Hemangioma
Conditions
Brief summary
This is a prospective randomized, double-blind study to compare the clinical efficacy of infantile hemangioma treatment using propranolol with corticosteroids as compared to therapy with corticosteroids and placebo. We hypothesize that a two-month treatment period with propranolol plus corticosteroids is more effective at reducing infantile hemangioma size and vascularity when compared to corticosteroids used without propranolol for the same time period.
Detailed description
Infantile hemangiomas (IH) are the most common head and neck pediatric tumors. Presence of these tumors can lead to complications of vision and airway compromise, bleeding and disfigurement. Medical treatment of these lesions has traditionally been focused on stopping new blood vessel growth with corticosteroids. Recent reports and our own experience have demonstrated that significant reduction in IH size and vascularity can also occur through the use of propranolol. Our initial experience with propranolol has demonstrated significant efficacy with fewer side effects than corticosteroids. Despite this experience, the standard of care for initial IH medical therapy remains corticosteroids. This Trial is a direct comparison of traditional IH therapy with corticosteroids to newer therapy with propranolol and corticosteroids.
Interventions
DRUGPrednisolone (Corticosteroid)
Oral liquid prednisolone. Dose: 1-2mg/kg/day for 7 days, and then dose will be slowly reduced and then stopped after 3 weeks.
DRUGPropranolol
Oral liquid propranolol will be dosed Oral liquid propranolol will be dosed at 2 mg/kg/day.
DRUGPlacebo
Liquid placebo will be given during the two month treatment trial.
Sponsors
Seattle Children's Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
No minimum to 9 Months
Healthy volunteers
No
Inclusion criteria
* Age 0 to \< 9months * Patients with clinical, radiographic or histologic diagnosis of infantile hemangioma (IH) requiring medical treatment * IH patients whose parents desire medical treatment for the IH * Stable cardiac function
Exclusion criteria
* IH patients over 9 months of age. * Hypersensitivity to propranolol * Untreated heart failure: If the patient has heart failure associated with the hemangioma, propranolol would be initiated after anti-congestive therapy and under observation as an in-patient. * Atrioventricular (AV) block * Resting heart \< 2 SD of normal\*(below) * Resting blood pressure \< 2 SD of normal\*\*(below) * Wolff-Parkinson-White syndrome (WPW) * History of unexplained syncope * Bronchial asthma * History of impaired renal or liver function * Diabetes mellitus
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Compare Changes in IH Size and Vascularity for the Two Treatment Groups | 1, 2, and 6 months after treatment initiation | This outcome cannot be evaluated. Trial limited by lack of patient family to have randomization, which limited the number of participants and made the project nonviable. |
| Lesion Regression | 12 months | measure of change in lesion area or volume |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Determine Therapeutic Response of IH to Propranolol Among Patients Who Switch to Corticosteroids Plus Propranolol Therapy After Failing to Respond to Corticosteroids Alone. | 1, 2, and 6 months after treatment initiation | This outcome cannot be evaluated. Trial limited by lack of patient family to have randomization, which limited the number of participants and made the project nonviable. |
| Assess the Safety of Propranolol With Corticosteroids and Corticosteroids Alone in the Treatment of IH. | 1, 2 and 6 months after treatment initiation | This outcome cannot be evaluated. Trial limited by lack of patient family to have randomization, which limited the number of participants and made the project nonviable. |
| Demonstrate How Duplex Scanning to Assess Blood Vessel Density and Qualitative Color Ratings of Cutaneous Lesions From Photographs Can be Used to Measure and Quantify Changes in IH Size and Vascularity in a Clinically Relevant Manner. | 1, 2 and 6 months after treatment initiation | This outcome cannot be evaluated. Trial limited by lack of patient family to have randomization, which limited the number of participants and made the project nonviable. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Group A: Corticosteroid With Placebo Group A will receive oral, liquid Prednisolone, which is the standard corticosteroid that we use here at Seattle Children's, and oral liquid placebo. The dose of prednisolone that Group A will receive will be 1-2mg/kg/day for 7 days and then the dose will be slowly reduced and then stopped after 3 weeks. This is a standard dose for IH treatment. Gastric prophylaxis (Zantac) will be given to help prevent any stomach problems associated with prednisolone. This treatment will be given for two months, as is our standard practice. | 4 |
| Group B: Corticosteroid With Propranolol Group B will receive oral liquid prednisolone, and oral propranolol. As in Group A, the dose of prednisolone will be 1-2mg/kg/day for 7 days and then the dose will be slowly reduced and then stopped after 3 weeks. Oral liquid propranolol will be dosed at 2 mg/kg/day, following initiation in the Cardiology Clinic. Gastric prophylaxis (Zantac) will be given to help prevent any stomach problems associated with prednisolone. | 5 |
| Total | 9 |
Baseline characteristics
| Characteristic | Group B: Corticosteroid With Propranolol | Group A: Corticosteroid With Placebo | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 5 Participants | 4 Participants | 9 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Continuous | 0.2 years STANDARD_DEVIATION 0.1 | 0.2 years STANDARD_DEVIATION 0.1 | 0.2 years STANDARD_DEVIATION 0.1 |
| Region of Enrollment United States | 5 participants | 4 participants | 9 participants |
| Sex: Female, Male Female | 3 Participants | 3 Participants | 6 Participants |
| Sex: Female, Male Male | 2 Participants | 1 Participants | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 4 | 0 / 5 |
| serious Total, serious adverse events | 0 / 4 | 0 / 5 |
Outcome results
Primary
Compare Changes in IH Size and Vascularity for the Two Treatment Groups
This outcome cannot be evaluated. Trial limited by lack of patient family to have randomization, which limited the number of participants and made the project nonviable.
Time frame: 1, 2, and 6 months after treatment initiation
Population: This outcome cannot be evaluated. Trial limited by lack of patient family to have randomization, which limited the number of participants and made the project nonviable.
Primary
Lesion Regression
measure of change in lesion area or volume
Time frame: 12 months
Population: Insufficient enrollment for data analysis to be meaningful.
Secondary
Assess the Safety of Propranolol With Corticosteroids and Corticosteroids Alone in the Treatment of IH.
This outcome cannot be evaluated. Trial limited by lack of patient family to have randomization, which limited the number of participants and made the project nonviable.
Time frame: 1, 2 and 6 months after treatment initiation
Population: This outcome cannot be evaluated. Trial limited by lack of patient family to have randomization, which limited the number of participants and made the project nonviable.
Secondary
Demonstrate How Duplex Scanning to Assess Blood Vessel Density and Qualitative Color Ratings of Cutaneous Lesions From Photographs Can be Used to Measure and Quantify Changes in IH Size and Vascularity in a Clinically Relevant Manner.
This outcome cannot be evaluated. Trial limited by lack of patient family to have randomization, which limited the number of participants and made the project nonviable.
Time frame: 1, 2 and 6 months after treatment initiation
Population: This outcome cannot be evaluated. Trial limited by lack of patient family to have randomization, which limited the number of participants and made the project nonviable.
Secondary
Determine Therapeutic Response of IH to Propranolol Among Patients Who Switch to Corticosteroids Plus Propranolol Therapy After Failing to Respond to Corticosteroids Alone.
This outcome cannot be evaluated. Trial limited by lack of patient family to have randomization, which limited the number of participants and made the project nonviable.
Time frame: 1, 2, and 6 months after treatment initiation
Population: This outcome cannot be evaluated. Trial limited by lack of patient family to have randomization, which limited the number of participants and made the project nonviable.