Trial Information

Preventing Growth of Hemangioma Tumors in Newborn: A Prospective Randomized Clinical Study

Hemangiomas affect 5-10% of all children born in the United States and up to 20% of
premature infants, with a higher incidence in girls. Most infantile hemangiomas (IHs)
appear within a few weeks of birth, grow rapidly for months to years and eventually
involute. "Benign neglect" (no treatment) is therefore recommended by most pediatricians.
However, about 1/3 of cases (2-3% of all children born in the US) eventually require medical
or surgical interventions for hemangiomas due to blocked vision, problems with breathing,
feeding, pain, ulceration, infection, profuse bleeding or disfigurement. None of the
interventions are benign. Occasionally, hemangiomas may be fatal.

The broad objective of this study is to prevent injury and disfigurement of millions of
children per year by developing a very safe, effective, and non-invasive treatment that
inhibits the growth of cutaneous hemangiomas in newborns. Historically, pulsed dye laser has
been known to bea very effective and safe treatment for hemangiomas; however, this treatment
modality has not been studied for the treatment of very early hemangiomas. Recently,
systemic beta-blockade with propanolol has also shown remarkable results in treating
threatening hemangiomas. However, systemic propanolol is not benign and requires inpatient
monitoring for cardiac side effects. Topical beta-blocker has been demonstrated in a case
report to prevent the growth of infantile eyelid hemangioma. We propose a prospective,
single blinded, randomized study of pulsed dye laser (PDL) and topical beta-blocker solution
(timolol maleate ophthalmic gel forming solution) in the treatment of very early
hemangiomas. Specifically the efficacy, side effects and outcome of PDL and timolol will be
compared with no treatment, the present standard of care for early stage hemangiomas. The
extent to which early laser treatment or topical timolol treatment prevents tumor growth and
the need for future medical or surgical treatments will be determined. Infants will be
recruited from the pediatric and neonatal practices at Massachusetts General Hospital, and
randomized to receive either: (1) a series of weekly to semi-weekly laser treatments, (2)
twice daily topical application of timolol ophthalmic gel-forming solution for six weeks, or
(3) no treatment. Hemangiomas will be assessed clinically and with digital photography for
serial repeated measures of hemangioma size at each study visit. A panel of blinded
evaluators will also provide assessment from photographs. Response, side effects and need
for additional treatments will be recorded for up to 2 years after PDL and topical timolol
treatment. This clinical trial fills a large gap in evidence-based medical therapy for IHs.
If indeed early laser treatment of hemangiomas with PDL or topical timolol, both relatively
harmless treatments, can eliminate the potential for complications by treating hemangiomas
prior to the growth phase, then this trial would present an attractive solution for the
problem of when and how to treat.