New hope for rare blood vessel disorders: everolimus trial launches

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: ① Diagnosis per the 2014 ISSVA classification: Group 1 (hemangioendothelioma, tufted angioma): histologically confirmed tumor, OR Kasabach-Merritt Syndrome (histologically confirmed or histologic diagnosis not feasible) Group 2 (vascular tumors not in Group 1, or vascular malformations): histologically confirmed, OR radiologically diagnosed when biopsy is not feasible * Age ≥1 year ③ Failure of at least one prior therapy (e.g., vincristine, corticosteroids, interferon), stratified as: Cohort 1: sirolimus-naïve Cohort 2: prior sirolimus failure * At least one measurable target lesion ≥1 cm in longest diameter per RECIST 1.1 on CT or MRI * ECOG Performance Score 0, 1, or 2 ⑥ WOCBP must have a negative pregnancy test prior to enrollment; adequate contraception required during the study and for 8 weeks after completion ⑦ Written informed consent obtained Exclusion Criteria: * Pregnancy or breastfeeding (WOCBP must use adequate contraception) * Documented allergy or hypersensitivity to everolimus ③ Inadequate organ function: Bone marrow: ANC \<1,000/µL or platelets \<75,000/µL Renal: serum creatinine \>1.5×ULN; if \>1.5×ULN, 24-hour creatinine clearance \<60 mL/min Hepatic: total bilirubin \>1.5×ULN or ALT \>3.0×ULN * KMP associated with vascular tumors or malformations is not an exclusion criterion, including: thrombocytopenia (\<100,000/µL), hypofibrinogenemia, anemia (Hb \<8 g/dL), consumptive coagulopathy, or overlying skin changes (edema, warmth, erythema, purplish/dark discoloration) * Uncontrolled hyperlipidemia (fasting cholesterol \>300 mg/dL or triglycerides \>2.5×ULN) * Uncontrolled diabetes (fasting glucose \>1.5×ULN) * Active uncontrolled infection * Hepatitis B (HBsAg positive) or hepatitis C (anti-HCV positive) ⑨ Known HIV infection (positive serology) * Clinically significant symptomatic pulmonary dysfunction; PFTs and room air SpO₂ performed if clinically indicated; exclusion if FEV₁ ≤70% or DLCO ≤70% of predicted (assessed in patients ≥8 years) ⑪ Prior solid organ or hematopoietic stem cell transplantation (bone marrow, liver, kidney, lung, or heart) ⑫ Concomitant investigational agents (e.g., mTOR inhibitors: sirolimus, temsirolimus) ⑬ Concurrent chemotherapy (e.g., mTOR inhibitors: sirolimus, temsirolimus) ⑭ Concurrent other malignancy not meeting eligibility criteria