Hemangiomas are relatively common lesions in infants. Most go away spontaneously after one year of life and do not need treatment. Others require treatment because they cause significant symptoms such as pain, or difficulty with breathing, eating or ambulating. Steroids have classically been used to treat hemangiomas and help to shrink them in 1/3 - 2/3 of patients. Unfortunately, steroids have many side effects in babies so physicians have sought other ways to treat them. Recently, the use of propranolol, a heart medication, was serendipitously found to reduce the size of hemangiomas. It appears to have many fewer side effects than steroids but it is not yet known if it works as well as steroids. This study seeks to compare the effect and the side effects of propranolol versus steroids for treating hemangiomas that cause symptoms in infants.
Infants with symptomatic hemangiomas will be enrolled. Magnetic resonance imaging will be completed before starting medication if the extent of the hemangioma is not evident on clinical examination alone. Infants will be randomized to receive either propranolol or steroids for 4-6 months. Hemangioma response will be measured and compared monthly as will tolerability of the medications. Additionally, urine specimens will be collected at each visit to determine if markers are present that can predict response to therapy. Additionally, any hemangiomas that are excised will be examined for genetic markers to aid in predicting response to therapy.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
19
propranolol 0.5 mg/kg orally, 4 per day - 4-6 months
1.0 mg/kg orally, 2 per day 4-6 months
Children's National Medical Center
Washington D.C., District of Columbia, United States
Decrease in Size of Hemangioma (Length x Width) in Square mm
A priori primary outcome was proportional change in the total surface area as measured by lesion's outer margin length x width at baseline minus the same measure at 4 months with surrogate data used at 5 months if 4 months not available.
Time frame: 4-5 months after initiating therapy
Tolerability of Medication
All adverse events relating to medication tolerability including: adrenal crisis, growth/development, constitutional (dehydration), allergy/immunology, dermatologic, endocrine, GI, infection, metabolism/labs, pulmonary, vascular.
Time frame: enrollment until study close out or withdrawal up to 9 months
Number of Serious Adverse Events (SAEs)
Number of serious adverse events experienced by the participants in each treatment arm within the categories adrenal crisis, growth/development, constitutional. Serious adverse events are defined as events that result in death, require either inpatient hospitalization or the prolongation of hospitalization, are life-threatening, result in a persistent or significant disability/incapacity, or result in a congenital anomaly/birth defect. Other important medical events, based upon appropriate medical judgment, may also be considered Serious Adverse Events if a trial participant's health is at risk and intervention is required to prevent an outcome mentioned.
Time frame: enrollment until study close out or withdrawal up to 9 months
Growth and Development Adverse Events
Number of Growth and Development AEs in each study arm
Time frame: enrollment to study withdrawal or close out up to 9 months
Pulmonary/Respiratory Adverse Events
Number of pulmonary/respiratory adverse events (CTCAE 22) in each study arm
Time frame: enrollment through study close out or withdrawal, up to 9 months
Allergy/Immunology Adverse Events
Number of allergy/immunology AE per study arm
Time frame: enrollment through study closeout or study withdrawal up to 9 months
Dermatologic Adverse Events
Number of Dermatologic Adverse Events in each study arm.
Time frame: enrollment to study close out or withdrawal up to 9 months
Endocrinologic Adverse Events
Number of Endocrinologic AEs (of which adrenal crisis does not overlap).
Time frame: enrollment to close out or study withdrawal up to 9 months
Gastrointestinal Adverse Events
Number of Gastrointestinal AEs in each arm
Time frame: enrollment to study withdrawal or study close out up to 9 months
Infectious Adverse Events
Number of infectious AEs in each study arm (i.e. conjunctivitis, thrush, fever)
Time frame: enrollment to study withdrawal or close out up to 9 months
Metabolic or Laboratory AEs
Number of Metabolic or Laboratory AEs in each study arm.
Time frame: enrollment to study withdrawal or close out up to 9 months
Vascular Adverse Events
Number of Vascular AEs in each study arm.
Time frame: enrollment to study withdrawal or close out up to 9 months
Constitutional Adverse Events
Number of constitutional AEs in each study arm.
Time frame: enrollment to study close out or withdrawal up to 9 months
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