyond Birthmarks
A new look at vascular malformations
While a pediatric hematology / oncology fellow in the mid-1990s, Denise Adams, MD, was asked to lunch by a concerned colleague who had discovered she planned to focus her work on vascular anomalies. “Don’t do it,” was the advice she recalls receiving at that lunch. “There’s no reason to have hematology / oncology physicians involved in this.”
Denise Adams, MD, is working to develop standards of care to treat vascular malformations.
Little more than a decade later, Dr. Adams, now medical director of the Comprehensive Hemangioma and Vascular Malformation Center at Cincinnati Children’s, is at the forefront of a new approach to studying and treating this large and complex group of diseases.
Creating Guideposts for Treatment
The term “vascular anomalies” encompasses a wide range of conditions, ranging from uncomplicated vascular tumors, such as hemangiomas, to much more serious vascular tumors and malformations. Many conditions do not require medical intervention, but the more complicated lesions require multiple specialists for optimum care.
“There are these conditions called kaposiform hemangioendotheliomas that used to be considered – and this is in the 1990s – hemangiomas that caused bleeding problems,” says Dr. Adams. “They’re not hemangiomas; they’re actually tumors and they can be devastating.”
Until recently, she says, there was no standard of care for a condition like this. “What was happening was, there were all these different people taking care of all these different diagnoses, and kids weren’t getting treated properly because they were being misdiagnosed and mistreated.”
By the late 1990s clinicians began to address these gaps in treatment. At Cincinnati Children’s, Richard Azizkahn, MD, and Anne Lucky, MD, formed the Hemangioma and Vascular Malformation Center in 2001; two years later they recruited Dr. Adams to be the group’s medical director.
Forging New Territory
Since arriving here, she has worked on establishing standards of care for vascular anomalies, an endeavor in which she and her colleagues find themselves in new territory. “Currently, there are no prospective research studies, there are no biomarkers out there that look specifcally at these diagnoses to help direct us with the therapy,” she says. “My goal is to develop clinical studies.”
Dr. Adams and her colleagues will be submitting a grant to develop a rare disease consortium consisting of Cincinnati Children’s and about nine other institutions that treat vascular anomalies. “We have a set of protocols that we hope to implement,” explains Dr. Adams. “Cincinnati Children’s will lead a protocol with the drug rapamycin to treat proliferating, complicated vascular lesions.”
After a decade of pursuing better treatments for vascular malformations, Dr. Adams is happy she didn’t follow her colleague’s advice. Far from hematology / oncology not having a role in treating vascular anomalies, she points out many of these conditions are tumors that can have problems such as coagulopathies and can be borderline malignant.
“Therapy needs to be targeted and directed against abnormal proliferation of these vessels. Angiogenesis plays a large role in these lesions, and oncologists are familiar with new drugs that are targeted anti-angiogenic agents,” she explains. “What better doctor to take care of a proliferating lesion? This has everything to do with hematology / oncology.”
