None listed
Conditions
Interventions
Drug : Everolimus (dose varies by age and concomitant use of CYP3A4/P-gp inducers)
-Starting dose:
Age <10 years: 6.0 mg/m²/day (9.0 mg/m²/day with CYP3A4/P-gp inducers)
Age 10 to <18 years: 5.0 mg/m²
Sponsors
Yonsei University Health System, Severance Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: ? Diagnostic criteria: Based on the 2014 ISSVA classification, -Group 1 (hemangioendothelioma, tufted angioma) Histologically confirmed tumor OR Kasabach-Merritt Syndrome (either histologically confirmed or histologic diagnosis not feasible) -Group 2 (vascular tumors not belonging to Group 1, or vascular malformations) Histologically confirmed OR radiologically diagnosed when biopsy is not feasible ? Age =1 year ? Prior treatment failure with at least one previous therapy (e.g., vincristine, corticosteroids, interferon), stratified into the following cohorts: -Cohort 1: Sirolimus-naïve -Cohort 2: Prior sirolimus — patients who failed previous sirolimus therapy ? Measurable disease per RECIST 1.1 criteria, with at least one target lesion =1 cm in longest diameter on CT or MRI ? Performance status: ECOG Performance Score 0, 1, or 2 ? Women of childbearing potential (WOCBP) must have a negative pregnancy test prior to enrollment. WOCBP with possibility of pregnancy (e.g., sexually active) must use adequate contraception during the study and for 8 weeks after study completion. ? Written informed consent obtained
Exclusion criteria
Exclusion criteria: ? Pregnant or breastfeeding patients (WOCBP must use adequate contraception during the study period) ? Documented allergy or hypersensitivity to the study drug ? Inadequate organ function, defined as any of the following: Bone marrow: ANC 1.5 × ULN; if >1.5 × ULN, 24-hour creatinine clearance 1.5 × ULN or ALT >3.0 × ULN ? Kasabach-Merritt Phenomenon (KMP) associated with vascular tumors or malformations is not an exclusion criterion, including: Thrombocytopenia (300 mg/dL or triglycerides >2.5 × ULN) ? Uncontrolled diabetes (fasting blood glucose >1.5 × ULN) ? Uncontrolled active infection ? Hepatitis B (HBsAg positive) or hepatitis C (anti-HCV positive) ? Known HIV infection (positive serology) ? Clinically significant and symptomatic pulmonary dysfunction. Pulmonary function tests (PFTs) and room air SpO2 should be performed if clinically indicated; exclusion if FEV1 =70% or DLCO =70% of predicted. PFTs are applicable for patients aged =8 years. ? Prior solid organ or hematopoietic stem cell transplantation (bone marrow, liver, kidney, lung, or heart) ? Concomitant use of other investigational agents (e.g., mTOR inhibitors such as sirolimus or temsirolimus) ? Concurrent chemotherapy (e.g., mTOR inhibitors such as sirolimus or temsirolimus) ? Concurrent other malignancy not meeting the eligibility criteria
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Overall Response Rate, ORR | — |
Secondary
| Measure | Time frame |
|---|---|
| Overall Response Rate, ORR;Overall Survival, OS;Progression Free Survival, PFS;Toxicity;Rate of platelet recovery (in cases of patients with Kasabach Merrit Syndrome) | — |
Countries
Korea, Republic of
Contacts
Public ContactWon Kee Ahn
Yonsei University Health System, Severance Hospital
Outcome results
None listed