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Brief Report
December 11, 2019

Evaluating the Safety of Oral Propranolol Therapy in Patients With PHACE Syndrome

Author Affiliations
  • 1Department of Dermatology, Medical College of Wisconsin, Milwaukee,
  • 2Department of Dermatology, University of California, San Francisco
  • 3Department of Dermatology, John Hopkins University School of Medicine, Baltimore, Maryland
  • 4Department of Dermatology, Intermountain Healthcare, Salt Lake City, Utah
  • 5Department of Dermatology, Mayo Clinic, Rochester, Minnesota
  • 6Department of Dermatology, Columbia University, New York, New York
  • 7Department of Pediatrics, Northwestern University and Ann and Robert H. Lurie Children’s Hospital of Chicago, Chicago, Illinois
  • 8Department of Dermatology, Northwestern University and Ann and Robert H. Lurie Children’s Hospital of Chicago, Chicago, Illinois
  • 9Division of Pediatric Dermatology, Department of Pediatrics, Sainte-Justine University Hospital Centre, University of Montreal, Montreal, Quebec, Canada
  • 10Department of Hematology, Boston Children’s Hospital, Boston, Massachusetts
  • 11Pediatric Dermatology Unit, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain
  • 12Division of Dermatology, Department of Pediatrics, Seattle Children’s Hospital, Seattle, Washington
  • 13Department of Pediatrics, Medical College of Wisconsin, Milwaukee
  • 14Division of Dermatology, Children’s Mercy Hospital, Kansas City, Missouri
  • 15Department of Radiology, Medical College of Wisconsin, Milwaukee
  • 16Department of Dermatology, University of Wisconsin–Madison School of Medicine and Public Health, Madison
JAMA Dermatol. 2020;156(2):186-190. doi:10.1001/jamadermatol.2019.3839
Key Points

Question  Is oral propranolol safe to use for the treatment of infantile hemangioma in patients with PHACE (posterior fossa malformations, hemangioma, arterial anomalies, cardiac defects, eye anomalies) syndrome?

Findings  This multicenter cohort study describes the use of oral propranolol in 76 infants with PHACE syndrome with no reports of serious adverse events. There was no significant difference in the rate of serious adverse events between patients with PHACE syndrome and patients without PHACE syndrome who received oral propranolol for infantile hemangioma.

Meaning  These data support the safety of oral propranolol in patients with PHACE syndrome.

Abstract

Importance  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.

Objective  To assess the incidence of adverse events among patients with PHACE syndrome receiving oral propranolol for infantile hemangioma.

Design, Setting, and Participants  This multicenter retrospective cohort study assessed the incidence of adverse events among 76 patients with PHACE syndrome receiving oral propranolol for infantile hemangioma at 11 tertiary care, academic pediatric dermatology practices. Medical records from January 1, 2010, through April 25, 2017, were reviewed.

Exposures  Patients received oral propranolol, 0.3 mg/kg/dose or more.

Main Outcomes and Measures  The main outcome was the rate and severity of adverse events occurring throughout the course of treatment with oral propranolol, as documented in the medical records. Adverse events were graded from 1 to 5 using a scale derived from the Common Terminology Criteria for Adverse Events and were considered to be serious if they were grade 3 or higher.

Results  A total of 76 patients (59 girls and 17 boys; median age at propranolol initiation, 56 days [range, 0-396 days]) met the inclusion criteria. There were no reports of serious adverse events (ie, stroke, transient ischemic attack, or cardiovascular events) during treatment with oral propranolol. A total of 46 nonserious adverse events were reported among 29 patients (38.2%); the most commonly reported nonserious adverse events were sleep disturbances and minor gastrointestinal tract and respiratory tract symptoms. In a comparison with 726 infants who received oral propranolol for hemangioma but did not meet criteria for PHACE syndrome, there was no significant difference in the rate of serious adverse events experienced during treatment (0 of 76 patients with PHACE syndrome and 3 of 726 patients without PHACE syndrome [0.4%]).

Conclusions and Relevance  This study found that oral propranolol was used to treat infantile hemangioma in 76 patients with PHACE syndrome and that no serious adverse events were experienced. These data provide support for the safety of oral propranolol in this patient population.

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