Oral propranolol may be safe in infants with PHACE syndrome

Oral propranolol yielded no serious adverse events and few nonserious events in a cohort of infants with PHACE syndrome, according to a study.

“Oral propranolol is widely considered to be first-line therapy for complicated infantile hemangioma, but its use in patients with PHACE (posterior fossa malformations, hemangioma, arterial anomalies, cardiac defects, eye anomalies) syndrome has been debated owing to concerns that the cardiovascular effects of the drug may increase the risk for arterial ischemic stroke,” Gerilyn M. Olsen, BA, of the department of dermatology at the Medical College of Wisconsin, and colleagues wrote.

In the retrospective cohort study, the researchers looked at adverse event incident rates in 76 patients with PHACE syndrome who had been treated with 0.3 mg/kg or more of oral propranolol for infantile hemangioma. Eligible participants were treated at one of 11 tertiary care centers or academic pediatric dermatology practices and had medical records for intervention between Jan. 1, 2010, and April 25, 2017.

The rate and severity of adverse events throughout oral propranolol treatment served as the primary endpoint.

Baseline data showed that 59 girls and 17 boys comprised the cohort. Propranolol therapy began at a median age of 56 days (range, 0 to 396 days).

Results indicated no occurrence of serious adverse events such as stroke, transient ischemic attack or other cardiovascular events while patients were receiving propranolol. The nonserious adverse event rate was 38.2%. Sleep disturbances topped the list of nonserious events, followed by minor gastrointestinal and respiratory tract symptoms.

The researchers also compared findings in the current cohort to those reported in 726 infants who underwent comparable oral propranolol therapy for the same indication but failed to meet criteria for PHACE syndrome. Results of this comparison showed adverse event rates of 0% for the PHACE syndrome group and 0.4% for the non-PHACE group.

Zeroing in on the key outcome of stroke incidence, other findings indicated that among the 29 patients who experienced a nonserious adverse event, 21% were at a high risk for stroke, 34% carried an intermediate risk for stroke, and 45% were low risk. The researchers observed no significant association between adverse event incidence and stroke risk categorization.

“To our knowledge, this multicenter study of 76 patients comprises the largest cohort to date of patients with PHACE syndrome treated with propranolol,” the researchers said. “These data provide support for the safety of oral propranolol in this patient population.” – by Rob Volansky

Disclosures: Olsen reports receiving grants from the National Institutes of Health National Heart, Lung, and Blood Institute during the conduct of the study. Please see the study for all other authors’ relevant financial disclosures.