Infantile hemangioma and vascular anomaly research
Recent cellular and genetic research has yielded several important clues about what causes infantile hemangiomas as well as important new tools for diagnosing them. The Birthmarks and Vascular Anomalies Center is conducting research on the causes and potential cures for dangerous and disfiguring hemangiomas. These efforts are lead by a team of internationally respected researchers:
Paula North, MD, PhD
Chief of Pediatric Pathology, Children's Hospital of Wisconsin; professor and chief of Pediatric Pathology, Medical College of Wisconsin.
Beth Ann Drolet, MD
Medical Director of the Dermatology Clinic and Birthmarks and Vascular Anomalies Center, Children's Hospital of Wisconsin;
Professor of Dermatology and Pediatrics, Medical College of Wisconsin.
Joseph Kerschner, MD, FACS, FAAP
Medical director, Otolaryngology, Children's Hospital of Wisconsin; chief, division of Pediatric Otolaryngology, professor of Otolaryngology and Communication Sciences, academic vice-chairman, senior associate dean for clinical affairs, Medical College of Wisconsin; CEO Children's Specialty Group.
Michael Kelly, MD, PhD
Medical director of Hematology/Oncology, Children's Hospital of Wisconsin; assistant professor of Pediatric Hematology/Oncology/BMT, Medical College of Wisconsin.
Kelly Duffy, PhD
Research scientist, Medical College of Wisconsin.
Current clinical research initiatives
Subjects for the following studies are recruited by faculty and staff in the Dermatology Clinic at Children's Hospital. To inquire about enrolling your child in any of the below studies, please contact Marcia Seefeldt, RN, at (414) 266-6430 or vac@chw.org.
- A Phase II Randomized Clinical Trial Assessing the Efficacy and Safety of Oral Prednisolone vs. Intravenous Vincristine in the Treatment of Infantile Hemangioma
Infants with large hemangiomas are often treated systemically with oral steroids (Prednisolone) to prevent complications. The best treatment for hemangiomas is not known. Also, the best method to measure the response of hemangioma to treatment is not known. The goals of this study, which will begin in early 2008, are to determine whether Prednisolone or Vincristine is more effective and safer for treating infants with hemangiomas and to determine if changes on MRI scan are more sensitive than changes on clinical exam for detecting responses to treatment. A total of 50 babies younger than 6 months old with hemangiomas requiring treatment will take part in this study. Participating hospitals include Children's Hospital of Wisconsin, Riley Hospital for Children in Indianapolis, and the University of California, San Francisco. We anticipate 20 patients from Children's Hospital during three years. Patients enrolling on this study will be randomly assigned to receive either daily Prednisolone by mouth or weekly Vincristine in a vein. Response to treatment will be monitored by clinical exams every two weeks and by an MRI at study entry and six and 12 weeks later. Patients with evidence of progressive disease (larger hemangiomas) on the week six MRI will be switched to the other drug to complete a total of 12 weeks of therapy. Side effects of each medication will be monitored closely determined from histories, physical exams, blood tests and other studies as necessary. Participation in this study will last up to 12 weeks and follow up for protocol. The funding for this clinical trial is through a Food and Drug Administration grant and a grant from the Greater Milwaukee Foundation. Click to see our study brochure.
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Lumbosacral Hemangioma and Association With Occult Spinal Dysraphism
Lumbosacral hemangiomas of infancy (a hemangioma in the lower back) can be associated with anomalies of the spine. These include tethered cord syndrome, a condition caused by an abnormally stretched spinal cord. Over time this condition can lead to neurological damage. Often, there are no symptoms until adulthood, but it can become apparent during childhood. Common symptoms are lower back pain, pain and weakness of the legs, walking problems, and loss of control of the bladder and bowel. "Occult spinal dysraphism" is the term used when the defect of the spine is hidden under normal skin. Infants with hemangioma in the lower back area seem to be more inclined to have a hidden spine defect. This study focuses on the presence of occult spinal dysraphism in infants with lumbosacral hemangiomas. The goal of this study is to better understand the risk of developing this condition in infants with lumbosacral hemangioma and create guidelines of care. Study participants will be recruited through the Dermatology Clinic at the Children's Hospital of Wisconsin. All patients up to age 18 who have a lumbosacral hemangioma that is greater than 2.5 cm in diameter overlying the spine will have an opportunity to participate in the study. Parents will be interviewed to obtain personal, medical and family history. Patients will have a skin and neurological examination, photograph, magnetic resonance imaging (MRI) of the back, and lab tests requiring samples of blood, urine or stool. This study is funded by Children's Research Institute, the Dermatology Foundation and the American Skin Association.
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Large Cutaneous and Multiple Hemangioma Study (No longer enrolling patients-Data Analysis)
Multiple or large skin hemangiomas frequently develop in premature and low birthweight infants. Children with multiple or large skin hemangiomas sometimes also develop hemangiomas on internal organs. When hemangiomas are present in internal organs, the liver is the most common location, followed by the brain, intestines, lungs and tongue. Hepatic (liver) hemangiomas are closely associated with heart disease because the hemangiomas force the heart to work harder. These factors ultimately can lead to death. This study focuses on the association of internal organ hemangiomas in infants with multiple or large skin hemangiomas. The goal of this study is to create guidelines for evaluation and care for children of this condition. Study subjects will be recruited through the Dermatology Clinic at Children's Hospital of Wisconsin. Parents will be interviewed to obtain personal, medical and family history. Patients will be examined for multiple or large skin hemangiomas. This includes skin and neurological examinations, a photograph, an ultrasound of the abdomen and lab tests requiring samples of blood, urine or stool.
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Large Facial Hemangioma Study (No longer enrolling patients-Data Analysis)
Large hemangiomas of the face can be associated with anomalies of the blood vessels of head and chest. The acronym PHACES indicates the association of Posterior fossa and other brain malformations, facial Hemangioma, Arterial anomalies, Coarctation of the aorta and other cardiac defects,Eye abnormalities and Sternal malformations. This study focuses on the presence of PHACE syndrome in children with large facial hemangiomas. The goal of this study is to better understand the risk of this syndrome and to develop guidelines for its evaluation and management. Study subjects will be recruited through the Dermatology Clinic at Children's Hospital of Wisconsin. Parents will be interviewed about their personal, medical and family history. Patients will be evaluated for facial hemangiomas with risk of PHACE syndrome. This includes a skin, eye and neurological examination, photograph, magnetic resonance imaging (MRI) of the head, neck and chest, and lab tests requiring samples of blood, urine or stool. This study is funded by Children's Research Institute, the Dermatology Foundation and the American Skin Association.
- Multifocal Lymphangioendotheliomatosis with Thrombocytopenia Registry
Multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT) is a rare vascular disorder characterized by multiple congenital skin and visceral lesions, profound thrombocytopenia, and gastrointestinal bleeding. Although a relatively newly described entity, MLT was likely previously reported as hemangiomas, blue rubber bleb nevus syndrome, diffuse hemangiomatosis, Kasabach-Merritt phenomenon, and hereditary hemorrhagic telangiectasia. The term cutaneovisceral angiomatosis with thrombocytopenia is also a term used for this same disease. We have designed a web-based registry to collect information and biopsy specimens on all patients diagnosed with MLT. Dr Paula North, who originally described this disease will review all biopsy specimens. The immediate goal of the registry is to collect long-term morbidity and mortality data. In addition, the registry will address many unanswered questions regarding risk factors and treatment options for this rare disease. This data will be used to better understand the disease, design diagnostic criteria, and create treatment guidelines. Families can contact our department directly and we will send information to them. Please do not hesitate to contact Kelly Duffy, Ph.D., at (414) 456-4078 for more information. This study is funded by the Children's Research Institute.
- Longitudinal Study of Neurologic, Cognitive and Radiologic Outcomes in PHACE Syndrome What is PHACE syndrome? A small group of patients with skin hemangiomas (non-cancerous growth of blood vessels appearing as a type of birthmark) on the head and neck may also have additional disorders associated with PHACE syndrome. PHACE refers to Posterior fossa anomalies (a structural brain abnormality); Hemangioma (growth of blood vessels) appearing on the skin; Arterial abnormalities; Cardiac (heart) abnormalities; and abnormalities of the Eye. Your child may have one or several of the abnormalities listed above as part of the PHACE syndrome. To participate in this trial: (1) Your child must be diagnosed with definite or probable PHACE syndrome (according to the 2009 criteria); (2) Child must be 4, 5, or 6 years of age; (3) You and your child must be available to travel to Children's Hosptial of Wisconsin for scheduled outpatient evaluations over a one or two day period (limited travel funds are provided by the study); (4) No drugs are used for this study. Why is this study important? While it is known that children with PHACE syndrome may have abnormalities of the blood vessels in the brain or structural brain abnormalities, it is not known what the significance of these abnormalities is or how this affects children as they get older. As a result, it is difficult for physicians to counsel parents of infants with PHACE syndrome regarding future expectations or problems that may be encountered in regards to development. This project is the first study to look at specific areas of development in children with PHACE syndrome through neurologic, psychological and cognitive evaluations. The data used from this study will be used in the development of standardized testing to establish clinical guidelines for the management of children with PHACE syndrome. About 30 children will be enrolled in this study. We believe that the information gained in this study will better characterize PHACE syndrome, and establish guidelines for diagnostic neuroimaging of at-risk infants. For more information about this study, please contact the research coordinator, Linda Gaertner, at 414-456-5769.
Past research
To search for research articles published by physicians in the Dermatology Clinic and the Birthmarks and Vascular Anomalies Center, click here. |
