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

NCT ID: NCT04684667

Last Updated: 2020-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-01-01

Study Completion Date

2021-12-31

Brief Summary

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

Detailed Description

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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.

Conditions

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Infantile Hemangioma

Keywords

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Hemangioma Propranolol Beta Blockers

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

This study will involve patients with infantile hemangiomas who will be admitted at the Assiut University Children Hospital between January 2021 and December 2021.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Propranolol Therapy

This arm will involve patients with infantile hemangiomas who will be admitted at the Assiut University Children Hospital between January 2021 and December 2021.

Group Type OTHER

Propranolol Therapy for Infantile Hemangioma

Intervention Type DRUG

If there are no contraindications to propranolol, it will be given at initial dose of 0.5 mg /kg/day in 2-3 divided doses with feeds and the patients will be observed for clinical signs of serious adverse reactions. If the dose is tolerated, it will be maintained for four to seven days, and then it will be increased by 0.5mg/kg/day every four days to the target dose of 2mg/kg/day. The patients will be observed for 2 hours after each dose increase for the clinical signs of severe side effects.

Interventions

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Propranolol Therapy for Infantile Hemangioma

If there are no contraindications to propranolol, it will be given at initial dose of 0.5 mg /kg/day in 2-3 divided doses with feeds and the patients will be observed for clinical signs of serious adverse reactions. If the dose is tolerated, it will be maintained for four to seven days, and then it will be increased by 0.5mg/kg/day every four days to the target dose of 2mg/kg/day. The patients will be observed for 2 hours after each dose increase for the clinical signs of severe side effects.

Intervention Type DRUG

Other Intervention Names

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Propranolol Treatment for Infantile Hemangioma

Eligibility Criteria

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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.
Minimum Eligible Age

1 Day

Maximum Eligible Age

1 Year

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Assiut University

OTHER

Sponsor Role lead

Responsible Party

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Essam Ezzat Anees Mowannas

Principal Investigator

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

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Assiut University Teaching Hospital

Asyut, , Egypt

Site Status

Countries

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Egypt

Central Contacts

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Essam EA Mowannas, MD

Role: CONTACT

Phone: +201285289188

Email: [email protected]

Facility Contacts

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Essam E.A Mowannas, MD

Role: primary

References

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Darrow DH, Greene AK, Mancini AJ, Nopper AJ; SECTION ON DERMATOLOGY, SECTION ON OTOLARYNGOLOGY-HEAD AND NECK SURGERY, and SECTION ON PLASTIC SURGERY. Diagnosis and Management of Infantile Hemangioma. Pediatrics. 2015 Oct;136(4):e1060-104. doi: 10.1542/peds.2015-2485.

Reference Type BACKGROUND
PMID: 26416931 (View on PubMed)

Darrow DH, Greene AK, Mancini AJ, Nopper AJ; SECTION ON DERMATOLOGY, SECTION ON OTOLARYNGOLOGY-HEAD & NECK SURGERY, AND SECTION ON PLASTIC SURGERY. Diagnosis and Management of Infantile Hemangioma: Executive Summary. Pediatrics. 2015 Oct;136(4):786-91. doi: 10.1542/peds.2015-2482. No abstract available.

Reference Type BACKGROUND
PMID: 26416928 (View on PubMed)

Leaute-Labreze C, Harper JI, Hoeger PH. Infantile haemangioma. Lancet. 2017 Jul 1;390(10089):85-94. doi: 10.1016/S0140-6736(16)00645-0. Epub 2017 Jan 13.

Reference Type BACKGROUND
PMID: 28089471 (View on PubMed)

HOLMDAHL K. Cutaneous hemangiomas in premature and mature infants. Acta Paediatr (Stockh). 1955 Jul;44(4):370-9. doi: 10.1111/j.1651-2227.1955.tb04151.x. No abstract available.

Reference Type BACKGROUND
PMID: 13292246 (View on PubMed)

Amir J, Metzker A, Krikler R, Reisner SH. Strawberry hemangioma in preterm infants. Pediatr Dermatol. 1986 Sep;3(4):331-2. doi: 10.1111/j.1525-1470.1986.tb00535.x.

Reference Type BACKGROUND
PMID: 3774653 (View on PubMed)

Hemangioma Investigator Group; Haggstrom AN, Drolet BA, Baselga E, Chamlin SL, Garzon MC, Horii KA, Lucky AW, Mancini AJ, Metry DW, Newell B, Nopper AJ, Frieden IJ. Prospective study of infantile hemangiomas: demographic, prenatal, and perinatal characteristics. J Pediatr. 2007 Mar;150(3):291-4. doi: 10.1016/j.jpeds.2006.12.003.

Reference Type BACKGROUND
PMID: 17307549 (View on PubMed)

Margileth AM, Museles M. Cutaneous hemangiomas in children. Diagnosis and conservative management. JAMA. 1965 Nov 1;194(5):523-6. No abstract available.

Reference Type BACKGROUND
PMID: 5897362 (View on PubMed)

Bennett ML, Fleischer AB Jr, Chamlin SL, Frieden IJ. Oral corticosteroid use is effective for cutaneous hemangiomas: an evidence-based evaluation. Arch Dermatol. 2001 Sep;137(9):1208-13. doi: 10.1001/archderm.137.9.1208.

Reference Type BACKGROUND
PMID: 11559219 (View on PubMed)

Greenberger S, Boscolo E, Adini I, Mulliken JB, Bischoff J. Corticosteroid suppression of VEGF-A in infantile hemangioma-derived stem cells. N Engl J Med. 2010 Mar 18;362(11):1005-13. doi: 10.1056/NEJMoa0903036.

Reference Type BACKGROUND
PMID: 20237346 (View on PubMed)

Crum R, Szabo S, Folkman J. A new class of steroids inhibits angiogenesis in the presence of heparin or a heparin fragment. Science. 1985 Dec 20;230(4732):1375-8. doi: 10.1126/science.2416056.

Reference Type BACKGROUND
PMID: 2416056 (View on PubMed)

Boon LM, MacDonald DM, Mulliken JB. Complications of systemic corticosteroid therapy for problematic hemangioma. Plast Reconstr Surg. 1999 Nov;104(6):1616-23. doi: 10.1097/00006534-199911000-00002.

Reference Type BACKGROUND
PMID: 10541160 (View on PubMed)

Barlow CF, Priebe CJ, Mulliken JB, Barnes PD, Mac Donald D, Folkman J, Ezekowitz RA. Spastic diplegia as a complication of interferon Alfa-2a treatment of hemangiomas of infancy. J Pediatr. 1998 Mar;132(3 Pt 1):527-30. doi: 10.1016/s0022-3476(98)70034-4.

Reference Type BACKGROUND
PMID: 9544915 (View on PubMed)

Deb G, Jenkner A, Donfrancesco A. Spastic diplegia and interferon. J Pediatr. 1999 Mar;134(3):382. doi: 10.1016/s0022-3476(99)70474-9. No abstract available.

Reference Type BACKGROUND
PMID: 10064684 (View on PubMed)

Leaute-Labreze C, Dumas de la Roque E, Hubiche T, Boralevi F, Thambo JB, Taieb A. Propranolol for severe hemangiomas of infancy. N Engl J Med. 2008 Jun 12;358(24):2649-51. doi: 10.1056/NEJMc0708819. No abstract available.

Reference Type BACKGROUND
PMID: 18550886 (View on PubMed)

Sans V, de la Roque ED, Berge J, Grenier N, Boralevi F, Mazereeuw-Hautier J, Lipsker D, Dupuis E, Ezzedine K, Vergnes P, Taieb A, Leaute-Labreze C. Propranolol for severe infantile hemangiomas: follow-up report. Pediatrics. 2009 Sep;124(3):e423-31. doi: 10.1542/peds.2008-3458. Epub 2009 Aug 10.

Reference Type BACKGROUND
PMID: 19706583 (View on PubMed)

Buckmiller LM, Munson PD, Dyamenahalli U, Dai Y, Richter GT. Propranolol for infantile hemangiomas: early experience at a tertiary vascular anomalies center. Laryngoscope. 2010 Apr;120(4):676-81. doi: 10.1002/lary.20807.

Reference Type BACKGROUND
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Bagazgoitia L, Torrelo A, Gutierrez JC, Hernandez-Martin A, Luna P, Gutierrez M, Bano A, Tamariz A, Larralde M, Alvarez R, Pardo N, Baselga E. Propranolol for infantile hemangiomas. Pediatr Dermatol. 2011 Mar-Apr;28(2):108-14. doi: 10.1111/j.1525-1470.2011.01345.x. Epub 2011 Mar 8.

Reference Type BACKGROUND
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Cushing SL, Boucek RJ, Manning SC, Sidbury R, Perkins JA. Initial experience with a multidisciplinary strategy for initiation of propranolol therapy for infantile hemangiomas. Otolaryngol Head Neck Surg. 2011 Jan;144(1):78-84. doi: 10.1177/0194599810390445.

Reference Type BACKGROUND
PMID: 21493392 (View on PubMed)

Holland KE, Frieden IJ, Frommelt PC, Mancini AJ, Wyatt D, Drolet BA. Hypoglycemia in children taking propranolol for the treatment of infantile hemangioma. Arch Dermatol. 2010 Jul;146(7):775-8. doi: 10.1001/archdermatol.2010.158.

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Harrison DC, Meffin PJ, Winkle RA. Clinical pharmacokinetics of antiarrhythmic drugs. Prog Cardiovasc Dis. 1977 Nov-Dec;20(3):217-42. doi: 10.1016/0033-0620(77)90022-6. No abstract available.

Reference Type BACKGROUND
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Other Identifiers

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Essam Mowannas

Identifier Type: -

Identifier Source: org_study_id