In an ongoing effort to find better and safer treatment for complicated infantile hemangiomas, researchers at The Medical College of Wisconsin and Children’s Hospital of Wisconsin, report outcomes on two treatment options. The first study examined the effects of oral steroids on a group of patients and the second highlights possible complication/side effect from propranolol, a novel therapy for hemangioma. Both studies published in the Archives of Dermatology, will facilitate safer therapy.
Infantile hemangiomas are benign tumors composed of blood vessel-like cells that grow in the first month of life. The duration and rate of growth is variable; some infants will have hemangiomas that grow very little while others grow at an alarming rate. Approximately 80,000 children annually are diagnosed with hemangiomas in the United States; 12 percent or 9,600 of those are significantly complex, requiring referral to specialists for consideration of treatment. Complications of hemangiomas include permanent disfigurement, ulceration, bleeding, loss of vision, airway obstruction, congestive heart failure and very rarely death.
“Despite the increasing incidence and the severity of potential complications, we lack uniform treatment guidelines for the treatment and response to treatment of infants with hemangiomas. There are no FDA-approved agents for the treatment of hemangiomas, thus physicians must rely on observational reports and clinical judgment to determine optimal treatment of infants with complicated hemangiomas,” points out Beth A. Drolet, M.D., senior author on both studies. Dr. Drolet is medical director, Dermatology, Birthmarks and Vascular Anomalies Center, Children’s Hospital, and professor and vice chairman of dermatology and professor pediatrics; and chief of pediatric dermatology at the Medical College.
There are no management guidelines or safety data on large groups of infants treated with these medications for infantile hemangioma as the safety of many of the existing therapies have not been studied in a scientific fashion. Medications with reported efficacy include corticosteroids, the mainstay of therapy, and more recently propranolol which is viewed as a safer alternative to traditional therapy.
To address this lack of data, the first study looked at 16 infants at Children’s Hospital with a mean age of 2.9 months, who were treated with corticosteroids for complicated hemangiomas. Over a period of 22 weeks, the researchers found profound, but reversible, suppression of the immune system. Other side effects included hypertension, sleep disturbance and weight gain.
The second publication describes three patients treated with oral propranolol who developed severe and symptomatic hypoglycemia during treatment, a condition in which the blood sugar is very low and can result in lethargy, confusion, and even seizures in these infants. The researchers also reviewed the literature to identify other reports of propranolol-associated hypoglycemia in children to highlight this rare side effect.
According to Dr. Drolet, “Given the lack of evidence-based standards for treatment of complicated infantile hemangiomas combined with the risks of accepted systemic therapies, care givers need to be aware of these side effects. We are committed to performing high quality studies to scientifically determine which therapies are more effective and to insure that these interventions are safe in these young babies”
Michael E. Kelly, MD, PhD, assistant professor of pediatrics—hematology/oncology, is the first author on the corticosteroids study and Kristen E. Holland, MD, assistant professor of dermatology, is the first author on the propranolol study.
These studies are part of an ongoing international research consortium, the Hemangioma Investigator Group (HIG). The HIG is a multi-institutional clinical research consortium established in 1999 and consists of a network of investigators in the U.S. and Canada who care for large patient populations with complex vascular lesions. The mission of the group is to advance knowledge about infantile hemangioma and improve outcomes for affected children through research, education, and advocacy.
This network has completed several successful projects and established a large registry of patients with – infantile hemangiomas. Since its inception, HIG has expanded to 10 sites, and has completed several longitudinal and observational studies addressing the incidence, demographics, complication rate, and outcomes of this disorder .Over the past ten years the HIG group has contributed over 20 publications in peer-reviewed journals as Journal of Pediatrics, Pediatrics, Archives of Dermatology, and American Journal of Medical Genetics.