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HEALTH PROFESSIONAL · SOURCE READING

Topical beta-blocker therapy

Source: Childhood Vascular Tumors Treatment (PDQ®)–Health Professional Version, National Cancer Institute.

Source updated: April 17, 2025 · Captured 2026-09-09.

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Context: Benign Tumors / Infantile Hemangioma / Treatment of infantile hemangioma

Topical beta-blockers are used mainly for the treatment of small, localized, superficial hemangiomas as an alternative to observation. They have also been used in combination with systemic therapy in complicated hemangiomas or to prevent rebound in hemangiomas being tapered off of systemic treatment.[127-129] The same precautions (assessment of comorbidities and family history), as noted previously for propranolol, should be followed for topical beta-blockers. Systemic absorption (plasma and urine) of timolol is variable and prescreening for normal cardiac, pulmonary, and endocrine issues are essential. Recent medical histories and physical examinations are also important. Cautious administration is necessary for ulcerated and deep hemangiomas because higher plasma concentrations of timolol can be seen.[130,131]

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The topical timolol that is used is the ophthalmic gel-forming solution 0.5%. One drop is applied to the hemangioma two times per day until stable response is achieved.

This treatment has limited side effects, but infants with a postmenstrual age of younger than 44 weeks and weight at treatment initiation of less than 2,500 grams may be at risk of adverse events, including bradycardia, hypotension, apnea, and hypothermia.[131,132] Close monitoring of temperature, blood pressure, and heart rate in premature and low birth weight infants with infantile hemangiomas at initiation of and during therapy with topical timolol is necessary.

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Evidence (topical timolol therapy):

A retrospective cohort study included 666 patients with infantile hemangioma who were treated with topical timolol for 12 months.[133]

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A retrospective cohort study included 666 patients with infantile hemangioma who were treated with topical timolol for 12 months.[133]

Of these patients, 583 (87.5%) had visible reductions in the size of their lesions.

A retrospective cohort study included 666 patients with infantile hemangioma who were treated with topical timolol for 12 months.[133]

A total of 188 children (28.2%) had excellent responses (no remaining visible abnormality), 127 of whom had complete responses earlier than 12 months.

A retrospective cohort study included 666 patients with infantile hemangioma who were treated with topical timolol for 12 months.[133]

Of the remaining patients, 292 (43.8%) had good outcomes (i.e., the hemangioma was less than half its original size), while 103 (15.5%) had fair outcomes (i.e., a visibly smaller hemangioma but not less than half its original size), and 83 (12.5%) had poor outcomes (i.e., there was no change in hemangioma size or it was larger).

A retrospective cohort study included 666 patients with infantile hemangioma who were treated with topical timolol for 12 months.[133]

Patients aged 3 months and younger were more likely to have better outcomes than those older than 3 months (P < .001).

A retrospective cohort study included 666 patients with infantile hemangioma who were treated with topical timolol for 12 months.[133]

Patients with small infantile hemangiomas (maximum diameter, 1.5–≤5 cm) also had better outcomes than those with large infantile hemangiomas (5–≤10 cm) (P = .046).

In a multicenter, retrospective, cohort study, 731 children with predominantly superficial hemangiomas were treated with topical timolol 0.5% twice daily.[129]

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In a multicenter, retrospective, cohort study, 731 children with predominantly superficial hemangiomas were treated with topical timolol 0.5% twice daily.[129]

Ninety-two percent of patients showed significant improvement in hemangioma color.

In a multicenter, retrospective, cohort study, 731 children with predominantly superficial hemangiomas were treated with topical timolol 0.5% twice daily.[129]

Seventy-seven percent of patients showed improvement in hemangioma size, extent, and volume.

In a multicenter, retrospective, cohort study, 731 children with predominantly superficial hemangiomas were treated with topical timolol 0.5% twice daily.[129]

Topical timolol is generally well tolerated; however, data on its safety are limited.

A Spanish consortium performed a prospective randomized trial to evaluate the efficacy and safety of topical timolol for the treatment of infantile hemangioma in the early proliferative stage.[134] This multicenter, randomized, double-blind, placebo-controlled, phase IIa pilot clinical trial included patients aged 10 to 60 days with focal or segmental hemangiomas (superficial, deep, mixed, or minimal/arrested growth). Patients were randomly assigned to treatment with either topical timolol maleate solution, 0.5%, or placebo, twice daily for 24 weeks.

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A Spanish consortium performed a prospective randomized trial to evaluate the efficacy and safety of topical timolol for the treatment of infantile hemangioma in the early proliferative stage.[134] This multicenter, randomized, double-blind, placebo-controlled, phase IIa pilot clinical trial included patients aged 10 to 60 days with focal or segmental hemangiomas (superficial, deep, mixed, or minimal/arrested growth). Patients were randomly assigned to treatment with either topical timolol maleate solution, 0.5%, or placebo, twice daily for 24 weeks.

At 24 weeks, there were no significant differences between the timolol treatment and the placebo for complete or nearly complete infantile hemangioma resolution (42% for timolol [n = 11] vs. 36% for placebo [n = 11]; P = .37).

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