Medical College of Wisconsin receives grant to study infantile hemangiomas

The Food and Drug Administration (FDA) has awarded the Medical College of Wisconsin in Milwaukee a three-year, $1 million Orphan Products Development grant to study infantile hemangiomas - a vascular tumor of the skin or internal organs.

The unique, interdisciplinary, and multi-institutional study is led by co-principal investigators Beth Drolet, M.D., professor of dermatology and pediatrics, at the Medical College and medical director of the vascular anomalies and dermatology program at Children's Hospital of Wisconsin; and Michael E. Kelly, M.D., Ph.D., assistant professor of pediatrics - hematology/oncology.

“This is a major achievement for Drs. Drolet and Kelly to receive an FDA grant to study a neglected but important health issue in infants,” says Dean and Executive Vice President Michael Dunn, M.D. “Dr. Drolet noted the increase in incidence of this disease and found a way to fund research to develop better treatment options.”

The new research builds on earlier Medical College studies supported by Children's Hospital and Health System Foundation, the Dermatology Foundation, Children's Research Institute, the Greater Milwaukee Foundation, and the NOVO Foundation.

“Partnering with Children's Hospital, Children's Research Institute, our patient families, and private donors in the community was truly inspiring,” says Dr. Drolet. “Their generosity and support has empowered our center to create a vision for our research that will change the way infants with this disorder are cared for around the country.”

In 2004, Children's Hospital and the Medical College created the Birthmarks and Vascular Anomalies Center to better care for infants with hemangiomas and other vascular anomalies. This interdisciplinary program, composed of surgeons, oncologists, radiologists, and pathologists, treats patients from around the country.

“Our preliminary studies show that the increased incidence of hemangiomas may be related to the increase in the rate of low birth weight infants in the United States,” says Dr. Drolet. “We are treating more of these infants while uniform guidelines for therapy and methods used to measure response to treatment of infants with hemangiomas are lacking.”

Infantile hemangiomas are a common, yet poorly understood vascular tumor. Most hemangiomas are found in the skin, but sometimes, they occur in other organs in the body such as the liver, spleen, intestine, airway, lungs and the central nervous system. Unlike most birthmarks, cutaneous hemangiomas are tumors that undergo cellular proliferation. They are either absent or barely evident at birth proliferating in the first few weeks to months of life, followed by a phase where they tend to decrease in size over several months to years.

Although most hemangiomas eventually resolve, many infants will suffer complications such as permanent disfigurement, ulceration, bleeding, loss of vision, airway obstruction, congestive heart failure and even death. Since hemangiomas can behave in vastly different ways and affect many different areas of the body, even physicians who are knowledgeable about hemangiomas and have access to diagnostic resources often find caring for affected infants challenging.

Drs. Drolet and Kelly will study infants diagnosed with large, complicated hemangiomas to determine and compare the effectiveness and safety of steroids in the current standard of care with a drug currently used for cancer.

Fifty infants with large and complicated hemangiomas will be randomly assigned to receive daily oral corticosteroid, prednisolone, or weekly IV vincristine for up to six months. The diagnostic, therapeutic and response criteria determined in this study may be used as a framework for future multi-institutional clinical trials to treat hemangiomas.

The study will provide answers as to which drug is more effective while at the same time providing opportunities for several additional investigators at the Medical College and at the Children's Research Institute to examine pathogenesis of hemangiomas and genetic factors that influence disease susceptibility and response treatment. These unique partnerships should help develop even better and safer treatment options for these infants.

Other research sites include the University of California-San Francisco and Indiana University.