Clinical Trial of Vincristine vs. Prednisolone for Treatment of Complicated Hemangiomas
Condition: Hemangioma
MeSH terms: Hemangioma
Intervention: Vincristine; Prednisone
Intervention description: Vincristine (0.05 mg/kg/dose) will be administered into a vein (PICC line) every week for 12 weeks. If assigned to receive Vincristine, a PICC line will be placed by a doctor who is a specialist in this procedure, an interventional radiologist. This will require sedation and when possible, will be coordinated with sedation for the MRI.; Prednisolone given at 3 mg/kg/day by mouth for 12 week
Intervention MeSH terms: Prednisone; Prednisolone; Methylprednisolone Acetate; Methylprednisolone; Methylprednisolone Hemisuccinate; Prednisolone acetate; Vincristine; Prednisolone hemisuccinate; Prednisolone phosphate
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 8.0
Sex: All
Maximum age: 6 Months
Healthy volunteers: No
Keywords: hemangioma, steroid, prednisolone, vincristine

Summary:

      The goal of this study is to determine the safety and efficacy of Prednisolone and
      Vincristine for treatment of large, complicated infantile hemangiomas. The diagnostic,
      therapeutic and response criteria experimentally determined in this study will be used as a
      framework for future infantile hemangioma studies.
    

Eligibility criteria:

        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
             disfigurement.

          -  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
             penetration.

          -  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.