PHACES syndrome

Would close observation be sufficient for this facial lesion?

Case:A 7-week-old girl is brought for evaluation of swelling and “rash” on the right side of the forehead that involved the right eyelid and extended to the right ear. The rash had been present at birth and had progressed. The infant also has small satellite lesions on the right upper lip. Ptosis of the right upper eyelid is evident.
There are no other visible deformities. A grade I/VI systolic ejection murmur with radiation to the axilla is appreciated.

Would close observation be sufficient for this facial lesion—or is further evaluation required?

(Answer on next page.)

Answer: Large segmental cervicofacial hemangiomas are associated with PHACES syndrome; further evaluation is required.

Hemangiomas are the most common benign tumor of infancy, with a prevalence of 2.5% to 10%.1,2 Infants with segmental cervicofacial hemangiomas, especially those greater than 5 cm, are at risk for systemic manifestations of PHACES, or PHACE, syndrome, a neurocutaneous disorder. PHACES stands for Posterior fossa malformations, Hemangiomas, Arterial cerebrovascular anomalies, Cardiovascular anomalies, Eye abnormalities, Sternal defects, and Supraumbilical raphe.

Although the exact prevalence of PHACES syndrome is unknown, it is believed to be more common than previously estimated. About 65% of infants with cervicofacial hemangiomas larger than 22 cm2 have at least 1 other manifestation of PHACES syndrome, of which CNS and cerebrovascular anomalies are the most common.3,4 Thus, the workup should include 2D transthoracic echocardiogram, MRI of the brain, magnetic resonance angiogram (MRA) of the head and neck, and complete ophthalmologic and neurological evaluations.

In this infant, laboratory studies were unremarkable. An MRI scan of the brain revealed a solid, enhancing, lobulated mass (2 3 2 3 3 cm) occupying most of the superficial lobe of the right parotid gland and a 2.3-cm mass in the right lacrimal gland extending into the extraconal space lateral to the right lateral rectus. MRA showed significant mild hypoplasia and stenosis of an otherwise patent right internal carotid artery. Echocardiogram revealed a patent foramen ovale, right aortic arch, retro-innominate vein, and bilateral peripheral pulmonary stenosis. Evaluation by a pediatric ophthalmologist revealed infiltration of the hemangioma into the right upper eyelid. Neurological examination findings were normal.

Most infantile hemangiomas spontaneously regress over several years. The necessity of medical management is dependent on the lesion’s size, location, and character. Some hemangiomas are prone to ulcerate through surrounding tissue while others may compress vital structures, including surrounding vessels, airways, and orbital structures. Oral corticosteroids are generally considered first-line treatment. However, because of side effects (eg, osteoporosis, growth failure, infection, and hypertension), other treatments have been used.

Although not FDA-approved for infantile hemangioma, oral propranolol has been used successfully for the past 4 years, is well-tolerated, and is rapidly becoming the preferred treatment.2,5,6 Use of propranolol is controversial in PHACES syndrome because of the theoretical increased risk of cerebral ischemia in the presence of intracerebral vascular anomalies.6,7 However, with inpatient monitoring for signs of cerebrovascular compromise, bradycardia, hypotension, and other possible side effects (eg, hypoglycemia, bronchospasm), propranolol may safely be administered.

This infant was initially treated with oral prednisolone, 2 mg/kg/d. After a month, this treatment was tapered and oral propranolol was started. The patient was monitored in the hospital for 2 days while the dosage was increased from 0.5 to 2 mg/kg/d. She tolerated the medication well. Within 2 weeks, the hemangioma began to involute and the accompanying ptosis decreased. Near complete resolution was evident at 2-month follow-up.