Faculty Collaboration Database

Topical Timolol Maleate Treatment of Infantile Hemangiomas. Pediatrics 2016 Sep;138(3)

Date

08/17/2016

Pubmed ID

27527799

DOI

10.1542/peds.2016-0355

Scopus ID

2-s2.0-84985993148 (requires institutional sign-in at Scopus site)   111 Citations

Abstract

BACKGROUND: There has been a dramatic increase in the off-label use of ophthalmic timolol maleate, a β-blocker used for infantile hemangioma (IH) treatment as a topical counterpart to oral propranolol. Its safety and efficacy in a pediatric population with IH have not been evaluated in a large cohort. Our goal was to retrospectively assess timolol's effectiveness, discern characteristics associated with response, and document reported adverse events.

METHODS: A multicenter retrospective cohort study of 731 patients treated with topical timolol was completed at 9 centers. Inclusion required an IH suitable for timolol in the treating physician's judgment and access to clinical details including photographs. Logistic regression analysis and descriptive statistics were performed. Primary outcome measures were efficacy assessed by using visual analog scales for color and for size, extent, and volume from review of digital photographs taken as standard of care.

RESULTS: Most IHs were localized (80.1%) and superficial (55.3%). Risk of disfigurement was the most common indication for therapy (74.3%). Duration of therapy (P < .0001), initial thinness (P = .008), and subtype (P = .031) were significant predictors of response. Best response occurred in superficial IHs <1 mm thick. Fifty-three (7.3%) required subsequent therapy with systemic β-blocker. Adverse events were mild, occurring in 25 (3.4%) patients. No cardiovascular side effects were documented.

CONCLUSIONS: Timolol seems to be a well-tolerated, safe treatment option with moderate to good effectiveness, demonstrating best response in thin, superficial IHs regardless of pretreatment size. Timolol can be recommended as an alternative to systemic β-blockers and watchful waiting for many patients.

Author List

Püttgen K, Lucky A, Adams D, Pope E, McCuaig C, Powell J, Feigenbaum D, Savva Y, Baselga E, Holland K, Drolet B, Siegel D, Morel KD, Garzon MC, Mathes E, Lauren C, Nopper A, Horii K, Newell B, Song W, Frieden I, Hemangioma Investigator Group

Author

Kristen E. Holland MD Associate Professor in the Dermatology department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Administration, Oral
Administration, Topical
Adrenergic beta-Antagonists
Cohort Studies
Female
Hemangioma
Humans
Infant
Infant, Newborn
Male
Off-Label Use
Propranolol
Retrospective Studies
Skin Neoplasms
Timolol
Visual Analog Scale