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

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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.

Inclusion Criteria:

1. Subjects aged less than 3 months, male or female.

2. Infant with one or more superficial hemangiomas in the preproliferative phase or very
early proliferative growth phase.

3. Absence or minimal appearance of the lesion at birth

4. More pronounced appearance within 1 month of birth.

5. Willingness of parent/guardian to participate in the study

6. Willingness of parent/guardian to receive EXPERIMENTAL treatment

7. Informed consent agreement signed by the parent/guardian

8. Willingness of parent/guardian to follow the treatment schedule and post treatment
care requirements

9. Willingness of parent/guardian to not use topical or systemic (oral) TREATMENT
medications of the hemangioma other than those prescribed by the investigators during
the study period.

Exclusion Criteria:

1. Infants already on other treatment prior to PDL or timolol treatments (including
topical, systemic steroids or other agents)

2. Any infant who, in the opinion of his or her pediatrician or the investigators, has a
major medical problem (such as cardiac pathology or airway obstruction) that makes
participation in the study difficult

3. Infants with hemangiomas that threaten vital functions (e.g. obstructing the airway
or impairing hearing or vision)

4. Scarring or infection of the area to be treated

5. Subjects who are immunocompromised

6. Subject whose parent/guardian is unable to comply with treatment, home care or
follow-up visits

7. Patients with asthma or a history of asthma, chronic obstructive pulmonary disease or
cardiovascular disease, including sinus bradycardia, second or third degree
atrioventricular block, overt cardiac failure, and cardiogenic shock;
hypersensitivity to any component of timolol; and in those patients receiving
systemic administration of beta-blockers or ace inhibitors.

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Single Blind (Outcomes Assessor), Primary Purpose: Prevention

Outcome Measure:

Proportion of lesions that are completely clear or with minimum residual signs

Outcome Description:

The primary outcome measurement will be the proportion of lesions that are completely clear or with minimum residual signs (defined as faint macular erythema with no palpable component). Three independent assessors (blinded to patient allocation) will be asked to evaluate photographs at each study visit compared to baseline using a 100-mm visual analog scale (VAS). Improvement in lesion size, thickness and color relative to baseline will be assessed by three independent observers.

Outcome Time Frame:

2 years

Safety Issue:


Principal Investigator

R. Rox Anderson, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Massachusetts General Hospital


United States: Institutional Review Board

Study ID:




Start Date:

February 2011

Completion Date:

December 2015

Related Keywords:

  • Hemangioma
  • hemangioma
  • infantile hemangioma
  • pulsed dye laser
  • PDL
  • topical beta blocker
  • timolol maleate
  • timolol
  • Hemangioma



Massachusetts General HospitalBoston, Massachusetts  02114-2617