Infantile Hemangioma (IH)
Conditions
Brief summary
This is a prospective, randomized controlled trial designed to evaluate the efficacy and safety of low-dose oral propranolol (1 mg/kg/day) combined with monthly intralesional bleomycin injections versus low-dose propranolol monotherapy for the treatment of infantile hemangioma (IH). A total of 260 infants were randomized to either the combination group or the control group. The study aims to determine if the combination therapy offers superior clinical outcomes, including faster regression, better color resolution, and reduced scarring over a 6-month treatment period.
Detailed description
Infantile hemangioma (IH) is the most common benign vascular tumor in infancy. While oral propranolol is the first-line systemic therapy, its efficacy can be limited or slow for certain IHs. Intralesional bleomycin offers a targeted local therapy. This study investigates whether combining low-dose propranolol with local bleomycin injections can enhance therapeutic effects while maintaining a good safety profile. This prospective, randomized, controlled, open-label trial with blinded outcome assessment enrolled 260 infants with IH. Participants were randomly assigned to receive either oral propranolol (1 mg/kg/day) plus monthly intralesional bleomycin (Combination Group) or oral propranolol (1 mg/kg/day) alone (Control Group) for 6 months. The primary objective is to compare the clinical therapeutic effect between the two groups at 6 months. Secondary objectives include evaluating early tumor response, changes in tumor volume and color, long-term scar formation, and the incidence of adverse events. The findings aim to provide robust evidence for a potentially more effective combination treatment strategy for IH.
Interventions
DRUGPropranolol + Bleomycin
Oral propranolol at 1 mg/kg/day for 6 months plus intralesional bleomycin (1 mg/mL solution) injected once monthly. The bleomycin dose was 0.2-0.5 mg per injection site, not exceeding a total of 1 mg/kg per session or a cumulative dose of 10 mg.
DRUGPropranolol
Oral propranolol hydrochloride solution administered at a dose of 1 mg/kg/day for 6 months.
Sponsors
Wuhan Integrated Traditional Chinese and Western Medicine Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
No minimum to 12 Months
Healthy volunteers
No
Inclusion criteria
* Age ≤12 months. * Clinically and/or imaging-diagnosed infantile hemangioma requiring treatment. * No prior treatment for IH. * Guardians willing to comply with the study protocol and provide informed consent.
Exclusion criteria
* Known hypersensitivity to propranolol or bleomycin. * Congenital or mixed hemangiomas distinct from IH. * Significant cardiopulmonary, hepatic, or renal dysfunction. * Presence of other severe systemic diseases. * PHACE syndrome or major congenital anomalies that could interfere with treatment or assessment.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Clinical Therapeutic Effect | 6 Months | Categorized as Complete Regression (\>95% volume reduction), Marked Effectiveness (≥75% to 95% volume reduction), Effective (30% to \<75% reduction), or Ineffective (\<30% reduction). The primary measure is the rate of participants achieving Complete Regression or Marked Effectiveness. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Early Tumor Surface Atrophy | 24 hours after the first treatment | Assessed by comparing photographs and graded as Obvious Atrophy, Mild Atrophy, or No Obvious Change. |
| Change in Hemangioma Color Score | Baseline, 6 Months | Rated on a 4-point subjective scale (0-3) based on standardized digital photographs, where a lower score indicates color closer to normal skin. The change from baseline is assessed. |
| Change in Tumor Volume | Baseline, 6 Months | Measured in cubic centimeters (cm³) using high-frequency color Doppler ultrasound (Volume = length × width × height × 0.52). The change from baseline is assessed. |
| Vancouver Scar Scale (VSS) Score | 6 Months | Assessed at the former hemangioma site. The VSS evaluates pigmentation, vascularity, pliability, and height, with a total score from 0 (normal skin) to 13 (worst scar). Lower scores indicate better outcomes. |
| Incidence of Adverse Events | Through study completion (6 months) | Number and type of local and systemic adverse events reported by guardians or observed clinically (e.g., injection site reactions, bradycardia, hypoglycemia, sleep disturbances). |
Countries
China
Outcome results
None listed