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A Phase II, Randomized, Clinical Trial Assessing Efficacy And Safety Of Oral Prednisolone vs Intravenous Vincristine In The Treatment Of Infantile

Phase 2
6 Months
Not Enrolling

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Trial Information

A Phase II, Randomized, Clinical Trial Assessing Efficacy And Safety Of Oral Prednisolone vs Intravenous Vincristine In The Treatment Of Infantile

Infants with large hemangiomas are often treated systemically with oral steroids
(Prednisolone) to prevent complications. The best treatment for hemangiomas is not known and
there are no medications approved by the FDA for treatment of hemangiomas. Also, the best
method to measure the response of hemangioma to treatment is not known. Patients enrolling
on this study will be randomly assigned to receive either daily Prednisolone by mouth or
weekly Vincristine in a vein. Response to treatment will be monitored by clinical exams
every two weeks and by an MRI at study entry and six and twelve weeks later. Patients with
evidence of progressive disease (larger hemangiomas) on the week 6 MRI will be switched to
the other drug to complete a total of 12 weeks of therapy. Side effects of each medication
will be monitored closely determined from histories, physical exams, blood tests and other
studies as necessary. Participation in this study will last up to 12 weeks and follow up for

Inclusion Criteria:

- Children age 0-6 months old.

- Infants with infantile hemangiomas with complications that require systemic therapy
to control their growth. To be eligible for enrollment infants must have clear
indications for systemic treatment.

- Clinical diagnosis of infantile hemangioma confirmed by tissue biopsy positive for
GLUT-1 Immunohistochemical staining. If the risk of bleeding or permanent
disfigurement from biopsy is believed to be too great then clinical and radiological
characteristics may be used to establish the diagnosis after discussion with the
study PI. Patients with GLUT-1 negative vascular tumors such as Kaposiform
hemangioendothelioma, tufted angioma, and angiosarcoma are not eligible.

- Hemangiomas must be greater than or equal to 50 cm2 clinically measured by taking the
product of the two largest perpendicular diameters and have one of the following
complications: ulceration, impairment of vision, impairment of hearing, obstruction
of the airway, high output cardiac failure, bleeding, abdominal distention and/or
compartment syndrome, compression of the spinal cord, or high risk of permanent

- Adequate liver function defined as:

- Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age, and

- SGPT(serum glutamate pyruvate transaminase) (ALT) < 2.5 x upper limit of normal
(ULN) for age.

- Patients who have received topical or intralesional corticosteroids are eligible to
be enrolled. A washout of one week is required prior to study enrollment. Patients
who have undergone surgical resection are eligible if they meet all inclusion
criteria after surgery.

- All patients' parents or legal guardians must sign a written informed consent. All
institutional and FDA requirements for human studies must be met.

Exclusion Criteria:

- Children greater then 6 months old.

- Contraindications to Vincristine: previously diagnosed neuropathy including sensory
neuropathy type 1, Charcot- Marie-Tooth or childhood poliomyelitis.

- Hemangioma involving the central nervous system (CNS) as Vincristine has poor CNS

- Infants who have received prior systemic therapy with corticosteroids (oral or
intravenous), interferon or Vincristine are not eligible for enrollment.

- Patients receiving Vincristine who concomitantly require oral steroids for treatment
of non-hemangioma indications such as asthma or atopic dermatitis will be removed
from study.

- A life-threatening intercurrent infection.

- Infants with an underlying illness that would require use of general anesthesia (as
opposed to sedation) for the MRI.

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Response of Hemangioma (IH) to Treatment

Outcome Description:

Response of IH not confined to the dermis will be coded using the following criteria: Progressive disease: >40% increase in volume by MRI, Partial response: >65% reduction in volume by MRI, Complete response: no visual or radiographic evidence of disease, Stable disease: none of the above or <40% increase or <65% decrease in volume by MRI. Response of superficial IH will be coded using the following criteria (based on RECIST): Progressive disease: >30% increase in IH size, Partial response: >30% reduction in size, Complete response: no evidence of disease, Stable disease: none of the above. Our first 3 patients showed limits to using MRI volume to measure IH size/response to therapy. Unlike other solid tumors, the superficial distribution of some IH made getting volume by MRI difficult, resulting in smaller tumor estimation compared to clinical assessment. Based on these observations, we amended the protocol to report response based on RECIST criteria instead of change in IH volume.

Outcome Time Frame:

6 weeks

Safety Issue:


Principal Investigator

Beth Drolet, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Medical College of Wisconsin


United States: Food and Drug Administration

Study ID:




Start Date:

November 2007

Completion Date:

December 2012

Related Keywords:

  • Hemangioma
  • hemangioma, steroid, prednisolone, vincristine
  • Hemangioma



Medical College of Wisconsin/Children's Hospital of Wisconsin Milwaukee, Wisconsin  53226