''Efficacy of Propranolol in the Treatment of Infantile Hemangioma"

Phase 2

• Conditions: Infantile Hemangioma
• Interventions: Drug: Propranolol Therapy for Infantile Hemangioma

• Registration Number: NCT04684667
• Lead Sponsor: Assiut University

Brief Summary

''Evaluation of the Efficacy of Propranolol in the Treatment of Infantile Hemangioma"

Detailed Description

Infantile hemangiomas (IH) are benign tumors of vascular endothelium.They are the most common tumors of infancy. They occur in about 4% of infants, though early studies were as high as 10%, probably due to the inclusion of other vascular lesions. The incidence is higher in premature infants, Caucasians, and females (by a 3 to 5:1 ratio). Advanced maternal age, multiple gestations, and placental abnormalities are also risk factors. IHs have a unique and characteristic life cycle consisting of three phases: proliferative, involuting, and involuted.

The majority of IHs do not require any specific treatment other than observation and reassurance of the parents. Even tumors that exhibit rapid growth or fiery red skin will spontaneously regress and leave behind little to no evidence of their presence. However, regular follow-up is important as the potential complications have few clinical indicators.

Reasons for treatment include dangerous locations (impinging on a vital structure such as the airway or eye), unusually large size or rapid growth, and local or endangering complications (skin ulceration or high-output heart failure). Hemangiomas exhibiting the aforementioned risk factors or complications should be considered for treatment. As hemangiomas are tumors of pure angiogenesis, pharmacologic therapy involves angiogenesis inhibition.

Historically, steroids have been used as the primary treatment for IH. Steroids have been shown to be antiangiogenic in a number of in vitro settings and also have shown good therapeutic effects clinically. However, the use of steroids may lead to various complications including gastroesophageal reflux and growth disorders, although these complications are associated with long-term use and high dose. A type of anticancer drug or immunomodulator, interferon alfa, may be used for severe IH in cases where patients did not respond to steroids. However, interferon alfa also has several possible adverse effects, including fever, muscle pain, systemic myalgia, and in severe cases, liver damage, blood toxic effects, thyroid hormonal abnormality, and neurological and neurodevelopmental toxic effects. Because of concerns about these adverse effects, many guardians of pediatric patients prefer to wait rather than accept treatment.

Propranolol, a nonselective beta blocker, has recently been recognized as an important treatment option for hemangiomas. In most centers, it has become first-line pharmacotherapy. A child with a nasal capillary hemangioma treated with propranolol for steroid-induced cardiomyopathy had regression of his lesion. This revelation led to the publication of several more studies supporting this finding. Propranolol is given orally at 2-3 mg/kg/day, in two or three divided doses, and discontinued following regression of the lesion. Treatment often leads to a consistent, rapid, therapeutic effect with softening of the lesion on palpation and color shift from intense red to purple. Propranolol is well tolerated but can cause rare side effects such as bradycardia, gastroesophageal reflux, hypoglycemia, hypotension, rash, somnolence, and wheezing.

Study Timeline

• Study First Submitted: 2020-04-29
• Status Verified: 2020-12
• Study First Submitted QC: 2020-12-23
• Last Update Submitted: 2020-12-23
• Study First Posted: 2020-12-24
• Last Update Posted: 2020-12-24
• Study Start: 2021-01-01
• Primary Completion: 2021-12-31
• Study Completion: 2021-12-31

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

• Children diagnosed with problematic infantile hemangiomas:

A-Large hemangiomas at increased risk of scarring or disfigurement at any site. B-Hemangiomas carrying functional risks near the eyes, nose, natural orifices, limbs, genitalia.

C-Ulcerated infantile hemangiomas. D-Uncomplicated progressive infantile hemangiomas with unpredictable future course.

E-Life-threatening hemangiomas.

• Multiple hemangiomas

Exclusion Criteria

• Patients older than 1 year of age.
• Patients with heart diseases.
• Patients with history of bronchspasm or wheezing.
• Patients with Hypotension.
• Patients with Hypertension.
• Premature infants with corrected age less than 5 weeks.
• Patients with conditions affecting blood glucose maintenance.
• Patients with liver failure.
• Patients with PHACES syndrome.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Propranolol Therapy	Propranolol Therapy for Infantile Hemangioma	This arm will involve patients with infantile hemangiomas who will be admitted at the Assiut University Children Hospital between January 2021 and December 2021.

Primary Outcome Measures

Name	Time	Method
Complete clinical clearance of hemangioma or reduction of hemangioma's volume.	within 9 months	The efficacy of the treatment will be evaluated by complete clinical clearance of lesion (defined arbitrarily as \>90% reduction in the size of Infantile Hemangioma as assessed by Physician Global Assessment) or after 9 months of treatment (primary end point) whichever is earlier for cutaneous infantile hemangiomas and by volume reduction of hemangiomas as evaluated by the proper imaging modalities ( Ultrasound, CT, or MRI) for non cutaneous hemangiomas.

Trial Locations

Locations (1)

Assiut University Teaching Hospital; 🇪🇬 Assiut, Egypt