ABOUT THIS STUDY
- Diagnosis: All patients must have one of the following vascular anomalies as determined by clinical, radiologic and histologic criteria (when possible). Biopsy strongly recommended (but not required) with suggested immunostains: CD34, PROX-1 or D240, Glut-1 and MIB-1.
1. Kaposiform Hemangioendotheliomas
2. Tufted angioma
High Risk Stratification: In addition to the above diagnosis, all of the following criteria need to be met:
a. Kasabach Merritt Syndrome defined at a platelet counts less than 50,000 K/µl and/or fibrinogen level < 100 mg/dl at the time of diagnosis.
- Age: Patients must be 0 - 31 years of age at the time of study entry. Enrollment includes patients of both genders and all ethnic groups.
- Organ function requirements:
1. Adequate liver function defined as:
1. Total bilirubin ≤ 1.5 x ULN for age, and
2. SGPT (ALT) ≤ 5 x ULN for age, and
3. Serum albumin >/= 2 g/dL.
4. Fasting LDL cholesterol of <160 mg/dL
5. Fasting triglyceride <400 mg/dl
2. Adequate Bone Marrow Function defined as:
1. Peripheral absolute neutrophil count (ANC) >/= 1000/uL
2. Hemoglobin >/= 8.0 g/dL (may receive RBC transfusions)
3. No Platelet requirement
3. Adequate Renal Function Defined as:
1. A serum creatinine based on age as follows:
Age (Years) Maximum Serum Creatinine (mg/dL)
- 5 0.8 6 to ≤10 1.0 11 to ≤15 1.2 >15 1.5
2. Urine protein to creatinine ratio (UPC) < 0.3 g/l
- Performance Status: Karnofsky >/= 50 (≥16 years of age) and Lansky >/= 50 for patients <16 years of age.
- Prior therapy
1. Patients who have undergone surgical resection or interventional radiology procedures for disease control are eligible if they meet all inclusion criteria after surgery/procedure
2. Surgery: At least 2 weeks since undergoing any major surgery
3. Radiation: > 6 months from involved field radiation
4. Prior vincristine therapy is permitted. Patients may also have received up to 2 doses of vincristine prior to randomization.
- Concurrent severe and/or uncontrolled medical disease that could compromise
participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension,
severe infection, severe malnutrition, chronic liver or renal disease, active upper GI
- Patients who require medications that are strong inhibitors/inducers CYP3A4 enzyme
activity, including anticonvulsants, (Appendix II) to control concurrent medical
conditions are not eligible. Patients who discontinue use of prohibited medications
with a one week washout prior to start of study treatment are eligible.
- Known history of HIV seropositivity or known immunodeficiency. Testing is not required
unless a condition is suspected.
- Impairment of gastrointestinal function or gastrointestinal disease that may
significantly alter the absorption of sirolimus (e.g. ulcerative disease, uncontrolled
nausea, vomiting, diarrhea, malabsorption syndrome or small bowel resection). A
gastric tube or nasogastric tube is allowed.
- Females who are pregnant or breast feeding.
- Males or females of reproductive potential may not participate unless they have agreed
to use an effective contraceptive method during the period they are receiving the
study drug and for 3 months thereafter. Abstinence is an acceptable method of birth
control. Females of childbearing potential will be given a pregnancy test within 7
days prior to administration of study treatment and must have a negative urine or
serum pregnancy test.
- Patients who have received prior treatment with an mTOR inhibitor.
- Patients unwilling or unable to comply with the protocol or who in the opinion of the
investigator may not be able to comply with the safety monitoring requirements of the
- Patients who currently have an uncontrolled infection, defined as receiving
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